Home mechanical ventilation for children with severe neurological impairment: Parents' perspectives on clinician counselling
children; home mechanical ventilation; parent perspectives; severe neurological impairment
AIM: To retrospectively explore the perspectives of parents of children with severe neurological impairment (SNI), such as those with severe cerebral palsy, epilepsy syndromes, and structural brain differences, on clinician counseling regarding home mechanical ventilation (HMV). METHOD: Inductive thematic analysis was performed on data from telephone interviews with parents who chose for and against HMV for their child with SNI at three academic children's hospitals across the USA. RESULTS: Twenty-six parents/legal guardians of 24 children were interviewed. Fourteen children had static encephalopathy, 11 received HMV, and 20 were alive at the time of parent interviews. Themes included how HMV related to the child's prognosis, risk of death, and integration with goals of care. Although clinicians voiced uncertainty about how HMV would impact their child, parents felt this was coupled with prescriptive/intimidating examples about the child's end of life and judgments about the child's quality of life. INTERPRETATION: While prognositc uncertainty exists, this study suggests that parents of children with SNI seek clinician counseling about HMV that considers their goals of care and views on their child's quality of life.
Bogetz JF; Munjapara V; Henderson CM; Raisanen JC; Jabre NA; Shipman KJ; Wilfond BS; Boss RD
Developmental Medicine & Child Neurology
2022
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<a href="http://doi.org/10.1111/dmcn.15151" target="_blank" rel="noreferrer noopener">10.1111/dmcn.15151</a>
Viral gene therapy for paediatric neurological diseases: progress to clinical reality
clinical; life-limiting; neurological diseases; paediatric; viral gene therapy
In the era of genomic medicine, diagnoses of rare paediatric neurological diseases are increasing. Many are untreatable and life-limiting, leading to an exceptional increase in gene therapy development. It is estimated that 20 gene therapy products will have received approval from the US Food and Drug Administration by 2025. With viral gene therapy considered a potential single-dose cure for patients with spinal muscular atrophy type 1 as one example, and contemporaneously tragically resulting in the deaths of three male children with X-linked myotubular myopathy receiving high-dose gene therapy in 2020, what is the current state of gene therapy? What is behind the decades of hype around viral gene therapy and is it high impact, but high risk? In this review, we outline principles of viral gene therapy development and summarize the most recent clinical evidence for the therapeutic effect of gene therapy in paediatric neurological diseases. We discuss adeno-associated virus and lentiviral vectors, antisense oligonucleotides, emerging genetic editing approaches, and current limitations that the field still faces.
Privolizzi R; Chu WS; Tijani M; Ng J
Developmental Medicine & Child Neurology
2021
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<a href="http://doi.org/10.1111/dmcn.14885" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14885</a>
Terminology and classification of cerebral palsy
Child; Cerebral Palsy; Terminology; Classification
1964
Bax M
Developmental Medicine & Child Neurology
1964
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/10.1111/j.1469-8749.1964.tb10791.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.1964.tb10791.x</a>
Functional Evaluation of Eating Difficulties Scale to predict oral motor skills in infants with neurodevelopmental disorders: a longitudinal study
eating difficulties; functional evaluation; infants; longitudinal study; neurodevelopmental disabilities; neurodevelopmental disorders; neurodevelopmental impairment; oral motor skills
Aim To assess the predictive validity of the Functional Evaluation of Eating Difficulties Scale (FEEDS) on long-term eating developmental outcomes in infants with neurodevelopmental disorders. Method In total, 144 infants (69 females, 75 males) aged 0 to 12 months (mean [SD] 5.34mo [3.42]) with neurodevelopmental disorders and requiring enteral nutrition support, hospitalized between January 2004 and December 2017, were included. The FEEDS was administered at the onset of hospitalization. Follow-up evaluations of feeding modalities occurred at discharge and at 6 months, 12 months, and 24 months after discharge. FEEDS score was tested as a predictor of infants’ feeding modality (percutaneous endoscopic gastrostomy, nasogastric tube, mixed, oral feeding) and time to autonomous oral feeding. Percentages of false-positive and negative cases were checked. Results Lower FEEDS scores significantly predicted infants’ feeding modality (0.40≤R2≤0.61). A 1-point increase in FEEDS score was associated with increased risk (6%–14%; p<0.05) of being non-autonomous feeders at the different follow-up points in infants who had a FEEDS score above the clinical cut-off. Interpretation The FEEDS appears to be a clinically valid assessment to predict the presence of eating difficulties in infants with neurodevelopmental disabilities. What this paper adds Functional Evaluation of Eating Difficulties Scale (FEEDS) significantly predicted eating difficulties in infants with neurodevelopmental disabilities. Lower FEEDS score is significantly associated with autonomous feeding at the 24-month follow-up. FEEDS cut-off identified infants at low-risk and high-risk for eating disorder.
