Clinical Sciences/Health Conditions
Tomoko Sugiyama, MD
Attending Physician
Chiba prefectural Chiba Rehabilitation Center
Koto-ku, Tokyo, Japan
Ryo Tanabe, MD
Director of Pediatric Neurology
Chiba Prefectural Chiba Rehabilitation Center
Chiba, Chiba, Japan
Mitsuko Ishii, MD
Director of the Comprehensive Developmental Support Center
Chiba Prefectural Chiba Rehabilitation Center
Chiba, Chiba, Japan
Botulinum toxin therapy has become widely adopted for spasticity management in children with cerebral palsy, and reports regarding its long-term outcomes are gradually increasing. However, reports on the very long-term course, extending beyond five years, remain limited. This study analyzed long-term treatment outcomes over six years to evaluate continuation rates and factors influencing treatment decisions.
Design:
We conducted a retrospective analysis of 351 patients who received botulinum toxin therapy at the Chiba Rehabilitation Center pediatric spasticity clinic between April 2019 and August 2025. Treatment continuation rates and reasons for discontinuation were evaluated based on Gross Motor Function Classification System (GMFCS) levels.
Results:
The patient distribution showed a predominance of severe cases: GMFCS Level I (n=34), II (n=38), III (n=35), IV (n=62), and V (n=182). The mean age in April 2019 was 14.5±9.5 years, with approximately one-third of the cohort being 18 years of age or older. The six-year continuation rates were 50% for the GMFCS I/II/III group (n=107) and 68% for the GMFCS IV/V group (n=244). Among GMFCS I/II/III patients, 40% discontinued treatment, with 20% transitioning to orthopedic surgery. Conversely, 73% of GMFCS IV/V patients continued treatment, often resuming therapy after lower limb surgery, frequently in combination with intrathecal baclofen therapy.
Conclusion:
Treatment outcomes and the subsequent role of botulinum toxin therapy varied by GMFCS level. In GMFCS I/II/III cases, treatment often led to a transition to definitive surgery or was discontinued due to natural functional improvement. For individuals with GMFCS IV/V, botulinum toxin therapy proved to be a valuable long-term strategy for spasticity management, yielding comprehensive benefits that include not only functional improvement but also a reduction in caregiver burden and enhancement of patient comfort.