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. 2024 Sep 25;16(19):3257.
doi: 10.3390/cancers16193257.

Improved Outcomes in Myelofibrosis after Allogeneic Stem-Cell Transplantation in the Era of Ruxolitinib Pretreatment and Intensified Conditioning Regimen-Single-Center Analysis

Sigrid Machherndl-Spandl  1   2 , Sarah Hannouf  1   2 , Alexander Nikoloudis  1   2 , Otto Zach  3 , Irene Strassl  1   2 , Emine Kaynak  1 , Gerald Webersinke  3 , Christine Gruber-Rossipal  4 , Holger Rumpold  1   2 , Wolfgang Schimetta  5 , Johannes Clausen  1   2 , Veronika Buxhofer-Ausch  1   2
Affiliations

Improved Outcomes in Myelofibrosis after Allogeneic Stem-Cell Transplantation in the Era of Ruxolitinib Pretreatment and Intensified Conditioning Regimen-Single-Center Analysis

Sigrid Machherndl-Spandl et al. Cancers (Basel). .

Abstract

(1) Background: Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) is the only treatment with the potential for cure in patients with myelofibrosis (MF). However, the risk of graft rejection, which is particularly high in MF, and the risk of significant non-relapse mortality must be considered. (2) Methods: In this retrospective, single-center study, we compared allo-HSCT outcomes in 36 adult patients with MF transplanted at two-time intervals (2001-2015 versus 2016-2021). (3) Results: The estimated median overall survival was 48.9 months (95%CI 0.00-98.2) in the cohort transplanted before 2016 and not reached in the more recent years (p = 0.04) due to markedly lower non-relapse mortality (p = 0.02). The 3-year relapse incidence was low in both cohorts (11.1% and 12.5%, p > 0.99). When comparing only subgroups within the more recent cohort based on the presence or absence of total body irradiation (TBI) or the use of sequential regimens, OS and PFS were comparable. (4) Conclusion: Pretreatment with ruxolitinib, intensified conditioning, and the preferential use of haploidentical related instead of mismatched unrelated donors for patients lacking an HLA-identical donor are most likely responsible for the improved outcome after allo-HCT in MF in recent years.

Keywords: allogeneic stem-cell transplantation; conditioning therapy; myelofibrosis.

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Conflict of interest statement

S.M.-S. has received a speaker honorarium from Amgen, Abbvie, Jazz, Novartis, Pfizer, Servier, BMS/Celgene, Janssen, V.B.-A. has received a speaker honorarium from Novartis, AOP, GSK; J.C. has received a speaker honorarium from Amgen, Abbvie, Servier, Jazz; I.S. has received a speaker honorarium from Janssen, Amgen; E.K. has received a speaker honorarium from Abbvie, Novartis, GSK, BMS/Celgene.

Figures

Figure 1
Figure 1
(a–d): OS, PFS, CIR, and NRM in the subcollectives (allo-HSCT until 31 December 2015, n = 15 and from 1 January 2016, n = 21). (a): Overall survival of the subcollectives. (b): Progression-free survival of the subcollectives. (c): Cumulative incidence of relapse of the subcollectives. (d): Non-relapse mortality of the subcollectives. * p-value with significance.
Figure 1
Figure 1
(a–d): OS, PFS, CIR, and NRM in the subcollectives (allo-HSCT until 31 December 2015, n = 15 and from 1 January 2016, n = 21). (a): Overall survival of the subcollectives. (b): Progression-free survival of the subcollectives. (c): Cumulative incidence of relapse of the subcollectives. (d): Non-relapse mortality of the subcollectives. * p-value with significance.
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