• Louise Laisne, Benedicte Neven, Jean-Hugues Dalle, Claire Galambrun, Maxime Esvan, Cecile Renard, Fanny Rialland, Anne Sirvent, Virginie Gandemer, and Pediatric Group of SFGM-TC.
• Department of Pediatric Hematology and Oncology, University Hospital of Rennes, Rennes, France.
• Pediatr Blood Cancer. 2020 Sep 1; 67 (9): e28233.

BackgroundWe conducted a national multicenter retrospective study in France to evaluate the efficacy and tolerance of ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplant.ProcedurePatients were recruited from the 15 pediatric transplantation centers. Transplanted patients were eligible if they met the following criteria: aged ≤ 18 years at transplantation, receiving a myeloablative allogeneic hematopoietic stem cell transplant, having an aGVHD of grade ≥2, and treated with ruxolitinib for steroid-refractory aGVHD.ResultsTwenty-nine patients received ruxolitinib for steroid-refractory aGVHD. Six patients achieved a complete response at day 28 after the start of treatment but finally 19 patients (65.5%) achieved a complete response (CR) with a median delay of 41 days (5-93 days). Two patients had a partial response. All patients who achieved CR or partial response discontinued corticosteroid treatment. Eight patients showed treatment failure. The overall response rate was 72.4%. Twenty-three of 29 patients were alive at a median follow-up of 685 days (177-1042 days) after the hematopoietic stem cell transplantation. Viral replication was observed in 41.4% of cases. We did not observe severe hematological adverse events and cytopenia requiring a modification of ruxolitinib doses always resolved. The median initial dose of ruxolitinib was 12.6 mg/m2 /day with an important range. We could not demonstrate any relationship between initial dose and effectiveness.ConclusionRuxolitinib may constitute a promising second-line treatment for children with steroid-refractory aGVHD that should be validated in a prospective large-scale pharmacokinetic and efficacy trial.© 2020 Wiley Periodicals, Inc.

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