When paediatricians and families can't agree
child; human; ethics; preschool child; terminal care; United Kingdom; medical ethics; treatment outcome; infant; conflict; treatment withdrawal; child health care; experimental therapy
Recent high-profile cases have highlighted the difficulties that professionals caring for terminally ill or technology dependent children face. I am a paediatrician. I see children with severe problems, often chronic and frequently without a cure, and I try as far as possible to help them and their families. Occasionally there is science and evidence behind the decisions we make, but not always. Medicine does have its black and white disciplines—it is either cancer or it isn’t; or you either need to have an operation or you don’t. But paediatrics is often a discipline of uncertainty dealing with many shades of grey. Paediatricians learn to live with uncertainty and similarly have to support parents and young people to accept this uncertainty. Sometimes we have to wait for nature to reveal itself or for the future to unfold as it should. Herein lies the art of paediatrics: the ability to watch and wait, intervening in a timely fashion when required to do so and recognising the sad eventuality of having to let go of life at times. This is our craft: to do what is in the child’s best interests and use therapies that will help with life’s quality but not unnecessarily prolong inevitable death. However there are competing interests in this delicate balance in doing what is right for the child. Juggling our way along the best route required in reaching the best outcome for the child makes my opening paragraphs easier said than done. First there is medical technology, what we can do, with advances developing at a breakneck speed. Home ventilation, home total parenteral nutrition, home dialysis and transplants are all now commonplace and can sustain life in children in whom death would otherwise have been inevitable. Gene editing and small molecule therapies have the potential to change the …
Wallis C
Archives of Disease in Childhood
2018
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1136/archdischild-2017-314414" target="_blank" rel="noreferrer noopener">10.1136/archdischild-2017-314414</a>
When a Child Dies in the PICU Despite Ongoing Life Support
death; treatment withdrawal; child; Child; family; human; human relation; long term care; Only Child; palliative therapy; pediatric intensive care unit; psychology; resuscitation; terminal care; treatment outcome; uncertainty
OBJECTIVES: To examine the circumstance of death in the PICU in the setting of ongoing curative or life-prolonging goals. DATA SOURCES: Multidisciplinary author group, international expert opinion, and use of current literature. DATA SYNTHESIS: We describe three common clinical scenarios when curative or life-prolonging goals of care are pursued despite a high likelihood of death. We explore the challenges to providing high-quality end-of-life care in this setting. We describe possible perspectives of families and ICU clinicians facing these circumstances to aid in our understanding of these complex deaths. Finally, we offer suggestions of how PICU clinicians might improve the care of children at the end of life in this setting. <br/>CONCLUSION(S): Merging curative interventions and optimal end-of-life care is possible, important, and can be enabled when clinicians use creativity, explore possibilities, remain open minded, and maintain flexibility in the provision of critical care medicine. When faced with real and perceived barriers in providing optimal end-of-life care, particularly when curative goals of care are prioritized despite a very poor prognosis, tensions and conflict may arise. Through an intentional exploration of self and others' perspectives, values, and goals, and working toward finding commonality in order to align with each other, conflict in end-of-life care may lessen, allowing the central focus to remain on providing optimal support for the dying child and their family.
Lewis-Newby M; Clark JD; Butt WW; Dryden-Palmer K; Parshuram CS; Truog RD
Pediatric Critical Care Medicine
2018
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1097/PCC.0000000000001611" target="_blank" rel="noreferrer noopener">10.1097/PCC.0000000000001611</a>
Real World Presentation and Treatment Outcomes with a Predominant Induction Chemotherapy Based Approach in Nasopharyngeal Carcinoma: A Sixteen Year Report from a Teaching Hospital in India
child; adult; article; cohort analysis; female; human; major clinical study; male; retrospective study; aged; hospitalization; India; follow up; cancer recurrence; overall survival; adolescent; hearing impairment; diplopia; adjuvant therapy; smoking; multiple cycle treatment; overall response rate; progression free survival; thrombocytopenia; headache; diarrhea; histopathology; intensity modulated radiation therapy; anemia; vomiting; cancer staging; teaching hospital; palliative chemotherapy; carboplatin/cb [Drug Combination]; carboplatin/dt [Drug Therapy]; cisplatin/ae [Adverse Drug Reaction]; cisplatin/cb [Drug Combination]; cisplatin/dt [Drug Therapy]; gemcitabine/dt [Drug Therapy]; paclitaxel/cb [Drug Combination]; paclitaxel/dt [Drug Therapy]; treatment outcome; cyclophosphamide/cb [Drug Combination]; cyclophosphamide/dt [Drug Therapy]; mucosa inflammation; antiemetic agent; treatment interruption; date of death; survival prediction; neck dissection; hypothyroidism; induction chemotherapy; nasopharynx carcinoma/dt [Drug Therapy]; nasopharynx carcinoma/rt [Radiotherapy]; nasopharynx carcinoma/su [Surgery]; albumin/ec [Endogenous Compound]; bone metastasis; capecitabine/dt [Drug Therapy]; cervical lymph node; chemoradiotherapy; cisplatin/to [Drug Toxicity]; cobalt therapy; cranial nerve paralysis; distant metastasis; docetaxel/cb [Drug Combination]; docetaxel/dt [Drug Therapy]; dysphasia; epirubicin/cb [Drug Combination]; epirubicin/dt [Drug Therapy]; exophthalmos; febrile neutropenia; fluorouracil/cb [Drug Combination]; fluorouracil/dt [Drug Therapy]; liver metastasis; lung metastasis; neck swelling; neutropenia; nose obstruction; peripheral neuropathy; primary tumor/rt [Radiotherapy]; radiotherapy dosage; salvage therapy; spinal cord; toxicity/si [Side Effect]; trismus; xerostomia
Introduction: Nasopharyngeal carcinoma (NPC) is a rare malignancy in India except in north-eastern states. We present our institutional experience of 16 years highlighting management, outcomes, responses and toxicities. Material(s) and Method(s): NPC patients registered at our center during the period of 2000-2015. The primary objective of the study was to assess the overall survival (OS). Secondary outcome included determinations of response rates, progression free survival (PFS) and to assess treatment-related toxicity (CTCAE v4.0). Institute ethics committee approval was obtained prior to initiation of this study. Result(s): Data was retrieved from complete records of 222 patients out of 390 registered during study period. There were 163 males (73.4%) and 59 females (26.6%) with a male to female ratio of 2.8:1. The median age was 35 years (range 6-73). Only 5.6% (n = 12) presented in early-stage disease (stage I and II) while 89.6% (n = 199) were advanced stage (stage III, IVA, IVB). Five patients (2.2%) presented as metastatic disease. Majority of patients were treated with induction chemotherapy followed by concurrent chemoradiation (CCRT) {76.1%, n = 169}. Relapses were documented in 10.4% patients. 5% patients had loco-regional relapse while distant metastases were seen in 4% patients. The 3-year PFS and OS rates are 60.9% and 68.4%, respectively. Achieving a CR predicted superior OS on multivariate analysis. Conclusion(s): NPC is a rare malignancy and majority presented with advanced stages. This data outlines our experience and outcomes with a predominantly induction chemotherapy followed by definitive CCRT based approach.Copyright © 2022 Taylor & Francis Group, LLC.
Gogi R; Sharma A; Mohanti BK; Pramanik R; Bhasker S; Biswas A; Thakar A; Singh AC; Sikka K; Kumar R; Thulkar S; Bahadur S
Cancer Investigation
2023
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1080/07357907.2022.2141771" target="_blank" rel="noreferrer noopener">10.1080/07357907.2022.2141771</a>
Use of paclitaxel to successfully treat children, adolescents, and young adults with Kaposi sarcoma in southwestern Tanzania
child; Adolescent; adult; article; cohort analysis; female; human; major clinical study; male; retrospective study; quality of life; treatment duration; young adult; follow up; cancer palliative therapy; cancer recurrence; cancer survival; adolescent; treatment outcome; trend study; virus load; multiple cycle treatment; cancer combination chemotherapy; doxorubicin/cb [Drug Combination]; doxorubicin/dt [Drug Therapy]; vincristine/cb [Drug Combination]; vincristine/dt [Drug Therapy]; treatment planning; long term survival; Tanzania; Kaposi sarcoma/dt [Drug Therapy]; paclitaxel/dt [Drug Therapy]; antiretroviral therapy; antiretrovirus agent/dt [Drug Therapy]; bleomycin/cb [Drug Combination]; bleomycin/dt [Drug Therapy]; bleomycin/iv [Intravenous Drug Administration]; dexamethasone/dt [Drug Therapy]; drug contraindication; Human immunodeficiency virus infection/dt [Drug Therapy]; lack of drug effect; vincristine/iv [Intravenous Drug Administration]; Paclitaxel
Treating Kaposi sarcoma (KS) in children, adolescents, and young adults (AYA) remains a challenge in low-and middle-income countries (LMIC) where chemotherapy options and availability are limited. We describe a retrospective cohort review of pediatric patients with KS treated with paclitaxel in Mbeya, Tanzania, between 1 March 2011 and 31 December 2019. Paclitaxel was given to patients who had KS relapse, a contraindication to bleomycin, vincristine, and doxorubicin (ABV), special circumstances in which a clinician determined that paclitaxel was preferable to ABV, or experienced treatment failure, defined as persistent KS symptoms at the completion of treatment. All patients also received multidisciplinary palliative care. Seventeen patients aged 5.1-21.3 years received paclitaxel, of whom 47.1% (8/17) had treatment failure, 29.4% (5/17) received paclitaxel as initial treatment, and 23.5% (4/17) had relapsed. All HIV positive patients (16/17) were given anti-retroviral therapy (ART) and 87.5% (14/16) achieved viral load <1000 cp/mL. At censure, 82.3% (14/17) of patients were alive-71.4% (10/14) achieved complete clinical remission and 28.6% (4/14) achieved a partial response. The median follow up was 37.3 months (range 8.0-83.5, IQR 19.7-41.6), and no patients were lost to follow up. In this cohort, high rates of long-term survival and favorable outcomes were possible with paclitaxel treatment.Copyright © 2021 by the authors. Licensee MDPI, Basel, Switzerland.
