Outcome Assessment and Function in Cerebral Palsy.

Care and research in childhood cerebral palsy (CP) continue to evolve. As our understanding of CP grows more nuanced, so grows our need to describe function, activities, challenges, adaptations of children with CP. In CP, robust means of measuring outcomes are vital to understanding utility of treatments. Research must accurately measure meaningful constructs of children with CP as a reliable ruler to establish if interventions produce useful effects. This article addresses the challenges of outcome measurement in CP, current status of outcome measurement in CP, and issues of understanding change in childhood CP.

International Classification of Functioning, Disability and Health (ICF) as a framework for change: revolutionizing rehabilitation.

The International Classification of Functioning, Disability and Health (ICF) utilizes domains of body functions and structures, activities and participation, as well as environmental and personal factors to fully encapsulate the concepts of health and disability. The International Classification of Functioning, Disability and Health provides a rich and holistic understanding of functioning that is particularly valuable in the setting of childhood disability and rehabilitation. With applicability that enhances a nuanced understanding of each child within their family, school, and community, the International Classification of Functioning, Disability and Health also ensures facile and meaningful communication between professionals. Use of the International Classification of Functioning, Disability and Health promotes improved treatment plans for individual children and for larger programmatic decisions. This article demonstrates how the International Classification of Functioning, Disability and Health has reinvented the language and understanding of childhood disability and rehabilitation.

The GMFM, PEDI, and CP-QOL and perspectives on functioning from children with CP, parents, and medical professionals.

OBJECTIVE: Many outcome measures assess function of children with cerebral palsy (CP), but establishing meaningful clinical change remains challenging. This study explored correlations between subjective status ratings in several functional domains, made by children, parents, and medical professionals. The ratings were compared with three outcome measures in preparation for longitudinal work to establish minimal clinically important change. METHOD: Children were assessed with the Gross Motor Function Measure (GMFM), Pediatric Evaluation of Disability Inventory (PEDI), and Cerebral Palsy Quality of Life Questionnaire for Children (CP-QOL). Respondents provided Likert scale and Linear Analogue Scale ratings of gross motor function, self care, social function, quality of life, and overall function. Correlations were calculated for outcome measure scores and ratings. RESULTS: 122 children with CP across all GMFCS and MACS levels, 79 male, aged 8.1 ± 2.9 years generated status ratings by 27 child reports, 122 parent reports, and 110 medical professional reports. Most ratings were moderately to highly correlated between parents and medical professionals. Outcome measure scores were frequently significantly correlated with pertinent ratings from medical professionals and parents but usually not with child ratings. CONCLUSIONS: Parents and medical professionals have similar perceptions of gross motor, self-care, quality of life, and overall status for children with CP and these perceptions correlate with standard outcome measures, but often do not agree with children's ratings. Longitudinal use of subjective status ratings from parents and professionals should contribute to establishing minimal clinically important differences for CP outcome measures.

Pharmacokinetics of amantadine in children with impaired consciousness due to acquired brain injury: preliminary findings using a sparse-sampling technique.

OBJECTIVE: To evaluate the pharmacokinetics of amantadine in children with impaired consciousness from acquired brain injury. DESIGN: Randomized, double-blind, placebo-controlled, crossover study with sparse sampling for pharmacokinetics. SETTING: Tertiary care pediatric hospital. PARTICIPANTS: Children, ages 6-18 years, with impaired consciousness 5-10 weeks after acquired brain injury. METHODS: Subjects received amantadine for 3 weeks. Subjects were randomized to placebo or amantadine 4 mg/kg/day for 7 days followed by 6 mg/kg/day for 14 days. Crossover was after a 7-day washout period. MAIN OUTCOME MEASURES: The Coma/Near-Coma Scale and Coma Recovery Scale-Revised were done 3 times per week to evaluate arousal and consciousness. Plasma concentrations of amantadine were determined for pharmacokinetic parameter estimation and evaluation of the exposure-response relationship. Adverse events were monitored. RESULTS: Nine subjects met the final inclusion and exclusion criteria, 7 of whom agreed to participate. Five subjects completed both arms of the study. Amantadine total body clearance was 0.17 L/h/kg with a half-life of 13.9 hours. Higher exposure of amantadine (average concentration of amantadine during 6 mg/kg/day > 1.5 mg/L) may be associated with better recovery of consciousness. CONCLUSIONS: Amantadine was well-tolerated in children with acquired brain injury and demonstrates pharmacokinetics similar to those reported for healthy young adults. Based on the preliminary data, higher dosing may be considered in the setting of brain injury.

