Pediatric physical functioning reference curves

Haley, Stephen M.; Fragala-Pinkham, Maria; Ni, Pengsheng; Skrinar, Alison; Kaye, Edward M.

doi:10.1016/j.pediatrneurol.2004.05.002

Cited by 19 publications

(12 citation statements)

References 33 publications

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“…The Pompe PEDI was specifically designed to evaluate functional skills in patients with Pompe disease 14,15 and includes three content domains: mobility, self-care, and social skills. The seven patients designated as walkers based on the AIMS assessments also acquired the most Kaplan skills in the Pompe PEDI, including (but not limited to) such skills as self-feeding, playing independently, climbing stairs, and naming objects.…”

Section: Results Disposition Of Patientsmentioning

confidence: 99%

“…14,15 Although not an efficacy endpoint, cognitive development (using Bayley's Scales of Infant Development, Second Edition [BSID-II]) was also serially evaluated. 16 Echocardiograms and glycogen content in muscle biopsy samples were centrally read by a pediatric cardiologist and a pathologist who were blinded to dose, patient, and time point.…”

Section: Methods Study Design and Treatmentmentioning

confidence: 99%

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Recombinant human acid α-glucosidase

Kishnani

Corzo²,

Nicolino³

et al. 2007

Neurology

675

457

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Abstract-Background:Pompe disease is a progressive metabolic neuromuscular disorder resulting from deficiency of lysosomal acid ␣-glucosidase (GAA). Infantile-onset Pompe disease is characterized by cardiomyopathy, respiratory and skeletal muscle weakness, and early death. The safety and efficacy of recombinant human (rh) GAA were evaluated in 18 patients with rapidly progressing infantile-onset Pompe disease. Methods: Patients were diagnosed at 6 months of age and younger and exhibited severe GAA deficiency and cardiomyopathy. Patients received IV infusions of rhGAA at 20 mg/kg (n ϭ 9) or 40 mg/kg (n ϭ 9) every other week. Analyses were performed 52 weeks after the last patient was randomized to treatment. Results: All patients (100%) survived to 18 months of age. A Cox proportional hazards analysis demonstrated that treatment reduced the risk of death by 99%, reduced the risk of death or invasive ventilation by 92%, and reduced the risk of death or any type of ventilation by 88%, as compared to an untreated historical control group. There was no clear advantage of the 40-mg/kg dose with regard to efficacy. Eleven of the 18 patients experienced 164 infusion-associated reactions; all were mild or moderate in intensity. Conclusions: Recombinant human acid ␣-glucosidase is safe and effective for treatment of infantile-onset Pompe disease. Eleven patients experienced adverse events related to treatment, but none discontinued. The young age at which these patients initiated therapy may have contributed to their improved response compared to previous trials with recombinant human acid ␣-glucosidase in which patients were older.

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Section: Results Disposition Of Patientsmentioning

confidence: 99%

Section: Methods Study Design and Treatmentmentioning

confidence: 99%

Recombinant human acid α-glucosidase

Kishnani

Corzo²,

Nicolino³

et al. 2007

Neurology

675

457

View full text Add to dashboard Cite

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“…Additionally, almost all patients in the study demonstrated the acquisition of functional skills as measured by the Pompe PEDI; this measure provides a more sensitive and comprehensive assessment of the ability to perform skills required for many activities of daily living than motor evaluations alone. 15,16 Failure to thrive is another typical finding in infants with untreated Pompe disease. 10,20 A natural history study found that, despite the frequent use of tube feeding (in 59.5% of patients), 53% of untreated Pompe patients failed to maintain normal growth in the first year of life.…”

Section: Discussionmentioning

confidence: 99%

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

Nicolino

Byrne

Wraith

et al. 2009

Genetics in Medicine

263

209

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Purpose: A clinical trial was conducted to evaluate the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease. Methods: Open-label, multicenter study of IV alglucosidase alfa treatment in 21 infants 3-43 months old (median 13 months) with minimal acid ␣-glucosidase activity and abnormal left ventricular mass index by echocardiography. Patients received IV alglucosidase alfa every 2 weeks for up to 168 weeks (median 120 weeks). Survival results were compared with an untreated reference cohort. Results: At study end, 71% (15/21) of patients were alive and 44% (7/16) of invasive-ventilator free patients remained so. Compared with the untreated reference cohort, alglucosidase alfa reduced the risk of death by 79% (P Ͻ 0.001) and the risk of invasive ventilation by 58% (P ϭ 0.02). Left ventricular mass index improved or remained normal in all patients evaluated beyond 12 weeks; 62% (13/21) achieved new motor milestones. Five patients were walking independently at the end of the study and 86% (18/21) gained functional independence skills. Overall, 52% (11/21) of patients experienced infusion-associated reactions; 95% (19/20) developed IgG antibodies to recombinant human lysosomal acid ␣-glucosidase; no patients withdrew from the study because of safety concerns. Conclusions: In this population of infants with advanced disease, biweekly infusions with alglucosidase alfa prolonged survival and invasive ventilation-free survival. Treatment also improved indices of cardiomyopathy, motor skills, and functional independence. Genet Med 2009:11(3):210-219.

