芦可替尼：慢性移植物抗宿主病的类固醇节约剂。
Ruxolitinib: a steroid sparing agent in chronic graft-versus-host disease.
机构信息
Department of Hematology and Medical Oncology Winship Cancer Institute of Emory, University Atlanta, Atlanta, GA, USA.
Division of Medical Oncology, Washington University School of Medicine, St. Louis, MO, USA.
出版信息
Bone Marrow Transplant. 2018 Jul;53(7):826-831. doi: 10.1038/s41409-017-0081-5. Epub 2018 Jan 24.
Abstract
Inhibition of the Janus-associated kinases (JAK) with ruxolitinib (RUX) reduces graft-versus-host disease (GVHD) in preclinical and clinical models. In total 19 allograft recipients with moderate/severe steroid-dependent chronic GVHD received RUX as ≥2nd line salvage. RUX was well tolerated, and led to complete/partial resolution of oral (92/7%), cutaneous (82/0%), hepatic (71/28%), gastro-intestinal (75/17%), musculoskeletal (33/67%), pulmonary (0/80%), scleroderma (0/75%), vaginal (0/75%), and ocular (0/100%) chronic GVHD. Overall 18 achieved partial response and 1 complete response according to NIH Consensus Criteria. Responses occurred early and were sustained which enabled discontinuation (68%) or reduction of steroids to physiologic doses (21%). We conclude that RUX is an effective steroid-sparing agent in chronic GVHD.
摘要
抑制 Janus 相关激酶（JAK）的芦可替尼（RUX）可减少临床前和临床模型中的移植物抗宿主病（GVHD）。共有 19 名接受同种异体移植且患有中度/重度类固醇依赖型慢性 GVHD 的患者接受了 RUX 作为二线挽救治疗。RUX 耐受性良好，导致口腔（92/7%）、皮肤（82/0%）、肝脏（71/28%）、胃肠（75/17%）、肌肉骨骼（33/67%）、肺（0/80%）、硬皮病（0/75%）、阴道（0/75%）和眼部（0/100%）慢性 GVHD 完全/部分缓解。根据 NIH 共识标准，18 例患者达到部分缓解，1 例完全缓解。反应发生得早且持续，从而能够停用（68%）或减少类固醇至生理剂量（21%）。我们得出结论，RUX 是慢性 GVHD 中一种有效的类固醇节约剂。
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