Beelen, Dietrich W., Stelljes, Matthias, Remenyi, Peter, Wagner-Drouet, Eva-Maria, Dreger, Peter, Bethge, Wolfgang, Ciceri, Fabio, Stoelzel, Friedrich, Junghanss, Christian, Labussiere-Wallet, Helene, Schaefer-Eckart, Kerstin, Grigoleit, Goetz U., Scheid, Christof, Patriarca, Francesca, Rambaldi, Alessandro ORCID: 0000-0002-3739-7502, Niederwieser, Dietger, Hilgendorf, Inken ORCID: 0000-0003-2038-9730, Russo, Domenico, Socie, Gerard, Holler, Ernst, Glass, Bertram, Casper, Jochen, Wulf, Gerald, Basara, Nadezda, Bieniaszewska, Maria, Stuhler, Gernot, Verbeek, Mareike, La Rocca, Ursula ORCID: 0000-0002-1990-9973, Finke, Juergen, Benedetti, Fabio, Pichlmeier, Uwe, Klein, Anja, Baumgart, Joachim and Markiewicz, Miroslaw ORCID: 0000-0002-8550-8908 (2022). Treosulfan compared with reduced-intensity busulfan improves allogeneic hematopoietic cell transplantation outcomes of older acute myeloid leukemia and myelodysplastic syndrome patients: Final analysis of a prospective randomized trial. Am. J. Hematol., 97 (8). S. 1023 - 1035. HOBOKEN: WILEY. ISSN 1096-8652

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Abstract

The phase III study was designed to compare event-free survival (EFS) after treosulfan-based conditioning with a widely applied reduced-intensity conditioning (RIC) busulfan regimen in older or comorbid patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). A previously reported confirmatory interim analysis of the randomized clinical study including 476 patients demonstrated statistically significant noninferiority for treosulfan with clinically meaningful improvement in EFS. Here, the final study results and pre-specified subgroup analyses of all 570 randomized patients with completed longer-term follow-up are presented. Patients presenting HCT-specific comorbidity index >2 or aged >= 50 years were randomly assigned (1:1) to intravenous (IV) fludarabine with either treosulfan (30 g/m(2) IV) or busulfan (6.4 mg/kg IV) after stratification by disease risk group, donor type, and participating institution. The primary endpoint was EFS with disease recurrence, graft failure, or death from any cause as events. EFS of patients (median age 60 years) was superior after treosulfan compared to RIC busulfan: 36-months-EFS rate 59.5% (95% CI, 52.2-66.1) vs. 49.7% (95% CI, 43.3-55.7) with a hazard ratio (HR) of 0.64 (95% CI, 0.49-0.84), p = 0.0006. Likewise, overall survival (OS) with treosulfan was superior compared to busulfan: 36-month-OS rate 66.8% vs. 56.3%; HR 0.64 (95% CI, 0.48-0.87), p = 0.0037. Post hoc analyses revealed that these differences were consistent with the confirmatory interim analysis, and thereby the treosulfan regimen appears particularly suitable for older AML and MDS patients.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Beelen, Dietrich W.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stelljes, MatthiasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Remenyi, PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wagner-Drouet, Eva-MariaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Dreger, PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bethge, WolfgangUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ciceri, FabioUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stoelzel, FriedrichUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Junghanss, ChristianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Labussiere-Wallet, HeleneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schaefer-Eckart, KerstinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Grigoleit, Goetz U.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Scheid, ChristofUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Patriarca, FrancescaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Rambaldi, AlessandroUNSPECIFIEDorcid.org/0000-0002-3739-7502UNSPECIFIED
Niederwieser, DietgerUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hilgendorf, InkenUNSPECIFIEDorcid.org/0000-0003-2038-9730UNSPECIFIED
Russo, DomenicoUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Socie, GerardUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Holler, ErnstUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Glass, BertramUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Casper, JochenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wulf, GeraldUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Basara, NadezdaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bieniaszewska, MariaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stuhler, GernotUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Verbeek, MareikeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
La Rocca, UrsulaUNSPECIFIEDorcid.org/0000-0002-1990-9973UNSPECIFIED
Finke, JuergenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Benedetti, FabioUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pichlmeier, UweUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Klein, AnjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Baumgart, JoachimUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Markiewicz, MiroslawUNSPECIFIEDorcid.org/0000-0002-8550-8908UNSPECIFIED
URN: urn:nbn:de:hbz:38-698056
DOI: 10.1002/ajh.26620
Journal or Publication Title: Am. J. Hematol.
Volume: 97
Number: 8
Page Range: S. 1023 - 1035
Date: 2022
Publisher: WILEY
Place of Publication: HOBOKEN
ISSN: 1096-8652
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
VERSUS-HOST-DISEASE; CONDITIONING INTENSITY; FLUDARABINE; RECOMMENDATIONS; MANAGEMENT; DIAGNOSIS; MELPHALAN; REGIMEN; MARROW; IMPACTMultiple languages
HematologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/69805

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