Botulinum toxin type A neuromuscular blockade in the treatment of equinus foot deformity in cerebral palsy: A multicenter, open-label clinical trial

L. Andrew Koman, Allison Brashear, Samuel Rosenfeld, Henry Chambers, Barry Russman, Mercer Rang, Leon Root, Eugenio Ferrari, J. Garcia De Yebenes Prous, Beth P. Smith, Catherine Turkel, Jennifer M. Walcott, Patricia T. Molloy

Research output: Contribution to journalArticle

129 Citations (Scopus)

Abstract

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. Conclusion. BTX-A proved both safe and effective in the chronic management of focal muscle spasticity in children with equinus gait.

Original languageEnglish (US)
Pages (from-to)1062-1071
Number of pages10
JournalPediatrics
Volume108
Issue number5
DOIs
StatePublished - Nov 12 2001
Externally publishedYes

Fingerprint

Equinus Deformity
Foot Deformities
Neuromuscular Blockade
Type A Botulinum Toxins
Cerebral Palsy
Gait
Clinical Trials
Ankle
Leg
Skeletal Muscle
Therapeutics
Neuromuscular Blocking Agents
Physicians
Muscle Cramp
Muscle Spasticity
Dystonia
Intramuscular Injections
Articular Range of Motion
Treatment Failure
Multicenter Studies

Keywords

  • BTX-A
  • Cerebral palsy
  • Equinus foot deformity
  • Neuromuscular blockade
  • Pediatric patients
  • Spasticity

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Botulinum toxin type A neuromuscular blockade in the treatment of equinus foot deformity in cerebral palsy : A multicenter, open-label clinical trial. / Koman, L. Andrew; Brashear, Allison; Rosenfeld, Samuel; Chambers, Henry; Russman, Barry; Rang, Mercer; Root, Leon; Ferrari, Eugenio; Garcia De Yebenes Prous, J.; Smith, Beth P.; Turkel, Catherine; Walcott, Jennifer M.; Molloy, Patricia T.

In: Pediatrics, Vol. 108, No. 5, 12.11.2001, p. 1062-1071.

Research output: Contribution to journalArticle

Koman, LA, Brashear, A, Rosenfeld, S, Chambers, H, Russman, B, Rang, M, Root, L, Ferrari, E, Garcia De Yebenes Prous, J, Smith, BP, Turkel, C, Walcott, JM & Molloy, PT 2001, 'Botulinum toxin type A neuromuscular blockade in the treatment of equinus foot deformity in cerebral palsy: A multicenter, open-label clinical trial', Pediatrics, vol. 108, no. 5, pp. 1062-1071. https://doi.org/10.1542/peds.108.5.1062
Koman, L. Andrew ; Brashear, Allison ; Rosenfeld, Samuel ; Chambers, Henry ; Russman, Barry ; Rang, Mercer ; Root, Leon ; Ferrari, Eugenio ; Garcia De Yebenes Prous, J. ; Smith, Beth P. ; Turkel, Catherine ; Walcott, Jennifer M. ; Molloy, Patricia T. / Botulinum toxin type A neuromuscular blockade in the treatment of equinus foot deformity in cerebral palsy : A multicenter, open-label clinical trial. In: Pediatrics. 2001 ; Vol. 108, No. 5. pp. 1062-1071.
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abstract = "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. Conclusion. BTX-A proved both safe and effective in the chronic management of focal muscle spasticity in children with equinus gait.",
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AU - Brashear, Allison

AU - Rosenfeld, Samuel

AU - Chambers, Henry

AU - Russman, Barry

AU - Rang, Mercer

AU - Root, Leon

AU - Ferrari, Eugenio

AU - Garcia De Yebenes Prous, J.

AU - Smith, Beth P.

AU - Turkel, Catherine

AU - Walcott, Jennifer M.

AU - Molloy, Patricia T.

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N2 - 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. Conclusion. BTX-A proved both safe and effective in the chronic management of focal muscle spasticity in children with equinus gait.

AB - 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. Conclusion. BTX-A proved both safe and effective in the chronic management of focal muscle spasticity in children with equinus gait.

KW - BTX-A

KW - Cerebral palsy

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KW - Neuromuscular blockade

KW - Pediatric patients

KW - Spasticity

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