Trends and off-label use of botulinum toxin type A in Chinese neurological practice from 2016 to 2023: a real-world study.

BACKGROUND: Botulinum toxin type A (BoNT-A) is an effective therapeutic tool for various neurological conditions, but it is frequently prescribed off-label in clinical practice. Currently, real-world prescribing trends and off-label utilization in China remain unclear. This study aimed to describe the trends in BoNT-A use, analyze off-label prescribing patterns, and evaluate the supporting scientific evidence. METHODS: In this retrospective study, prescription data were extracted to analyze patient demographics, monotherapy and co-prescribing therapy of BoNT-A, and trends in BoNT-A usage from 2016 to 2023. We categorized the prescriptions into on-label and off-label groups according to the indications approved by the National Medical Products Administration of China. Additionally, the scientific evidence supporting off-label indications was evaluated based on randomized controlled trials (RCTs). RESULTS: Our study included 9,561 BoNT-A prescriptions from patients with a median age of 59 years. Of these, 35.82% (3,425) were off-label. Over the study period, the number of BoNT-A prescriptions and the proportion of off-label use generally increased. Most prescriptions were issued for dystonia or facial nerve disorders, accounting for 51.00% and 43.48%, respectively. BoNT-A monotherapy comprised 93.54% of prescriptions, whereas among polypharmacy regimens, antiepileptics were the most frequently prescribed comedications. The majority of off-label prescriptions were issued for extrapyramidal and movement disorders, with cervical dystonia being the most frequent specific indication. Based on RCT evidence, 89.99% of off-label prescriptions were evidence-supported, peaking within extrapyramidal and movement disorders. CONCLUSION: This study indicated that BoNT-A prescriptions and off-label prescribing increased in Chinese neurological practice. Although such off-label use was often evidence-supported, unsubstantiated utilization may still raise potential safety concerns, highlighting the need for continuing efforts to align regulatory approvals with evolving clinical practice.