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

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...

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Published in:Bone marrow transplantation (Basingstoke) 2018-07, Vol.53 (7), p.826-831
Main Authors: Khoury, Hanna Jean, Langston, Amelia A., Kota, Vamsi K., Wilkinson, Jennifer A., Pusic, Iskra, Jillella, Anand, Bauer, Stephanie, Kim, Audrey S, Roberts, Danielle, Al-Kadhimi, Zaid, Bodo, Imre, Winton, Elliott, Arellano, Martha, DiPersio, John F.
Format: Article
Language:English
Subjects:
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Adult
Aged
Analysis
Cell Biology
Chronic Disease
Dosage and administration
Female
Graft vs Host Disease - drug therapy
Graft vs Host Disease - pathology
Graft-versus-host reaction
Hematology
Humans
Inflammation
Internal Medicine
Intestine
Janus Kinases - pharmacology
Janus Kinases - therapeutic use
Kinases
Male
Medical schools
Medicine
Medicine & Public Health
Middle Aged
Public Health
Pyrazoles - pharmacology
Pyrazoles - therapeutic use
Ruxolitinib
Salvage
Scleroderma
Scleroderma (Disease)
Stem cell transplantation
Stem Cells
Steroid hormones
Steroids
Transplantation
Vagina
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Summary: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.
ISSN:0268-3369
1476-5365
DOI:10.1038/s41409-017-0081-5