Tefferi A.Gangat N.Mudireddy M.Lasho T.L.Finke C.Begna K.H.Elliott M.A.Al-Kali A.Litzow M.R.Hook C.C.Wolanskyj A.P.Hogan W.J.Patnaik M.M.Pardanani A.Zblewski D.L.He R.Viswanatha D.Hanson C.A.Ketterling R.P.JIH-LUH TANGWEN-CHIEN CHOUCHIEN-CHIN LINCHENG-HONG TSAIHWEI-FANG TIENHSIN-AN HOU2022-01-122022-01-1220180025-6196https://www.scopus.com/inward/record.uri?eid=2-s2.0-85047819256&doi=10.1016%2fj.mayocp.2018.04.013&partnerID=40&md5=f115dffb38acebbb072d9cba90a0e8fahttps://scholars.lib.ntu.edu.tw/handle/123456789/592171Objective: To develop a new risk model for primary myelodysplastic syndromes (MDS) that integrates information on mutations, karyotype, and clinical variables. Patients and Methods: Patients with World Health Organization–defined primary MDS seen at Mayo Clinic (MC) from December 28, 1994, through December 19, 2017, constituted the core study group. The National Taiwan University Hospital (NTUH) provided the validation cohort. Model performance, compared with the revised International Prognostic Scoring System, was assessed by Akaike information criterion and area under the curve estimates. Results: The study group consisted of 685 molecularly annotated patients from MC (357) and NTUH (328). Multivariate analysis of the MC cohort identified monosomal karyotype (hazard ratio [HR], 5.2; 95% CI, 3.1-8.6), “non-MK abnormalities other than single/double del(5q)” (HR, 1.8; 95% CI, 1.3-2.6), RUNX1 (HR, 2.0; 95% CI, 1.2-3.1) and ASXL1 (HR, 1.7; 95% CI, 1.2-2.3) mutations, absence of SF3B1 mutations (HR, 1.6; 95% CI, 1.1-2.4), age greater than 70 years (HR, 2.2; 95% CI, 1.6-3.1), hemoglobin level less than 8 g/dL in women or less than 9 g/dL in men (HR, 2.3; 95% CI, 1.7-3.1), platelet count less than 75 × 109/L (HR, 1.5; 95% CI, 1.1-2.1), and 10% or more bone marrow blasts (HR, 1.7; 95% CI, 1.1-2.8) as predictors of inferior overall survival. Based on HR-weighted risk scores, a 4-tiered Mayo alliance prognostic model for MDS was devised: low (89 patients), intermediate-1 (104), intermediate-2 (95), and high (69); respective median survivals (5-year overall survival rates) were 85 (73%), 42 (34%), 22 (7%), and 9 months (0%). The Mayo alliance model was subsequently validated by using the external NTUH cohort and, compared with the revised International Prognostic Scoring System, displayed favorable Akaike information criterion (1865 vs 1943) and area under the curve (0.87 vs 0.76) values. Conclusion: We propose a simple and contemporary risk model for MDS that is based on a limited set of genetic and clinical variables. ? 2018 Mayo Foundation for Medical Education and Research[SDGs]SDG3hemoglobin; transcription factor RUNX1; ASXL1 protein, human; hemoglobin; phosphoprotein; repressor protein; RNA splicing factor; RUNX1 protein, human; SF3B1 protein, human; transcription factor RUNX1; adult; age; aged; Article; ASXL1 gene; cancer prognosis; cancer risk; cancer survival; cohort analysis; cytogenetics; diagnostic test accuracy study; female; follow up; gene deletion; hemoglobin blood level; high risk patient; human; International Prognostic Scoring System; karyotype; major clinical study; male; median survival time; monosomy; myelodysplastic syndrome; overall survival; platelet count; prognostic assessment; RUNX1 gene; sex difference; SF3B1 gene; survival prediction; survival rate; Taiwan; treatment outcome; tumor-related gene; blood; bone marrow examination; genetics; karyotyping; mortality; mutation; myelodysplastic syndrome; procedures; prognosis; proportional hazards model; reproducibility; risk assessment; risk factor; sex factor; statistics and numerical data; Age Factors; Aged; Bone Marrow Examination; Core Binding Factor Alpha 2 Subunit; Female; Hemoglobins; Humans; Karyotyping; Male; Mutation; Myelodysplastic Syndromes; Phosphoproteins; Platelet Count; Prognosis; Proportional Hazards Models; Repressor Proteins; Reproducibility of Results; Risk Assessment; Risk Factors; RNA Splicing Factors; Sex Factorsjournal article10.1016/j.mayocp.2018.04.013298664192-s2.0-85047819256