Table_1_Erenumab for Migraine Prevention in a 1-Year Compassionate Use Program: Efficacy, Tolerability, and Differences Between Clinical Phenotypes.DOCX

During a 1-year compassionate use program, 156 patients with migraine self-administered a monthly dose of erenumab 140 mg with a subcutaneous autoinjector. Main inclusion criteria were: ≥ 4 migraine days/month and ≥two prior prophylactic treatment failures. The patients covered the migraine severity spectrum from episodic migraine (EM) (n = 80) to chronic migraine (CM) (n = 76). During the 3rd month of treatment, monthly headache days decreased by 45.7% in EM and 35.5% in CM. The 50% responder rate for reduction in monthly headache days was significantly higher in EM (55%) than in CM (43%) (p = 0.05). In both the migraine subgroups, the clinical improvement vs. baseline was already significant during the 1st month of treatment (p < 0.001). There were also significant reductions in mean headache severity, duration, and monthly days with acute drug intake. The 30% responder rate at 3 months was 60% in CM and 54.1% of patients reversed from CM to EM. The therapeutic effect was maintained at 12 months when 50% responder rates, considering discontinuation for lack of efficacy or adverse effects as 0% response, still were 51% in EM and 41% in CM. A total of 10 patients with EM (12.5%) and 23 patients with CM (30.3%) had discontinued treatment, considering the treatment as ineffective. At 3 months, 48% of patients reported non-serious adverse events among which the most frequent was constipation (20.5%); corresponding figures at 12 months were 30 and 15%. Discontinuation due to an adverse effect for the entire 12 month period was rare (3.8%). The lower efficacy in CM than in EM was mainly due to a very low 50% responder rate in patients with CM with continuous pain (13%) as compared to CM with pain-free periods (58%) (p < 0.001). Similarly, the 50% responder rate was lower in patients with ≥two prior prophylactic treatment failures (40.5%) compared to those with two failures (70%) (p < 0.05). There was no significant efficacy difference between low (4–7 migraine days/month, n = 22) and high frequency (8–14 days, n = 59) EM nor between patients with CM with (n = 50) or without (n = 26) acute medication overuse. Erenumab had no effect on the frequency of auras. Taken together, erenumab 140 mg monthly was highly effective for migraine prophylaxis over the whole severity spectrum of the disease, except in patients with continuous headaches. Its effect is significant after the first injection, quasi-maximal after the second injection, and does not wear off after 12 months. The most frequent adverse effect was constipation. These results are compared to those published for erenumab in the pivotal randomized placebo-controlled trials and to those reported in several recent real-world studies.

在一项为期一年的同情使用项目中，156名患有偏头痛的患者通过皮下自动注射器自行每月给予厄洛替尼单抗140毫克。主要纳入标准为：每月≥4天偏头痛发作和≥两次预防性治疗失败。患者涵盖了从周期性偏头痛（EM）（n = 80）到慢性偏头痛（CM）（n = 76）的偏头痛严重程度范围。在治疗的第3个月，EM组每月头痛天数减少了45.7%，CM组减少了35.5%。减少每月头痛天数的50%响应率在EM组（55%）显著高于CM组（43%）（p = 0.05）。在两组偏头痛患者中，与基线相比，临床改善在治疗的第1个月就已经显著（p < 0.001）。平均头痛严重程度、持续时间和每月急性药物摄入天数也均有显著降低。3个月时的30%响应率为CM组60%，其中54.1%的患者从CM逆转为EM。在12个月时，考虑因疗效不足或不良反应而停药的患者不计为响应，50%的响应率在EM组仍为51%，在CM组为41%。总共有10名EM患者（12.5%）和23名CM患者（30.3%）因认为治疗无效而中断治疗。在3个月时，48%的患者报告了非严重不良事件，其中最常见的是便秘（20.5%）；在12个月时的相应数字为30和15%。在整个12个月期间，因不良反应而停药的情况很少（3.8%）。与CM组中持续疼痛的患者（13%）相比，CM组中无疼痛期患者（58%）的50%响应率非常低（p < 0.001）。同样，≥两次预防性治疗失败的患者（40.5%）的50%响应率低于两次失败的患者（70%）（p < 0.05）。在低频（每月4-7天偏头痛发作，n = 22）和高频（每月8-14天，n = 59）EM患者之间，以及在CM患者中有（n = 50）或无（n = 26）急性药物滥用之间，均没有显著的疗效差异。厄洛替尼对视觉症状的频率没有影响。综合来看，厄洛替尼140毫克每月一次在治疗偏头痛的整个严重程度范围内均显示出高度的有效性，除对持续头痛患者无效外。其效果在首次注射后显著，在第二次注射后几乎达到最大，并在12个月后不会减弱。最常见的不良反应是便秘。这些结果与厄洛替尼在关键随机安慰剂对照试验中发布的结果以及最近几项现实世界研究的报告结果进行了比较。