C3与pegcetacoplan抑制与阵发性夜间血红蛋白尿eculizumab处理对象。

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德卡斯特罗C, Grossi F, Weitz IC, Maciejewski J, Sharma V,罗马E,布罗斯基RA,谭L, Di Casoli C, El Mehdi D, Deschatelets P,弗朗索瓦•C

C3与pegcetacoplan抑制与阵发性夜间血红蛋白尿eculizumab处理对象。

J内科杂志。2020年11月,95 (11):1334 - 1343。doi: 10.1002 / ajh.25960。Epub 2020年9月11日。

PubMed ID

33464651 (在PubMed

]

文摘

阵发性夜间血红蛋白尿(PNH)是一种获得,危及生命的血液疾病,其特征是慢性complement-mediated溶血和血栓形成。尽管eculizumab治疗,C5抑制剂,72%的人仍然乏力。Pegcetacoplan(可调整规划贷款二期项目),聚乙二醇C3抑制剂,有可能提供更完整的溶血控制PNH患者。这非盲、阶段Ib研究旨在评估安全、耐受性和药物动力学的pegcetacoplan科目PNH eculizumab治疗期间保持脆弱。药效学端点也评估作为一种探索性研究的目标。数据提出了六个学科组4人接受治疗长达2年。总共有427治疗诱发不良事件(流泪)报道,68年的可能与研究药物有关。八个严重流泪发生在两个科目;这三个事件被认为可能与研究药物有关。Pegcetacoplan药代动力学与重复剂量浓度累积,并在大约6 - 8周时达到稳定状态。 Lactate dehydrogenase levels were well controlled by eculizumab at baseline. Pegcetacoplan increased hemoglobin levels and decreased both reticulocyte count and total bilirubin in all six subjects. Improvements were observed in Functional Assessment of Chronic Illness Therapy Fatigue scores. Two subjects discontinued for reasons unrelated to pegcetacoplan. All four subjects who completed the study transitioned to pegcetacoplan monotherapy following eculizumab discontinuation and avoided transfusions. In this small study, pegcetacoplan therapy was generally well-tolerated, and resulted in an improved hematological response by achieving broad hemolysis control, enabling eculizumab discontinuation.

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药物

Pegcetacoplan