Adinani H; Campbell L; El-Mallawany NK; Slone J; Mehta P; Bacha J
Children
2021
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.3390/children8040275" target="_blank" rel="noreferrer noopener">10.3390/children8040275</a>
Effectiveness of Time-Limited Eye Movement Desensitization Reprocessing Therapy for Parents of Children with a Rare Life-Limiting Illness: A Randomized Clinical Trial
Eye Movement Desensitization Reprocessing/methods; Stress Disorders Post-Traumatic/diagnosis/psychology/therapy; Child; Comorbidity; Emdr; Humans; Life-limiting illness; Parents; PTSD; Trauma; Treatment Outcome; Waiting Lists
BACKGROUND: Parents of children with a rare progressive life-limiting illness are at risk for parental posttraumatic stress disorder (PTSD). Studies on the treatment of parental PTSD with eye movement and desensitization reprocessing (EMDR) therapy in pediatric practice are lacking. Therefore this study aims to evaluate the feasibility and effectiveness of time-limited EMDR therapy in reducing PTSD symptoms, comorbid psychological symptoms, distress, and parental stress. METHODS: Mono-center randomized clinical trial conducted between February 2020 and April 2021. Fourteen parents (N = 7 mothers, N = 7 fathers) of mucopolysaccharidosis type III patients reporting PTSD symptoms on a (sub)clinical level were assigned to EMDR or a wait-list control condition followed by EMDR. Four sessions of EMDR (each 90 min) divided over two half-days were offered. Measurements were conducted at baseline, post-treatment/post-waitlist, and 3-months post-treatment. The primary outcome was PTSD symptom severity (PTSD Check List for DSM-5). Secondary outcomes included comorbid psychological symptoms (Brief Symptom Inventory), distress (Distress Thermometer for Parents) and parenting stress (Parenting Stress Questionnaire). Between-group comparisons pre-to-post treatment (N = 7 EMDR vs. N = 7 wait-list) and within-group comparisons (EMDR, N = 14) from pre-to-post treatment and from pre-treatment to 3-months follow-up were carried out per intent-to-treat linear mixed model analyses. RESULTS: Compared to wait-list, EMDR resulted in a significant reduction on total PTSD symptom severity (d = 1.78) and on comorbid psychological symptoms, distress and parenting stress (d = .63-1.83). Within-group comparisons showed a significant effect on all outcomes at post-treatment (d = 1.04-2.21) and at 3-months follow-up (d = .96-2.30) compared to baseline. EMDR was well-tolerated, associated with a low drop-out rate, a high therapy adherence and no adverse events. CONCLUSION: Time-limited EMDR reduces PTSD symptoms, psychological comorbidity, distress and parenting stress in parents of children with a rare progressive life-limiting illness. This treatment was feasible for these overburdened parents. Recurrent monitoring of PTSD symptoms, and, if needed, offering this time-limited type of trauma treatment should be introduced in everyday pediatric practice. Trial registration Netherlands Trial Register, NL8496. Registered 01-04-2020, https://trialsearch.who.int/Trial2.aspx?TrialID=NL8496 .
Conijn T; De Roos C; Vreugdenhil HJI; Van Dijk-Lokkart EM; Wijburg FA; Haverman L
Orphanet Journal of Rare Diseases
2022
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s13023-022-02500-9" target="_blank" rel="noreferrer noopener">10.1186/s13023-022-02500-9</a>
Examining key sociodemographic characteristics of adolescents and young adults with cancer: A post hoc analysis of the Promoting Resilience in Stress Management randomized clinical trial
child; human; palliative therapy; controlled study; female; major clinical study; male; article; adolescent; school child; young adult; quality of life; total quality management; outcome assessment; randomized controlled trial; treatment outcome; cancer center; coping behavior; socioeconomics; Pediatric Quality of Life Inventory; stress management; cancer specific quality of life; Connor-Davidson Resilience Scale; Hope Scale; patient-reported outcome; Psychological Distress Scale; psychosocial development; environmental resilience; post hoc analysis
Background: The "Promoting Resilience in Stress Management" intervention is a skills-based, early palliative care intervention with demonstrated efficacy in adolescents and young adults with cancer.
Lau N; Bradford M C; Steineck A; Scott S; Bona K; Yi-Frazier J P; McCauley E; Rosenberg A R
Palliative Medicine
2020
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1177/0269216319886215" target="_blank" rel="noreferrer noopener">10.1177/0269216319886215</a>
Clinical practice and outcomes of palliative radiation therapy in pediatric oncology patients: An international comparison of experiences from two distinct countries and health care systems
anorexia; article; bradycardia; brain metastasis; cancer palliative therapy; cancer radiotherapy; cancer survival; child; childhood cancer/rt [Radiotherapy]; clinical outcome; clinical practice; cohort analysis; follow up; human; leukemia/rt [Radiotherapy]; lymphoma/rt [Radiotherapy]; major clinical study; mucosa inflammation; neuroblastoma/rt [Radiotherapy]; opiate/dt [Drug Therapy]; opiate/pv [Special Situation for Pharmacovigilance]; osteosarcoma/rt [Radiotherapy]; pain/dt [Drug Therapy]; Palliative radiation therapy; Palliative therapy; pediatric patient; Pediatric radiation therapy; preschool child; priority journal; radiation dermatitis; radiation dose fractionation; radiation induced neoplasm; rectum hemorrhage; treatment outcome; treatment response; tumor growth
Background and purpose: This study describes clinical outcomes of palliative radiation therapy (RT)for children treated in distinct health-care environments-the US where there is advanced integration of palliative resources and Brazil, a country in the process of developing provisions for pediatric palliative care. Methods and materials: Palliative RT cases of pediatric oncology patients aged <=21-years from 2010 to 2016 in two Brazil-based and one US-based (Johns Hopkins Hospital, JHH)academic centers were reviewed in this study. Result(s): Eighty-eight pediatric patients were treated to 131 lesions with palliative RT. Forty-nine patients from the JHH cohort comprised 84 cases and 39 patients from the Brazil cohort comprised 46 cases. The most common indication for palliative RT was pain (55% overall, 39% Brazil, 63% JHH). Sixty-seven percent of patients experienced a complete (CR)or partial response (PR)to palliative RT, 12% reported stable symptoms (SS), and 22% reported progressive symptoms (PS). The median survival from the end of palliative RT was 3.6 months (95% confidence interval (CI), 2.3-4.8 months). When treated with palliative RT for pain, 83% of patients experience CR/PR, facilitating reduction or discontinuation of opiates in 46% of these patients. Conclusion(s): Despite different practices, the clinical results using palliative RT for pediatric patients treated in two unique healthcare environments demonstrated it is an effective tool for pediatric oncology patients across systems. Copyright © 2019 Elsevier B.V.
Rao A D; Figueiredo M L S; Yegya-Raman N; Sehgal S; Chen Q; Alcorn S R; Chen M J; Ladra M; Villar R; Terezakis S A
Radiotherapy and Oncology
2019
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.radonc.2019.05.017" target="_blank" rel="noreferrer noopener">10.1016/j.radonc.2019.05.017</a>
A two-step multidisciplinary approach to treat recurrent esophageal strictures in children with epidermolysis bullosa dystrophica
Male; Treatment Outcome; Follow-Up Studies; Child; Humans; Adolescent; Female; Child Preschool; Infant; Recurrence; Combined Modality Therapy; Gastrostomy; Dilatation/methods; Epidermolysis Bullosa Dystrophica/complications; Esophageal Stenosis/etiology/therapy; feeding difficulties; Epidermolysis Bullosa; surgical intervention; orthograde balloon dilation; retrograde dilation; growth improvement; nutrition
In children with severe generalized recessive dystrophic epidermolysis bullosa (RDEB), esophageal scarring leads to esophageal strictures with dysphagia, followed by malnutrition and delayed development. We describe a two-step multidisciplinary therapeutic approach to overcome malnutrition and growth retardation. In Step 1, under general anesthesia, orthograde balloon dilation of the esophagus is followed by gastrostomy creation using a direct puncture technique. In Step 2, further esophageal strictures are treated by retrograde dilation via the established gastrostomy; this step requires only a short sedation period. A total of 12 patients (median age 7.8 years, range 6 weeks to 17 years) underwent successful orthograde balloon dilation of esophageal strictures combined with direct puncture gastrostomy. After 12 and 24 months in 11 children, a substantial improvement of growth and nutrition was achieved (body mass index [BMI] standard deviation score [SDS] + 0.59 and + 0.61, respectively). In one child, gastrostomy was removed because of skin ulcerations after 10 days. Recurrent esophageal strictures were treated successfully in five children. The combined approach of balloon dilation and gastrostomy is technically safe in children with RDEB, and helps to promote catch-up growth and body weight. In addition, recurrent esophageal strictures can be treated successfully without general anesthesia in a retrograde manner via the established gastrostomy.
Vowinkel T; Laukoetter M; Mennigen R; Hahnenkamp K; Gottschalk A; Boschin M; Frosch M; Senninger N; Tubergen D
Endoscopy
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1055/s-0034-1391308" target="_blank" rel="noreferrer noopener">10.1055/s-0034-1391308</a>
Dystonia in neurodegeneration with brain iron accumulation: outcome of bilateral pallidal stimulation
Male; Treatment Outcome; Young Adult; Child; Humans; Adult; Adolescent; Female; Child Preschool; Infant; Retrospective Studies; Brain Diseases/physiopathology/therapy; Brain/physiopathology; Deep Brain Stimulation/adverse effects/methods; Dystonia/physiopathology/therapy; Functional Laterality; Globus Pallidus/physiopathology; Iron/metabolism; Neurodegenerative Diseases/physiopathology/therapy; tone and motor problems; IND; surgical intervention; bilateral pallidal stimulation; dystonia
Neurodegeneration with brain iron accumulation encompasses a heterogeneous group of rare neurodegenerative disorders that are characterized by iron accumulation in the brain. Severe generalized dystonia is frequently a prominent symptom and can be very disabling, causing gait impairment, difficulty with speech and swallowing, pain and respiratory distress. Several case reports and one case series have been published concerning therapeutic outcome of pallidal deep brain stimulation in dystonia caused by neurodegeneration with brain iron degeneration, reporting mostly favourable outcomes. However, with case studies, there may be a reporting bias towards favourable outcome. Thus, we undertook this multi-centre retrospective study to gather worldwide experiences with bilateral pallidal deep brain stimulation in patients with neurodegeneration with brain iron accumulation. A total of 16 centres contributed 23 patients with confirmed neurodegeneration with brain iron accumulation and bilateral pallidal deep brain stimulation. Patient details including gender, age at onset, age at operation, genetic status, magnetic resonance imaging status, history and clinical findings were requested. Data on severity of dystonia (Burke Fahn Marsden Dystonia Rating Scale-Motor Scale, Barry Albright Dystonia Scale), disability (Burke Fahn Marsden Dystonia Rating Scale-Disability Scale), quality of life (subjective global rating from 1 to 10 obtained retrospectively from patient and caregiver) as well as data on supportive therapy, concurrent pharmacotherapy, stimulation settings, adverse events and side effects were collected. Data were collected once preoperatively and at 2-6 and 9-15 months postoperatively. The primary outcome measure was change in severity of dystonia. The mean improvement in severity of dystonia was 28.5% at 2-6 months and 25.7% at 9-15 months. At 9-15 months postoperatively, 66.7% of patients showed an improvement of 20% or more in severity of dystonia, and 31.3% showed an improvement of 20% or more in disability. Global quality of life ratings showed a median improvement of 83.3% at 9-15 months. Severity of dystonia preoperatively and disease duration predicted improvement in severity of dystonia at 2-6 months; this failed to reach significance at 9-15 months. The study confirms that dystonia in neurodegeneration with brain iron accumulation improves with bilateral pallidal deep brain stimulation, although this improvement is not as great as the benefit reported in patients with primary generalized dystonias or some other secondary dystonias. The patients with more severe dystonia seem to benefit more. A well-controlled, multi-centre prospective study is necessary to enable evidence-based therapeutic decisions and better predict therapeutic outcomes.
