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2025-03-14T11:36:56.000Z

The role of molecular risk in predicting survival benefit and guiding transplant decisions in primary MF

Mar 14, 2025
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Learning objective: After reading this article, learners will be able to cite a new clinical development in primary myelofibrosis.

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Insights into primary myelofibrosis (PMF) driver mutations have led to the development of several classifications for the prediction of OS and transformation to AML, including DIPSS, MIPSS, and MTSS.1 However, optimal patient selection and timing for transplantation remain unclear.

Paz et al.1 conducted a cohort study to analyze the potential survival benefit associated with HSCT according to clinicobiological scores, incorporating MIPSS, to facilitate decision-making in this context. Data from a transplant cohort of patients who received transplantation between 2001 and 2022 (n = 239) and a matched non-transplant cohort (n = 105) were used to determine whether patients with PMF and a higher risk molecular score benefit from HSCT. Their findings were published in Blood Advances.1 


Key learnings
HSCT was associated with improved 6-year OS vs non-transplant across intermediate-2 (60.1% vs 41.5%) and high-risk DIPSS (44.4% vs 6.55%), MIPSS70 (46.5% vs 23.9%), high-risk (73.2% vs 39.7%), and very high-risk MIPSS70+V2 (51.8% vs 24%) groups.

The survival gain with transplantation in DIPSS intermediate-2 and high-risk patients was +18.6% and +37.9%, respectively. Patients with intermediate MIPSS70 scores achieved an OS benefit with HSCT only if their MTSS scores were low or intermediate.

Following the 1-year landmark, transplantation reduced the risk of mortality in patients with DIPSS high or intermediate-2, MIPSS70 high, HMR mutations, and extended HMR mutations.
These findings show that MIPSS70 and MIPSS70+V2 risk scores, in addition to MTSS, are effective in predicting the survival benefit of HSCT in patients with PMF, offering reliable scores for transplant decision-making.

Abbreviations: AML, acute myeloid leukemia; DIPSS, Dynamic International Prognostic Scoring System; HSCT, hematopoietic stem cell transplantation; HMR, high molecular risk; MIPSS, Mutation-Enhanced International Prognostic Scoring System for patients aged ≥70 years; MIPSS70+V2, Mutation-Enhanced International Prognostic Scoring System for patients aged ≥70 years Version 2; MTSS, Myelofibrosis Transplant Scoring system; OS, overall survival; PMF, primary myelofibrosis.

  1. Paz DL, Gagelmann N, Benajiba L, et al. Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis. Blood Adv. 2025;9(4):797-807. DOI: 10.1182/bloodadvances.2024014368

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