Objectives To explore the efficacy and safety of ruxolitinib in the treatment of steroid refractory/refractory acute graft versus host disease (aGVHD).

Methods A prospective analysis was conducted in the 31 steroid refractory/refractory aGVHD cases who treated with ruxolitinib from October 2017 to June 2018. The first dose of ruxolitinib was 5-10mg bid, and the dose was adjusted to 5mg qd for maintenance treatment when complete remission (CR) status lasted for 2 weeks after steroid refractory/refractory aGVHD turned into CR.

Results Of all the 29 steroid refractory/refractory aGVHD patients, 15 patients were treated with ruxolitinib in case of disease progression after receiving at least one second-line treatment, and 14 cases directly with ruxolitinib after the occurrence of corticosteroid refractoriness. The time of starting ruxolitinib treatment after the occurrence of aGVHD was 10 (4-81) d, the median time of onset was 8 (3-18) d. The overall response rate was 89.6% (26/29) including 22 complete responses (75.9%), and 4 partial responses (13.8%). 12 patients with thrombocytopenia need to reduce the dose of ruxolitinib, and 1 patient withdrew treatment for obvious bleeding tendency. With the median follow-up 3 (2-8) months after treatment with ruxolitinib, 28 patients survived and 1 died of severe pneumonia related to aGVHD, and the overall survival was 96.6% (28/29).

Conclusion Ruxolitinib is safe and effective in the treatment of steroid refractory/refractory aGVHD, and can be used as a second-line treatment for refractory aGVHD.

Disclosures

No relevant conflicts of interest to declare.

Topics:
graft-versus-host disease, acute, ruxolitinib, steroids, second line treatment, adrenal corticosteroids, bleeding diathesis, complete remission, disease progression, follow-up, glucocorticoids

Author notes

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Asterisk with author names denotes non-ASH members.

© 2018 by The American Society of Hematology
2018
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