A retrospective validation of the IPSS-M molecular score in primary and therapy-related myelodysplastic syndromes (MDS).

A molecular scoring system (IPSS-M) was recently proposed for myelodysplastic syndrome (MDS). We conducted a retrospective study of adults with MDS referred 2019-2021. The primary outcomes were leukemia-free survival (LFS) and overall survival (OS). One hundred and forty-four patients diagnosed between 2011 and 2021 were analyzed. After IPSS-M re-stratification, 33% of patients were up-staged and 11% down-staged. Median follow-up was 2.8 years and 53 patients died (37%). Cumulative incidence of acute myeloid leukemia (AML) transformation was 20% at 3 years post-diagnosis. International Prognostic Scoring System (IPSS), revised version (IPSS-R) was significantly associated with LFS (log-rank  = 9.2e-05; 'very high' vs. 'low' risk HR = 3.85,  = 5.8e-04) and OS (log-rank  = 7.2e-06; 'very high' vs. 'low' HR = 5.09,  = 1.7e-04). IPSS-M was also a significant predictor of LFS (log-rank  = 1.1e-06; 'very high' vs. 'low' HR = 4.97,  = 2.2e-05) and OS (log-rank  = 4.8e-07; 'very high' vs. 'low' HR = 6.42,  = 2.5e-05) while providing better discrimination than IPSS-R for both outcomes. This mutation-incorporating prognostic index has greater discriminative potential than IPSS-R to predict AML transformation and any-cause mortality.