Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam-Part 2: Experience from a nusinersen clinical study.

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.

• Authors: Kathie M Bishop, Jacqueline Montes, Richard S Finkel
• Journal: Muscle & nerve (Muscle Nerve) Vol. 57 Issue 1 Pg. 142-146 (Jan 2018) ISSN: 1097-4598 [Electronic] United States
• PMID: 28556387 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study)
• Copyright: © 2017 Wiley Periodicals, Inc.

Chemical References

• Oligonucleotides
• nusinersen

Topics

• Child, Preschool
• Developmental Disabilities (pathology)
• Disease Progression
• Female
• Humans
• Infant
• Male
• Movement
• Neurologic Examination (methods)
• Observer Variation
• Oligonucleotides (therapeutic use)
• Reference Standards
• Reproducibility of Results
• Sensitivity and Specificity
• Spinal Muscular Atrophies of Childhood (drug therapy, pathology)