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“…Information pertaining to the difficulty in carrying out these activities is highly relevant because this is usually the main complaint of affected children, parents and relatives 28 . Children of the SG showed higher scores in the PEDI 17 and a better position in the PPFRC 18 , which, in everyday life, means greater independence in activities related to self-care and mobility and less of a need for caregiver assistance. It should be noted that although the results do not show significance, the SG had better functional outcomes than the CG, especially in the GMFM scores 13 , which is considered the gold standard for the development of motor skills.…”

Section: Discussionmentioning

confidence: 92%

“…Th e GMFM 13 score allowed comparing the best positioning in relation to the median in the motor development curves (MDC) for CP for CP 14 . Th e Pediatric Evaluation of Disability Inventory (PEDI) 17 was used to assess the child' s actual performance during day-to-day life and the positioning in relation to the third percentile for the Pediatric Physical Functioning Reference Curves (PPFRC) for self-care and mobility 18 . All PEDI 17 evaluations were performed by the same trained author of this study, MP.…”

Section: Outcomes Measures Used In Comparing the Groupsmentioning

confidence: 99%

Effects of early spasticity treatment on children with hemiplegic cerebral palsy: a preliminary study

Zonta

Bruck

Puppi

et al. 2013

Arq. Neuro-Psiquiatr.

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Objective: To compare motor and functional performance of two groups of children with hemiplegic cerebral palsy (HCP). Only the study group (SG) received early treatment of spasticity with botulinum neurotoxin type A (BXT-A). Methods: Gross Motor Function Measure (GMFM), functional performance (Pediatric Evaluation of Disability Inventory -PEDI), range of movement, gait pattern (Physician Rating Scale -PRS) and the speed of hand movements were considered. Results: The SG, composed of 11 HCP (45.64±6.3 months), was assessed in relation to the comparison group, composed of 13 HCP (45.92±6.4 months). SG showed higher scores in four of the five GMFM dimensions, which included scores that were statistically significant for dimension B, and higher scores in five of the six areas evaluated in the PEDI. Active wrist extension, the speed of hand movements and PRS score were higher in the SG. Conclusion: Children who received early BXT-A treatment for spasticity showed higher scores in motor and functional performance.Key words: child, botulinum toxins, type A, cerebral palsy, hemiplegia. RESUMOObjetivo: Comparar a performance motora e funcional de dois grupos de crianças com paralisia cerebral hemiplégica (PCH). Apenas o grupo de estudo (GE) recebeu tratamento precoce da espasticidade com toxina botulínica do tipo A (BXT-A). Métodos: Foram considerados a Função Motora Grossa (Gross Motor Function Measure -GMFM), performance funcional (Pediatric Evaluation of Disability Inventory -PEDI), amplitude de movimento, padrão da marcha (Physician Rating Scale -PRS) e a velocidade de movimento das mãos. Resultados: O GE, composto de 11 PCH (45,64±6,3 meses), foi analisado em relação ao grupo de comparação, composto por 13 PCH (45,92±6,4 meses). O GE mostrou maiores escores em quatro das cinco dimensões da GMFM, sendo a diferença estatisticamente significativa na dimensão B, e melhores escores em cinco das seis áreas avaliadas na PEDI. A extensão ativa do punho, a velocidade de movimento das mãos e o escore na PRS foram maiores no GE. Conclusão: As crianças que receberam tratamento precoce da espasticidade com BXT-A mostraram melhores escores motores e funcionais.Palavras-Chave: criança, toxinas botulínicas tipo A, paralisia cerebral, hemiplegia.The ultimate goal for the treatment of children with cerebral palsy (CP) is an improvement in functionality and increased independence, which will prepare these children for adult life 1 . The prognosis of a child with CP often depends on the type and severity of the palsy and the accompanying medical comorbidities 2 . In hemiplegic cerebral palsy (HCP), different severities and combinations of impairments in sensory function, perception, muscle tone, muscle strength and range of motion contribute to their motor dysfunction. Furthermore, these factors should be evaluated and managed because functionality is a component of overall health, and the appropriate combinations of interventions can improve function 3 .

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Pediatric physical functioning reference curves

Cited by 19 publications

References 33 publications

Recombinant human acid α-glucosidase

Recombinant human acid α-glucosidase

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

Effects of early spasticity treatment on children with hemiplegic cerebral palsy: a preliminary study

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