Timmermann L; Pauls K A; Wieland K; Jech R; Kurlemann G; Sharma N; Gill S S; Haenggeli C A; Hayflick S J; Hogarth P; Leenders K L; Limousin P; Malanga C J; Moro E; Ostrem J L; Revilla F J; Santens P; Schnitzler A; Tisch S; Valldeoriola F; Vesper J; Volkmann J; Woitalla D; Peker S
Brain
2010
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1093/brain/awq022" target="_blank" rel="noreferrer noopener">10.1093/brain/awq022</a>
Comparison of treatment modalities in syndromic children with Obstructive Sleep Apnea-A randomized cohort study
quality of life; major clinical study; priority journal; follow up; randomized controlled trial; mucopolysaccharidosis; outcome assessment; clinical effectiveness; human; article; child; female; male; controlled study; adenotonsillectomy; apnea hypopnea index; Continuous Positive Airway Pressure; CPAP device; Down syndrome; Epworth sleepiness scale; ess-c; Mucopolysaccharidoses; osa-18; Pediatric obstructive sleep apnea; positive end expiratory pressure; radiofrequency ablation device; sleep disordered breathing/su [Surgery]; sleep disordered breathing/th [Therapy]; treatment outcome; breathing difficulties; MPSI; MPSII; MPSIII; MPSIIIA; MPSIIIB; MPSVI; MPSVII; surgical interventions; physical interventions; adenotonsillectomy
Introduction: Obstructive Sleep Apnea (OSA) is a common medical problem in adults that is becoming increasingly recognized in children. It occurs in the pediatric age group, from newborns to teens. More recently, many specialists have estimated OSA prevalence to be between 5 and 6%. However, in syndromic children, the prevalence of OSA can be from 50 to 100%, having a significant effect on their Quality-of-Life. As they are a challenging population for management, it is essential to evaluate them thoroughly before planning appropriate intervention. Objective: To compare the efficacy of Adenotonsillectomy (T&A) and Continuous Positive Airway Pressure (CPAP) in syndromic children [Down syndrome (DS) and Mucopolysaccharidoses (MPS)] with Obstructive Sleep Apnea (OSA). Materials and methods: In a prospective, randomized, cohort comparative study, 124 syndromic children (DS and MPS) aged between 6 and 12 years were recruited from a private MPS support group and the Down Syndrome Society, Chennai. A standard assessment was performed on all children who entered the study including a full overnight Polysomnogram (PSG), Epworth Sleepiness Scale-Children (ESS-C) and Quality-of-Life (QOL) tool OSA-18. The children with positive PSG who consented for the study (n= 80) were randomly distributed to two groups, T&A group & CPAP group. The children were followed up with repeat PSG, clinical evaluation, ESS-C and Quality-of-Life (QOL) tool OSA-18 for a period of 1 year. Observation and results: Follow-up was available for 73 syndromic children. Both the groups, T&A group and CPAP group, showed statistically significant (p<. 0.05) improvement in Apnea-Hypoapnea Index (AHI), ESS-C, QOL from the intervention. In our study, T&A showed equal outcome compared to CPAP. The contrasting feature between the two groups was that CPAP use gave immediate sustained improvement while T&A gave gradual progressive improvement of symptoms over a period of 1 year. Conclusion: On average, T&A gives equal outcomes as CPAP and it can be suggested as a first-line treatment in this group of syndromic children. © 2014.
Sudarsan S S; Paramasivan V K; Arumugam S V; Murali S; Kameswaran M
International Journal of Pediatric Otorhinolaryngology
2014
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.ijporl.2014.06.027" target="_blank" rel="noreferrer noopener">10.1016/j.ijporl.2014.06.027</a>
Efficacy and tolerance of gastrostomy feeding in pediatric forms of neuromuscular diseases
Male; Treatment Outcome; Infant Newborn; Follow-Up Studies; Child; Humans; Adult; Adolescent; Female; Child Preschool; Infant; Nutritional Status; Weight Gain; Gastrostomy; Gastroesophageal Reflux/complications; Enteral Nutrition; Neuromuscular Diseases/complications/therapy; feeding difficulties; SMA type I; surgical intervention; percutaneous endoscopic gastrostomy; gastroesophageal reflux; GERD
BACKGROUND: Percutaneous endoscopic gastrostomy (PEG) is a simple and reliable method, but there are few data about its use in pediatric forms of neuromuscular diseases (NMD). The aim of this study was to evaluate the nutritional effects and tolerance of gastrostomy feeding in NMD patients. METHODS: Twelve patients with NMD, ranging in age from 1 month to 25.5 years, underwent a gastrostomy placement (PEG, n = 11; surgical gastrostomy, n = 1) between January 1990 and December 2000. Diseases were muscular dystrophies (n = 5), infantile spinal muscular atrophies (n = 3), congenital myopathies (n = 3), and polyradiculoneuritis (n = 1). Height-for-age (Z(H/A)), weight-for-age (Z(W/A)), and weight-for-height (Z(W/H)) z scores were assessed at birth, at the time of gastrostomy placement, after a 6-month and 1-year follow-up, and at the end of follow-up. Complications that occurred during the gastrostomy feeding period were recorded. Comparisons of z scores at the different times were performed with repeated-measures analyses of variance. RESULTS: Z(W/A) (p < .05) and Z(W/H) (p < .001) improved in the whole group after gastrostomy. For the 10 patients for whom follow-up was more than 1 year, Z(W/A) (p < .03) and Z(W/H) (p < .001) increased from the time of gastrostomy to the end of follow-up. Among 5 patients who had a gastroesophageal reflux diagnosed before gastrostomy placement, 3 had at least 1 episode of pneumonia and 2 died of respiratory distress caused by the worsening of NMD. No other major complication was encountered. CONCLUSIONS: Gastrostomy feeding is well tolerated and results in an improvement of nutritional indices in NMD patients. Special care should be taken in patients with preexisting gastroesophageal reflux.
Seguy D; Michaud L; Guimber D; Cuisset J; Devos P; Turck D; Gottrand F
Journal of Parenteral and Enteral Nutrition
2002
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1177/0148607102026005298" target="_blank" rel="noreferrer noopener">10.1177/0148607102026005298</a>
Improving functional skills and physical fitness in children with Rett syndrome
Treatment Outcome; Child; Humans; Female; Motor Skills; Statistics as Topic; Feasibility Studies; Social Behavior; Physical Fitness; Activities of Daily Living/psychology; Education of Intellectually Disabled; Exercise/psychology; Rett Syndrome/psychology/rehabilitation; tone and motor problems; Rett syndrome; psychological intervention; daily conductive educational program
BACKGROUND: To investigate the feasibility of a physical exercise programme with treadmill for persons with Rett syndrome (RS) in order to promote fitness and health. METHODS: A daily training programme on a treadmill was designed for four females with RS over a period of 2 months with tests performed in three intervals, at time 1, 2 and 3, 2 months apart with intervention taking place between tests 2 and 3. Participants were four girls with RS aged 8.5-11 years (mean: 10 years) attending the educational facility Beit Issie Shapiro, Raanana, Israel, all with independent mobility and with typical characteristics of RS stage III. The training took place at the educational facility, on a 1400 model treadmill (Trimline, capable of very low speeds < 0.5 k/h), with very long side rails. Special low side rails were adapted to the treadmill in order to fit the height of the children and velcro straps were added to assist in safely placing the hands. Pulse was monitored constantly during exercise by an A3 polar pulse belt. Pulse measurements at rest during training were considered as evaluators of aerobic physical condition. Functional measurement was based on a scale specially established for the present study. The scale was a 31-item motor-functioning tool that measures the ability of participants to knee walk and knee stand, to get up to a standing position, duration of walking different paths, and to go up and down stairs and slopes. RESULTS: The study showed that physical fitness of the children at the end of the training programme had improved considerably (P < 0.05). Tests showed that general functional abilities had improved considerably (P < 0.0001). Although all items of the functional ability measure showed impressive positive change, some of the 31 items on it showed statistically significant improvement (knee walking, going up and down stairs and speed of walking for 25 m. Pearson correlation showed high linkage (r = -0.76) between functional improvement and change in physical fitness. CONCLUSIONS: Physical fitness programme executed on a daily basis is capable of improving functional ability of children with RS. Nonprofessional personnel can execute such a programme under supervision of a qualified physical therapist.
Lotan M; Isakov E; Merrick J
Journal of Intellectual Disability Research
2004
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1111/j.1365-2788.2003.00589.x" target="_blank" rel="noreferrer noopener">10.1111/j.1365-2788.2003.00589.x</a>
Subthalamic Nuclei Stimulation in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
adolescent; subthalamic nucleus; follow up; scoring system; human; article; female; male; adult; middle aged; dystonia; case report; treatment outcome; brain depth stimulation; Deep brain stimulation; neurologic disease assessment; Burke Fahn Marsden Dystonia Rating Scale movement rating scale; fluency disorder; globus pallidus internus; neurodegeneration with brain iron accumulation/th [Therapy]; neurologic examination; pantothenate kinase-associated neurodegeneration; subthalamic nuclei; tone and motor problems; IND; surgical intervention; subthalamic nuclei stimulation
Introduction: Pantothenate kinase-associated neurodegeneration (PKAN) is a rare autosomal recessive genetic disease that leads to extrapyramidal symptoms, such as dystonia, ataxia, dysarthria, and involuntary movements. Treatment of PKAN with deep brain stimulation (DBS) has been reported, but mainly focuses on targeting the globus pallidus internus (GPi). Subthalamic nuclei (STN) may also be a potential target for treatment of PKAN. Methods: In this study, we reviewed three patients with PKAN (two with typical PKAN and one with atypical PKAN) treated by bilateral STN stimulation and present a review of the literature. All patients received neurological evaluation using the Burke-Fahn-Marsden Dystonia Rating Scale-movement (BFMDRS) scoring system before and after surgery. Patients were then subject to regular clinical follow-ups (ranging from 22 to 44 months). Results: The mean stimulation amplitude, pulse width and frequency was 2.65 +/- 0.45 V, 91.7 +/- 21.9 mus, and 146.7 +/- 12.5 Hz, respectively. BFMDRS scores were improved in all patients after surgery, ranging from 41.6 to 73.1%. Improvements of appendicular symptoms ranged from 46.2 to 94.1%, and improvements of axial symptoms ranged from 27.3 to 33.3%. No side effects were reported in patients 1 and 2; whereas patient 3 exhibited a mild decline in verbal fluency one year after surgery. Conclusion: STN stimulation could serve as a candidate DBS target in the treatment of PKAN, especially for patients with prominent appendicular symptoms. Copyright © 2017 International Neuromodulation Society
Liu Z; Liu Y; Yang Y; Wang L; Dou W; Guo J; Wang Y; Guo Y; Wan X; Ma W; Wang R
Neuromodulation
2017
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1111/ner.12549" target="_blank" rel="noreferrer noopener">10.1111/ner.12549</a>
Effect of melatonin dosage on sleep disorder in tuberous sclerosis complex
Male; Treatment Outcome; Dose-Response Relationship Drug; Child; Humans; Adult; Cross-Over Studies; Female; Child Preschool; Infant; Administration Oral; Double-Blind Method; Tuberous Sclerosis/complications; Antioxidants/pharmacology/therapeutic use; Melatonin/pharmacology/therapeutic use; Sleep Wake Disorders/drug therapy/etiology; sleep disturbance/disorders; tuberous sclerosis; pharmacologic intervention; melatonin
We report a randomized, double-blind, controlled, crossover trial investigating the response to oral melatonin using two dose regimens in patients with sleep disorders associated with tuberous sclerosis complex. Eight outpatients with tuberous sclerosis complex and sleep disorder received either 5 or 10 mg of melatonin. Sleep latency, total sleep time, number of awakenings, and seizure frequency were recorded in sleep and seizure diaries. No evidence of a dose effect between 5 and 10 mg was seen with respect to any outcome measure. (The 5 mg results are given first: sleep latency, 86 and 76 minutes; total sleep time, 8 hours, 57 minutes and 9 hours, 4 minutes; and sleep fragmentation, 0.8 and 1.0). This study might have missed a small beneficial effect of 10 mg melatonin. We propose that an initial trial of 5 mg melatonin is worth considering in patients with tuberous sclerosis complex and sleep disorder.
