Botulinum toxin use in patients with neurological disorders: A U.S.-based claims database analysis.

[BACKGROUND AND OBJECTIVE] Real-world evidence describing long-term persistency with botulinum toxin (BoNT) therapy is limited. We assessed treatment patterns and persistency with BoNT in clinical practice over 2 years.

[DESIGN] Retrospective, longitudinal, claims database analysis.

[SETTING] Medical claims data from the Merative MarketScan database in the United States.

[PARTICIPANTS AND INTERVENTION] Patients with an index medical claim for any BoNT therapy between October 1, 2016 and October 31, 2019, and at least 2 follow-up years.

[MAIN OUTCOMES MEASURES] Number of treatments, time between treatments, and persistency (based on a 120-day look-forward from treatment date) were evaluated over the 2-year follow-up period. Using the 120-day look-forward from treatment date, 31.1% of all patients (n = 19,711) received only one BoNT injection cycle in the 2-year follow-up, indicating a notable drop in the proportion of patients who persisted with BoNT treatment. This prompted further analysis of cohorts by indication: patients with adult spasticity (n = 1912), pediatric spasticity (n = 638), stroke (n = 611), cervical dystonia (n = 688), migraine (n = 7778), or bladder dysfunction (n = 1791) diagnoses. [Correction added on 02 April 2026, after first online publication: In the previous sentence, "decrease" has been changed to "drop" in this version.] RESULTS: A large proportion of patients treated with BoNT stopped after 1-2 cycles. Across cohorts, the mean (SD) number of treatments administered was 2.1-4.7 (1.0-3.0). The time between treatments (80th percentile reported) ranged from 105 days to 319 days depending on the cohort.

[CONCLUSIONS] Increased understanding of BoNT treatment persistency in clinical practice is essential to help to optimize patient care. This study opens the discussion and field for future studies about why many patients discontinue BoNT therapy after only one or two treatment cycles. Further research will help to determine the underlying reasons for cessation of treatment.