Role of gabapentin in spinal muscular atrophy: Results of a multicenter, randomized Italian study

Luciano Merlini, Alessandra Solari, Giuseppe Vita, Enrico Bertini, Carlo Minetti, Tiziana Mongini, Elena Mazzoni, Corrado Angelini, Lucia Morandi

Research output: Contribution to journalArticle

Abstract

Recent studies suggest that gabapentin has a neuroprotective effect in experimental models of motoneuron disease. We carried out a multicenter, randomized, controlled trial of gabapentin versus no treatment in 120 patients with type II or III spinal muscular atrophy for 12 months. We assessed maximum voluntary isometric contraction with a handheld myometer and calculated an arm megascore (summing elbow flexion, hand grip, and three-point pinch scores), and a leg megascore (summing knee flexion, knee extension, and foot extension scores). Forced vital capacity and timed tasks were also evaluated. Arm megascore improved by at least 30% in 24.6% of treated and 16.9% of untreated patients (relative risk = 1.45; 95% confidence interval = 0.71-2.97). The leg megascore improved by at least 30% in 37.7% of treated and 20.3% of untreated patients (relative risk = 1.85; 95% confidence interval = 1.02-3.37). We conclude that gabapentin produced a significant improvement in leg megascore at 6 months, which was more evident at 12 months, with a trend for improvement in arm megascore at 12 months. The treatment had no effect on forced vital capacity or timed functional tests.

Original languageEnglish
Pages (from-to)537-541
Number of pages5
JournalJournal of Child Neurology
Volume18
Issue number8
Publication statusPublished - Aug 1 2003

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health

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