Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study.

Autor:	Bader P; Goethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany., Pötschger U; St. Anna Children's Cancer Research Institute, Vienna, Austria., Dalle JH; Pediatric Hematology and Immunology Department, Robert Debré Hospital, Groupe Hospitalo-Universitaire Assistance Publique Hôpitaux de Paris (GHU AP-HP) Nord, Université Paris Cité, Paris, France., Moser LM; Goethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany., Balduzzi A; Università degli Studi di Milano-Fondazione, FONDAZIONE MONZA E BRIANZA PER IL BAMBINO E LA SUA MAMMA (MBBM), Department for Pediatric Hematology and Oncology, Monza, Italy., Ansari M; CANSEARCH Research Platform for Pediatric Oncology and Hematology, Faculty of Medicine, Department of Pediatrics, Gynecology and Obstetrics, University of Geneva, Switzerland.; Division of Pediatric Oncology and Hematology, Department of Women, Child and Adolescent, University Geneva Hospitals, Geneva, Switzerland., Buechner J; Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway., Güngör T; Department of Hematology/Oncology/Immunology, Gene Therapy, and Stem Cell Transplantation, University Children's Hospital Zürich, Eleonore Foundation & Children's Research Center, Zürich, Switzerland., Ifversen M; Copenhagen University Hospital Rigshospitalet, Department for Pediatric Hematology and Oncology, Copenhagen, Denmark., Krivan G; Pediatric Hematology and Stem Cell Transplantation Department, Central Hospital of Southern Pest, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Pichler H; St. Anna Children's Hospital, University Vienna, Vienna, Austria., Renard M; Department of Paediatric Oncology, University Hospital Leuven, Leuven, Belgium., Staciuk R; Hospital de Pediatría 'Prof. Dr. Juan P. Garrahan,' Buenos Aires, Buenos Aires, Argentina., Sedlacek P; Department of Pediatric Hematology and Oncology, Motol University Hospital, Prague, Czech Republic., Stein J; Schneider Children's Medical Center of Israel and Sackler Faculty of Medicine, Tel Aviv University, Petah Tikva, Israel., Heusel JR; Goethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany., Truong T; Division of Pediatric Oncology and Cellular Therapy, Alberta Children's Hospital, Calgary, Alberta, Canada., Wachowiak J; Department of Pediatric Oncology, Hematology and Transplantology, Poznań University of Medical Sciences, Poznań, Poland., Yesilipek A; Medical Park Antalya Hospital, Antalya, Turkey., Locatelli F; Department of Pediatric Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy., Peters C; St. Anna Children's Cancer Research Institute, Vienna, Austria.; St. Anna Children's Hospital, University Vienna, Vienna, Austria.
Jazyk:	angličtina
Zdroj:	Blood advances [Blood Adv] 2024 Jan 23; Vol. 8 (2), pp. 416-428.
DOI:	10.1182/bloodadvances.2023010591
Abstrakt:	Abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129. (© 2024 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.)
Databáze:	MEDLINE
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