Cavallini A; Provenzi L; Minico GSD; Sacchi D; Gavazzi L; Amorelli V; Borgatti R
Developmental Medicine & Child Neurology
2019
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<a href="http://doi.org/10.1111/dmcn.14154" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14154</a>
Children and Adolescents with Cancer Assisted in a Support Institution in Juiz de Fora, Minas Gerais, Brazil - Preliminary Study
Low resource setting; oncology
Introduction: The most frequent tumors in childhood are leukemias, central nervous system tumors, lymphomas, and neuroblastoma. According to Brazilian National Institute of Cancer (INCA), these patients should maintain follow-up with a multidisciplinary team for early recognition and appropriate care of possible late complications. Patients and Methods: Children and adolescents, from 3 to 21 years of age, diagnosed with cancer, in outpatient follow-up, supported by the Ricardo Moyses Junior Cancer Foundation (Juiz de Fora, Minas Gerais, Brazil) were invited to participate. These are preliminary results of a cross-sectional observational study, approved by the University's ethics committee (opinion: 28699920.2.0000.5147). The data collections were performed remotely (WhatsApp), with personal and environmental information and application of physical activity level instruments. Descriptive Frequency Analysis was performed for data analysis. Result(s): Nineteen participants participated in this preliminary study. Of these, 57.9% are female; mean age 11.37 years (5.84). The diagnosese were: 36.8% acute lymphoid leukemia, 10.5% chronic myeloid leukemia, 21.1% Hodgkin lymphoma, 5.3% neuroblastoma; 52.6% of the participants were in intermediate phase of treatment, 42.1% in control phase and 5.3% in palliative care; 36.8% of the participants in chemotherapy treatment; 26.3% chemotherapy and radiotherapy and 10.5% surgical approach. The participants were mostly physically inactive (78.9%). Regarding the follow-up with professionals from the multidisciplinary team, 57.9% had nutritional follow-up and 42.1% received physiotherapy. Conclusion(s): The study suggests that the prevalence of cancer in the assisted population agrees with INCA estimates. More attention and care are needed regarding nutritional care of the participants and guidelines for physical inactivity.
Chagas P; Marques R; Goretti P; Castilho J; El'corab B; Costa M; Taroco AP; Oliveira I; Costa H; Fronio J
Developmental Medicine and Child Neurology
2021
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<a href="http://doi.org/Issues%20in%20adolescent%20and%20young%20adult%20oncology%20in%20a%20developing%20country%20like%20India" target="_blank" rel="noreferrer noopener">Issues in adolescent and young adult oncology in a developing country like India</a>
Pain coping strategies in children with cerebral palsy
AIM: To describe coping strategies in children and adolescents with cerebral palsy (CP), relative to age. METHOD: Patients were prospectively recruited from two paediatric rehabilitation centres in France. The Pediatric Pain Coping Inventory - French and Structured Pain Questionnaire were completed by an experienced professional for each child. RESULTS: One hundred and forty-two children with CP were included (80 males, 62 females; median age 12y; IQR=8-15y). They generally used fewer coping strategies than typically developing children ('Seeks social support and action': 12.47 vs 12.85, p=0.477; 'Cognitive self-instruction': 9.28 vs 10.90, p<0.001; 'Distraction': 4.89 vs 7.00, p<0.001; 'Problem solving': 4.43 vs 5.19, p<0.001). In the CP group, 'Seeks social support and action' decreased with age (p=0.021) and 'Cognitive self-instruction' increased with age (p<0.001). 'Problem solving' and 'Distraction' did not change with age. Coping strategies were influenced by Gross Motor Function Classification System level (p=0.022) and history of surgery (p=0.002). INTERPRETATION: Children with CP generally used fewer coping strategies than typically developing children and tended to rely on social support. Use of active strategies increased with age; however, they appeared later than in typically developing children and were used to a lesser extent. WHAT THIS PAPER ADDS: Children with cerebral palsy (CP) use fewer pain-coping strategies than typically developing children. Children with CP tend to use social support to cope with pain. Children with CP learn more appropriate strategies from previous painful experiences. Active coping strategies appear later but remain underused in children with CP.
