The role of botulinum toxin in multimodal treatment of spasticity in ambulatory children with spastic Cerebral Palsy: extensive evaluation of a cost-effectiveness trial

Abstract

Recently, we performed a study on the added value of botulinum toxin injections (BoNT-A) for ambulatory children with spastic cerebral palsy (the Space Bop study). The results showed that BoNT-A did not add any value in this context, but this counterintuitive finding was met with scepticism. The aim of this paper is to describe and discuss some crucial aspects of, and barriers to, the Space Bop study. The paper also discusses the design, interpretation and presentation of previous research; the role of clinician's own experience and interpretation; the aims of (BoNT-A) treatment; conflict of interest, role of industry, and the role of history; and optimal treatment modalities and dose-response relationships. We conclude that several factors hindered acceptance of the Space Bop results. Awareness of these factors is important when performing rehabilitation research and disseminating and implementing research findings. Background: A cost-effectiveness trial (the Space Bop study) on the added value of botulinum toxin injections (BoNT-A) in the leg muscles, as part of a multimodal intervention for ambulatory children with spastic cerebral palsy in the context of a single distinct cycle of care was performed recently by our group. For a broad set of effect outcomes, we found that BoNT-A had no added value if children received comprehensive rehabilitation. However, this counterintuitive finding was met with scepticism. Objective: Since several noteworthy facts and experiences were recorded during the course of the trial and the dissemination phase, the aim of this paper was to describe and discuss some crucial aspects of, and barriers to, the Space Bop study, related to context and perspective, design and results, as well as publication and implementation. Methods: This paper discusses 5 issues: (i) the design, interpretation and presentation of previous research; (ii) the role of one's own clinical experience and interpretation; (iii) the aims of (BoNT-A) treatment; (iv) conflict of interest, role of industry, and the role of history; (v) optimal treatment modalities and dose-response relationships. Conclusion: Despite the unambiguous findings from the Space Bop study, several factors hindered acceptance of the results. Awareness of these factors is important when performing rehabilitation research and disseminating and implementing research findings.

The state of the art in therapeutic administration of botulinum toxin in children with cerebral palsy: an integrative review

“…As of the absence of relevant data in the included articles, three studies (2.9%) did not specify the age range, 36 (34.3%) did not indicate whether the patient was undergoing rehabilitation treatment, and 53 (50.5%) did not provide information on adverse effects ( Tables 1 – 6 ). 4 - 105 …”

“…Referring to systematic reviews, three (30%) originated from Euro-Asia (Turkey), two (20%) from Europe, two (20%) from North America, two (20%) from South America, and one (10%) was from Asia ( Tables 1 – 6 ) 4 - 105 …”

“…The study conducted by Bussmann et al, which evaluated the cost-effectiveness of BTX in 60 children with walking capacity, found that BTX was efficacious when administered by specialist doctors in physical medicine and rehabilitation and/or trained neurologists. 4 …”

“…Out of these, seven (53.8%) were treated with Botox ® , two (15.4%) with both Botox ® and Dysport ® , one (7.7%) with Botulax ® and Botox ® , one (7.7%) was treated with Botox ® , Dysport ® , and Xeomin ® , and one (7.7%) did not specify which BTX was used ( Tables 1 - 6 ). 4 - 105 …”

“…BTX administration for cerebral palsy (CP) has proved to be of great therapeutic value in reducing spasticity, particularly in gait improvement, upper limb function enhancement, hygiene care facilitation, pain relief, and prevention of myo-articular deformities. 3 , 4 …”