High-dose biotin (HDB) is a therapy used in non-active progressive multiple sclerosis (PMS). Some reports have suggested that HDB treatment may be associated with an increased risk of relapse. We evaluate the relationship between exposure to HDB for treating PMS and the risk of relapse. We screened for PMS patients prospectively registered in a French regional cohort being part of the OFSEP national registry. In a case-crossover design among patients who received HDB, we first compared number of relapses before and after initiation of HDB. Second, time to the first clinical relapse was compared between patients who received HDB (biotin group) and a control group using a Cox survival analysis after a propensity score (PS) matching (1:1) and inverse probability of treatment weighting (IPTW) method. In the 42 PMS patients who received HDB, the number of relapses was statistically and clinically significant higher after biotin initiation than before biotin initiation (incident rate ratio [IRR] 7.4, 95% confidence interval [CI] 3.5–15.9, p < 0.0001). With the PS matching method, the risk of relapse was significantly higher in the biotin group compared to the control group (hazard ratio [HR] 4.3, 95% CI 1.4–13.3, p = 0.01). The IPTW method with 440 control patients revealed consistent results (HR 5.1, 95% CI 2.3–11.3, p < 0.0001). In our non-randomized study, HDB treatment for PMS was associated with an increased risk of relapse. The follow-up of PMS patients initiating HDB should include careful assessment of clinical and radiological activity to monitor the potential pro-inflammatory effect of biotin.

高剂量生物素 (HDB) 是一种用于非活动性进行性多发性硬化症 (PMS) 的疗法。一些报告表明 HDB 治疗可能与复发风险增加有关。我们评估暴露于 HDB 以治疗 PMS 与复发风险之间的关系。我们筛选了在法国区域队列中前瞻性注册的 PMS 患者，该队列是 OFSEP 国家注册的一部分。在接受 HDB 的患者的病例交叉设计中，我们首先比较了 HDB 开始前后的复发次数。其次，在倾向评分 (PS) 匹配 (1:1) 和治疗加权的逆概率 (IPTW) 后，使用 Cox 生存分析比较接受 HDB（生物素组）和对照组的患者首次临床复发的时间方法。在接受 HDB 治疗的 42 名 PMS 患者中，p  < 0.0001)。使用 PS 匹配方法，与对照组相比，生物素组的复发风险显着更高（风险比 [HR] 4.3，95% CI 1.4-13.3，p  = 0.01）。对 440 名对照患者进行的 IPTW 方法显示出一致的结果（HR 5.1，95% CI 2.3–11.3，p  < 0.0001）。在我们的非随机研究中，HDB 治疗 PMS 与复发风险增加有关。启动 HDB 的 PMS 患者的随访应包括仔细评估临床和放射学活动，以监测生物素的潜在促炎作用。