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Ruxolitinib as a Steroid-Sparing Agent for cGVHD

Biol Blood Marrow Transplant; 2017 Mar; Khoury, et al

Ruxolitinib was effective in patients with hematological malignancies who previously experienced steroid-dependent severe chronic graft versus host disease (cGVHD), according to a small study involving 19 individuals.

Participants had received unrelated donor (n=13) or matched sibling (n=6) transplant involving either blood (n=17) or marrow (n=2). After experiencing cGVHD, ruxolitinib was administered over a median of 18 months as a 2nd, 3rd, 4th, or 5th line salvage at 5 mg BID. Dosage was increased to 20 mg BID in 10 patients due to physician preference, patient weight, or cGVHD flare. Among the results:

  • Complete resolution of clinical manifestations of cGVHD was observed in the skin (n=14), mouth (n=13), GI (n=8), liver (n=7), and lungs (n=1).
  • Subjective improvement was reported in sclerodermatous, vaginal, ocular, and musculoskeletal cGVHD.
  • Prednisone therapy was either discontinued (n=15) or reduced (n=4) after a median of 86 days of ruxolitinib treatment.


Khoury H, Kota K, Arellano M, et al. Ruxolitinib as sparing agent for steroid-dependent chronic graft-versus-host disease (cGVHD). Biol Blood Marrow Transplant. 2017; 23(3):S373. doi:10.1016/j.bbmt.2016.12.324.