Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes.
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Sauta Elisabetta, Robin Marie, Bersanelli Matteo, Travaglino Erica, Meggendorfer Manja, Zhao Lin-Pierre, Caballero Berrocal Juan Carlos, Sala Claudia, Maggioni Giulia, Bernardi Massimo, Di Grazia Carmen, Vago Luca, Rivoli Giulia, Borin Lorenza, D'Amico Saverio, Tentori Cristina Astrid, Ubezio Marta, Campagna Alessia, Russo Antonio, Mannina Daniele, Lanino Luca, Chiusolo Patrizia, Giaccone Luisa, Voso Maria Teresa, Riva Marta, Oliva Esther Natalie, Zampini Matteo, Riva Elena, Nibourel Olivier, Bicchieri Marilena, Bolli Niccolo', Rambaldi Alessandro, Passamonti Francesco, Savevski Victor, Santoro Armando, Germing Ulrich, Kordasti Shahram, Santini Valeria, Diez-Campelo Maria, Sanz Guillermo, Sole Francesc, Kern Wolfgang, Platzbecker Uwe, Ades Lionel, Fenaux Pierre, Haferlach Torsten, Castellani Gastone, Della Porta Matteo Giovanni
Аннотация
METHODS: A total of 2,876 patients with primary MDS from the GenoMed4All consortium were retrospectively analyzed.
RESULTS: IPSS-M improved prognostic discrimination across all clinical end points with respect to IPSS-R (concordance was 0.81 v 0.74 for overall survival and 0.89 v 0.76 for leukemia-free survival, respectively). This was true even in those patients without detectable gene mutations. Compared with the IPSS-R based stratification, the IPSS-M risk group changed in 46% of patients (23.6% and 22.4% of subjects were upstaged and downstaged, respectively).In patients treated with hematopoietic stem cell transplantation (HSCT), IPSS-M significantly improved the prediction of the risk of disease relapse and the probability of post-transplantation survival versus IPSS-R (concordance was 0.76 v 0.60 for overall survival and 0.89 v 0.70 for probability of relapse, respectively). In high-risk patients treated with hypomethylating agents (HMA), IPSS-M failed to stratify individual probability of response; response duration and probability of survival were inversely related to IPSS-M risk.Finally, we tested the accuracy in predicting IPSS-M when molecular information was missed and we defined a minimum set of 15 relevant genes associated with high performance of the score.
CONCLUSION: IPSS-M improves MDS prognostication and might result in a more effective selection of candidates to HSCT. Additional factors other than gene mutations can be involved in determining HMA sensitivity. The definition of a minimum set of relevant genes may facilitate the clinical implementation of the score.