Hematopoietic stem cell transplantation (HSCT) represents the cornerstone of treatment in pediatric high-risk and relapsed acute myeloid leukemia (AML). The aim of the present study was to compare outcomes of pediatric patients with AML undergoing HSCT using 3 different conditioning regimens: total body irradiation (TBI) and cyclophosphamide (Cy); busulfan (Bu) and Cy; or Bu, Cy, and melphalan (Mel). In this retrospective study, registry data for patients > 2 and <18 years age undergoing matched allogeneic HSCT for AML in first complete remission (CR1) in 204 European Group for Blood and Marrow Transplantation centers between 2000 and 2010 were analyzed. Data were available for 631 patients; 458 patients received stem cells from a matched sibling donor and 173 from a matched unrelated donor. For 440 patients, bone marrow was used as stem cell source, and 191 patients received peripheral blood stem cells. One hundred nine patients received TBICy, 389 received BuCy, and 133 received BuCyMel as their preparatory regimen. Median follow-up was 55 months. Patients receiving BuCyMel showed a lower incidence of relapse at 5 years (14.7% versus 31.5% in BuCy versus 30% in TBICy, P < .01) and higher overall survival (OS) (76.6% versus 64% versus 64.5%, P = .04) and leukemia-free survival (LFS) (74.5% versus 58% versus 61.9%, P < .01), with a comparable nonrelapse mortality (NRM) (10.8% versus 10.5% versus 8.1%, P = .79). Acute graft-versus-host disease (GVHD) grades III and IV but not chronic GVHD, was higher in patients receiving BuCyMel. Older age at HSCT had an adverse impact on NRM and the use of peripheral blood as stem cell source was associated with increased chronic GVHD and NRM as well as lower LFS and OS. Among pediatric patients receiving HSCT for AML in CR1, the use of BuCyMel conditioning proved superior to TBICy and BuCy in reducing relapse and improving LFS.

BMT Statistical Unit European Group for Blood and Marrow Transplantation Office Universite' Pierre et Marie Curie Paris France

Bone Marrow and Stem Cell Transplantation Department King Hussein Cancer Centre Amman Jordan

Bone Marrow Transplant Department Great Ormond Street Hospital London United Kingdom

Haematology Department Royal Hospital for Sick Children Glasgow United Kingdom

Hemato immunology Department Hôpital Robert Debre Paris France

Hematology and Transplantology Department Saint Petersburg State Medical Pavlov University Ratsa Gorbacheva Memorial Children's Institute St Petersburg Russia

Hematology Department Centre Pierre et Marie Curie Alger Algeria

Hematology Immunology SCT Department Astrid Lindgren Children's Hospital Karolinska University Hospital Stockholm Sweden

Hematology Transplantation Department Hôpital St Louis Paris France

Paediatric Haematology and Oncology Department University Hospital Motol Prague Czech Republic

Paediatric Haematology Oncology Department Bristol Royal Hospital for Children Bristol United Kingdom

Paediatrics and Adolescent Medicine Department Rigshospitalet Copenhagen Denmark

Pediatric Hematology and Oncology Department Institut d'Hématologie et d'Oncologie Pédiatriqu Lyon France

Pediatric Hematology Oncology Department Fondazione IRCCS Policlinico San Matteo Pavia Italy

Pediatric Hematology Oncology Department IRCCS Bambino Gesu' Children Hospital Rome Italy

Pediatrics Department Division of Immuno Hematology and Stem Cell Transplantation Leiden University Hospital The Netherlands

Stem Cell Transplantation and Immunology Department Universitätsklinikum Frankfurt Goethe Universität Frankfurt am Main Germany

Stem Cell Transplantation Department St Anna Kinderspital Vienna Austria

Stem Cell Transplantation Department University College Hospital London United Kingdom

Citace poskytuje Crossref.org

Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies