Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators

Research output: Contribution to journalArticle

48 Citations (Scopus)

Abstract

Objective: This study prospectively assessed putative promising biomarkers for use in assessing infants with spinal muscular atrophy (SMA). Methods: This prospective, multi-center natural history study targeted the enrollment of SMA infants and healthy control infants less than 6 months of age. Recruitment occurred at 14 centers within the NINDS National Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) Network. Infant motor function scales and putative electrophysiological, protein and molecular biomarkers were assessed at baseline and subsequent visits. Results: Enrollment began November, 2012 and ended September, 2014 with 26 SMA infants and 27 healthy infants enrolled. Baseline demographic characteristics of the SMA and control infant cohorts aligned well. Motor function as assessed by the Test for Infant Motor Performance Items (TIMPSI) and the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) revealed significant differences between the SMA and control infants at baseline. Ulnar compound muscle action potential amplitude (CMAP) in SMA infants (1.4 ± 2.2 mV) was significantly reduced compared to controls (5.5 ± 2.0 mV). Electrical impedance myography (EIM) high-frequency reactance slope (Ohms/MHz) was significantly higher in SMA infants than controls SMA infants had lower survival motor neuron (SMN) mRNA levels in blood than controls, and several serum protein analytes were altered between cohorts. Interpretation: By the time infants were recruited and presented for the baseline visit, SMA infants had reduced motor function compared to controls. Ulnar CMAP, EIM, blood SMN mRNA levels, and serum protein analytes were able to distinguish between cohorts at the enrollment visit.

Original languageEnglish (US)
Pages (from-to)132-145
Number of pages14
JournalAnnals of Clinical and Translational Neurology
Volume3
Issue number2
DOIs
StatePublished - Feb 1 2016

Fingerprint

Spinal Muscular Atrophy
Biomarkers
Myography
Motor Neurons
Electric Impedance
Action Potentials
Blood Proteins
National Institute of Neurological Disorders and Stroke
Muscles
Messenger RNA
Neurosciences
Natural History

ASJC Scopus subject areas

  • Neuroscience(all)
  • Clinical Neurology

Cite this

The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators (2016). Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Annals of Clinical and Translational Neurology, 3(2), 132-145. https://doi.org/10.1002/acn3.283

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. / The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators.

In: Annals of Clinical and Translational Neurology, Vol. 3, No. 2, 01.02.2016, p. 132-145.

Research output: Contribution to journalArticle

The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators 2016, 'Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study', Annals of Clinical and Translational Neurology, vol. 3, no. 2, pp. 132-145. https://doi.org/10.1002/acn3.283
The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Annals of Clinical and Translational Neurology. 2016 Feb 1;3(2):132-145. https://doi.org/10.1002/acn3.283
The NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. / Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. In: Annals of Clinical and Translational Neurology. 2016 ; Vol. 3, No. 2. pp. 132-145.
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