Ruxolitinib: a steroid sparing agent in chronic graft-versus-host disease

• Type of Material: Article
• Authors: Hanna Khoury, Emory University; Amelia Langston, Emory University; Vamsi Kota, Emory University; Jennifer A. Wilkinson, Emory University; Iskra Pusic, Washington University; Anand Jillella, Emory University; Stephanie Bauer, Washington University; Audrey S Kim, Emory University; Danielle Roberts, Emory University; Zaid Al-Kadhimi, Emory University; Imre Bodo, Emory University; Elliott Winton, Emory University; Martha Arellano, Emory University; John F. DiPersio, Washington University
• Language: English
• Date: 2018-07-01
• Publisher: Springer Nature [academic journals on nature.com]: Hybrid Journals
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2018 Macmillan Publishers Limited, part of Springer Nature.
• Final Published Version (URL): http://dx.doi.org/10.1038/s41409-017-0081-5
• Title of Journal or Parent Work: Bone Marrow Transplantation
• ISSN: 0268-3369
• Volume: 53
• Issue: 7
• Start Page: 826
• End Page: 831
• Grant/Funding Information: None.
• Keywords: WORKING GROUP-REPORT; CONSENSUS DEVELOPMENT PROJECT; CLINICAL-TRIALS; Science & Technology; CRITERIA; Immunology; Transplantation; Oncology; Biophysics; GVHD; Life Sciences & Biomedicine; Hematology
• Research Categories: Health Sciences, Oncology; Health Sciences, Immunology; Health Sciences, Medicine and Surgery

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.