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Updated: Feb 9, 2026
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Mayo Alliance Prognostic Model for Myelodysplastic Syndromes: Integration of Genetic and Clinical Information
Ayalew Tefferi1, Naseema Gangat1, Mythri Mudireddy1
1Division of Hematology, Mayo Clinic, Rochester, MN.
Objective:
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.
Insights
A new risk model for myelodysplastic syndromes (MDS) integrates genetic mutations and clinical factors. This Mayo alliance model improves prognostic accuracy over existing systems, aiding patient stratification and treatment decisions.
Area of Science:
- Hematology
- Oncology
- Genetics
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Accurate prognostication is crucial for managing MDS and guiding treatment decisions.
Purpose of the Study:
- To develop and validate a novel risk stratification model for primary MDS.
- The model integrates cytogenetic, molecular, and clinical variables for enhanced accuracy.
Main Methods:
- A cohort of 685 patients with primary MDS from Mayo Clinic and National Taiwan University Hospital were analyzed.
- Multivariate analysis identified key predictors of overall survival.
- A 4-tiered prognostic model (Mayo alliance model) was developed and validated.
Main Results:
- Key predictors of inferior survival included monosomal karyotype, RUNX1/ASXL1 mutations, absence of SF3B1 mutations, age >70, low hemoglobin, low platelets, and bone marrow blasts.
- The Mayo alliance model demonstrated superior performance compared to the revised International Prognostic Scoring System (IPSS-R) in terms of Akaike information criterion and area under the curve.
- Median survival ranged from 85 months (low risk) to 9 months (high risk).
Conclusions:
- The Mayo alliance prognostic model offers a simple, contemporary, and accurate tool for risk stratification in primary MDS.
- This model integrates essential genetic and clinical data, improving upon existing prognostic systems.
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