VEO Specialty Care, Value Evidence Outcome, Japan Medical and Development, GlaxoSmithKline K.K., Tokyo 107-0052, Japan.
PMS Data Management, Value Evidence Outcome, Japan Medical and Development, GlaxoSmithKline K.K., Tokyo 107-0052, Japan.
Toxins (Basel). 2023 Sep 5;15(9):553. doi: 10.3390/toxins15090553.
This postmarketing surveillance study was conducted to evaluate the safety and effectiveness of onabotulinumtoxinA in Japanese patients with laryngeal dystonia (LD). Patients receiving onabotulinumtoxinA for the first time were enrolled and observed for up to 12 months following the first injection. Safety assessment included adverse drug reactions (ADRs), and effectiveness assessments included the Voice Handicap Index-10 (VHI-10) and physician's global assessment (PGA). ADRs were observed in 48 (5.8%) of 834 patients in the safety analysis set, including dysphonia in 43 (5.2%) patients and dysphagia in 7 (0.8%) patients. The change in total VHI-10 score (mean) in 790 patients included in the effectiveness analysis set showed that improvement in adductor LD peaked at 2 months after the first injection, while patients with abductor or mixed LD showed a gradual attenuation of effect 2-4 weeks post-injection. The change in total VHI-10 score in subsequent injections was generally similar to that following the first injection. The overall effectiveness rate according to the PGA was 93.4% (738/790 patients). The results demonstrate that onabotulinumtoxinA is a well-tolerated and effective treatment for LD in real-world clinical practice.
本上市后监测研究旨在评估在日本喉肌张力障碍(LD)患者中使用肉毒毒素 A (onabotulinumtoxinA)的安全性和有效性。首次接受肉毒毒素 A 治疗的患者被纳入研究,并在首次注射后最多观察 12 个月。安全性评估包括药物不良反应(ADR),有效性评估包括嗓音障碍指数-10 (VHI-10)和医生总体评估(PGA)。在安全性分析集中,834 例患者中有 48 例(5.8%)出现 ADR,包括 43 例(5.2%)患者出现发音障碍和 7 例(0.8%)患者出现吞咽困难。790 例有效性分析集患者的总 VHI-10 评分(均值)变化显示,内收肌 LD 在首次注射后 2 个月达到最大改善,而外展肌或混合性 LD 患者的疗效在注射后 2-4 周逐渐减弱。随后注射的总 VHI-10 评分变化通常与首次注射相似。根据 PGA 的总体有效率为 93.4%(738/790 例)。结果表明,在真实临床实践中,肉毒毒素 A 是一种耐受良好且有效的 LD 治疗方法。