Chaleat-Valayer E; Roumenoff F; Bard-Pondarre R; Ganne C; Verdun S; Lucet A; Bernard JC
Developmental Medicine & Child Neurology
2019
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<a href="http://doi.org/10.1111/dmcn.14204" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14204</a>
Severe gastric distension in seven patients with cerebral palsy
Child; Female; Male; Adult; Enteral Nutrition; Gastrostomy; Human; Case Report; Cerebral Palsy/co [Complications]; Adolescence; Weight Loss; Stomach Dilatation/et [Etiology]; Vomiting/et [Etiology]; Cerebral Palsy/th [Therapy]; Mental Retardation/co [Complications]; Scoliosis/co [Complications]; Stomach Dilatation/th [Therapy]; Vomiting/th [Therapy]
This is a report of two children who had severe recurrent gastric distension and vomiting, and five who experienced severe gastric distension without vomiting. Two of the five died during an episode of acute gastric distension. All had marked nutritional depletion, and severe spastic quadriplegia due to either cerebral palsy or acquired brain injury. None of the patients had significant gastroesophageal reflux. Positioning the patients in the left lateral decubitus position temporarily relieved their obstructions. Complete resolution of the distension and/or vomiting did not occur until after adequate weight gain. Loss of fat stores may lead to this type of recurrent gastric distension.
1991
Del Beccaro MA; McLaughlin JF; Polage DL
Developmental Medicine & Child Neurology
1991
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/10.1111/j.1469-8749.1991.tb14802.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.1991.tb14802.x</a>
Cardiac monitoring and treatment for children and adolescents with neuromuscular disorders
PedPal Lit; Adolescent Age of Onset Arrhythmia/diagnosis/etiology Cardiomyopathy; Dilated/diagnosis/etiology Cardiomyopathy; Duchenne/complications Muscular Dystrophy; Emery-Dreifuss/complications Myotonic Dystrophy/complications; Hypertrophic/diagnosis/etiology Child Echocardiography Electrocardiography Friedreich Ataxia/complications Heart Diseases/diagnosis/etiology Humans Muscular Dystrophies/complications Muscular Dystrophy
Dilated cardiomyopathy, hypertrophic cardiomyopathy, and cardiac rhythm disturbances are important features of certain neuromuscular disorders in children, adolescents, and young adults. This article summarizes the cardiac features seen in patients with Duchenne muscular dystrophy, Becker muscular dystrophy, myotonic dystrophy, Friedreich's ataxia, and Emery-Dreifuss muscular dystrophy. The optimal management of these cardiac features remains contentious, but increasingly these patients are referred for routine cardiological assessment in the absence of symptoms. This article examines the value of routine screening and drug interventions for cardiac complications in asymptomatic and symptomatic individuals with neuromuscular disorders. We recommend a pragmatic approach, actively looking for cardiac conditions which will benefit from early intervention, but avoiding routine screening for asymptomatic conditions in which there is no evidence of benefit from early intervention.