Hancock E; O'Callaghan F; Osborne J P
Journal of Child Neurology
2005
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<a href="http://doi.org/10.1177/08830738050200011302" target="_blank" rel="noreferrer noopener">10.1177/08830738050200011302</a>
Melatonin for sleep problems in children with neurodevelopmental disorders: randomised double masked placebo controlled trial
Male; Treatment Outcome; Severity of Illness Index; Dose-Response Relationship Drug; Child; Humans; Adolescent; Family Health; Female; Child Preschool; Drug Monitoring; Child Behavior/drug effects; Melatonin/administration & dosage/adverse effects; Sleep Wake Disorders/diagnosis/drug therapy/etiology; Central Nervous System Depressants/administration & dosage/adverse effects; Central Nervous System Diseases/complications; Developmental Disabilities/complications; Polysomnography/methods; Sleep/drug effects; sleep disturbance/disorders; unspecified Q3 conditions; Q3 conditions; pharmacologic intervention; melatonin
OBJECTIVE: To assess the effectiveness and safety of melatonin in treating severe sleep problems in children with neurodevelopmental disorders. DESIGN: 12 week double masked randomised placebo controlled phase III trial. SETTING: 19 hospitals across England and Wales. PARTICIPANTS: 146 children aged 3 years to 15 years 8 months were randomised. They had a range of neurological and developmental disorders and a severe sleep problem that had not responded to a standardised sleep behaviour advice booklet provided to parents four to six weeks before randomisation. A sleep problem was defined as the child not falling asleep within one hour of lights out or having less than six hours' continuous sleep. INTERVENTIONS: Immediate release melatonin or matching placebo capsules administered 45 minutes before the child's bedtime for a period of 12 weeks. All children started with a 0.5 mg capsule, which was increased through 2 mg, 6 mg, and 12 mg depending on their response to treatment. MAIN OUTCOME MEASURES: Total sleep time at night after 12 weeks adjusted for baseline recorded in sleep diaries completed by the parent. Secondary outcomes included sleep onset latency, assessments of child behaviour, family functioning, and adverse events. Sleep was measured with diaries and actigraphy. RESULTS: Melatonin increased total sleep time by 22.4 minutes (95% confidence interval 0.5 to 44.3 minutes) measured by sleep diaries (n=110) and 13.3 (-15.5 to 42.2) measured by actigraphy (n=59). Melatonin reduced sleep onset latency measured by sleep diaries (-37.5 minutes, -55.3 to -19.7 minutes) and actigraphy (-45.3 minutes, -68.8 to -21.9 minutes) and was most effective for children with the longest sleep latency (P=0.009). Melatonin was associated with earlier waking times than placebo (29.9 minutes, 13.6 to 46.3 minutes). Child behaviour and family functioning outcomes showed some improvement and favoured use of melatonin. Adverse events were mild and similar between the two groups. CONCLUSIONS: Children gained little additional sleep on melatonin; though they fell asleep significantly faster, waking times became earlier. Child behaviour and family functioning outcomes did not significantly improve. Melatonin was tolerable over this three month period. Comparisons with slow release melatonin preparations or melatonin analogues are required. TRIAL REGISTRATION: ISRCT No 05534585.
Gringras P; Gamble C; Jones A P; Wiggs L; Williamson P R; Sutcliffe A; Montgomery P; Whitehouse W P; Choonara I; Allport T; Edmond A; Appleton R
British Medical Journal
2012
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1136/bmj.e6664" target="_blank" rel="noreferrer noopener">10.1136/bmj.e6664</a>
Sleep disturbance in Sanfilippo syndrome: a parental questionnaire study
Treatment Outcome; Infant Newborn; Humans; Adolescent; Child Preschool; Infant; Questionnaires; Age of Onset; Incidence; Behavior Therapy; Melatonin/therapeutic use; Mucopolysaccharidosis III/psychology; Sleep Disorders/epidemiology/etiology/therapy; Q3 Literature Search; child; adult; Adolescent; Adult; Age of Onset; Behavior Therapy; Child; Preschool; Humans; Incidence; Infant; sleep disturbance/disorders; MPS III; trajectory; characteristics; melatonin; benzodiazepines
AIMS: To determine the incidence, manifestations, and best management of sleep disturbance in Sanfilippo syndrome (mucopolysaccharidosis (MPS) type III). METHODS: Families were ascertained through the MPS societies of Australasia, the UK, and the USA. Questionnaires were sent by mail and were answered anonymously. Identical questions regarding sleep disturbance were asked about unaffected siblings to provide control data. Sleep disturbance was quantified by a total sleep disturbance score. RESULTS: A total of 141 responses were received; 91.5% of children with Sanfilippo syndrome had sleep disturbance and this was significantly higher than for their unaffected sibs; 77.5% of parents had used medication for this problem, with melatonin and antihistamines being most commonly used. Melatonin and benzodiazepines were reported as the most efficacious. Many different environmental modifications had been employed for this problem and some parents reported success with behavioural therapies. CONCLUSIONS: Sleep disturbance is common, severe, and difficult to manage in Sanfilippo syndrome. Based on the parental responses and its safety profile, melatonin is the first line drug that should be tried. Behavioural therapy should be tried in all with Sanfilippo syndrome and sleep disturbance.
Fraser J; Gason A A; Wraith J E; Delatycki M B
Archives of Disease in Childhood
2005
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1136/adc.2004.065482" target="_blank" rel="noreferrer noopener">10.1136/adc.2004.065482</a>
Melatonin treatment in individuals with intellectual disability and chronic insomnia: a randomized placebo-controlled study
Saliva; Middle Aged; Treatment Outcome; Humans; Adolescent; Child Preschool; Double-Blind Method; Time Factors; Central Nervous System Depressants/adverse effects/therapeutic use; Melatonin/adverse effects/therapeutic use; Mental Retardation/epidemiology/psychology; Sleep Initiation and Maintenance Disorders/drug therapy/epidemiology/psychology; Q3 Literature Search; chronic disease; child; female; male; adult; comorbidity; aged; sleep disturbance/disorders; chromosome 18q deletion; MPS III; pharmacologic intervention; melatonin
BACKGROUND: While several small-number or open-label studies suggest that melatonin improves sleep in individuals with intellectual disabilities (ID) with chronic sleep disturbance, a larger randomized control trial is necessary to validate these promising results. METHODS: The effectiveness of melatonin for the treatment of chronic sleep disturbance was assessed in a randomized double-blind placebo-controlled trial with 51 individuals with ID. All of these individuals presented with chronic ideopatic sleep disturbance for more than 1 year. The study consisted of a 1-week baseline, followed by 4 weeks of treatment. Parents or other caregivers recorded lights off time, sleep onset time, night waking, wake up time and epileptic seizures. Endogenous melatonin cycle was measured in saliva before and after treatment. RESULTS: Compared with placebo, melatonin significantly advanced mean sleep onset time by 34 min, decreased mean sleep latency by 29 min, increased mean total sleep time by 48 min, reduced the mean number of times the person awoke during the night by 0.4, decreased the mean duration of these night waking periods by 17 min and advanced endogenous melatonin onset at night by an average of 2.01 h. Lights off time, sleep offset time and the number of nights per week with night waking did not change. Only few minor or temporary adverse reactions and no changes in seizure frequency were reported. CONCLUSIONS: Melatonin treatment improves some aspects of chronic sleep disturbance in individuals with ID.
Braam W; Didden R; Smits M; Curfs L
Journal of Intellectual Disability Research
2008
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1111/j.1365-2788.2007.01016.x" target="_blank" rel="noreferrer noopener">10.1111/j.1365-2788.2007.01016.x</a>
Esophageal strictures in children with recessive dystrophic epidermolysis bullosa: an 11-year experience with fluoroscopically guided balloon dilatation
Adolescent; Barium; Catheterization; Child; Preschool; Epidermolysis Bullosa; Dystrophica/co [Complications]; Esophageal Stenosis/et [Etiology]; Esophageal Stenosis/th [Therapy]; Female; Humans; Male; Retrospective Studies; Treatment Outcome; 24GP945V5T (Barium); feeding difficulties; surgical intervention; fluoroscopically guided balloon dilatation
BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is an inherited blistering skin disorder that is associated with significant esophageal strictures, resulting in dysphagia and nutritional failure. Although endoscopically guided balloon dilatation is a widely used treatment, the use of an endoscope carries the risk of oropharyngeal trauma. To minimize this risk, we have eliminated its use. METHOD: We reviewed the charts of all RDEB patients who underwent balloon dilatation for esophageal strictures between August 1993 and March 2005. Balloon dilatation procedures were performed under anesthesia and with fluoroscopic control. RESULTS: We performed 92 dilatations on 25 RDEB patients. Most patients reported immediate relief of symptoms, rapid recovery, and resumption of adequate food intake within 1 day. The mean interval between dilatations was 1 year. Six patients (24%) have required only 1 dilatation, and 1 of these 6 has had a dilatation-free interval of 25 months. One patient with a history of multiple dilatations has remained dilatation-free for 5 years. No procedure-related complications have occurred. CONCLUSIONS: Fluoroscopically guided balloon dilatation is a gentle, safe, effective, and repeatable technique that should be considered as a first line of treatment.