Measuring what matters in cerebral palsy: a breadth of important domains and outcome measures.

UNLABELLED: Vargus-Adams JN, Martin LK. Measuring what matters in cerebral palsy: a breadth of important domains and outcome measures. OBJECTIVE: To establish domains of importance for evaluating therapeutic effects in childhood cerebral palsy (CP) and the best means of evaluating those domains. DESIGN: Delphi iterative survey. SETTING: Tertiary pediatric hospital with assessments conducted via mail or Internet. PARTICIPANTS: Youth with CP (n=21), parents of children with CP (n=23), and medical professionals (n=39). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Identification of "the things you find most important to consider when you evaluate the effects of an intervention for yourself/your child/your patient with cerebral palsy" as domains of concern, followed by the best outcome measures to assess each of the domains, including preference rankings of those measures. RESULTS: Eight domains of importance were identified: impairment, general health, gross motor skills, self-care/fine motor skills, speech/communication, integration/participation, quality of life, and caregiver issues. All domains were viewed as important with no clear demarcation to allow reduction in number. Only quality of life emerged as being most important. Medical professionals suggested 10 to 27 different outcome measures for assessing each domain. Few of these measures, including the Gross Motor Function Measure and the Cerebral Palsy Quality of Life Questionnaire, were broadly endorsed by the medical professionals. CONCLUSIONS: Parents, youth, and medical professionals identify a wide range of arenas that they would like to see impacted by interventions for CP. These arenas can be consolidated into 8 domains. Although many outcome measures are available to evaluate these domains, few of the measures are widely preferred. Further work may determine optimal assessment strategies and provide guidance for therapeutic decisions.

Effects of amantadine in children with impaired consciousness caused by acquired brain injury: a pilot study.

OBJECTIVE: To conduct a pilot study of amantadine in children with impaired consciousness caused by acquired brain injury, to establish design feasibility, and to assess the effect on level of arousal and consciousness. DESIGN: Randomized, double-blind, placebo-controlled crossover trial. Seven subjects (mean age, 12.7 yrs) with an acquired brain injury (mean duration, 6 wks) were randomized to receive either 3 wks of placebo or amantadine, followed by a 1-wk washout period and then 3 wks of the other agent. Main outcome measures were the Coma/Near-Coma Scale and Coma Recovery Scale-Revised, each done three times per week. Subjective evaluations of change in arousal and consciousness by the parent and physician were done weekly. RESULTS: Five subjects completed the study. There was no significant difference in the slopes of recovery during either arm for the Coma/Near-Coma Scale (P = 0.24) or the Coma Recovery Scale-Revised (P = 0.28), although improvements in consciousness were noted by the physician during weeks when amantadine was given (P = 0.02). CONCLUSIONS: This study suggests that amantadine facilitates recovery of consciousness in pediatric acquired brain injury and provides important information necessary to design future more definitive studies.

Inconsistencies with physical functioning and the child health questionnaire in children with cerebral palsy.

OBJECTIVES: To explore the performance of the Physical Functioning (PF) subscale of the Child Health Questionnaire (CHQ) in children with cerebral palsy (CP). STUDY DESIGN: Parents of 177 children and adolescents (age 3 to 18 years) with CP completed the CHQ -Parent Form 50. Severity of CP was assessed using the 5-level Gross Motor Function Classification System (GMFCS), in which higher levels reflect more severe impairment. RESULTS: PF scores were negatively correlated with GMFCS classification (R = -0.62) and were distributed bimodally in subjects with severe motor impairment. For GMFCS classifications IV and V (n = 59), PF scores were very low (means, 9 to 28; medians, 0 to 8); however, 12% of these subjects had excellent PF scores (> 88) despite being nonambulatory. CONCLUSIONS: Although the CHQ PF subscale correlated well with the GMFCS, the CHQ questions on physical functioning resulted in unexpected responses in approximately 1 in 8 subjects with severe CP. These unanticipated responses to the PF subscale questions may be due to ambiguity in the questions (which do not differentiate between health problems and disability) or to alternative parental interpretation of physical functioning. Confusion in differentiating health status and functional status may make the CHQ less useful in children with significant disabilities.

Hyperhidrosis in pediatric spinal cord injury: a case report and gabapentin therapy.

Hyperhidrosis is a relatively common condition with a multitude of causes. Spinal cord injury may be complicated by hyperhidrosis. Many forms of therapy have been reported for this phenomenon but few have been demonstrated to be efficacious. We describe a case of a child with hyperhidrosis from a spinal cord injury and report the first therapeutic use, to our knowledge, of gabapentin for hyperhidrosis.