2006
English KM; Gibbs JL
Developmental Medicine & Child Neurology
2006
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Journal Article
<a href="http://doi.org/10.1017/s0012162206000491" target="_blank" rel="noreferrer">10.1017/s0012162206000491</a>
Status epilepticus in children: aetiology, treatment, and outcome
Child; Female; Male; Prevalence; Follow-Up Studies; Treatment Outcome; Risk Factors; Time Factors; Chi-Square Distribution; Anticonvulsants; Drug Administration Schedule; Anesthetics; Preschool; Non-U.S. Gov't; infant; retrospective studies; Human; Age Distribution; Electroencephalography; Sex Distribution; Neuropsychological Tests; Support; Adolescence; Shock; Status Epilepticus/dt [Drug Therapy]; Status Epilepticus/et [Etiology]; Barbiturates/ad [Administration & Dosage]; Diazepam/ad [Administration & Dosage]; Encephalitis/co [Complications]; Intravenous/ad [Administration & Dosage]; Meningitis; Nervous System Diseases/ep [Epidemiology]; Septic/co [Complications]; Status Epilepticus/di [Diagnosis]; Status Epilepticus/ep [Epidemiology]; Streptococcal Infections/co [Complications]; Viral/co [Complications]
This retrospective study includes 65 children treated for status epilepticus at Tampere University Hospital in Finland. Aetiology of the condition, effectiveness of the treatment protocol, including short barbiturate anaesthesia to prevent prolonged status epilepticus episodes, and neurological outcome were evaluated. Symptomatic aetiology was present in 40% of status epilepticus episodes, and 37% of episodes were induced by fever. Neurological sequelae secondary to status epilepticus were identified in 15% of the cases and subsequent epilepsy in 23% during the mean follow-up time of 3.6 years. There were no status epilepticus-related deaths. The cut-off point of status epilepticus duration for significant risk for permanent neurological sequelae was 2 hours. Our treatment protocol, including short barbiturate anaesthesia in refractory cases, was able to abort status epilepticus in less than 2 hours in 75% of cases. We conclude that early and prompt use of barbiturate anaesthesia should be encouraged, and may explain our low morbidity figures.
1997
Eriksson KJ; Koivikko MJ
Developmental Medicine & Child Neurology
1997
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Journal Article
<a href="http://doi.org/10.1111/j.1469-8749.1997.tb07358.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.1997.tb07358.x</a>
Family-provider consensus outcomes for children with medical complexity
Aim To describe the process of obtaining consensus of outcome priorities between families of children with medical complexity (CMC) and their healthcare providers (HCPs) for the purpose of evaluating changes to service delivery. Method The consensus of outcomes involved surveying families of CMC and HCPs and an in-person consensus meeting. Priorities were obtained from the survey using a stratified ranking approach ensuring equal representation among unequally sized subgroups. An in-person meeting was held using the survey results to inform Delphi voting. Results Families of CMC (n=40) and HCPs (n=74) responded to the survey. Consensus generated three main target areas (child health, family health, experience of care) covered by 15 specific outcomes needed to evaluate care. Differences between family and HCP perceptions of importance were found for child self-care, play, social skills, and recreation as well as emotional health (for both parent and child) outcomes. Interpretation Families of CMC and HCPs identified common priorities for outcome evaluation of CMC initiatives. Outcomes that differ in importance between families of CMC and HCPs should be studied further. What this paper adds Families of children with medical complexity and their providers can reach consensus on important outcomes. Stratifying subgroups ensures diverse representation, which is important to outcome prioritization.
Fayed N; Guttmann A; Chiu A; Gardecki M; Orkin J; Hamid JS; Major N; Lim A; Cohen E
Developmental Medicine & Child Neurology
2018
<a href="http://doi.org/10.1111/dmcn.14110" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14110</a>
Epilepsy and evidence-based medicine: a vote of confidence in expert opinion from the National Institute for Clinical Excellence?