Azizkhan R G; Stehr W; Cohen A P; Wittkugel E; Farrell M K; Lucky A W; Hammelman B D; Johnson N D; Racadio J M
Journal of Pediatric Surgery
2006
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.jpedsurg.2005.10.007" target="_blank" rel="noreferrer noopener">10.1016/j.jpedsurg.2005.10.007</a>
Efficacy and safety of endoscopic dilation of esophageal strictures in epidermolysis bullosa
adolescent; dysphagia; major clinical study; priority journal; follow up; scoring system; school child; human; article; female; male; adult; morbidity; treatment outcome; balloon dilatation; barium; body weight; contrast enhancement; endoscopic therapy; epidermolysis bullosa/th [Therapy]; epidermolysis bullosa; esophagus dilatation; esophagus stricture/th [Therapy]; nutritional status; propofol; feeding difficulties; surgical intervention; endoscopic balloon dilatation
Background: Epidermolysis bullosa is a rare genetically determined disorder of the stratified squamous epithelium. Patients with the most severe forms develop scarring of the esophagus after ingestion of food. This results in dysphagia, which severely compromises the ability to eat. Maintenance of adequate nutritional intake is a central aim, but the most appropriate method is unknown. Methods: The results of endoscopic through-the-scope balloon dilation under propofol anesthesia in 53 patients with epidermolysis bullosa and esophageal strictures are reported. Results: Seventy-five percent of patients had a single stricture (range 1 to 6 strictures), most often in the proximal esophagus (median 20 cm from incisors). A total of 182 dilations were performed (median two per patient) over a median follow-up period of 3.5 years. For all but 3 patients, there was an improvement in the dysphagia score. There was a mean increase in weight after the procedure of 2.9 kg: 95% CI[2.0, 3.8]; p < 0.001, over a median 29 days. There was no significant post-procedure morbidity. Conclusions: Endoscopic balloon dilation is a safe and effective treatment for the esophageal strictures of epidermolysis bullosa. In the majority of patients, dilation relieves dysphagia and improves nutritional status.
Anderson S H C; Meenan J; Williams K N; Eady R A J; Prinja H; Chappiti U; Doig L; Thompson R P H
Gastrointestinal Endoscopy
2004
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/S0016-5107%2803%2902379-4" target="_blank" rel="noreferrer noopener">10.1016/S0016-5107%2803%2902379-4</a>
Respiratory events and obstructive sleep apnea in children with achondroplasia: investigation and treatment outcomes
Cross-Sectional Studies; Age Factors; Male; Treatment Outcome; Comorbidity; Child; Humans; Adolescent; Cohort Studies; Female; Child Preschool; Infant; Polysomnography; Body Mass Index; Achondroplasia/di [Diagnosis]; Achondroplasia/th [Therapy]; Sleep Apnea Central/di [Diagnosis]; Sleep Apnea Central/th [Therapy]; Sleep Apnea Obstructive/di [Diagnosis]; Sleep Apnea Obstructive/th [Therapy]; Achondroplasia/ep [Epidemiology]; Airway Obstruction/di [Diagnosis]; Airway Obstruction/ep [Epidemiology]; Airway Obstruction/th [Therapy]; Sleep Apnea Central/ep [Epidemiology]; Sleep Apnea Obstructive/ep [Epidemiology]; breathing difficulties; achondroplasia; trajectory; characteristics; obstructive sleep apnea
PURPOSE: We report aspects of sleep-disordered breathing in a cohort of achondroplastic children who attended our hospital. METHODS: A retrospective chart review was conducted for a 15-year period to further evaluate the diagnosis and treatment of sleep-disordered breathing in children with achondroplasia. RESULTS: A review of the medical records was undertaken for 46 children (63%, mean age 3.9 years) with achondroplasia that had overnight polysomnography. Among them, 25 (54.3%) had obstructive sleep apnea (OSA). For 19 out of 46 patients (follow-up rate, 41.3%) with a mean follow-up of 31.3 months (range, 3 month to 11 years), 13 had undergone adenotonsillectomy, while nine were treated with continuous positive airway pressure. CONCLUSIONS: Prospective evaluation of our clinic population confirms a high incidence of SDB in achondroplastic children. OSA has been linked to raise intracranial pressure as well as neurocognitive deficits in children and we hypothesize that associations between neurological and respiratory abnormalities in this disorder are a consequence of the early onset of associated respiratory, rather than the neurological complications.
Afsharpaiman S; Sillence D O; Sheikhvatan M; Ault J E; Waters K
Sleep and Breathing
2011
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<a href="http://doi.org/10.1007/s11325-010-0432-6" target="_blank" rel="noreferrer noopener">10.1007/s11325-010-0432-6</a>
Incompatible with Care: Examining Trisomy 18 Medical Discourse and Families' Counter-discourse for Recuperative Ethos
psychology; child parent relation; human; Edwards syndrome; treatment outcome; blogging
Parents whose child is diagnosed with a serious disease such as trisomy 18 first rely on the medical community for an accurate description and prognosis. In the case of trisomy 18, however, many families are told the disease is "incompatible with life" even though some children with the condition live for several years. This paper considers parents' response to current medical discourse concerning trisomy 18 by examining blogs written by the parents of those diagnosed. Using interpretive humanistic reading and foregrounding Cathryn Molloy's recuperative ethos theory (2015), we find that parents demonstrate recuperative ethos in response to physicians' descriptions of trisomy 18, particularly in rhetoric addressing survival, medicalized language, and religious and/or spiritual rhetoric. We argue that, by using language such as "incompatible with life," physicians distance themselves from families, creating not care, but the very gulf that requires recuperation. We conclude that medical professionals would do well to engage with the trisomy 18 community-including learning from blogs and online forums- employ palliative care practices, and seek more accurate, descriptive language that is compatible with care.
Thorvilson M J; Copeland A J
The Journal of medical humanities
2018
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<a href="http://doi.org/10.1007/s10912-017-9436-6" target="_blank" rel="noreferrer noopener"> 10.1007/s10912-017-9436-6</a>
Parents of children diagnosed with cancer: work situation and sick leave, a five-year post end-of-treatment or a child's death follow-up study
Survival Rate; Male; Treatment Outcome; Infant Newborn; Bereavement; Follow-Up Studies; Child; Humans; Adult; Adolescent; Female; Child Preschool; Infant; Survivors; Sweden; Sick Leave/statistics & numerical data; Fathers/psychology/statistics & numerical data; Mothers/psychology/statistics & numerical data; Neoplasms/economics/mortality/psychology/therapy
BACKGROUND: Cancer in a child is associated with a significant impact on parental employment. We assessed the proportions of parents of survivors and bereaved parents working and reporting sick leave five years after end of successful treatment (ST)/child's death (T7) compared with one year after end of ST/child's death (T6) and the association between partial post-traumatic stress disorder (PTSD) and work situation and sick leave at T7. Participants and procedure: The sample included 152 parents of survivors (77 mothers, 75 fathers) and 42 bereaved parents (22 mothers, 20 fathers) of children diagnosed with cancer in Sweden. RESULTS: The proportions of parents working or reporting sick leave did not differ among mothers and fathers of survivors (92% vs. 96% working, 20% vs. 18% on sick leave) or among bereaved mothers and fathers (91% vs. 90% working, 14% vs. 20% on sick leave) at T7. There was no change from T6 to T7 in the proportion of fathers working (fathers of survivors 91% vs. 96%, bereaved fathers 95% vs. 90%). Although more mothers of survivors (92% vs. 82%) and bereaved mothers (91% vs. 77%) worked at T7 than at T6, this increase was not significant. Fewer bereaved mothers reported sick leave at T7 than at T6 (14% vs. 59%, p < 0.05). Although more fathers reported sick leave at T7 than at T6 (fathers of survivors 18% vs. 8%, bereaved fathers 20% vs. 15%), this was not significant. Partial PTSD was not associated with parents' work situation or sick leave at T7. CONCLUSION: Results suggest little adverse effect on work situation and sick leave among parents of survivors and bereaved parents five years after end of ST/child's death from cancer. However, the pattern of change observed differed between parents, which could potentially indicate possible delayed consequences for fathers not captured in the present paper.
Wikman A; Hoven E; Cernvall M; Ljungman G; Ljungman L; von Essen L
Acta Oncologica
2016
<a href="http://doi.org/%2010.3109/0284186x.2016.1167956" target="_blank" rel="noreferrer noopener">10.3109/0284186x.2016.1167956</a>
Experts' Perspectives Toward a Population Health Approach for Children With Medical Complexity
OBJECTIVE: Because children with medical complexity (CMC) display very different health trajectories, needs, and resource utilization than other children, it is unclear how well traditional conceptions of population health apply to CMC. We sought to identify key health outcome domains for CMC as a step toward determining core health metrics for this distinct population of children. METHODS: We conducted and analyzed interviews with 23 diverse national experts on CMC to better understand population health for CMC. Interviewees included child and family advocates, health and social service providers, and research, health systems, and policy leaders. We performed thematic content analyses to identify emergent themes regarding population health for CMC. RESULTS: Overall, interviewees conveyed that defining and measuring population health for CMC is an achievable, worthwhile goal. Qualitative themes from interviews included: 1) CMC share unifying characteristics that could serve as the basis for population health outcomes; 2) optimal health for CMC is child specific and dynamic; 3) health of CMC is intertwined with health of families; 4) social determinants of health are especially important for CMC; and 5) measuring population health for CMC faces serious conceptual and logistical challenges. CONCLUSIONS: Experts have taken initial steps in defining the population health of CMC. Population health for CMC involves a dynamic concept of health that is attuned to individual, health-related goals for each child. We propose a framework that can guide the identification and development of population health metrics for CMC.
Barnert ES; Coller R J; Nelson BB; Thompson LR; Chan V; Padilla C; Klitzner TS; Szilagyi M; Chung P J
Academic Pediatrics
2017
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.acap.2017.02.010" target="_blank" rel="noreferrer noopener">10.1016/j.acap.2017.02.010</a>
Inpatient-based intensive interdisciplinary pain treatment for highly impaired children with severe chronic pain: randomized controlled trial of efficacy and economic effects
adolescent; Child; Female; Humans; Male; Pain Management; Pain Measurement; Follow-Up Studies; Treatment Outcome; Chronic Pain; Inpatients; Depression; Catastrophization; Anxiety; Disability Evaluation
Pediatric chronic pain, which can result in deleterious effects for the child, bears the risk of aggravation into adulthood. Intensive interdisciplinary pain treatment (IIPT) might be an effective treatment, given the advantage of consulting with multiple professionals on a daily basis. Evidence for the effectiveness of IIPT is scarce. We investigated the efficacy of an IIPT within a randomized controlled trial by comparing an intervention group (IG) (n=52) to a waiting-list control group (WCG) (n=52). We made assessments before treatment (PRE), immediately after treatment (POST), as well as at short-term (POST6MONTHS) and long-term (POST12MONTHS) follow-up. We determined a combined endpoint, improvement (pain intensity, disability, school absence), and investigated 3 additional outcome domains (anxiety, depression, catastrophizing). We also investigated changes in economic parameters (health care use, parental work absenteeism, subjective financial burden) and their relationship to the child's improvement. Results at POST showed that significantly more children in the IG than in the WCG were assigned to improvement (55% compared to 14%; Fisher P<.001; 95% confidence interval for incidence difference: 0.21% to 0.60%). Although immediate effects were achieved for disability, school absence, depression, and catastrophizing, pain intensity and anxiety did not change until short-term follow-up. More than 60% of the children in both groups were improved long-term. The parents reported significant reductions in all economic parameters. The results from the present study support the efficacy of the IIPT. Future research is warranted to investigate differences in treatment response and to understand the changes in economic parameters in nonimproved children.
