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Can molecular data improve prognostication in MDS patients undergoing HSCT?

Presented By
Dr Carmelo Gurnari, Cleveland Clinic, OH, USA
EBMT 2023

In the prognostication of patients with myelodysplastic syndromes (MDS) undergoing hematopoietic stem cell transplant (HSCT), findings from a large, international study showed that molecular data improved the prediction of overall survival (OS) modestly.

Dr Carmelo Gurnari (Cleveland Clinic, OH, USA) and co-investigators conducted a real-world study among 416 participants with MDS to assess the links between transplantation and the Molecular International Prognostic Scoring System (IPSS-M) [1]. In the study cohort, 36% of the participants underwent the transplant upfront and 85% of the participants used hypomethylating agents as bridge therapy.

The IPSS-R, a prognostic scoring system that uses haematological and cytogenetic parameters, not including molecular information, stratified participants according to risk: very low (6%), low (17%), intermediate (19%), high (26%), and very high (32%) risk. The calculated IPSS-M scores, which differs from the IPSS-R by adding molecular data, resulted in a significant redistribution of participants with respect to these risk categories: very low (27%), low (18%), moderately low (14%), moderately high (15%), high (10%), and very high (16%). Furthermore, the c-index for overall survival was 0.583 for the IPSS-M and 0.547 for the IPSS-R, suggesting that the IPSS-M is slightly better at predicting OS outcomes than the IPSS-R. The c-index of IPSS-M for OS was lower than the observed c-indexes in the study by Bernard et al. and comparable with existing specific HSCT MDS risk scores [2].

    1. Gurnari C, et al. Outcome prediction in patients with myelodysplastic syndrome undergoing hematopoietic cell transplant in the molecular era of IPSS-M. OS17-05, European Society for Blood and Marrow Transplantation (EBMT) 49th annual meeting, 23–26 April 2023, Paris, France.
    2. Bernard E, et al. N Engl J Med Evid. 2022;1(7).


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