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Role of Gabapentin in Spinal Muscular Atrophy

Results of a Multicenter, Randomized Italian Study

Neuromuscular Unit, Istituto Ortopedico Rizzoli, Bologna, Italy

Alessandra Solari, MD

Unit of Epidemiology

Giuseppe Vita, MD

Dipartimento di Neuroscienze, Scienze Psichiatriche ed Anestesiologiche, Università di Messina

Enrico Bertini, MD

Department of Neurosciences, Bambino Gesù Children's Hospital IRCCS, Roma, Italy

Carlo Minetti, MD

Istituto Gaslini, Università di Genova, Genova, Italy

Tiziana Mongini, MD

Centro per le Malattie Neuromuscolari, Università di Torino, Torino, Italy

Elena Mazzoni, MD

Neuromuscular Unit, Istituto Ortopedico Rizzoli, Bologna, Italy

Corrado Angelini

Dipartimento di Scienze Neurologiche e Psichiatriche, Università di Padova, Padova, Italy

Lucia Morandi, MD

Department of Neuromuscular Diseases, Istituto Nazionale Neurologico "Carlo Besta," Milano, Italy

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. (J Child Neurol 2003;18:537—541).

Journal of Child Neurology, Vol. 18, No. 8, 537-541 (2003)
DOI: 10.1177/08830738030180080501


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