2014-01
Hechler T; Ruhe A-K; Schmidt P; Hirsch J; Wager J; Dobe M; Krummenauer F; Zernikow B
Pain
2014
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1016/j.pain.2013.09.015" target="_blank" rel="noreferrer">10.1016/j.pain.2013.09.015</a>
Causes and palliation of transfusion-associated vaginal bleeding in patients with metastatic cancer
adolescent; Female; Humans; retrospective studies; Young Adult; Palliative Care; Neoplasms; Adult; Aged; Middle Aged; Treatment Outcome; Neoplasm Metastasis; Uterine Hemorrhage
PURPOSE: The current study was undertaken (1) to capture a clinically relevant, systematically collected series of patients with metastatic cancer and transfusion-associated vaginal bleeding and (2) to provide insight into how best to palliate this bleeding. METHODS/RESULTS: As part of a single-institution review, 46 patients with metastatic cancer and transfusion-associated vaginal bleeding were identified. In a minority, 14 (30%), the cancer itself was directly responsible for the bleeding, and under these circumstances, gynecological cancer was the most frequent cause. In 13 patients (28%), more than 1 palliative intervention was attempted. Of all the interventions, a hysterectomy was performed most frequently and was successful in 11 patients. The use of ablation or embolization procedures was rarely tried but successful in 4 patients. However, 2 patients died of vaginal bleeding, despite multiple palliative procedures to control bleeding, including tumor embolization in one. CONCLUSIONS: Transfusion-associated vaginal bleeding in patients with metastatic cancer can arise from nonmalignant causes and often assumes an uneventful course but can, at times, be serious and difficult to control.
2014-01
Jackson AE; Stephens EK; Jatoi A
International Journal Of Gynecological Cancer
2014
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Journal Article
<a href="http://doi.org/10.1097/IGC.0000000000000017" target="_blank" rel="noreferrer">10.1097/IGC.0000000000000017</a>
Effectiveness of Music Intervention in Ameliorating Cancer Patients' Anxiety, Depression, Pain, and Fatigue: A Meta-analysis
Treatment Outcome; oncology; Music therapy
BACKGROUND:: This is the first study to use meta-analysis as a scientific technique to provide an integrated analysis of the effectiveness of music intervention in cancer patients. OBJECTIVES:: The purpose of this study was, using the meta-analysis method, to present a summary of existing research and explore the effectiveness of music intervention in ameliorating anxiety, depression, pain, and fatigue in cancer patients. METHODS:: The present study collected quantitative study designs sought of music intervention for cancer patients published from 2002 to 2012. These studies were then cross-referenced using Medical Subject Headings for topics on music intervention and cancer patients. Outcome indicators were anxiety, depression, pain, and fatigue. The quality of the studies was evaluated using Cochrane Collaboration Guidelines. The effect size on outcome indicators used the formula devised by Hedges and Olkin (1985). RESULTS:: Results showed that music interventions were significantly effective in ameliorating anxiety (g = -0.553), depression (g = -0.510), pain (g = -0.656), and fatigue (g = -0.422) in cancer patients. Subgroup analyses revealed that age and who selected the music were major factors influencing the effect size on anxiety reduction. CONCLUSIONS:: Music interventions significantly ameliorate anxiety, depression, pain, and fatigue in cancer patients, especially adults. Music interventions were more effective in adults than in children or adolescents and more effective when patients, rather than researchers, chose the music. IMPLICATIONS FOR PRACTICE:: Our findings provide important information for future music-intervention planners to improve the design and processes that will benefit patients in such programs.
2014-03
Tsai Hsiu F; Chen YR; Chung MH; Liao Yuan M; Chi MJ; Chang CC; Chou KR
Cancer Nursing
2014
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Journal Article
<a href="http://doi.org/10.1097/NCC.0000000000000116" target="_blank" rel="noreferrer">10.1097/NCC.0000000000000116</a>
Functional electrical stimulation therapy for recovery of reaching and grasping in severe chronic pediatric stroke patients
Child; Female; Humans; Male; Pediatrics; Chronic disease; Treatment Outcome; Severity of Illness Index; Disability Evaluation; Articular; Range of Motion; Recovery of Function; Chronic; Electric Stimulation Therapy; functional electrical stimulation therapy; grasping; Hand Strength; Psychomotor Performance; severe; Stroke; Upper Extremity
Stroke affects 2.7 children per 100,000 annually, leaving many of them with lifelong residual impairments despite intensive rehabilitation. In the present study the authors evaluated the effectiveness of 48 hours of transcutaneous functional electrical stimulation therapy for retraining voluntary reaching and grasping in 4 severe chronic pediatric stroke participants. Participants were assessed using the Rehabilitation Engineering Laboratory Hand Function Test, Quality of Upper Extremity Skills Test, Pediatric Evaluation of Disability Inventory, and Assisting Hand Assessment. All participants improved on all measures. The average change scores on selected Rehabilitation Engineering Laboratory Hand Function Test components were 14.5 for object manipulation (P = .042), 0.78 Nm for instrumented cylinder (P = .068), and 14 for wooden blocks (P = .068) and on the grasp component of Quality of Upper Extremity Skills Test was 25.93 (P = .068). These results provide preliminary evidence that functional electrical stimulation therapy has the potential to improve upper limb function in severe chronic pediatric stroke patients.
2014-04
Kapadia Naaz M; Nagai MK; Zivanovic V; Bernstein J; Woodhouse J; Rumney P; Popovic MR
Journal Of Child Neurology
2014
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Journal Article
<a href="http://doi.org/10.1177/0883073813484088" target="_blank" rel="noreferrer">10.1177/0883073813484088</a>
Change in parent-identified goals in young children with cerebral palsy receiving a context-focused intervention: associations with child, goal and intervention factors
Child; Female; Humans; Male; Treatment Outcome; Goals; Ontario; Cerebral Palsy; Disability Evaluation; Alberta; Preschool; Motor Skills; Physical Therapy Modalities
The purpose of this study was to examine the relationship between goal achievement measured by the Canadian Occupational Performance Measure (COPM) and child, goal, and intervention factors. Participants were 41 preschool children with cerebral palsy (CP) who were in the context-focused therapy arm of a randomized controlled trial. Factors including child age, Gross Motor Function Classification System (GMFCS) level, type and complexity of goals, and intervention strategies were analyzed. Children made large, positive mean changes on the COPM over 6 months (Performance = 3.8, SD = 1.9; Satisfaction = 4.3, SD 4.3) with younger children showing greater change. The COPM scores had low to moderate correlations with change on the Pediatric Evaluation of Disability Inventory and the Gross Motor Function Measure (GMFM-66). Regression analysis indicated that age, but not GMFCS level influenced COPM change scores. Goal complexity and intervention strategies were not significantly related to COPM change scores. The results provide support for using the COPM as an individualized measure of change in young children with CP receiving intervention.
2014-02
Pollock N; Sharma N; Christenson C; Law M; Gorter JW; Darrah J
Physical & Occupational Therapy In Pediatrics
2014
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Journal Article
<a href="http://doi.org/10.3109/01942638.2013.799627" target="_blank" rel="noreferrer">10.3109/01942638.2013.799627</a>
Improving the Care of Children With Advanced Cancer by Using an Electronic Patient-Reported Feedback Intervention: Results From the PediQUEST Randomized Controlled Trial
Child; Female; Humans; Male; Palliative Care; Neoplasms; Treatment Outcome; Electronic Health Records; Pilot Projects; Symptom Assessment; Feedback; Intervention Studies; Sickness Impact Profile; quality of life; Preschool
Purpose This study aimed to determine whether feeding back patient-reported outcomes (PROs) to providers and families of children with advanced cancer improves symptom distress and health-related quality of life (HRQoL). Patients and Methods This study was a parallel, multicentered pilot randomized controlled trial. At most once per week, children age ≥ 2 years old with advanced cancer or their parent completed the computer-based Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) survey consisting of age- and respondent-adapted versions of the Memorial Symptom Assessment Scale (MSAS), Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL4.0), and an overall Sickness question. In the intervention group (n = 51), oncologists and families received printed reports summarizing PROs; e-mails were sent to oncologists and subspecialists when predetermined scores were exceeded. No feedback was provided in the control group (n = 53). Primary outcomes included linear trends of MSAS, PedsQL4.0 total and subscale scores, and Sickness scores during 20 weeks of follow-up, along with child, parent, and provider satisfaction with PediQUEST feedback. Results Feedback did not significantly affect average MSAS, PedsQL4.0, or Sickness score trends. Post hoc subgroup analyses among children age ≥ 8 years who survived 20 weeks showed that feedback improved PedsQL4.0 emotional (+8.1; 95% CI, 1.8 to 14.4) and Sickness (−8.2; 95% CI, −14.2 to −2.2) scores. PediQUEST reports were valued by children, parents, and providers and contributed at least sometimes to physician initiation of a psychosocial consult (56%). Conclusion Although routine feedback of PROs did not significantly affect the child’s symptoms or HRQoL, changes were in expected directions and improvements observed in emotional HRQoL through exploratory analyses were encouraging. Importantly, children, parents, and providers value PRO feedback.
2014-03
Wolfe J; Orellana L; Cook EF; Ullrich CK; Kang TI; Geyer JR; Feudtner C; Weeks Jane C; Dussel V
Journal Of Clinical Oncology
2014
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Journal Article
<a href="http://doi.org/10.1200/JCO.2013.51.5981" target="_blank" rel="noreferrer">10.1200/JCO.2013.51.5981</a>
Neurogenetic disorders and treatment of associated seizures.
Humans; Treatment Outcome; Epilepsies; Angelman Syndrome/dt [Drug Therapy]; Anticonvulsants/tu [Therapeutic Use]; Epilepsies; Rett Syndrome/dt [Drug Therapy]; Seizures/dt [Drug Therapy]; Tuberous Sclerosis/dt [Drug Therapy]; Angelman Syndrome/co [Complications]; Anticonvulsants/ad [Administration & Dosage]; Anticonvulsants/ae [Adverse Effects]; KT Synthesis; Myoclonic/co [Complications]; Myoclonic/dt [Drug Therapy]; Rett Syndrome/co [Complications]; Seizures/co [Complications]; Tuberous Sclerosis/co [Complications]
Seizures are a frequent complication associated with several neurogenetic disorders. Antiepileptic medications remain the mainstay of treatment in these patients. We summarized the available data associated with various antiepileptic therapies used to treat patients with neurogenetic disorders who experienced recurrent seizures. A MEDLINE search was conducted to identify articles and abstracts describing the use of antiepileptic therapy for the treatment of various neurogenetic syndromes. Of all the neurogenetic syndromes, only autism spectrum disorders, Angelman syndrome, Rett syndrome, Dravet syndrome, and tuberous sclerosis complex were identified as having sufficient published information to evaluate therapy. Some efficacy trends were identified, including frequent successes with valproic acid with clonazepam for epilepsy with Angelman syndrome; valproic acid, stiripentol, and clobazam (triple combination therapy) for epilepsy with Dravet syndrome; and vigabatrin for infantile spasms associated with tuberous sclerosis complex. Due to a paucity of information regarding the mechanisms by which seizures are generated in the various disorders, approach to seizure control is primarily based on clinical experience and a limited amount of study data exploring patient outcomes. Although exposure of the developing brain to antiepileptic medications is of some concern, the control of epileptic activity is an important undertaking in these individuals, as the severity of eventual developmental delay often appears to correlate with the severity of seizures. As such, early aggressive therapy is warranted. 2013 Pharmacotherapy Publications, Inc.