PedPal Lit; Adult Child Drug Industry Drug Resistance Epilepsy/diagnosis/drug therapy/etiologyEvidence-Based Medicine Great Britain Humans Outcome Assessment (Health Care) Practice GuidelinesProfessional CompetenceQuality Assurance; Health Care Randomized Controlled Trials Research SupportState Medicine
2005
Ferrie CD; Livingston JH
Developmental Medicine & Child Neurology
2005
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Journal Article
<a href="http://doi.org/10.1111/j.1469-8749.2005.tb01118.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.2005.tb01118.x</a>
Long-term survival for a cohort of adults with cerebral palsy
PedPal Lit; Adolescent AdultCause of Death Cerebral Palsy/mortality/pathology Child Child; Newborn Life Expectancy Male Prognosis Research Support; Non-U.S. Gov't Sex Factors Survival Analysis; Preschool Cohort StudiesDisabled Persons Female Humans Infant Infant
The aim of this study was to investigate long-term survival and examine causes of death in adult patients with cerebral palsy (CP). A 1940-1950 birth cohort based on paediatric case referral allows for long-term survival follow-up. Survival is analyzed by birth characteristics and severity of disability from age 20 years (and age 2y for a subset of the data). Survival outcome compared with that expected in the general population based on English life tables. The main cohort consisted of 341 individuals, with 193 males and 148 females. Conditional on surviving to age 20 years, almost 85% of the cohort survived to age 50 years (a comparable estimate for the general population is 96%). Very few deaths were attributed to CP for those people dying over 20 years of age. Females survived better than males. However, females faced a greater increase in risk relative to the general population than did males. We conclude that survival outlook is good though lower than in the general population. The relative risk of death compared with the UK population decreases with age, although it shows some indication of rising again after age 50 years. Many more deaths were caused by diseases of the respiratory system among those dying in their 20s and 30s than would be expected in the general population. Many fewer deaths than expected in this age group are caused by injuries and accidents. For those people who die in their 40s and 50s, an increase in deaths due to diseases of the circulatory system and neoplasms is observed. More deaths than expected in this age group are due to diseases of the nervous system.
2006
Hemming K; Hutton JL; Pharoah PO
Developmental Medicine & Child Neurology
2006
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Journal Article
<a href="http://doi.org/10.1017/s0012162206000211" target="_blank" rel="noreferrer">10.1017/s0012162206000211</a>
Outpatient appointment non-attendance and unplanned health care for children and young people with neurological conditions: a retrospective cohort study
Aim To test the hypothesis that children and young people with neurological conditions who missed outpatient appointments have more emergency inpatient admissions and Accident and Emergency centre (A&E) visits than those who missed none. Method Retrospective cohort of individuals aged up to 19 years with neurological conditions, identified from routine hospital data in England, UK from April 1st, 2003 to March 31st, 2015 using an International Statistical Classification of Diseases and Related Health Problems, coding framework. Counts of emergency inpatient admissions and A&E visits per person per year were modelled (random intercept negative binomial regression) with outpatient attendance the independent variable of interest. Results The cohort numbered 524 613 individuals. Those who missed outpatient appointments had 19 per cent (95% confidence interval [CI] 18-19) more emergency inpatient admissions and 16 per cent (95% CI 15-17) more A&E visits per year than those who missed none. 'Did not attends' had a larger increase in unplanned health care than patient or provider cancellations. If no appointments were missed, the models predict there would have been 107 000 fewer A&E visits from 2007/2008 to 2014/2015 and 104 000 fewer emergency inpatient admissions from 2003/2004 to 2014/2015.
Jarvis S; Livingston J; Childs AM; Fraser L
Developmental Medicine & Child Neurology
2018
<a href="http://doi.org/10.1136/bmjspcare-2017-001413" target="_blank" rel="noreferrer noopener">10.1136/bmjspcare-2017-001413</a>
Outpatient appointment non-attendance and unplanned health care for children and young people with neurological conditions: a retrospective cohort study
Aim To test the hypothesis that children and young people with neurological conditions who missed outpatient appointments have more emergency inpatient admissions and Accident and Emergency centre (A&E) visits than those who missed none. Method Retrospective cohort of individuals aged up to 19 years with neurological conditions, identified from routine hospital data in England, UK from April 1st, 2003 to March 31st, 2015 using an International Statistical Classification of Diseases and Related Health Problems, coding framework. Counts of emergency inpatient admissions and A&E visits per person per year were modelled (random intercept negative binomial regression) with outpatient attendance the independent variable of interest. Results The cohort numbered 524 613 individuals. Those who missed outpatient appointments had 19 per cent (95% confidence interval [CI] 18–19) more emergency inpatient admissions and 16 per cent (95% CI 15–17) more A&E visits per year than those who missed none. ‘Did not attends’ had a larger increase in unplanned health care than patient or provider cancellations. If no appointments were missed, the models predict there would have been 107 000 fewer A&E visits from 2007/2008 to 2014/2015 and 104 000 fewer emergency inpatient admissions from 2003/2004 to 2014/2015. Interpretation Missed outpatient appointments were associated with increased unplanned health care. Improving outpatient attendance may have the potential to reduce emergency inpatient admissions and A&E visits. What this paper adds Missed outpatient appointments by children and young people with neurological conditions are associated with increased unplanned health care. Both emergency inpatient admissions and Accident and Emergency centre visits are increased. ‘Did not attends’ are more strongly associated with unplanned health care than cancellations.
