1. Reduced-toxicity myeloablative conditioning regimen using fludarabine and full doses of intravenous busulfan in pediatric patients not eligible for standard myeloablative conditioning regimens: Results of a multicenter prospective phase 2 trial
- Author
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Fanny Rialland, Audrey Grain, Myriam Labopin, Gerard Michel, Virginie Gandemer, Catherine Paillard, Cécile Pochon, Laurence Clement, Eolia Brissot, Charlotte Jubert, Anne Sirvent, Pierre Simon Rohrlich, Dominique Plantaz, Jean-Hugues Dalle, Mohamad Mohty, Centre hospitalier universitaire de Nantes (CHU Nantes), Centre de Recherche Saint-Antoine (CRSA), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Hôpital de la Timone [CHU - APHM] (TIMONE), Aix Marseille Université (AMU), CHU Pontchaillou [Rennes], Université de Rennes (UR), CHU Strasbourg, Hôpital Brabois, CHU Bordeaux [Bordeaux], CHU Montpellier, Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Centre Hospitalier Universitaire de Nice (CHU Nice), CHU Grenoble, Hôpital Robert Debré, and This trial was supported by the French national cancer institute (INCa) through a grant as part of the 'Programme Hospitalier de Recherche Clinique' (PHRC-Cancer)
- Subjects
MESH: Adolescent ,Transplantation ,MESH: Humans ,Transplantation Conditioning ,Adolescent ,MESH: Busulfan ,[SDV]Life Sciences [q-bio] ,MESH: Child, Preschool ,Hematopoietic Stem Cell Transplantation ,Hematology ,MESH: Prospective Studies ,Young Adult ,MESH: Young Adult ,MESH: Child ,Child, Preschool ,MESH: Vidarabine ,Humans ,Prospective Studies ,Child ,Busulfan ,MESH: Hematopoietic Stem Cell Transplantation ,Vidarabine ,MESH: Transplantation Conditioning - Abstract
International audience; Data regarding the safety and efficacy of reduced-toxicity conditioning regimen (RTC) prior to allogeneic stem cell transplantation (allo-SCT) to treat hematological malignancies in pediatric patients are limited. This prospective multicenter, phase 2 trial investigated a RTC regimen based on the combination of intravenous busulfan (3.2 mg/kg/d x 4 days), fludarabine (30 mg/m2/d x 5 days) and antithymocyte globulin (Thymoglobulin®, Genzyme; 5 mg/kg total dose) with the aim of delivering high dose myeloablation that would allow optimal disease control while minimizing toxicity, in a subgroup of children at very high risk of non-relapse mortality (NRM). The primary endpoint was NRM at 1 year after allo-SCT. A total of 48 high risk patients were included (median age, 13 years; range, 3-24). At 1 year, the cumulative incidence of recurrence/disease progression and NRM were 33% and 8%, respectively. With a median follow-up of 23 months, the Kaplan-Meier estimates of overall survival (OS) and disease-free survival (DFS) at 1 year were 69% and 58%, respectively. We conclude that the RTC regimen used in this prospective trial is safe, with a
- Published
- 2022
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