特立氟胺对成人T细胞白血病病毒1型相关脊髓病/热带痉挛性截瘫患者临床病程及实验室检查结果的安全性和有效性：一项三盲研究
Safety and efficacy of teriflunomide on clinical course, and laboratory findings in patients with HTLV-1-associated myelopathy/tropical spastic paraparesis: a triple-blind study.
作者信息
Jozan Neda Ghadiri, Vahidi Zohreh, Rafatpanah Houshang, Boostani Reza, Zemorshidi Fariba, Sahraian Mohammadali, Tabeshpour Jamshid, Baghaei Mahdieh, Nahayati Mohammadali
机构信息
Department of Neurology, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.
Inflammation and Inflammatory Diseases Division, Immunology Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
出版信息
J Neurol. 2025 May 10;272(6):386. doi: 10.1007/s00415-025-13134-9.
BACKGROUND
HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP) is a chronic inflammatory disease of the central nervous system (CNS). Teriflunomide is an oral agent developed for the treatment of multiple sclerosis (MS) by suppressing the proliferation of autoreactive lymphocytes. This study was conducted to evaluate the efficacy of teriflunomide in HAM/TSP patients in Northeast Iran.
METHODS
This study was a triple-blind, randomized, placebo-controlled trial involving 22 patients with HAM/TSP. The intervention group (n = 11) received one tablet of teriflunomide (14 mg daily), while the control group (n = 11) received one placebo tablet for 12 months. Muscle strength, spasticity, motor disability, urinary disorders, walking speed, laboratory factors, and drug complications were examined during the study.
RESULTS
In the intervention group, consumption of teriflunomide decreased the duration of walking according to the T25FW test (p = 0.01). The severity of OMDS disability also significantly decreased (P < 0.001). Additionally, the total score of UDS in the intervention group decreased. The levels of HTLV-1 proviral load significantly decreased (p = 0.003). No adverse effects were observed, and the increase in liver enzyme levels was tolerable and controllable.
CONCLUSIONS
Teriflunomide effectively reduced the proviral load, improved the severity of disability and walking speed, and better controlled urinary and constipation symptoms without any adverse effects. Therefore, teriflunomide can be considered a disease-modifying therapy for patients with HAM/TSP. However, further studies with a large number of patients and longer duration, along with the determination of specific HAM/TSP-associated biomarkers, are needed to validate the results of the present study.
TRIAL REGISTRATION
IRCT20180618040127N3; November 19, 2021.
背景
人类嗜T淋巴细胞病毒1型相关脊髓病/热带痉挛性截瘫（HAM/TSP）是一种中枢神经系统（CNS）的慢性炎症性疾病。特立氟胺是一种口服药物，通过抑制自身反应性淋巴细胞的增殖来治疗多发性硬化症（MS）。本研究旨在评估特立氟胺在伊朗东北部HAM/TSP患者中的疗效。
方法
本研究是一项三盲、随机、安慰剂对照试验，涉及22例HAM/TSP患者。干预组（n = 11）每天服用一片特立氟胺（14毫克），而对照组（n = 11）服用一片安慰剂，持续12个月。在研究期间检查肌肉力量、痉挛、运动功能障碍、泌尿系统疾病、步行速度、实验室指标和药物并发症。
结果
在干预组中，根据T25FW测试，服用特立氟胺缩短了步行时间（p = 0.01）。OMDS残疾严重程度也显著降低（P < 0.001）。此外，干预组UDS总分降低。HTLV-1前病毒载量水平显著降低（p = 0.003）。未观察到不良反应，肝酶水平的升高是可耐受且可控的。
结论
特立氟胺有效降低了前病毒载量，改善了残疾严重程度和步行速度，并更好地控制了泌尿系统和便秘症状，且无任何不良反应。因此，特立氟胺可被视为HAM/TSP患者的疾病修饰治疗药物。然而，需要进行更多患者、更长时间的进一步研究，并确定特定的HAM/TSP相关生物标志物，以验证本研究结果。
试验注册
IRCT20180618040127N3；2021年11月19日。
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