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Long-term outcomes of ruxolitinib therapy in steroid-refractory graft-versus-host disease in children and adults


Acute and chronic steroid-refractory graft-versus-host disease (srGVHD) is a life-threatening complication of allogeneic stem cell transplantation. There are a number of reports on case series describing efficacy of ruxolitinib in both acute and chronic srGVHD. We conducted a prospective study (NCT02997280) in 75 patients with srGVHD (32 acute, 43 chronic, 41 adults, and 34 children). Patients with chronic GVHD had severe disease in 83% of cases, and acute GVHD patients had grade III–IV disease in 66% of cases. The overall response rate (ORR) was 75% (95% CI 57–89%) in acute GVHD and 81% (95% CI 67–92%) in chronic. Overall survival was 59% (95% CI 49–74%) in acute group and 85% (95% CI 70–93%). The major risk factors for lower survival were grade III–IV gastrointestinal involvement (29% vs 93%, p = 0.0001) in acute form and high disease risk score in chronic (65% vs 90%, p = 0.038). Toxicity was predominantly hematologic with 79% and 44% of grade III–IV neutropenia in acute and chronic groups, respectively. There was no difference between adults and children in terms of ORR (p = 0.31, p = 0.35), survival (p = 0.44, p = 0.12) and toxicity (p > 0.93). The study demonstrated that ruxolitinib is an effective option in acute and chronic srGVHD and can be used both in adults and children.

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The study was supported clinical approbation program No.2016–29–1 by the Ministry of Health of Russian Federation. The laboratory assays were performed with the support of Russian Science Fund, grant No 17–75–20145. We thank Valerii Beklenischev for managing the frozen samples.

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Correspondence to I. S. Moiseev.

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ISM received consultancy and lecturer honoraria from Novartis. EVM received lecturer honoraria from Novartis. The other authors have no conflicts of interest.

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Moiseev, I.S., Morozova, E.V., Bykova, T.A. et al. Long-term outcomes of ruxolitinib therapy in steroid-refractory graft-versus-host disease in children and adults. Bone Marrow Transplant 55, 1379–1387 (2020).

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