Clinical practice guidelines on botulinum toxin for spasticity in children with cerebral palsy

Project Summary This research project represents a comprehensive evidence synthesis initiative designed to inform the development of high-quality clinical practice guidelines for botulinum neurotoxin (BoNT) use in children and adolescents with cerebral palsy (CP). The project employs a dual methodological approach: (1) an umbrella review systematically synthesizing existing systematic reviews and clinical practice guidelines, and (2) targeted systematic reviews of primary randomized controlled trials addressing specific clinical questions. Purpose The primary purpose is to consolidate the current evidence base on BoNT for spasticity management in pediatric CP populations through rigorous systematic methodology. Specifically, the project aims to: Synthesize existing evidence syntheses: Evaluate the quality, conclusions, and recommendations from published systematic reviews and evidence-based clinical practice guidelines concerning BoNT efficacy, effectiveness, and safety Map patient-important outcomes: Systematically examine evidence on functional outcomes, participation, pain, quality of life, and caregiver burden—outcomes that matter most to children with CP and their families Assess methodological rigor: Apply validated quality appraisal instruments (AMSTAR 2 for systematic reviews; AGREE II for guidelines) to evaluate the trustworthiness of existing evidence syntheses Identify evidence gaps: Determine areas where additional primary research is needed and where existing evidence requires updating Inform guideline development: Provide a consolidated, readily actionable evidence map to guide the prioritization and scoping of key clinical questions for new, high-quality clinical practice guideline development Methodological Approach The project utilizes the umbrella review methodology—a systematic review of systematic reviews and guidelines—combined with focused systematic reviews of primary studies. This approach adheres to international reporting standards (PRISMA-P for protocols; PRIOR for the final umbrella review) and incorporates: Comprehensive database searching (Epistemonikos, MEDLINE, Cochrane Database of Systematic Reviews) without language or date restrictions Targeted gray literature searches of guideline repositories and professional societies Duplicate independent screening, data extraction, and quality assessment by trained reviewers Standardized, validated quality appraisal tools GRADE (Grading of Recommendations Assessment, Development and Evaluation) methodology to evaluate certainty of evidence Expected Outcomes This research is expected to produce several important deliverables: Comprehensive evidence map: A structured synthesis of all available systematic reviews and guidelines on BoNT for pediatric CP-related spasticity, organized by clinical question, population characteristics, and outcome domains Quality assessment profile: A critical appraisal of the methodological rigor of existing evidence syntheses, identifying which reviews and guidelines can be confidently used to inform clinical decision-making Recommendation synthesis: A systematic compilation and comparison of clinical recommendations across multiple guidelines, highlighting areas of consensus and controversy Evidence gap identification: Clear delineation of questions where evidence is absent, insufficient, or outdated, directly informing future research priorities Foundation for guideline development: A consolidated evidence base structured specifically to support the development of new, evidence-based clinical practice recommendations using GRADE Evidence-to-Decision frameworks Clinical and research impact: By synthesizing evidence on patient-important outcomes beyond traditional clinical measures (e.g., muscle tone), this work will help shift the focus of CP spasticity management toward outcomes that meaningfully impact children's daily function, participation, and quality of life Significance This research addresses a critical need in pediatric rehabilitation medicine. Despite two decades of research on BoNT for CP-related spasticity and multiple published guidelines, clinicians face challenges navigating the extensive, sometimes contradictory literature. This project will provide a definitive, methodologically rigorous synthesis that consolidates existing knowledge, critically appraises its quality, and creates a clear roadmap for both clinical practice improvement and future research—ultimately supporting evidence-based, patient-centered care for children and adolescents living with cerebral palsy.