Jarvis Stuart; Livingston J; Childs A-M; Fraser L
Developmental Medicine & Child Neurology
2018
<a href="http://doi.org/10.1111/dmcn.14070" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14070</a>
Management of scoliosis in Duchenne muscular dystrophy: a large 10-year retrospective study
PedPal Lit; Adolescent Comparative Study Decision Making Disability Evaluation Female Humans Male Muscular Dystrophy; Duchenne/complications Postoperative Period Research Support; Non-U.S. Gov't Retrospective Studies Scoliosis/etiology/surgery/therapy Time Factors Treatment Outcome
Scoliosis affects 75 to 90% of patients with non-ambulant Duchenne muscular dystrophy (DMD). Spinal surgery is the treatment of choice but the indication varies among centres. Some offer surgery to all non-ambulant patients, irrespective of scoliosis severity. Early surgery has the advantage of targeting DMD when cardiorespiratory function is preserved, but not all patients develop scoliosis. We report our 10-year experience of scoliosis management in 123 patients with DMD who were at least 17 years old at the time of the study. Scoliosis was absent in 10%, and mild, non-progressive (at least 30 degrees ) in 13% of patients. Another 13% had moderate scoliosis (31-50 degrees ) and were managed conservatively. Surgery was considered in 57% (70/123) of patients with scoliosis greater than 50 degrees and eventually performed in 35%. The remaining patients either refused surgery (9%) or were unfit because of cardiorespiratory compromise (13%). In a further 7%, scoliosis (greater than 50 degrees ), first noted after 14 years of age, was progressing slowly and surgery was not performed. At 17 years there was no difference in survival, respiratory impairment, or sitting comfort among patients managed conservatively or with surgery. One-third (44/123) of our patients were managed satisfactorily without receiving spinal surgery. We provide insight into the natural history of scoliosis in DMD that should help families and clinicians with decision-making when surgery is considered.
2006
Kinali M; Messina S; Mercuri E; Lehovsky J; Edge G; Manzur AY; Muntoni F
Developmental Medicine & Child Neurology
2006
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Journal Article
<a href="http://doi.org/10.1017/s0012162206001083" target="_blank" rel="noreferrer">10.1017/s0012162206001083</a>
Parents' perceptions of caregiving: development and validation of a measure of processes
Cohen 2006 BMC HSR Refs
1996
King SM; Rosenbaum PL; King GA
Developmental Medicine & Child Neurology
1996
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/10.1111/j.1469-8749.1996.tb15110.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.1996.tb15110.x</a>
Sleep abnormalities in patients with severe cerebral palsy
Child; Preschool; infant; Human; Electroencephalography; Seizures/co [Complications]; Cerebral Palsy/co [Complications]; Sleep Disorders/et [Etiology]; Sleep Apnea Syndromes/di [Diagnosis]; Sleep Apnea Syndromes/et [Etiology]; Sleep Disorders/di [Diagnosis]
The all-night polysomnographic findings of nine patients with spastic quadriparesis (mean age 36.7 months) were analysed retrospectively and compared with those of nine age-matched controls (mean age 37.4 months). The cerebral palsy group had significantly more respiratory disturbances per hour of sleep, with five of nine being diagnosed as having obstructive sleep apnea. They also had fewer changes in body position during the night. Interictal epileptiform discharges averaged 23.3 per cent of the total arousals in the cerebral palsy group. Obstructive apnea, decreased ability to change body position, and interictal epileptiform discharges are prevalent in the sleep of patients with severe cerebral palsy, and contribute towards its disruption.
