Blood Cancer Journal (Aug 2024)
Busulfan-fludarabine versus busulfan-cyclophosphamide for allogeneic transplant in acute myeloid leukemia: long term analysis of GITMO AML-R2 trial
- Gianluca Cavallaro,
- Anna Grassi,
- Chiara Pavoni,
- Maria Caterina Micò,
- Alessandro Busca,
- Irene Maria Cavattoni,
- Stella Santarone,
- Carlo Borghero,
- Attilio Olivieri,
- Giuseppe Milone,
- Patrizia Chiusolo,
- Pellegrino Musto,
- Riccardo Saccardi,
- Francesca Patriarca,
- Fabrizio Pane,
- Giorgia Saporiti,
- Paolo Rivela,
- Elisabetta Terruzzi,
- Raffaella Cerretti,
- Giuseppe Marotta,
- Angelo Michele Carella,
- Arnon Nagler,
- Domenico Russo,
- Paolo Corradini,
- Paolo Bernasconi,
- Anna Paola Iori,
- Luca Castagna,
- Nicola Mordini,
- Elena Oldani,
- Carmen Di Grazia,
- Andrea Bacigalupo,
- Alessandro Rambaldi
Affiliations
- Gianluca Cavallaro
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- Anna Grassi
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- Chiara Pavoni
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- Maria Caterina Micò
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- Alessandro Busca
- SSD Trapianto Cellule Staminali, AOU Città della Salute e della Scienza
- Irene Maria Cavattoni
- Ematologia e TMO, Ospedale di Bolzano, Azienda Sanitaria Alto Adige
- Stella Santarone
- Ospedale Civile
- Carlo Borghero
- Dipartimento di Ematologia, Ospedale San Bortolo
- Attilio Olivieri
- Università Politecnica Delle Marche
- Giuseppe Milone
- Hematology and BMT Unit, Azienda Ospedaliero Universitaria Policlinico “G. Rodolico-San Marco”
- Patrizia Chiusolo
- Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS
- Pellegrino Musto
- Department of Precision and Regenerative Medicine and Ionian Area, “Aldo Moro” University School of Medicine, and Unit of Hematology and Stem Cell Transplantation, AOUC Policlinico
- Riccardo Saccardi
- Azienda Ospedaliero Universitaria Careggi
- Francesca Patriarca
- Division of Hematology and Transplant Unit, Azienda Sanitaria Universitaria Friuli Centrale, Department of Medicine, Udine University
- Fabrizio Pane
- Dipartimento di Medicina Clinica e Chirurgia, UOC Ematologia e Trapianti di Midollo, Azienda Ospedaliera Università Federico II
- Giorgia Saporiti
- Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano
- Paolo Rivela
- Division of Hematology, A.O. SS Antonio e Biagio and Cesare Arrigo
- Elisabetta Terruzzi
- Hematology Division, San Gerardo Hospital
- Raffaella Cerretti
- Hematology Division-Stem Cell Transplant Unit, Policlinico Tor Vergata
- Giuseppe Marotta
- Azienda Ospedaliera Universitaria Senese
- Angelo Michele Carella
- Ematologia e Centro Trapianto CSE, Fondazione Casa Sollievo della Sofferenza
- Arnon Nagler
- Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center
- Domenico Russo
- Unit of Blood Diseases and Stem Cell Transplantation, University of Brescia, ASST Spedali Civili
- Paolo Corradini
- Università degli Studi di Milano and Fondazione Istituti di Ricovero e Cura a Carattere Scientifico (IRCSS) Istituto Nazionale dei Tumori
- Paolo Bernasconi
- HSCT Unit, UOC Ematologia, Fondazione IRCCS Policlinico San Matteo
- Anna Paola Iori
- Division of Hematology, AOU Policlinico Umberto I, University “Sapienza”
- Luca Castagna
- UOSD Trapianto di Midollo, Ospedale Villa Sofia Cervello
- Nicola Mordini
- Santa Croce e Carle Hospital, Division of Haematology
- Elena Oldani
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- Carmen Di Grazia
- UO Ematologia e Terapie Cellulari, IRCCS Ospedale Policlinico San Martino
- Andrea Bacigalupo
- Ematologia, Fondazione Universitaria Policlinico Gemelli IRCCS
- Alessandro Rambaldi
- Department of Oncology and Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII
- DOI
- https://doi.org/10.1038/s41408-024-01116-5
- Journal volume & issue
-
Vol. 14,
no. 1
pp. 1 – 7
Abstract
Abstract We report the long-term results of a randomized trial (GITMO, AML-R2), comparing 1:1 the combination of busulfan and cyclophosphamide (BuCy2, n = 125) and the combination of busulfan and fludarabine (BuFlu, n = 127) as conditioning regimen in acute myeloid leukemia patients (median age 51 years, range 40–65) undergoing allogeneic hematopoietic stem cell transplantation. With a median follow-up of 6 years, significantly better non-relapse mortality (NRM) was confirmed in BuFlu recipients, which is sustained up to 4 years after transplant (10% vs. 20%, p = 0.0388). This difference was higher in patients older than 51 years (11% in BuFlu vs. 27% in BuCy2, p = 0.0262). The cumulative incidence of relapse, which was the first cause of death in the entire study population, did not differ between the two randomized arms. Similarly, the leukemia-free survival (LFS) and overall survival (OS) were not different in the two cohorts, even when stratifying patients per median age. Graft-and relapse-free survival (GRFS) in BuFlu arm vs. the BuCy2 arm was 25% vs. 20% at 4 years and 20% vs. 17% at 10 years. Hence, the benefit gained by NRM reduction is not offsets by an increased relapse. Leukemia relapse remains a major concern, urging the development of new therapeutic approaches.
