Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes

被引：47

作者：

Sauta, Elisabetta ^{[1
]}

Robin, Marie ^{[2
]}

Bersanelli, Matteo ^{[3
]}

Travaglino, Erica ^{[1
]}

Meggendorfer, Manja ^{[4
]}

Zhao, Lin-Pierre ^{[2
]}

Caballero Berrocal, Juan Carlos ^{[5
]}

Sala, Claudia ^{[6
]}

Maggioni, Giulia ^{[1
]}

Bernardi, Massimo ^{[7
]}

Di Grazia, Carmen ^{[8
]}

Vago, Luca ^{[7
]}

Rivoli, Giulia ^{[8
]}

Borin, Lorenza ^{[9
]}

D'Amico, Saverio ^{[1
]}

Tentori, Cristina Astrid ^{[1
]}

Ubezio, Marta ^{[1
]}

Campagna, Alessia ^{[1
]}

Russo, Antonio ^{[1
]}

Mannina, Daniele ^{[1
]}

Lanino, Luca ^{[1
]}

Chiusolo, Patrizia ^{[10
,11
]}

Giaccone, Luisa ^{[12
,13
]}

Voso, Maria Teresa ^{[14
,15
]}

Riva, Marta ^{[16
]}

Oliva, Esther Natalie ^{[17
]}

Zampini, Matteo ^{[1
]}

Riva, Elena ^{[1
]}

Nibourel, Olivier ^{[18
]}

Bicchieri, Marilena ^{[1
]}

Bolli, Niccolo' ^{[19
,20
]}

Rambaldi, Alessandro ^{[21
,22
]}

Passamonti, Francesco ^{[23
,24
]}

Savevski, Victor ^{[1
]}

Santoro, Armando ^{[1
,3
]}

Germing, Ulrich ^{[25
]}

Kordasti, Shahram ^{[26
,27
,28
,29
]}

Santini, Valeria ^{[30
,31
]}

Diez-Campelo, Maria ^{[5
]}

Sanz, Guillermo ^{[32
]}

Sole, Francesc ^{[33
]}

Kern, Wolfgang ^{[4
]}

Platzbecker, Uwe ^{[34
]}

Ades, Lionel ^{[2
]}

Fenaux, Pierre ^{[2
]}

Haferlach, Torsten ^{[4
]}

Castellani, Gastone ^{[6
]}

Della Porta, Matteo Giovanni ^{[1
,3
]}

机构：

[1] IRCCS, Humanitas Clin & Res Ctr, Milan, Italy

[2] Univ Paris 07, Hop St Louis, Assistance Publ Hop Paris AP HP, Dept Hematol & Bone Marrow Transplantat, Paris, France

[3] Humanitas Univ, Dept Biomed Sci, Milan, Italy

[4] MLL Munich Leukemia Lab, Munich, Germany

[5] Hosp Univ Salamanca, Hematol Dept, Salamanca, Spain

[6] DIMES, Expt Diagnost & Specialty Med, Bologna, Italy

[7] Univ Vita Salute San Raffaele, IRCCS San Raffaele Sci Inst, Hematol & Bone Marrow Transplantat, Milan, Italy

[8] IRCCS Osped Policlin San Martino, Hematol & Transplant Ctr, Genoa, Italy

[9] Osped San Gerardo, Hematol, Monza, Italy

[10] IRCCS Fdn Policlin Univ Gemelli, Hematol, Rome, Italy

[11] Univ Cattolica Sacro Cuore, Rome, Italy

[12] AOU Citta Salute Sci Torino, Dept Oncol, Stem Cell Transplant Program, Turin, Italy

[13] Univ Turin, Dept Mol Biotechnol & Hlth Sci, Turin, Italy

[14] Tor Vergata Univ, Hematol, Policlin Tor Vergata, Rome, Italy

[15] Tor Vergata Univ, Dept Biomed & Prevent, Rome, Italy

[16] ASST Grande Osped Metropolitano Niguarda, Hematol, Milan, Italy

[17] Grande Osped Metropolitano Bianchi Melacrino More, Hematol, Reggio Di Calabria, Italy

[18] CHU Lille, Lab Hematol, Lille, France

[19] Fdn IRCCS Ca Granda Osped Maggiore Policlin, Hematol Unit, Milan, Italy

[20] Univ Milan, Dept Oncol & Hemato Oncol, Milan, Italy

[21] Azienda Osped Papa Giovanni XXIII, Hematol, Bergamo, Italy

[22] Univ Milan, Dept Oncol & Hemato Oncol, Milan, Italy

[23] Osped Circolo Varese, ASST Sette Laghi, Hematol, Varese, Italy

[24] Univ Insubria, Dept Med & Surg, Varese, Italy

[25] Univ Clin, Dept Hematol Oncol & Clin Immunol, Heinrich Heine Univ, Dusseldorf, Germany

[26] Kings Coll London, Guys Hosp, Haematol, London, England

[27] Kings Coll London, Ctr Comprehens Canc, London, England

[28] Univ Politecn Marche, Hematol Dept, Ancona, Italy

[29] Univ Politecn Marche, Stem Cell Transplant Unit, DISCLIMO, Ancona, Italy

[30] Azienda Osped Univ Careggi, Hematol, Florence, Italy

[31] Univ Florence, Florence, Italy

[32] Hosp Univ La Fe, Hematol, Valencia, Spain

[33] Inst Recerca Leucemia Josep Carreras, Barcelona, Spain

[34] Univ Hosp Leipzig, Med Clin & Policlin 1, Hematol & Cellular Therapy, Leipzig, Germany

来源：

JOURNAL OF CLINICAL ONCOLOGY | 2023年 / 41卷 / 15期

关键词：

STEM-CELL TRANSPLANTATION; HEALTH-ORGANIZATION CLASSIFICATION; ACUTE MYELOID-LEUKEMIA; DECISION-ANALYSIS; WORKING GROUP; MUTATIONS; RECOMMENDATIONS; AZACITIDINE; OUTCOMES;

D O I：

10.1200/JCO.22.01784

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

PURPOSE Myelodysplastic syndromes (MDS) are heterogeneous myeloid neoplasms in which a risk-adapted treatment strategy is needed. Recently, a new clinical-molecular prognostic model, the Molecular International Prognostic Scoring System (IPSS-M) was proposed to improve the prediction of clinical outcome of the currently available tool (Revised International Prognostic Scoring System [IPSS-R]). We aimed to provide an extensive validation of IPSS-M. 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.

引用

页码：2827 / +

页数：17

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