2013
Faulkner MA; Singh Sanjay P
Pharmacotherapy
2013
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Journal Article
<a href="http://doi.org/10.1002/phar.1201" target="_blank" rel="noreferrer">10.1002/phar.1201</a>
GRADE guidelines: 15. Going from evidence to recommendation-determinants of a recommendation's direction and strength
Humans; United States; Canada; Practice Guidelines as Topic; Treatment Outcome; Research Design; Risk Assessment; Evidence-Based Medicine; Treatment Failure; Germany; Pulmonary Disease; Health Care; Quality Assurance; Chronic Obstructive
In the GRADE approach, the strength of a recommendation reflects the extent to which we can be confident that the composite desirable effects of a management strategy outweigh the composite undesirable effects. This article addresses GRADE's approach to determining the direction and strength of a recommendation. The GRADE describes the balance of desirable and undesirable outcomes of interest among alternative management strategies depending on four domains, namely estimates of effect for desirable and undesirable outcomes of interest, confidence in the estimates of effect, estimates of values and preferences, and resource use. Ultimately, guideline panels must use judgment in integrating these factors to make a strong or weak recommendation for or against an intervention.
2013-07
Andrews JC; Schunemann HJ; Oxman A; Pottie K; Meerpohl Joerg J; Coello PA; Rind D; Montori VM; Brito JP; Norris S; Elbarbary M; Post P; Nasser M; Shukla V; Jaeschke R; Brozek J; Djulbegovic B; Guyatt G
Journal Of Clinical Epidemiology
2013
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Journal Article
<a href="http://doi.org/10.1016/j.jclinepi.2013.02.003" target="_blank" rel="noreferrer">10.1016/j.jclinepi.2013.02.003</a>
Pulmonary metastasectomy in pediatric/adolescent patients with synovial sarcoma: an institutional review
adolescent; Child; Female; Humans; infant; retrospective studies; Survival Rate; Young Adult; Treatment Outcome; Lung Neoplasms; Preschool; Metastasectomy; Reoperation; Sarcoma; Synovial
PURPOSE: Synovial sarcoma (SS) often metastasizes to the lung; however, the indications for and outcomes of pulmonary metastasectomy have not been evaluated in pediatric/adolescent patients. METHODS: The records of pediatric patients (age <22years) with pathologically confirmed SS and pulmonary metastasis treated between June 1971 and May 2011 at our institution were retrospectively reviewed for the number and type of surgical metastasectomies, tumor characteristics, and survival outcomes. RESULTS: Forty-one patients (mean age: 15.9years) were identified and 31 (76%) underwent at least one metastasectomy. Seventy-two resections (range, 1-8/patient) were performed. Two- and 5-year survival rates after metastasis diagnosis were 65% and 24% for patients who underwent metastasectomy. Patients who did not undergo a metastasectomy survived no more than 2years from the diagnosis of pulmonary disease (P<0.001). Longer time to progression after primary tumor resection (>1year) and complete resection of pulmonary disease correlated with greater OS (P=0.02 and P<0.001, respectively). Palliative debulking did not improve OS. Survival was unaffected by tumor histological subtype, bilateral pulmonary disease, number of surgical resections, or number and size of resected metastatic lesions. CONCLUSION: Pulmonary metastasectomy may be associated with improved survival in pediatric/adolescent patients with SS and pulmonary metastases if complete resection is achieved.
2013-04
Stanelle EJ; Christison-Lagay ER; Wolden SL; Meyers PA; La Quaglia MP
Journal Of Pediatric Surgery
2013
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Journal Article
<a href="http://doi.org/10.1016/j.jpedsurg.2012.09.042" target="_blank" rel="noreferrer">10.1016/j.jpedsurg.2012.09.042</a>
Evaluation of the effect of a comprehensive multidisciplinary care pathway for hip fractures: design of a controlled study.
Humans; Middle Aged; Interdisciplinary Communication; Treatment Outcome; Netherlands; Program Evaluation; Activities of Daily Living; Time Factors; Academic Medical Centers; Hip Fractures/th [Therapy]; Recovery of Function; patient care team; Critical Pathways; Research Design; Hip Fractures/di [Diagnosis]; Hip Fractures/pp [Physiopathology]; Propensity Score
BACKGROUND: Hip fractures constitute an economic burden on healthcare resources. Most persons with a hip fracture undergo surgery. As morbidity and mortality rates are high, perioperative care leaves room for improvement. Improvement can be achieved if it is organized in comprehensive care pathways, but the effectiveness of these pathways is not yet clear. Hence the objective of this study is to compare the clinical effectiveness of a comprehensive care pathway with care as usual on self-reported limitations in Activities of Daily Living., METHODS/DESIGN: A controlled trial will be conducted in which the comprehensive care pathway of University Medical Center Groningen will be compared with care as usual in two other, nonacademic, hospitals. In this trial, propensity scores will be used to adjust for differences at baseline between the intervention and control group. Propensity scores can be used in intervention studies where a classical randomized controlled trial is not feasible. Patients aged 60 years and older will be included. The hypothesis is that 15% more patients at University Medical Center Groningen compared with patients in the care-as-usual condition will have recovered at least as well at 6 months follow-up to pre-fracture levels for Activities of Daily Living., DISCUSSION: This study will yield new knowledge with respect to the clinical effectiveness of a comprehensive care pathway for the treatment of hip fractures. This is relevant because of the growing incidence of hip fractures and the consequent massive burden on the healthcare system. Additionally, this study will contribute to the growing knowledge of the application of propensity scores, a relatively novel statistical technique to simulate a randomized controlled trial in studies where it is not possible or difficult to execute this kind of design., TRIAL REGISTRATION: Nederlands Trial Register NTR3171.
2013
Izaks GJ; Reininga Inge HF; Wendt KW; Stevens M
Bmc Musculoskeletal Disorders
2013
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Journal Article
<a href="http://doi.org/10.1186/1471-2474-14-291" target="_blank" rel="noreferrer">10.1186/1471-2474-14-291</a>
Percutaneous CT-guided cordotomy for the treatment of pediatric cancer pain
Child; Humans; Male; Palliative Care; Pain; hospice care; Treatment Outcome; Palliative Care; Tomography; X-Ray Computed; Intractable; Cordotomy; Groin; Lower Extremity; Medulloblastoma
Percutaneous cordotomy using CT guidance has been shown to be a safe and effective means of reducing pain in adults with cancer in 2 large case series. Its effectiveness in pediatric patients, however, has not been reported. Here, the authors present a case of CT-guided percutaneous cordotomy being used effectively for the treatment of unilateral limb pain in a 9-year-old boy suffering from metastatic medulloblastoma. The efficacy and minimally invasive nature of this procedure support its use in selected pediatric cases.
2013-07
Reddy GD; Okhuysen-Cawley R; Harsh V; Viswanathan A
Journal Of Neurosurgery. Pediatrics
2013
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Journal Article
<a href="http://doi.org/10.3171/2013.4.PEDS12474" target="_blank" rel="noreferrer">10.3171/2013.4.PEDS12474</a>
Effects of a palliative care intervention on clinical outcomes in patients with advanced cancer: the Project ENABLE II randomized controlled trial
Female; Humans; Male; Aged; Middle Aged; Treatment Outcome; Length of Stay; Patient Participation; Affect; Sickness Impact Profile; Patient Education as Topic; Emergency Service; quality of life; Hospital/utilization; Adaptation; Psychological; patient care team; Terminally Ill/psychology; social support; Health Services/utilization; Hospitalization/statistics & numerical data; Intensive Care Units/utilization; Intervention; Interventions; Kaplan-Meiers Estimate; Neoplasms/mortality/nursing/psychology/therapy; Outcomes; Palliative Care/methods
CONTEXT: There are few randomized controlled trials on the effectiveness of palliative care interventions to improve the care of patients with advanced cancer. OBJECTIVE: To determine the effect of a nursing-led intervention on quality of life, symptom intensity, mood, and resource use in patients with advanced cancer. DESIGN, SETTING, AND PARTICIPANTS: Randomized controlled trial conducted from November 2003 through May 2008 of 322 patients with advanced cancer in a rural, National Cancer Institute-designated comprehensive cancer center in New Hampshire and affiliated outreach clinics and a VA medical center in Vermont. INTERVENTIONS: A multicomponent, psychoeducational intervention (Project ENABLE [Educate, Nurture, Advise, Before Life Ends]) conducted by advanced practice nurses consisting of 4 weekly educational sessions and monthly follow-up sessions until death or study completion (n = 161) vs usual care (n = 161). MAIN OUTCOME MEASURES: Quality of life was measured by the Functional Assessment of Chronic Illness Therapy for Palliative Care (score range, 0-184). Symptom intensity was measured by the Edmonton Symptom Assessment Scale (score range, 0-900). Mood was measured by the Center for Epidemiological Studies Depression Scale (range, 0-60). These measures were assessed at baseline, 1 month, and every 3 months until death or study completion. Intensity of service was measured as the number of days in the hospital and in the intensive care unit (ICU) and the number of emergency department visits recorded in the electronic medical record. RESULTS: A total of 322 participants with cancer of the gastrointestinal tract (41%; 67 in the usual care group vs 66 in the intervention group), lung (36%; 58 vs 59), genitourinary tract (12%; 20 vs 19), and breast (10%; 16 vs 17) were randomized. The estimated treatment effects (intervention minus usual care) for all participants were a mean (SE) of 4.6 (2) for quality of life (P = .02), -27.8 (15) for symptom intensity (P = .06), and -1.8 (0.81) for depressed mood (P = .02). The estimated treatment effects in participants who died during the study were a mean (SE) of 8.6 (3.6) for quality of life (P = .02), -24.2 (20.5) for symptom intensity (P = .24), and -2.7 (1.2) for depressed mood (P = .03). Intensity of service did not differ between the 2 groups. CONCLUSION: Compared with participants receiving usual oncology care, those receiving a nurse-led, palliative care-focused intervention addressing physical, psychosocial, and care coordination provided concurrently with oncology care had higher scores for quality of life and mood, but did not have improvements in symptom intensity scores or reduced days in the hospital or ICU or emergency department visits. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00253383.
