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Master Thesis

Utilisation du ruxolitinib dans la GVH aigüe cortico-résistante de l’enfant, une étude multicentrique du groupe pédiatrique de la SFGM-TC

Abstract : We conducted a national multicenter retrospective study in France to evaluate efficacy and tolerance of ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplant. Patients were recruited from the 15 pediatric transplantation centers in France. Transplanted patients were eligible if they met the following criteria: aged ≤ 18 years at transplantation, receiving a myeloablative allogeneic haematopoietic stem cell transplant irrespective of the stem cell source, having an acute GVHD of grade ≥ 2 and treated with ruxolitinib for steroid refractory acute GVHD. Treatment failure was defined by the absence of improvement of aGVHD, deterioration of aGVHD in any organ by at least one stage, the development of acute GVHD manifestations in a previously unaffected organ, and the use of any additional agents to control the disease. 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 CR with a median delay of 41 days (5 to 93 days). Two patients had a partial response. All patients who achieved CR or PR discontinued corticosteroid treatment. Eight patients showed treatment failure. Overall response rate was 72.4%. Twenty three of 29 patients were alive at a median follow-up of 685 days (177 to 1042 days) after the HSCT. Viral replication (including CMV, EBV and ADV) was observed in 51.7% 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 a huge range (6.3 to 28.7 mg/m2/d). We could not demonstrate any relationship between initial dose and effectiveness. Ruxolitinib may constitute a promising second line treatment for children with steroid-refractory aGVHD that should be validated in prospective large-scale pharmacokinetic and efficacy trial.
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Master Thesis
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Submitted on : Friday, July 2, 2021 - 3:11:14 PM
Last modification on : Thursday, July 8, 2021 - 3:37:12 AM
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  • HAL Id : dumas-03276929, version 1



Louise Laisné. Utilisation du ruxolitinib dans la GVH aigüe cortico-résistante de l’enfant, une étude multicentrique du groupe pédiatrique de la SFGM-TC. Sciences du Vivant [q-bio]. 2019. ⟨dumas-03276929⟩



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