Provider Perceptions for Withdrawing Life Sustaining Therapies at a Large Pediatric Hospital
child; human; female; male; article; perception; software; qualitative; health; hospital; life; analysis; therapy; practice; treatment; quality; study; erratum; clinical; withdrawal; drug; pediatric; neonatal; experiment; palliative; care; intensive; major; unit; sustaining; quantitative; guideline; coronary; data
Context: More than 74% of pediatric deaths occur in an intensive care unit (ICU), with 40% occurring after withdrawal of life-sustaining therapies (WOLST). No needs assessment has described provider needs or suggestions for improving the WOLST process in pediatrics.
Sawyer K E; Carpenter AT; Coleman RD; Tume SC; Crawford CA; Casas JA
Journal of Pain and Symptom Management
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/10.1016/j.jpainsymman.2022.05.009" target="_blank" rel="noreferrer noopener">10.1016/j.jpainsymman.2022.05.009</a>
Decision-making experiences of health professionals in withdrawing treatment for children and young people: A qualitative study
decision making; healthcare; pediatric; professionals; qualitative study; treatment; withdrawing treatment
OBJECTIVE: To explore factors that influence professionals in deciding whether to withdraw treatment from a child and how decision-making is managed amongst professionals as an individual and as a team. STUDY DESIGN: Semi-structured interviews were conducted with a purposive sample of health professionals working at a UK Children's Hospital, with children with life-limiting illnesses whose treatment has been withdrawn. Data was transcribed verbatim, anonymised, and analysed using a thematic framework method. RESULT(S): A total of fifteen participants were interviewed. Five interrelated themes with associated subthemes were generated to help understand the experiences of health professionals in decision-making on withdrawing a child's treatment: 1) Understanding the Child's Best Interests (2) Multidisciplinary Approach (3) External Factors (4) Psychological Wellbeing (5) Recommendations to Support Shared Decision-making. CONCLUSION(S): A shared decision-making approach should be adopted to support professionals, children, and their families to make decisions collectively. Copyright This article is protected by copyright. All rights reserved.
Abdin S; Heath G; Neilson S; Byron-Daniel J; Hooper N
Child: care, health and development.
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/10.1111/cch.12956" target="_blank" rel="noreferrer noopener">10.1111/cch.12956</a>
Cannabis Use in Children With Pantothenate Kinase-Associated Neurodegeneration
children; developmental disability; dystonia; pediatric; treatment
BACKGROUND: Pantothenate kinase-associated neurodegeneration is characterized by severe, progressive dystonia. This study aims to describe the reported usage of cannabis products among children with pantothenate kinase-associated neurodegeneration. METHODS: A cross-sectional, 37-item survey was distributed in April 2019 to the families of 44 children who participate in a clinical registry of individuals with pantothenate kinase-associated neurodegeneration. RESULTS: We received 18 responses (40.9% response rate). Children were a mean of 11.0 (SD 4.3) years old. The 15 respondents with dystonia or spasticity were on a median of 2 tone medications (range 0-9). Seven children had ever used cannabis (38.9%). The most common source of information about cannabis was other parents. Children who had ever used cannabis were on more tone medications, were more likely to have used opiates, were less likely to be able to roll, and less likely to sit comfortably, than children who had never used cannabis. Four children reported moderate or significant improvement in dystonia with cannabis. Other areas reported to be moderate or significantly improved were pain (n = 3), sleep (n = 4), anxiety (n = 3), and behavior (n = 2). Adverse effects included sadness (n = 1), agitation/behavior change (n = 1), and tiredness (n = 1). CONCLUSION: Cannabis use was commonly reported among children with pantothenate kinase-associated neurodegeneration whose parents responded to a survey, particularly when many other dystonia treatments had been tried. Physicians should be aware that parents may treat their child with severe, painful dystonia with cannabis. Placebo-controlled studies of products containing cannabidiol and 9-tetrahydrocannabinol are needed for pediatric tone disorders.