2009
Bakitas M; Lyons KD; Hegel MT; Balan S; Brokaw FC; Seville J; Hull JG; Li Z; Tosteson TD; Byock IR; Ahles TA
Jama
2009
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Journal Article
<a href="http://doi.org/10.1001/jama.2009.1198" target="_blank" rel="noreferrer">10.1001/jama.2009.1198</a>
Behavioral, social, and educational outcomes after pediatric stem cell transplantation and related factors
adolescent; Child; Female; Humans; Male; mothers; Neoplasms; Adult; Parent-Child Relations; Treatment Outcome; Longitudinal Studies; Social Adjustment; Behavior; Mental Health; Depression; Internal-External Control; Survivors; Teaching; Adaptation; Psychological; Cranial Irradiation; Social Behavior; Stem Cell Transplantation
BACKGROUND: The purpose of the current study was to investigate longitudinally children's behavioral and social competence outcomes up to 2 years after pediatric stem cell transplantation (SCT) and related factors. METHODS: Ninety-nine mothers and 24 youths completed standardized questionnaires (Child Behavior Checklist [CBCL] and Youth Self-Report [YSR]) pre-SCT, and 12 and 24 months after SCT; 26 teachers completed Teacher Report Form (TRF) at 24 months. Information regarding clinical (eg, diagnosis, cranial radiation, graft vs host disease [GVHD]), child (eg, age, sex, physical health), and familial (eg, maternal age, education, distress) factors was also obtained. RESULTS: Linear mixed regression models with compound covariance structure followed by adjusted pairwise analyses yielded significant improvements from pre-SCT to 1 and 2 years after SCT in total scores; in externalizing and internalizing scores from pre-SCT to 2 years after SCT; and in total competence from pre-SCT to 1 year after SCT. Child's physical health, maternal age, and depression were found to be significantly associated with the total, internalizing, and externalizing CBCL scores, whereas GVHD, mother's age, and time since diagnosis were associated with CBCL competence scores. Diagnosis, cranial radiation, GVHD, child's physical health, and maternal age and education were associated with YSR total behavioral and competence scores. Finally, total TRF scores were associated with time since diagnosis; TRF educational and adaptability scores were associated with maternal education, age, and distress. CONCLUSIONS: Clinical, personal, and familial factors must be considered to understand the psychosocial outcomes of these survivors up to 2 years after SCT. This study has important implications for psychosocial interventions for this population.
2009-02
Barrera M; Atenafu E; Pinto J
Cancer
2009
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Journal Article
<a href="http://doi.org/10.1002/cncr.24109" target="_blank" rel="noreferrer">10.1002/cncr.24109</a>
Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy
Child; Humans; Male; Survival Rate; Treatment Outcome; Cognition; Predictive Value of Tests; Motor Activity; Preschool; Outcomes; Adrenoleukodystrophy/complications/mortality/physiopathology/therapy; Cord Blood Stem Cell Transplantation/methods; Graft Survival; Graft vs Host Disease; Histocompatibility Testing; Language Development; Myeloablative Agonists/therapeutic use; Neurophysiology; Transplantation Conditioning/methods
Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marrow transplantation has been used to halt progression of the disease. However, many patients lack suitable HLA- matched related donors and must rely on unmatched donors for a source of stem cells. The purpose of this study was to evaluate outcomes of unrelated donor umbilical cord blood transplantation after chemotherapy-based myeloablative conditioning and retrospectively determine if baseline studies correlate and help predict outcome. Between November 22, 1996, and November 3, 2005, 12 boys with X-linked ALD who lacked HL- matched related donors were referred to Duke University Medical Center for transplantation. These children were conditioned with myeloablative therapy including busulfan, cyclophosphamide, and antithymocyte globulin before receiving umbilical cord-blood transplants from unrelated donors. Baseline studies of neurophysiologic, neuroimaging, and neurodevelopmental status were performed and patients were subsequently evaluated for survival, engraftment, graft-versus-host disease, and neurodevelopmental outcomes. A substudy evaluated whether baseline neuroimaging and neurophysiologic studies correlated with cognitive and motor function and if these studies were predictive of posttransplantation outcomes. The umbilical cord blood grafts had normal levels of very long chain fatty acids. They delivered a median of 6.98 x 10(7) nucleated cells per kilogram of recipient body weight and were discordant for up to 4 of 6 HLA markers. Neutrophil engraftment occurred at a median of 22.9 days after transplantation. Three patients had grade II-IV acute graft-versus-host disease; 2 had extensive chronic graft-versus-host disease. Cumulative incidence of overall survival of the group at 6 months is 66.7% (95% confidence interval 39.9-93.3%). Median follow-up was 3.3 years (range 12 days to 6.3 years). As previously reported with bone marrow transplantation, symptomatic patients faired poorly with lower survival and rapid deterioration of neurologic function. This study included 3 patients transplanted at a very young age (2.6-3.5 years) before the onset of clinical symptoms who continue to develop at a normal rate for 3-5 years posttransplant. Although baseline Loes scores correlated with cognitive and motor outcome, neurophysiologic studies failed to show statistically significant differences. Transplantation of boys with X-linked ALD using partial HLA-matched umbilical cord blood yields similar results to those previously reported after bone marrow transplantation. Superior outcomes were seen in neurologically asymptomatic boys less than 3.5 years of age at the time of transplantation. Baseline Loes scores were a strong predictor of cognitive and motor outcome.
2007
Beam D; Poe MD; Provenzale JM; Szabolcs P; Martin PL; Prasad V; Parikh S; Driscoll T; Mukundan S; Kurtzberg J; Escolar ML
Biology Of Blood And Marrow Transplantation : Journal Of The American Society For Blood And Marrow Transplantation
2007
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Journal Article
<a href="http://doi.org/10.1016/j.bbmt.2007.01.082" target="_blank" rel="noreferrer">10.1016/j.bbmt.2007.01.082</a>
Implementation of transition programs can prevent another lost generation of patients with congenital heart disease
Child; Humans; Adult; Health Services Needs and Demand; Treatment Outcome; Program Development; Longevity; Morbidity; adolescent; Adolescent Transitions; Heart Defects; Continuity of Patient Care/organization & administration; Aftercare/organization & administration; Congenital/complications/epidemiology/prevention & control; Patient Education as Topic/organization & administration
Congenital heart disease is the most frequently occurring birth defect. To date, more than 90% of the children born with a heart defect reach adulthood. Since many patients are prone to residua and sequelae, lifelong specialized care is required. However, studies indicate that about one-half to three-quarters of the patients are lost to follow-up when they have grown up. This has resulted in a virtual lost generation. Lapse of care is associated with significant morbidity. Therefore, implementation of strategies to prevent patients from failing to continue regular follow-up is critical. It is argued that transition programs that inform patients about the rationale for ongoing follow-up and that teach them how to navigate the medical system can avoid another lost generation.
2008
Moons P; Hilderson D; Van Deyk K
European Journal Of Cardiovascular Nursing
2008
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Journal Article
<a href="http://doi.org/10.1016/j.ejcnurse.2008.10.001" target="_blank" rel="noreferrer">10.1016/j.ejcnurse.2008.10.001</a>
Does palliative care improve quality? A survey of bereaved family members
Female; Humans; Male; Terminal Care; Family; Adult; Data Collection; Aged; Middle Aged; Treatment Outcome; New York; adolescent; 80 and over; bereavement; Palliative Care/statistics & numerical data; Patient Satisfaction/statistics & numerical data; Health Care; Quality Assurance; Pain/epidemiology/nursing
Palliative care is the interdisciplinary specialty that aims to relieve suffering and improve the quality of care for patients with serious illness and their families. Although palliative care programs are becoming increasingly prevalent in U.S. hospitals, the impact of hospital palliative care consultation programs on the quality of care received by family members is not well understood. We conducted prospective quantitative telephonic interviews of family members of patients who died at Mount Sinai Medical Center between April and December 2005 using the validated "After-Death Bereaved Family Member Interview," to assess quality of medical care at the end of life. Multivariable techniques were used to compare family satisfaction of palliative care patients vs. usual care patients controlling for age, race (white vs. nonwhite), diagnosis (cancer vs. noncancer), socioeconomic status (Medicaid vs. non-Medicaid), and functional status (number of dependent activities of daily living). One hundred ninety eligible subjects were contacted and successful interviews were completed with 149 (78.4%) family members (54 palliative care and 95 usual care patients). Palliative care showed benefit, with 65% of palliative care patients' family members reporting that their emotional or spiritual needs were met, as compared to 35% of usual care patients' family members (P=0.004). Sixty-seven percent of palliative care patients' family members reported confidence in one or more self-efficacy domains, as compared to 44% of usual care patients' family members (P=0.03). Our study shows that palliative care consultation is associated with improved satisfaction, with attention to family and enhanced self-efficacy. Palliative care offers a unique approach by integrating the needs of the family into the care of the patient.
2008
Gelfman LP; Meier DE; Morrison RS
Journal Of Pain And Symptom Management
2008
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Journal Article
<a href="http://doi.org/10.1016/j.jpainsymman.2007.09.008" target="_blank" rel="noreferrer">10.1016/j.jpainsymman.2007.09.008</a>
Outcomes and causes of death in children on home mechanical ventilation via tracheostomy: an institutional and literature review
Child; Humans; Cohort Studies; home care services; Positive-Pressure Respiration; Tracheostomy; Treatment Outcome; adolescent; Preschool; infant; retrospective studies; cause of death; Hospital-Based
OBJECTIVE: To describe outcomes and causes of death in children on chronic positive-pressure ventilation via tracheostomy. STUDY DESIGN: We conducted a retrospective observational cohort analysis of 228 children enrolled in an university-affiliated home mechanical ventilation (HMV) program over 22 years (990 person-years). Cumulative incidences of survival and liberation from HMV are presented. Time-to-events were compared by reason for chronic respiratory failure (CRF) and age and date of HMV initiation with Kaplan-Meier and Cox regression analyses. Circumstances of death are described. RESULTS: Of our cohort, 47 of 228 children died, and 41 children were liberated from HMV. The 5-year cumulative incidences of survival and liberation were 80% and 24%, respectively. Being placed on HMV for chronic pulmonary disease was independently associated with liberation from HMV (hazard ratio, 7.38; 95% CI, 3.0-18.2; P < .001). Neither age nor reasons for CRF were associated with shortened survival. Progression of underlying condition accounted for only 34% of deaths; 49% of deaths were unexpected. CONCLUSION: Most children on HMV survive or were weaned off. However, a sizable number of children in our cohort died, and many deaths were unexpected and from causes not directly related to their primary reason for CRF.
2010
Edwards JD; Kun SS; Keens TG
The Journal Of Pediatrics
2010
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Journal Article
<a href="http://doi.org/10.1016/j.jpeds.2010.06.012" target="_blank" rel="noreferrer">10.1016/j.jpeds.2010.06.012</a>