Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study.
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.
D000085:Acetates / Q000627:therapeutic use*; D000293:Adolescent; D000328:Adult; D000588:Amines; D001132:Arm; D002648:Child; D003509:Cyclohexanecarboxylic Acids; D005260:Female; D000077206:Gabapentin; D006801:Humans; D007537:Isometric Contraction / Q000187:drug effects; D007558:Italy; D007866:Leg; D008297:Male; D008875:Middle Aged; D009068:Movement / Q000187:drug effects; D018482:Muscle, Skeletal / Q000473:pathology / Q000503:physiopathology; D018696:Neuroprotective Agents / Q000627:therapeutic use*; D014897:Spinal Muscular Atrophies of Childhood / Q000188:drug therapy* / Q000503:physiopathology; D016896:Treatment Outcome; D014797:Vital Capacity; D005680:gamma-Aminobutyric Acid
D000085:Acetates; D000588:Amines; D003509:Cyclohexanecarboxylic Acids; D018696:Neuroprotective Agents; D005680:gamma-Aminobutyric Acid; D000077206:Gabapentin