1994
Kotagal S; Gibbons VP; Stith JA
Developmental Medicine & Child Neurology
1994
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Journal Article
<a href="http://doi.org/10.1111/j.1469-8749.1994.tb11850.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.1994.tb11850.x</a>
Behavioural problems in children and adolescents with spinal muscular atrophy and their siblings
2003
Laufersweiler-Plass C; Rudnik-Schoneborn S; Zerres K; Backes M; Lehmkuhl G; von Gontard A
Developmental Medicine & Child Neurology
2003
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Journal Article
<a href="http://doi.org/10.1017/s0012162203000082" target="_blank" rel="noreferrer">10.1017/s0012162203000082</a>
A systematic review of interventions for children with cerebral palsy: state of the evidence
2013-10
Novak I; Mcintyre S; Morgan C; Campbell L; Dark L; Morton N; Stumbles E; Wilson Salli-Ann; Goldsmith S
Developmental Medicine & Child Neurology
2013
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Journal Article
<a href="http://doi.org/10.1111/dmcn.12246" target="_blank" rel="noreferrer">10.1111/dmcn.12246</a>
Inborn errors of metabolism causing epilepsy
2013
Rahman S; Footitt EJ; Varadkar S; Clayton PT
Developmental Medicine & Child Neurology
2013
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Journal Article
<a href="http://doi.org/10.1111/j.1469-8749.2012.04406.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.2012.04406.x</a>
Family and quality of life: key elements in intervention in children with cerebral palsy: Review
Rosenbaum P
Developmental Medicine & Child Neurology
2011
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Journal Article
<a href="http://doi.org/10.1111/j.1469-8749.2011.04068.x" target="_blank" rel="noreferrer">10.1111/j.1469-8749.2011.04068.x</a>
Measurement in Duchenne muscular dystrophy: considerations in the development of a neuromuscular assessment tool
PedPal Lit; Child HumansMuscle; Duchenne/diagnosis/pathology/physiopathology Questionnaires Severity of Illness Index; Skeletal/innervation/pathology/physiopathology Muscular Dystrophy
A review of the measures relating to the monitoring of disease progression in Duchenne muscular dystrophy was undertaken as part of the work of the North Star Clinical Network for Paediatric Neuromuscular Disease Management developing a standardized assessment protocol for ambulant children in the UK. This article outlines the process of identifying possible measures. Detailed consideration has been given to key measures of muscle strength and function. As well as the usual assessment of the validity and reliability of the measures, three key characteristics were identified as necessary to the assessment of scales used in health care: (1) the type of scale used; (2) the clinical significance of the attribute being measured; and (3) the mathematical properties of the data provided. Consideration of such aspects in the early stage of a study facilitates the choice of measures, and the analysis and interpretation of data in the longer term.
2006
Scott E; Mawson SJ
Developmental Medicine & Child Neurology
2006
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Journal Article
<a href="http://doi.org/10.1017/s0012162206001137" target="_blank" rel="noreferrer">10.1017/s0012162206001137</a>
A novel classification system for research reporting in rare and progressive genetic conditions
Aim To create a classification system for severe, rare, and progressive genetic conditions for use in research reporting. Method A modified Delphi consensus technique was used to create and reach agreement on a new system of condition categories. Interrater reliability was tested via two rounds of an online survey whereby physicians classified a subset of conditions using our novel system. Overall percentage agreement and agreement above chance were calculated using Fleiss’ kappa (κ). Results Eleven physicians completed the first Delphi, with an overall agreement of 76.4%, the κ value was 0.57 (95% confidence interval 0.51–0.63), indicating moderate agreement (0.41–0.60) above chance. Based on the first survey several categories were described in more detail. The second survey confirmed a classification system with 12 categories, with an overall percentage agreement among the participants of 82.6%. The overall mean κ value was 0.71 (95% confidence interval 0.65–0.77), indicating substantial agreement (0.61–0.80). Interpretation Our new system was useful in categorizing a broad range of rare childhood diseases and may be applicable to other rare disease studies; further validation in larger cohorts is required. What this paper adds This novel 12-category classification system can be used in research reporting in rare and progressive genetic conditions.
van Karnebeek CDM; Beumer D; Pawliuk C; Goez Helly; Mostafavi S; Andrews G; Steele R; Siden H
Developmental Medicine & Child Neurology
2019
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<a href="http://doi.org/10.1111/dmcn.14180" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14180</a>