Department of Physical Medicine and Rehabilitation, Dong-A University College of Medicine, Busan, Republic of Korea.
Department of Rehabilitation Medicine, School of Medicine, Kyungpook National University, Kyungpook National University Chilgok Hospital, Daegu, Republic of Korea.
J Neurol. 2021 Sep;268(9):3344-3351. doi: 10.1007/s00415-021-10503-y. Epub 2021 Mar 6.
This study aimed to investigate the effect of androgen suppression therapy using leuprorelin focused on the bulbar function of patients with spinal and bulbar muscular atrophy (SBMA).
Genetically confirmed SBMA patients who consented to participate in this observational study were enrolled. Leuprorelin was subcutaneously injected every 12 weeks. Videofluoroscopic swallowing study was performed at baseline and after androgen suppression therapy for 1 year. The primary outcome measures were the changes in the vallecular residue and pyriform sinus residue. The videofluoroscopic swallowing study data were analyzed and interpreted by two experienced physiatrists.
A total of 40 patients with SBMA were analyzed in this study. The inter-rater reliability testing showed good agreement for the pharyngeal residue (ICC = 0.84) and videofluoroscopic dysphagia scale (ICC = 0.75). The vallecular residue and pyriform sinus residue after swallowing 9 mL yogurt were significantly reduced (26.8 ± 22.6 to 14.6 ± 14.5, p < 0.001, 14.9 ± 16.9 to 7.6 ± 9.9, p < 0.001, respectively). The swallowing subscore of amyotrophic lateral sclerosis functional rating scale-revised improved after androgen suppression therapy (3.3 ± 0.5 to 3.5 ± 0.6, p = 0.041).
Leuprorelin significantly reduced the pharyngeal residue in patients with SBMA after 1 year of treatment without any serious adverse events and longitudinal studies are needed to confirm these results.
本研究旨在探讨亮丙瑞林雄激素抑制治疗对脊髓延髓肌萎缩症（SBMA）患者球部功能的影响。
纳入同意参加这项观察性研究的遗传性 SBMA 患者。亮丙瑞林每 12 周皮下注射一次。在基线和雄激素抑制治疗 1 年后进行视频荧光透视吞咽研究。主要结局指标为会厌谷残留和梨状窦残留的变化。由两名经验丰富的物理治疗师对视频荧光透视吞咽研究数据进行分析和解释。
本研究共分析了 40 例 SBMA 患者。两位有经验的物理治疗师对咽后残留物的评估具有良好的组内一致性（ICC=0.84）和视频荧光透视吞咽障碍量表（ICC=0.75）。吞咽 9 毫升酸奶后会厌谷残留和梨状窦残留明显减少（26.8±22.6 至 14.6±14.5，p<0.001；14.9±16.9 至 7.6±9.9，p<0.001）。改良肌萎缩侧索硬化功能评定量表吞咽亚评分在雄激素抑制治疗后得到改善（3.3±0.5 至 3.5±0.6，p=0.041）。
亮丙瑞林治疗 1 年后，SBMA 患者的咽部残留物明显减少，且无严重不良事件。需要进行纵向研究来证实这些结果。
