Levodopa helps with gait problems in Dravet syndrome

In patients with Dravet syndrome, treatment with levodopa appears to produce improvements in gait disturbance, as shown in a randomized crossover trial.

The trial included nine patients with Dravet syndrome between 6 and 20 years of age (median, 15 years). For 4–6 weeks, the patients received either levodopa/carbidopa hydrate (5 mg/kg/day for those with body weight <60 kg or 300 mg/day for those with body weight ≥60 kg) or no treatment. Then, the patients switched interventions after a 4-week washout period.

The primary outcome was the change in the Gait Deviation Index (GDI), assessed using the three-dimensional gait analysis (3DGA). Other outcomes included spatiotemporal gait parameters, 6-minute walking distance (6MD), and balance. A mixed-effects model was used in the analyses.

Among the participants, the number of status epilepticus cases before study enrolment ranged from 0 to 5 times (median, 2 times). Intellectual disability was moderate in two participants and severe in seven. The median age when the participants began to walk without support was 14 months, and the median total scores on the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) was 26 points. Generalized rigidity, ataxia (appendicular, axial, and gait), pes planovalgus, and scoliosis were observed in one, four, nine, and three participants, respectively.

During levodopa treatment, GDI increased by 4.2 points (p=0.029), 6MD by 52 m (p=0.002), and balance test result by 4.1 mm (p=0.011). Younger participants with a higher baseline gait performance obtained greater benefit with levodopa.

None of the participants experienced severe adverse events, except for one participant who had fever and consequently discontinued levodopa treatment.