Wilson J L; Gregory A; Wakeman K; Freed A; Rai P; Roberts C; Hayflick S J; Hogarth P
Journal of Child Neurology
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/10.1177/0883073819890516" target="_blank" rel="noreferrer noopener">10.1177/0883073819890516</a>
Deep Brain Stimulation in Rare Inherited Dystonias
adolescent; clinical assessment; disease duration; time to treatment; dystonic disorder/su [Surgery]; priority journal; follow up; school child; outcome assessment; Dystonia; clinical effectiveness; gabapentin/dt [Drug Therapy]; human; article; child; adult; clinical article; aged; surgery; middle aged; disease severity; dystonia; phenotype; rating scale; ataxia telangiectasia/di [Diagnosis]; ataxia telangiectasia/dt [Drug Therapy]; atypical dopa responsive dystonia/di [Diagnosis]; atypical dopa responsive dystonia/dt [Drug Therapy]; baclofen/dt [Drug Therapy]; benzodiazepine derivative/dt [Drug Therapy]; brain depth stimulation; Burke Fahn Marsden Dystonia Rating Scale; cerebellar ataxia/di [Diagnosis]; cerebellar ataxia/dt [Drug Therapy]; chorea/di [Diagnosis]; chorea/dt [Drug Therapy]; clobazam/dt [Drug Therapy]; clonazepam/dt [Drug Therapy]; Deep brain stimulation; diazepam/dt [Drug Therapy]; dystonia/di [Diagnosis]; dystonia/dt [Drug Therapy]; dystonic disorder/th [Therapy]; entacapone/cb [Drug Combination]; entacapone/dt [Drug Therapy]; escitalopram/dt [Drug Therapy]; extrapyramidal syndrome/di [Diagnosis]; extrapyramidal syndrome/dt [Drug Therapy]; haloperidol/dt [Drug Therapy]; Inherited dystonia; levodopa/dt [Drug Therapy]; lorazepam/dt [Drug Therapy]; methylmalonic aciduria/di [Diagnosis]; methylmalonic aciduria/dt [Drug Therapy]; mirtazapine/dt [Drug Therapy]; motor dysfunction assessment; nemaline myopathy/di [Diagnosis]; nemaline myopathy/dt [Drug Therapy]; neuronal ceroid lipofuscinosis/di [Diagnosis]; neuronal ceroid lipofuscinosis/dt [Drug Therapy]; olanzapine/dt [Drug Therapy]; pramipexole/cb [Drug Combination]; pramipexole/dt [Drug Therapy]; preoperative care; risperidone/dt [Drug Therapy]; selegiline/cb [Drug Combination]; selegiline/dt [Drug Therapy]; tetrabenazine/dt [Drug Therapy]; therapy effect; tizanidine/dt [Drug Therapy]; trazodone/dt [Drug Therapy]; Treatment; trihexyphenidyl/cb [Drug Combination]; trihexyphenidyl/dt [Drug Therapy]; trisomy/di [Diagnosis]; trisomy/dt [Drug Therapy]; Wilson disease/di [Diagnosis]; Wilson disease/dt [Drug Therapy]; woodhouse sakati syndrome/di [Diagnosis]; woodhouse sakati syndrome/dt [Drug Therapy]; x trisomy/di [Diagnosis]; x trisomy/dt [Drug Therapy]; tone and motor problems; ataxia telangiectasia; MCM deficiency; NCL; Nemaline myopathy; surgical intervention; Deep Brain Stimulation
Background Rare causes of inherited movement disorders often present with a debilitating phenotype of dystonia, sometimes combined with parkinsonism and other neurological signs. Since these disorders are often resistant to medications, DBS may be considered as a possible treatment. Methods Patients with identified genetic diseases (ataxia-telangiectasia, chorea-achantocytosis, dopa-responsive dystonia, congenital nemaline myopathy, methylmalonic aciduria, neuronal ceroid lipofuscinosis, spinocerebellar ataxia types 2 and 3, Wilson's disease, Woodhouse-Sakati syndrome, methylmalonic aciduria, and X trisomy) and disabling dystonia underwent bilateral GPi DBS (bilateral thalamic Vim nucleus in 1 case). The primary outcome was the difference in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) between baseline, 1 year and last available follow-up. Preoperative factors such as age at surgery, disease duration at surgery, proportion of life lived with dystonia and severity of dystonia were correlated to the primary outcome. Results Eleven patients were operated between February 2003 and December 2013. Age and duration of disease at time of surgery were 30+/-19 and 12.5+/-15.7 years, respectively. DBS effects on dystonia severity were variable but overall marginally effective, with a mean improvement of 7.9% (p=0.39) at 1-year follow-up and 16.7% (p=0.46) at last follow-up (mean 47.3+/-19.9 months after surgery). No preoperative factors were identified to predict the surgical outcome. Conclusion Our findings support the current knowledge that DBS is modestly effective in treating rare inherited dystonias with a combined phenotype. However, the BFMDRS might not be the best tool to measure outcome in these severely affected patients. Copyright © 2016 Elsevier Inc.
