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芦可替尼-ECP 联合治疗难治性重度慢性移植物抗宿主病。

Ruxolitinib-ECP combination treatment for refractory severe chronic graft-versus-host disease.

机构信息

Department of Medicine I, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.

Clinical Trials Unit, Faculty of Medicine, Medical Center - University of Freiburg, Freiburg, Germany.

出版信息

Bone Marrow Transplant. 2021 Apr;56(4):909-916. doi: 10.1038/s41409-020-01122-8. Epub 2020 Nov 17.

Abstract

Glucocorticoid-refractory (SR) chronic (c) graft-versus-host disease (GVHD) is a multisystem immunological disease and the leading cause of non-relapse mortality (NRM) in patients surviving longer than 2 years after allogeneic hematopoietic cell transplantation. Both ruxolitinib (RUX) and extracorporeal photopheresis (ECP) have shown activity for SR-cGVHD which motivated us to treat refractory cGHVD patients with the RUX-ECP combination. In this retrospective survey, 23 patients received RUX-ECP as salvage therapy for SR-cGVHD. The best response (CR or PR) at any time point during treatment was 74% (17/23) including 9% (2/23) CR and 65% (15/23) PR. The 24-months-survival was 75% (CI 56.0-94.1). Newly diagnosed cytopenia occurred in 22% (5/23) and CMV reactivation was observed in 26% (6/23) of the patients. Serum levels of soluble interleukin-2 receptor (sIL-2R) correlated with response. Our retrospective analysis shows that the RUX-ECP combination is safe and has activity in a fraction of patients with SR-cGVHD, which needs validation in a prospective trial.

摘要

糖皮质激素难治性(SR)慢性(c)移植物抗宿主病(GVHD)是一种多系统免疫性疾病,是异体造血细胞移植后存活时间超过 2 年的患者非复发死亡率(NRM)的主要原因。鲁索利替尼(RUX)和体外光化学疗法(ECP)均显示出对 SR-cGVHD 的活性,这促使我们用 RUX-ECP 联合治疗难治性 cGHVD 患者。在这项回顾性调查中,23 名患者接受 RUX-ECP 作为 SR-cGVHD 的挽救性治疗。在治疗过程中的任何时间点,最佳反应(CR 或 PR)为 74%(23 例中的 17 例),包括 9%(23 例中的 2 例)CR 和 65%(23 例中的 15 例)PR。24 个月的生存率为 75%(95%CI56.0-94.1)。22%(5/23)的患者新诊断为血细胞减少症,26%(6/23)的患者发生 CMV 再激活。血清可溶性白细胞介素-2 受体(sIL-2R)水平与反应相关。我们的回顾性分析表明,RUX-ECP 联合治疗在一部分 SR-cGVHD 患者中是安全且有效的,这需要在前瞻性试验中进一步验证。

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