Treosulfan compared to busulfan in allogeneic haematopoietic stem cell transplantation for myelofibrosis: a registry-based study from the Chronic Malignancies Working Party of the EBMT

We aimed to compare outcomes following treosulfan (TREO) or busulfan (BU) conditioning in a large cohort of myelofibrosis (MF) patients from the EBMT registry. A total of 530 patients were included; 73 received TREO and 457 BU (BU ≤ 6.4 mg/kg in 134, considered RIC, BU > 6.4 mg/kg in 323 consider...

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Hauptverfasser: Robin, Marie (VerfasserIn) , Iacobelli, Simona (VerfasserIn) , Koster, Linda (VerfasserIn) , Passweg, Jakob R. (VerfasserIn) , Avenoso, Daniele (VerfasserIn) , Wilson, Keith M. O. (VerfasserIn) , Salmenniemi, Urpu (VerfasserIn) , Dreger, Peter (VerfasserIn) , von dem Borne, Peter (VerfasserIn) , Snowden, John A. (VerfasserIn) , Robinson, Stephen (VerfasserIn) , Finazzi, Maria Chiara (VerfasserIn) , Schroeder, Thomas (VerfasserIn) , Collin, Matthew (VerfasserIn) , Eder, Matthias (VerfasserIn) , Forcade, Edouard (VerfasserIn) , Loschi, Michael (VerfasserIn) , Bramanti, Stefania (VerfasserIn) , Pérez-Simón, Jose Antonio (VerfasserIn) , Czerw, Tomasz (VerfasserIn) , Polverelli, Nicola (VerfasserIn) , Drozd-Sokolowska, Joanna (VerfasserIn) , Raj, Kavita (VerfasserIn) , Hernández-Boluda, Juan Carlos (VerfasserIn) , McLornan, Donal (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 15 March 2024
In: Bone marrow transplantation
Year: 2024, Jahrgang: 59, Heft: 7, Pages: 928-935
ISSN:1476-5365
DOI:10.1038/s41409-024-02269-4
Online-Zugang:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41409-024-02269-4
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41409-024-02269-4
Volltext
Verfasserangaben:Marie Robin, Simona Iacobelli, Linda Koster, Jakob Passweg, Daniele Avenoso, Keith M.O. Wilson, Urpu Salmenniemi, Peter Dreger, Peter von dem Borne, John A. Snowden, Stephen Robinson, Maria Chiara Finazzi, Thomas Schroeder, Matthew Collin, Matthias Eder, Edouard Forcade, Michael Loschi, Stefania Bramanti, Jose Antonio Pérez-Simón, Tomasz Czerw, Nicola Polverelli, Joanna Drozd-Sokolowska, Kavita Raj, Juan Carlos Hernández-Boluda and Donal P. McLornan

MARC

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520 |a We aimed to compare outcomes following treosulfan (TREO) or busulfan (BU) conditioning in a large cohort of myelofibrosis (MF) patients from the EBMT registry. A total of 530 patients were included; 73 received TREO and 457 BU (BU ≤ 6.4 mg/kg in 134, considered RIC, BU > 6.4 mg/kg in 323 considered higher dose (HD)). Groups were compared using adjusted Cox models. Cumulative incidences of engraftment and acute GVHD were similar across the 3 groups. The TREO group had significantly better OS than BU-HD (HR:0.61, 95% CI: 0.39-0.93) and a trend towards better OS over BU-RIC (HR: 0.66, 95% CI: 0.41-1.05). Moreover, the TREO cohort had a significantly better Progression-Free-Survival (PFS) than both the BU-HD (HR: 0.57, 95% CI: 0.38-0.84) and BU-RIC (HR: 0.60, 95% CI: 0.39-0.91) cohorts, which had similar PFS estimates. Non-relapse mortality (NRM) was reduced in the TREO and BU-RIC cohorts (HR: 0.44, 95% CI: 0.24-0.80 TREO vs BU-HD; HR: 0.54, 95% CI: 0.28-1.04 TREO vs BU-RIC). Of note, relapse risk did not significantly differ across the three groups. In summary, within the limits of a registry-based study, TREO conditioning may improve PFS in MF HSCT and have lower NRM than BU-HD with a similar relapse risk to BU-RIC. Prospective studies are needed to confirm these findings. 
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