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Can molecular data improve prognostication in MDS patients undergoing HSCT?

Presented by
Dr Carmelo Gurnari, Cleveland Clinic, OH, USA & Tor Vergata University, Italy
Conference
EMBT 2023
Doi
https://doi.org/10.55788/b5380e78

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

The Molecular International Prognostic Scoring System for myelodysplastic syndromes (IPSS-M) added molecular parameters into the prognostication for MDS [1,2]. However, since only 9% of the patients in the cohort of the study by Bernard et al. underwent HSCT, the applicability of the IPSS-M in the context of transplantation is unclear. Dr Carmelo Gurnari (Cleveland Clinic, OH, USA & Tor Vergata University, Italy) and co-investigators conducted a real-world study among 416 participants with MDS to evaluate the value of IPSS-M in transplantated patient. In the study cohort, 36% of the participants underwent the transplant upfront, and in 85% of treated cases, hypomethylating agents were given as bridge therapy.

The Revised International Prognostic Scoring System (IPSS-R), which uses haematological and cytogenetic instead of molecular parameters, stratified participants according to risk: very low (6%), low (17%), intermediate (19%), high (26%), and very high (32%) risk. The calculated IPSS-M scores 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 [3].

The authors concluded that molecular data adds value to the prognostication of patients with MDS undergoing HSCT. Dr Gurnari argued that the lower c-indexes that were reported in the current study, as compared with the original IPSS-M study by Bernard et al., point out that there are transplant-related factors to consider beyond disease-specific variables and molecular information.

  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. Gurnari C, et al. Leukemia. 2023;37(3):717–719.
  3. Bernard E, et al. N Engl J Med Evid. 2022;1(7).

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