Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comparative Study
. 2024 Aug;99(8):1540-1549.
doi: 10.1002/ajh.27363. Epub 2024 May 14.

Impact of busulfan versus treosulfan dose intensity in myelofibrosis undergoing hematopoietic cell transplantation

Affiliations
Free article
Comparative Study

Impact of busulfan versus treosulfan dose intensity in myelofibrosis undergoing hematopoietic cell transplantation

Nico Gagelmann et al. Am J Hematol. 2024 Aug.
Free article

Abstract

One key aspect of allogeneic hematopoietic cell transplantation (HCT) is pretransplant conditioning, balancing risk for relapse versus non-relapse mortality. Conditioning regimens with different alkylators at different doses can influence outcome, but data are missing for myelofibrosis, a challenging cohort of patients usually presenting at older age and with comorbidities. We evaluated in a multicenter retrospective study the comparative efficacy and safety of busulfan versus treosulfan in combination with fludarabine for myelofibrosis patients undergoing HCT. This study included 1115 patients (busulfan, n = 902; treosulfan, n = 213) receiving first HCT between 2005 and 2021. Patients were generally balanced for key patient characteristics. Overall survival at 4 years was 62% for the busulfan group versus 58% for the treosulfan group (p = .22). Impact on outcome was dose-dependent. Overall survival was 65% (95% CI, 61%-69%) for reduced intensity busulfan versus 69% (95% CI, 54%-84%) for reduced intensity treosulfan, 53% (95% CI, 44%-63%) for higher intensity busulfan, and 55% (95% CI, 46%-63%) for higher intensity treosulfan. Incidence of relapse was similar across intensity groups. In multivariable analysis, the hazard for death (with reduced intensity busulfan as reference) was 0.88 (95% CI, 0.39-2.01) for reduced intensity treosulfan (p = .77), 1.42 (95% CI, 0.96-2.10) for higher intensity busulfan (0.08), and 1.61 (95% CI, 1.14-2.26) for higher intensity treosulfan (p = .006). In terms of non-relapse mortality, comparison was not significantly different, while the hazard ratio for higher intensity treosulfan was 1.48 (95% CI, 0.98-2.23; p = .06). Here, we showed comparable outcomes and improved survival in myelofibrosis undergoing HCT with reduced intensity busulfan or treosulfan.

PubMed Disclaimer

References

REFERENCES

    1. Kroger N, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic haematopoietic stem‐cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group. Lancet Haematol. 2024;11(1):e62‐e74. doi:10.1016/S2352‐3026(23)00305‐8
    1. Gagelmann N, Kroger N. Dose intensity for conditioning in allogeneic hematopoietic cell transplantation: can we recommend “when and for whom” in 2021? Haematologica. 2021;106(7):1794‐1804. doi:10.3324/haematol.2020.268839
    1. Gagelmann N, Salit RB, Schroeder T, et al. High molecular and cytogenetic risk in myelofibrosis does not benefit from higher intensity conditioning before hematopoietic cell transplantation: an international collaborative analysis. Hemasphere. 2022;6(10):e784. doi:10.1097/HS9.0000000000000784
    1. McLornan D, Szydlo R, Koster L, et al. Myeloablative and reduced‐intensity conditioned allogeneic hematopoietic stem cell transplantation in myelofibrosis: a retrospective study by the chronic malignancies working Party of the European Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2019;25(11):2167‐2171. doi:10.1016/j.bbmt.2019.06.034
    1. Hessling J, Kroger N, Werner M, et al. Dose‐reduced conditioning regimen followed by allogeneic stem cell transplantation in patients with myelofibrosis with myeloid metaplasia. Br J Haematol. 2002;119(3):769‐772. doi:10.1046/j.1365‐2141.2002.03884.x

Publication types

MeSH terms

LinkOut - more resources