Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam-Part 2: Experience from a nusinersen clinical study.
Muscle Nerve
; 57(1): 142-146, 2018 Jan.
Article
em En
| MEDLINE
| ID: mdl-28556387
ABSTRACT
INTRODUCTION:
In this study we examined the feasibility of assessing motor milestone performance of infants with spinal muscular atrophy (SMA) using the Hammersmith Infant Neurological Exam-Part 2 (HINE-2) in a phase 2 study of nusinersen.METHODS:
Nineteen SMA infants were assessed using the HINE-2 at baseline (≤7 months of age), and periodically up to 39 months of age. We evaluated whether the HINE-2 was feasible, reliable, and sensitive to change.RESULTS:
Motor milestone assessments in SMA infants were feasible using the HINE-2. Baseline test-retest reliability was excellent (R = 0.987; P < 0.0001). SMA infants were extremely low functioning at baseline and the HINE-2 was able to detect changes over time in 16 of 19 infants within all 8 domains. HINE-2 improvements were correlated with changes in other neuromuscular outcome measures.CONCLUSION:
Results support the use of the HINE-2 motor milestone assessment in clinical trials of SMA infants. Muscle Nerve 57 143-146, 2017.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
/
Atrofias Musculares Espinais da Infância
/
Deficiências do Desenvolvimento
/
Exame Neurológico
Tipo de estudo:
Clinical_trials
/
Diagnostic_studies
Limite:
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
Estados Unidos