Department of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Chang Gung Memorial Foundation-Linkou, Taiwan.
J Formos Med Assoc. 2021 Jan;120(1 Pt 2):542-550. doi: 10.1016/j.jfma.2020.07.002. Epub 2020 Jul 12.
BACKGROUND/PURPOSE: Multiple sclerosis is classified as a rare disease in Taiwan. This study evaluated the safety and effectiveness of fingolimod in patients with relapsing-remitting multiple sclerosis (RRMS) from routine clinical practice in Taiwan.
In this retrospective, multicentre, observational study, we collected clinical data of patients treated with fingolimod 0.5 mg/day in routine clinical practice between September 2012 and December 2015. Primary outcome was the overall safety of fingolimod; secondary outcome was the annualized relapse rate (ARR).
Overall, 62/69 (86.1%) patients were on fingolimod by the end of data collection period. Mean age (±standard deviation [SD]) at inclusion was 37.7 ± 10.10 years; mean duration of MS was 5.4 ± 4.52 years and mean duration of fingolimod exposure was 135.8 patient-years. The most common adverse events (AEs) were bradycardia (21.7%; first-dose related), upper respiratory tract infection, dizziness, and hypoaesthesia (numbness) (11.6% each), followed by urinary tract infection and back pain (7.2% each). Seven patients had liver enzyme-related AEs. Eight patients had absolute lymphocyte counts <0.2 × 10/uL over the study period. One patient developed second degree AV block after first-dosing. Serious AEs were observed in 11 patients (15.9%; mild-to-moderate). No newly developed macular oedema was detected. The ARR was 0.3 ± 0.74 in fingolimod-treated patients and 66.7% of patients were relapse-free. The mean (SD) change from baseline in expanded disability status scale score was -0.30 ± 1.353.
Fingolimod 0.5 mg/day treatment with an average of 2 years of exposure was associated with a manageable safety profile, and maintained/improved effectiveness in RRMS patients from Taiwan.
背景/目的：多发性硬化症在台湾被归类为罕见疾病。本研究评估了在台湾常规临床实践中使用 fingolimod 治疗复发缓解型多发性硬化症（RRMS）患者的安全性和有效性。
在这项回顾性、多中心、观察性研究中，我们收集了 2012 年 9 月至 2015 年 12 月期间接受 fingolimod 0.5mg/天治疗的患者的临床数据。主要结局是 fingolimod 的总体安全性；次要结局是年化复发率（ARR）。
总体而言，在数据收集结束时，69 例患者中有 62 例（86.1%）正在接受 fingolimod 治疗。纳入时的平均年龄（±标准差[SD]）为 37.7±10.10 岁；多发性硬化症的平均病程为 5.4±4.52 年，fingolimod 暴露的平均时间为 135.8 患者年。最常见的不良事件（AE）是心动过缓（21.7%；与首剂量相关）、上呼吸道感染、头晕和感觉迟钝（麻木）（各 11.6%），其次是尿路感染和背痛（各 7.2%）。7 例患者出现肝酶相关 AE。研究期间，有 8 例患者的绝对淋巴细胞计数<0.2×10/uL。1 例患者在首次给药后出现 II 度房室传导阻滞。11 例患者（15.9%；轻度至中度）发生严重 AE。未发现新的黄斑水肿。fingolimod 治疗组的 ARR 为 0.3±0.74，66.7%的患者无复发。扩展残疾状况量表评分从基线的平均（SD）变化为-0.30±1.353。
在台湾， fingolimod 0.5mg/天的治疗，平均暴露时间为 2 年，与可管理的安全性相关，并维持/改善 RRMS 患者的疗效。
