Using the PODCI to Measure Motor Function and Parent Expectations in Children With Cerebral Palsy.

OBJECTIVE: Involving parents in the evaluation of their child with cerebral palsy (CP) is associated with enhanced neurodevelopmental outcomes. The pediatric outcomes data collection instrument (PODCI) is a patient-reported outcome measure primarily used to assess motor function following orthopedic surgical intervention or for older children with more independent motor function. The PODCI expectations scale has infrequently been reported in previous studies. This study aims to determine the relationship between parent-reported motor performance using the PODCI and motor capacity assessed by pediatric therapists for young children with CP across all ability levels and to explore the use of the PODCI expectations scale for quantifying therapy-related parent expectations. METHODS: This prospective cohort study included 108 participants with CP, 2 to 8 years of age, gross motor function classification systems (GMFCS) levels I to V. Measures included the PODCI, gross motor function measure (GMFM), and GMFCS. RESULTS: There were moderate (r = 0.513) to large (r = 0.885) relationships between PODCI and GMFM scores. PODCI scores were significantly different across GMFCS levels. Weak, significant relationships (r = -0.28) were found between function expectations scores and measures of function. CONCLUSION: The PODCI, GMFM, and GMFCS provide different, but strongly related, information about the abilities of young children. The GMFM measures motor capacity. Parents report daily function and health-related quality of life for their child using the PODCI. Parent expectations for intervention outcomes may relate to a child's motor function. IMPACT: These study results are consistent with those for older children with greater independent mobility, indicating an opportunity for expanded use of the PODCI for measuring motor performance for younger children with CP across all ability levels. A strategy is provided for using the PODCI expectations scale to quantify parent therapy-related expectations in future research and clinical settings. Therapy-related expectations may relate to child outcomes.

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy, and causes severe developmental disability and lifelong motor, behavioral, and autonomic symptoms including oculogyric crises (OGC), sleep disorder, and mood disturbance. We investigated the safety and efficacy of delivery of a viral vector expressing AADC (AAV2-hAADC) to the midbrain in children with AADC deficiency (ClinicalTrials.gov Identifier NCT02852213). Seven (7) children, aged 4-9 years underwent convection-enhanced delivery (CED) of AAV2-hAADC to the bilateral substantia nigra (SN) and ventral tegmental area (VTA) (total infusion volume: 80 µL per hemisphere) in 2 dose cohorts: 1.3 × 1011 vg (n = 3), and 4.2 × 1011 vg (n = 4). Primary aims were to demonstrate the safety of the procedure and document biomarker evidence of restoration of brain AADC activity. Secondary aims were to assess clinical improvement in symptoms and motor function. Direct bilateral infusion of AAV2-hAADC was safe, well-tolerated and achieved target coverage of 98% and 70% of the SN and VTA, respectively. Dopamine metabolism was increased in all subjects and FDOPA uptake was enhanced within the midbrain and the striatum. OGC resolved completely in 6 of 7 subjects by Month 3 post-surgery. Twelve (12) months after surgery, 6/7 subjects gained normal head control and 4/7 could sit independently. At 18 months, 2 subjects could walk with 2-hand support. Both the primary and secondary endpoints of the study were met. Midbrain gene delivery in children with AADC deficiency is feasible and safe, and leads to clinical improvements in symptoms and motor function.

Daily and Weekly Rehabilitation Delivery for Young Children With Gross Motor Delay: A Randomized Clinical Trial Protocol (the DRIVE Study).

PURPOSE: The proposed project tests the principle that frequency of rehabilitation is an important regulator of therapeutic response in infants. METHODS: We will randomize 75 infants with cerebral palsy, 6 to 24 months of age and/or Gross Motor Function Classification System levels III to V (higher severity), to determine the short-term and long-term effects of 3 dosing protocols consisting of an identical number of 2-hour sessions of the same motor learning-based therapy applied over a different total number of calendar weeks. RESULTS AND CONCLUSIONS: The results will inform clinicians, families, and scientists about dosing and will provide needed recommendations for frequency of rehabilitation to optimize motor function and development of young children with cerebral palsy.

O efeito de um programa de equoterapia no desenvolvimento psicomotor de crianças com indicativos de transtorno de déficit de atenção e hiperatividade / The effect of a horseback riding therapy program on psychomotor development of children with indicators of attention deficit hyperactivity disorder

O objetivo do presente estudo foi verificar o efeito de um programa de equoterapia sobre o desenvolvimento psicomotor de crianças com indicativos de transtorno de déficit de atenção e hiperatividade (TDAH). Com relação ao método, foi desenvolvido um estudo exploratório do tipo manipulação experimental. Participaram do estudo cinco crianças com idade entre sete e 10 anos, com indicativos de TDAH obtidos por meio do SNAP IV. Antes e após o período de intervenção os participantes da pesquisa foram submetidos a uma avaliação baseada na Escala de Desenvolvimento Motor (EDM). A intervenção foi constituída por um programa de equoterapia caracterizado como educação/reeducação, tendo sido composta por 24 sessões individuais, com duração de 30 minutos, registradas sistematicamente por meio de filmagens e diário de campo. Por meio da análise dos resultados observou-se que o programa exerceu influência em todas as habilidades motoras estudadas, sendo que aspectos psicomotores mais influenciados pelo programa de equoterapia, de acordo com a escala de desenvolvimento motor (EDM), foram respectivamente: organização espacial, equilíbrio, motricidade fina e esquema corporal; a menor influência foi verificada nos aspectos motricidade global e organização temporal. Considerando que, no pós-teste, a idade motora geral foi estatisticamente significante, conclui-se, de forma geral, que o programa de equoterapia influenciou positivamente o desenvolvimento dos aspectos psicomotores das crianças com indicativos de TDAH participantes do estudo.

The aim of this study was to investigate the effect of a horseback riding therapy program on the psychomotor development of children with indicators of Attention Deficit Hyperactivity Disorder. Regarding the method, for this exploratory study an experimental manipulation procedure was developed. The study included five children aged seven to 10 years, with ADHD indicators obtained through the SNAP IV. An evaluation based on the Motor Development Scale (MDS) was carried out with the study participants before and after the intervention period. The intervention consisted of a horseback riding therapy program with education/reeducation aims. Twenty four individual sessions were carried out, lasting 30 minutes each; these sessions were systematically recorded through videotaping and field diary notes. The analysis of the results showed that the horseback riding therapy program affected all motor skills under investigation, and the greatest improvement of psychomotor aspects, according to the motor development scale (MDS) involved, respectively: spatial organization, balance, fine motor skills and body schema; global motor coordination and temporal organization aspects showed less improvement. Since at posttest, overall motor age was statistically significant, it was concluded that, generally speaking, the horseback riding therapy program had a positive influence on the development of psychomotor aspects of children with ADHD involved in the study.