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计划中，156名患有偏头痛的患者使用皮下自动注射器每月自行注射厄洛替尼单抗140毫克。主要纳入标准为：每月≥4天偏头痛发作和≥两次预防治疗失败。患者涵盖了从发作性偏头痛（EM）（n = 80）到慢性偏头痛（CM）（n = 76）的偏头痛严重程度谱。在治疗的第3个月，EM组的每月头痛天数减少了45.7%，CM组减少了35.5%。在减少每月头痛天数方面，50%的响应率在EM组（55%）中显著高于CM组（43%）（p = 0.05）。在偏头痛的两组患者中，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有所显著减少。3个月时的30%响应率为CM组的60%和从CM转为EM的患者的54.1%。在12个月时，考虑由于疗效不足或不良事件而停药的情况，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%）因认为治疗无效而停止治疗。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%）；12个月时的相应数字为30和15%。在整个12个月期间，因不良事件而停药的情况很少（3.8%）。与持续头痛的CM患者（13%）相比，CM患者中50%的响应率非常低（13%），而CM患者中无疼痛期的50%（p < 0.001）。同样，≥两次预防治疗失败的患者的50%响应率（40.5%）低于两次治疗失败的患者（70%）（p < 0.05）。在低频（每月4-7天，n = 22）和高频（每月8-14天，n = 59）EM患者之间，以及在CM患者中是否使用急性药物过度（n = 50）或未使用（n = 26）之间，均没有显著的治疗效果差异。厄洛替尼对晕厥的发生频率没有影响。综合来看，厄洛替尼140毫克每月注射在偏头痛预防方面对整个疾病严重程度谱都非常有效，除了对持续头痛患者无效。其效果在首次注射后显著，在第二次注射后几乎达到最大值，并且在12个月后不会减弱。最常见的不良反应是便秘。这些结果与已发表的厄洛替尼关键随机安慰剂对照试验结果以及最近几项真实世界研究的报道结果进行了比较。