Original articleA French observational study of botulinum toxin use in the management of children with cerebral palsy: BOTULOSCOPE
Introduction
Cerebral Palsy (CP) is a non-progressive and non-contagious condition leading to motor disability. It is a consequence of damages to the developing brain and leads to movement and posture disorders with the frequent clinical form of focal spasticity. Every year in France, prevalence of CP is 2–3 in 1000 live births, i.e. around 1500 new cases each year. Among these, approximately 1300 suffer from spastic disorders.1 The primary troubles of CP are tone and spasticity problems which can be treated by botulinum toxin type A (BTX-A), and balance, selectivity of motor control and strength problems which are improved with occupational and physical therapy combined with BTX-A. The secondary troubles, gradually occurring in growing children, are muscle shortening and associated soft tissue and skeletal deformations requiring orthopaedic surgery. Tertiary troubles are disorders such as postural compensations occurring when children counterbalance the impact of the above-mentioned symptoms.
It is now well accepted that conservative treatments should preferably be used before considering orthopaedic surgery in order to avoid repeated operations and major disturbances in child development (gait acquisition for example). The main conservative treatments for children with CP are currently serial casting and physiotherapy in order to gradually increase muscle length and to limit muscle retraction, and systemic anti-spastic medications or local anti-spastic products to reduce spastic muscle contractions.2 Today in France, the use of anti-spastic medications is limited as they are better indicated in the management of overall spasticity and might induce secondary cognitive effects.3 Yet, serial casting with static and/or dynamic ortheses and physiotherapy represent the standard management of spastic disorders. The use of local anti-spastic products which can be directly injected into the relevant muscles has been a field of development for a few decades and began with the use of alcohol by Tardieu.4
The first report on the use of BTX-A to treat spasticity in CP children was published in 1993.5 A first meta-analysis of the use of BTX-A in the treatment of lower limb spasticity of CP children was published in 2000,6 reviewing results of randomized controlled trials that showed significant decrease of injected muscle hypertonia, better tolerance and efficacy compared to serial casting.7, 8 Also, randomized controlled trials were performed showing the efficacy of BTX-A injections combined with physiotherapy on pathological gait patterns.9, 10 It is well accepted today that BTX-A administration is of major interest to improve gait patterns of children11, 12, 13, 14 as well as upper limb function.15, 16, 17 However, the studies hardly show any long-term significant improvement of gait. Still, a strong positive impact of BTX-A use in CP children is that incidence and prevalence of orthopaedic surgery are significantly lower for children who receive BTX-A injections.9, 11
The actual number of paediatric patients who could benefit from BTX-A treatment is not known in France. In any case the specialized centers that administer BTX-A to patients are already not numerous enough to manage all the patients, whose treatments are usually delayed by at least six months. This situation is worsened further because of the cost of the treatment.
In France between 2000 and 2009, BTX-A marketing authorization for children restricted its use to the sural triceps in dynamic spastic equinus in ambulant paediatric CP patients, 2 years of age or older. However, spasticity also touches a large number of other muscles in CP children, either in lower or in upper limbs. The present prospective observational study aimed at describing the use of BTX-A in routine clinical practice in French specialized rehabilitation centers, in order to provide up-to-date data on the management of CP spastic children. Herein are described all the aspects of treatment modalities and the efficacy of BTX-A on spasticity and on muscle function.
Section snippets
Population
This prospective observational study included children over a period from January 2004 to December 2005, with a 12-month follow-up period that ended in December 2006. After acceptance, children who needed a specific treatment for focal spasticity (true equinus or other spastic patterns) aged between 2 and 16, who had been previously treated with BTX-A injections or not were included in the study by 11 centers in France (representing all the French centers prescribing BTX-A injections for CP
Population and disease characteristics
A total of 572 children were eligible for BTX-A injections in the centers during the study. Among them, 309 children were included in the study upon acceptance by their parents and 27 children prematurely withdrew from the assessments. Overall, 282 children (54% of paediatric patients treated with BTX-A) were included in the analysis and followed up for 12 months after an initial BTX-A injection. More than half of the children (166/282) included in the study were boys (59%), and their mean age
Discussion
This multi-center and prospective cohort study is the first work about BTX-A use in paediatric CP on such a large population and long-term follow-up, as the other studies were meta-analyses or retrospective studies. The study reported here is observational, with the ambition of describing the current use of BTX-A in children with spastic disorders in France. The results present the advantages of a naturalistic study, showing the actual practices and the gap with the marketing authorization
Acknowledgments/Funding:
This study was funded by the Soutien aux Thérapeutiques Innovantes et Coûteuses 2003, France. The authors wish to thank Audrey Favre for the English editing of the manuscript.
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E. Chaleat-Valayer and B. Parratte contributed equally to this work.