Sutimlimab在寒冷的凝集素疾病。

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罗斯,Barcellini W, D是一个年代,Miyakawa Y,布鲁姆)厘米,米歇尔•M库特DJ, Jilma B, THA Tvedt Fruebis J,江X,林年代,路透社C, Morales-Arias J,霍布斯W, Berentsen年代

Sutimlimab在寒冷的凝集素疾病。

郑传经地中海J。2021年4月8日,384 (14):1323 - 1334。doi: 10.1056 / NEJMoa2027760。

PubMed ID

33826820 (在PubMed

]

文摘

背景:冷凝集素病是一种少见的自身免疫性溶血性贫血的特点通过溶血是由于激活补体的经典。人源化单克隆抗体Sutimlimab, C1蛋白选择性地目标,C1复杂丝氨酸蛋白酶负责激活这个途径。方法:我们进行了一次26周的多中心、非盲、单一群体研究的有效性和安全性评估患者的静脉sutimlimab冷凝集素疾病和最近的一次输血史。复合主要终点是一个规范化的血红蛋白水平12克每公升以上或增加血红蛋白水平的2 g以上每分升从基线,没有红细胞输血和药物禁止的协议。结果:总共有24例登记和接收至少一个剂量的sutimlimab;13例(54%)符合复合主要终点的标准。最小二乘均值增加血红蛋白水平是2.6克/分升的时候治疗评估(周23、25和26)。平均血红蛋白水平超过11 g每分升是维护患者一周3到研究结束的时期。在第三个星期里平均胆红素水平正常化。共有17个病人(71%)没有收到从星期5 - 26周输血。 Clinically meaningful reductions in fatigue were observed by week 1 and were maintained throughout the study. Activity in the classic complement pathway was rapidly inhibited, as assessed by a functional assay. Increased hemoglobin levels, reduced bilirubin levels, and reduced fatigue coincided with inhibition of the classic complement pathway. At least one adverse event occurred during the treatment period in 22 patients (92%). Seven patients (29%) had at least one serious adverse event, none of which were determined by the investigators to be related to sutimlimab. No meningococcal infections occurred. CONCLUSIONS: In patients with cold agglutinin disease who received sutimlimab, selective upstream inhibition of activity in the classic complement pathway rapidly halted hemolysis, increased hemoglobin levels, and reduced fatigue. (Funded by Sanofi; CARDINAL ClinicalTrials.gov number, NCT03347396.).

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药物

Sutimlimab