Botulinum toxin type a neuromuscular blockade in the treatment of equinus foot deformity in cerebral palsy: a multicenter, open-label clinical trial.
BACKGROUND
Focal spasticity of the gastrocnemius-soleus muscles causes equinus gait in children with cerebral palsy (CP). Botulinum toxin type A (BTX-A), a neuromuscular blocking agent, reduces muscle tone/overactivity in dystonia, stroke, and CP.


OBJECTIVE
A prospective, open-label, multicenter clinical trial evaluated the long-term safety and efficacy of repeated intramuscular injections of BTX-A on equinus gait in CP children.


METHODS
Nine centers enrolled 207 children. BTX-A injections (4 U/Kg) were given approximately every 3 months (maximum dose 200 U per treatment). Outcome measures included a Physician Rating Scale of gait, ankle range of motion measurements, and the incidence and profile of adverse events.


RESULTS
One hundred fifty-five (75%) of 207 children completed at least 1 year with a total of 302 patient years of BTX-A treatment. The mean duration of BTX-A exposure was 1.46 years per patient. Dynamic gait pattern on the Physician Rating Scale improved in 46% of patients (86/185) at first follow-up. The response was maintained in 41% to 58% of patients for 2 years. Both gait pattern and ankle position improved at every visit. The most common treatment-related adverse events included increased stumbling, leg cramps, leg weakness, and calf atrophy in 1% to 11% of patients. No treatment-related serious adverse events were reported. Only 6% (7/117) of patients with pre- and postantibody samples had both detectable antibodies and a subsequent treatment failure.


CONCLUSIONS
BTX-A proved both safe and effective in the chronic management of focal muscle spasticity in children with equinus gait.
Koman
L A
LA
Department of Orthopaedic Surgery, Wake Forest University School of Medicine, Winston-Salem, North Carolina 27157-1070, USA. lakoman@wfubmc.edu
Brashear
A
A
Rosenfeld
S
S
Chambers
H
H
Russman
B
B
Rang
M
M
Root
L
L
Ferrari
E
E
Garcia de Yebenes Prous
J
J
Smith
B P
BP
Turkel
C
C
Walcott
J M
JM
Molloy
P T
PT
D000293:Adolescent; D019274:Botulinum Toxins, Type A / Q000627:therapeutic use*; D002547:Cerebral Palsy / Q000150:complications*; D004863:Equinus Deformity / Q000209:etiology / Q000628:therapy*; D005260:Female; D005684:Gait; D006801:Humans; D008297:Male; D009465:Neuromuscular Agents / Q000627:therapeutic use*; D019148:Neuromuscular Blockade / Q000379:methods*; D011446:Prospective Studies
D009465:Neuromuscular Agents; D019274:Botulinum Toxins, Type A