Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: A retrospective multicenter study of the pediatric group of SFGM-TC - Université de Rennes Accéder directement au contenu
Article Dans Une Revue Pediatric Blood and Cancer Année : 2020

Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: A retrospective multicenter study of the pediatric group of SFGM-TC

Résumé

Background We 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. Procedure Patients 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. Results Twenty-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/m(2)/day with an important range. We could not demonstrate any relationship between initial dose and effectiveness. Conclusion Ruxolitinib 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.
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Dates et versions

hal-02927302 , version 1 (22-10-2020)

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Louise Laisne, Bénédicte Neven, J-H Dalle, Claire Galambrun, Maxime Esvan, et al.. Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: A retrospective multicenter study of the pediatric group of SFGM-TC. Pediatric Blood and Cancer, 2020, 67 (9), pp.e28233. ⟨10.1002/pbc.28233⟩. ⟨hal-02927302⟩
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