Clinical Sciences/Health Conditions
Amandeep Mann, PhD
Head of Medical Affairs
Ipsen, Cambridge, United States
Cambridge, Massachusetts, United States
Jinming Zhang, MBBS, PhD
Associate Director, Epidemiology and RWE Sciences
Ipsen, Cambridge, United States
Cambridge, Massachusetts, United States
Simon Page, PhD
Global Medical Affairs Director
Ipsen, London, United Kingdom
London, England, United Kingdom
Jonathan Bouchard, MS
Health Economics and Outcomes Research Director
Ipsen, Cambridge, United States
Cambridge, Massachusetts, United States
Mathieu Beneteau, BA
Biometry
Ipsen, Paris, France
Paris, Ile-de-France, France
Billie A. schultz, M.D., MD
Consultant
Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, Minnesota
Rochester, Minnesota, United States
Michael C. Wainberg, MD
Spasticity Section Chair
Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, Minnesota
Rochester, Minnesota, United States
To investigate real-world botulinum toxin A (BoNT-A) treatment persistence in patients with neurological disorders.
Design: Electronic health records (January 2010–June 2023) were used to characterize BoNT-A persistence in adult patients with ≥1 treatment cycle, overall and across mutually-exclusive International Classification of Diseases 9/10-defined cohorts: stroke, migraine, bladder dysfunction (BD), cervical dystonia (CD), and adult cerebral palsy (CP). Index date was first BoNT-A treatment; patients were followed for two years post-index. Persistence/non-persistence were defined by evaluating if all treatment intervals post-index were ≤80th percentile of all patients in the cohort (persistent) or if any were >80th percentile (non-persistent). Reasons for discontinuation were identified by AI natural language processing (NLP) analysis of physicians’ free-text notes, and further stratified by BoNT-A types (onabotulinumtoxinA, incobotulinumtoxinA and abobotulinumtoxinA).
Results: Of 24,212 patients (mean age 52 years, 73.6% female) persistency was evaluable for 23,746; 11.1% were persistent. Cross-cohorts persistency was 5.7% (stroke, 23/407), 7.4% (migraine, 408/5543), 10.5% (BD, 62/593), 10.3% (CD, 102/989), 14.8% (CP, 8/54). Persistent (vs non-persistent) patients were generally younger (stroke, BD) and more often female (stroke, BD, CD); neither trend was seen in the migraine cohort. Mean (95% confidence interval) BoNT-A treatment number was 3.35 (3.32–3.39) cycles overall; lowest in BD (1.99 [1.87–2.11]); highest in migraine (3.90 [3.83–3.98]). Cardiovascular comorbidities were highest for the stroke cohort; depression and anxiety were common across cohorts (17.6–35.4% and 28.5–57.4%, respectively). Cross-cohorts antidepressant use ranged from 16.7–39.5%; higher among persistent (41.0%) vs non-persistent (28.4%) patients overall. Most common reasons for discontinuation across cohorts were patient- or physician-described lack of treatment effectiveness (17.4–40.4% and 12.3–32.6%, respectively); reasons for discontinuation were comparable between BoNT-As.
Conclusion: BoNT-A persistence was low across disease cohorts over a 2-year follow-up. Understanding drivers of discontinuation may inform strategies to manage BoNT-A persistence.
