Upper extremity spasticity in children with cerebral palsy: a randomized, double-blind, placebo-controlled study of the short-term outcomes of treatment with botulinum A toxin.

PURPOSE: Botulinum A toxin (BoNT-A) injections are used widely to manage lower extremity spasticity in children with cerebral palsy. However, their use in the upper extremity is less well defined. This randomized, double-blind, placebo-controlled clinical trial evaluated the safety and efficacy of upper extremity intramuscular injections of BoNT-A in a cross-section of children with varying levels of function.

METHODS

Upper extremity function of study participants (N = 73; M:F = 47:26; age range, 3-18 y) was evaluated using the House Classification system (scores, 0-8, where a higher score indicates higher functional ability). Three groups of children were identified based on their House scores: 0-2 (n = 10), 3-5 (n = 54), and 6-8 (n = 9). Following randomization, children received a BoNT-A or placebo injection at baseline. Injections were administered at 8 and 20 weeks if clinically indicated. Occupational therapists evaluated study participants at screening, at baseline, and at 4, 8, 14, 20, and 26 weeks. Physician evaluations occurred at baseline and at 8, 20, and 26 weeks. The Melbourne Assessment of Unilateral Upper Limb Function evaluated the quality of upper extremity function before and after injections and served as the primary outcome variable.

RESULTS

The majority of study participants underwent 3 injection sessions. Muscles injected were individualized based on each child's particular spasticity pattern. A statistically higher percentage of children receiving BoNT-A injections showed an improvement in the Melbourne assessment at 26 weeks compared with the children receiving placebo. The range, frequency, and severity of postinjection adverse events were similar in both groups.

CONCLUSIONS

Children receiving BoNT-A injections demonstrated clinically meaningful short-term improvements in upper extremity function. Injections were well tolerated and safe. In contrast to other studies, study participants underwent multiple injection sessions based on their individual spasticity patterns.