GVHD and GRFS in patients with acute myeloid leukemia undergoing allogeneic HCT with treosulfan- versus reduced-intensity busulfan-based conditioning: a subgroup analysis of a randomized phase 3 trial
Year: 2026
Type: article
Abstract: Acute myeloid leukemia (AML) is the most common indication for allogeneic hematopoietic cell transplantation (alloHCT), yet graft-versus-host disease (GvHD) remains a major post-transplant complication. Conditioning regimens, particularly reduced-intensity approaches, are critical in optimizing outcomes. This subgroup analysis of the phase III MC-FludT.14/L trial compared treosulfan-fludarabine with reduced-intensity busulfan-fludarabine in 352 AML patients (aged 31-70 years) undergoing alloHCT. The primary endpoint was 24-month event-free survival; secondary endpoints included overall survival, GvHD incidence, relapse/progression, and non-relapse mortality. Treosulfan compared to busulfan demonstrated superiority: 24-month event-free survival was 65% vs. 53% (P=0.01), and overall survival was 73% vs. 65%. Event-free survival benefits were consistent across AML risk categories and notably higher in patients with Hematopoietic Cell Transplantation Comorbidity Index score >2 (62% vs. 42%, P=0.02). Treosulfan also showed lower non-relapse mortality and relapse rates. GvHD outcomes favored treosulfan, with a significantly lower incidence of extensive chronic GvHD at 24 months (15.1% vs. 28.1%, P=0.01). GvHD-free and relapse-free survival was also improved (53% vs. 40%, P=0.02). The safety profile was more favorable with treosulfan. These findings support treosulfan-fludarabine as a more effective and safer conditioning regimen than busulfan-fludarabine for AML patients undergoing alloHCT, particularly those at higher risk.
Source: Haematologica
Authors: Friedrich Stölzel, Matthias Stelljes, Dietrich Wilhelm Beelen, Miroslaw Markiewicz, Péter Reményi, Thomas Luft, Fabio Ciceri, Eva-Maria Wagner-Drouet, Christian Junghanss, Christoph Scheid, Francesca Patriarca, Gerard Socié, Inken Hilgendorf, Alessandro Rambaldi, Kerstin Schaefer-Eckart, Domenico Russo, Gerald Wulf, Bertram Glass, Hélène Labussiere-Wallet, Gernot Stuhler, Maria Bieniaszewska, Jochen Casper, Ernst Holler, Fabio Benedetti, Anna Paola Iori, Desiree Kunadt, Jelena Mihajlović, Bettina Schild, Imran Khan, Xieran Li, Wolfgang Bethge
Institutions: Christian-Albrechts-Universität zu Kiel, University Hospital Schleswig-Holstein, University Hospital Carl Gustav Carus, University of Münster, University Hospital Münster, Essen University Hospital, University of Duisburg-Essen, University of Rzeszów, Unified Szent István and Szent László Hospital, Heidelberg University, IRCCS Ospedale San Raffaele, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele, University Medical Center of the Johannes Gutenberg University Mainz, University of Rostock, University Hospital Cologne, University of Udine, Hôpital Saint-Louis, Jena University Hospital, University of Milan, Ospedale Papa Giovanni XXIII, Nuremberg Hospital, Paracelsus Medizinische Privatuniversität, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, University of Brescia, Universitätsmedizin Göttingen, University of Göttingen, Asklepios Klinik St. Georg, Hospices Civils de Lyon, Hôpital Lyon Sud, University of Würzburg, Gdańsk Medical University, Klinikum Oldenburg, University Hospital Regensburg, University of Regensburg, Policlinico Umberto I, Sapienza University of Rome, Technische Universität Dresden, Medac (Germany), University of Tübingen
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,157
Funder: Medac
OpenAlex ID: W7126054465