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Myelodysplastic Syndromes: 2026 Update on Diagnosis, Risk-Stratification and Management
1Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Disease Overview:
The myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by peripheral blood cytopenias and increased risk of transformation to acute myelogenous leukemia (AML). MDS occurs more frequently in older males and in individuals with prior exposure to cytotoxic therapy.
Diagnosis:
Diagnosis of MDS is based on morphological evidence of dysplasia upon visual examination of a bone marrow aspirate and biopsy. Information obtained from additional studies such as karyotype, flow cytometry, and molecular genetics is usually complementary and may help refine diagnosis. Under the 2022 WHO classification of MDS, this entity is now termed myelodysplastic neoplasms.
Risk-Stratification:
Prognosis of patients with MDS can be calculated using a number of scoring systems. All these scores include analysis of peripheral cytopenias, percentage of blasts in the bone marrow, and cytogenetic characteristics. The most commonly used systems are the Revised International Prognostic Scoring System (IPSS-R) and the molecular IPSS-M.
Risk-Adapted Therapy:
Therapy is selected based on risk, transfusion needs, percent of bone marrow blasts, cytogenetic and mutational profiles, comorbidities, potential for allogeneic stem cell transplantation (alloSCT), and prior exposure to hypomethylating agents (HMA). Goals of therapy are different in lower-risk patients than in higher-risk and in those with HMA failure. In lower risk, the goal is to decrease transfusion needs and transformation to higher-risk disease or AML, as well as to improve survival. In higher-risk, the goal is to prolong survival. In 2020, two agents were approved in the US for patients with MDS: luspatercept and oral decitabine/cedazuridine. Imetelstat was approved in 2024 for transfusion-dependent lower-risk MDS. Other available therapies include growth factors, lenalidomide, HMAs, intensive chemotherapy, and alloSCT. A number of combinations studies have been completed at the time of this report; none of them was superior to single-agent azacitidine. There are no approved interventions for patients with progressive or refractory disease, particularly after HMA-based therapy. AlloSCT still remains the only curative approach in MDS.