Beaulieu-Boire I; Aquino C C; Fasano A; Poon Y Y; Fallis M; Lang A E; Hodaie M; Kalia S K; Lozano A; Moro E
Brain Stimulation
2016
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.brs.2016.07.009" target="_blank" rel="noreferrer noopener">10.1016/j.brs.2016.07.009</a>
Quantitative Evaluation of the Clinical Efficacy of Attention Bias Modification Treatment for Anxiety Disorders
Anxiety Disorders; Cognition; Confidence Intervals; Depression; Anxiety; Psychology; Children; Clinical Trials; computer; Anxieties; Anxiety Disorders - therapy; COGNITIVE-BEHAVIORAL THERAPY; PSYCHIATRY; Psychology; THREAT; INDIVIDUALS; Clinical; GENERALIZED SOCIAL PHOBIA; Internet technology; Meta-Analysis; MODIFICATION PROGRAM; RANDOMIZED CONTROLLED-TRIAL; STIMULI; treatment
Attention bias modification treatment (ABMT) is a novel treatment for anxiety disorders. Although a number of other meta-analytic reviews exist, the purpose of the present meta-analysis is to examine issues unaddressed in prior reviews. Specifically, the review estimates the efficacy of ABMT in clinically anxious patients and examines the effect of delivery context (clinic vs. home) on symptom reduction. A literature search using PsychInfo and Web of Science databases was performed. Only randomized controlled trials (RCTs) examining dot-probe-based ABMT in clinically diagnosed anxious patients were included. From 714 articles located through the search, 36 ABMT studies were identified and 11 studies met inclusion criteria (N = 589 patients). ABMT was associated with greater clinician-rated reductions in anxiety symptoms relative to control training: between-groups effect (d = 0.42, P = .001, confidence interval (CI) = 0.18-0.66), contrast of within-group effects (Q = 7.25, P < .01). More patients in the treatment group no longer met formal diagnostic criteria for their anxiety disorder posttreatment relative to patients in the control condition (P < .05). Analyses of patients' self-reported anxiety were nonsignificant for the between-groups contrast (P = .35), and were at a trend level of significance for the contrast between the within-group effects (P = .06). Moderation analysis of the between-groups effect revealed a significant effect for ABMT delivered in the clinic (d = 0.34, P = 0.01, CI = 0.07-0.62), and a nonsignificant effect for ABMT delivered at home (d = -0.10, P = 0.40, CI = -0.33-0.13). The current meta-analysis provides support for ABMT as a novel evidenced-based treatment for anxiety disorders. Overall, ABMT effects are mainly evident when it is delivered in the clinic and when clinical outcome is evaluated by a clinician. More RCTs of ABMT in specific anxiety disorders are warranted.
2015-06
Linetzky M; Pergamin-HL; Pine DS; Bar‐Haim Yair
Depression And Anxiety
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).
Journal Article
<a href="http://doi.org/10.1002/da.22344" target="_blank" rel="noreferrer">10.1002/da.22344</a>
Mucopolysaccharidosis type I, II and VI and response to enzyme replacement therapy: Results from a single-center case series study
Glycosaminoglycans; Lysosomal Storage Disorders; Prognosis; Treatment
Mucopolysaccharidoses (MPS) types I, II and VI are associated with deficiencies in alpha-L-iduronidase, iduronate-2-sulfatase and N-acetylgalactosamine-4-sulfatase, respectively, and generally involve progressive and multi-systemic clinical manifestations. Enzyme replacement therapy (ERT) appears to be reasonably well tolerated. The aim of this study was to examine clinical and diagnostic findings of a series of pediatric and adult MPS patients, and assess the safety and efficacy of ERT in children and adults with MPS type I, II and VI. Pediatric and adult patients were treated weekly with 1 mg/kg recombinant human N-acetylgalactosamine-4-sulphatase (rhASB), 0.45 mg/kg alpha-L-iduronidase, or 0.5 mg/kg iduronate-2-sulfatase. Clinical and biochemical parameters with ERT were evaluated for a mean duration of 5 years. Mantel-Haenszel risk ratios and associated 95% confidence intervals (CIs) were calculated for rates of death among different types of enzyme replacement therapies (ERTs). Twenty-seven patients (mean ages pediatric: 6.8 years; adult: 29 years) were included. ERT was found to be consistently well tolerated and effective in attenuating symptoms, but did not prevent the progression of the disease or reduce mortality rates. Our findings demonstrated that early diagnosis and initiation of ERT are critical for improvements in patient-important outcomes and quality of life, although disease progression and mortality rates remain high.
Franco Jfds; El Dib R; Agarwal A; Soares D; Milhan NVM; Albano LMJ; Kim CA
Intractable Rare Dis Res
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).
10.5582/irdr.2017.01036