From the Guidelines
Treatment options for myelodysplastic syndrome (MDS) should prioritize supportive care, including red blood cell transfusions at a sufficiently high hemoglobin threshold, and iron chelation therapy in patients with iron overload, as recommended by the most recent guidelines 1. The goal of treatment is to address cytopenias, reduce transfusion dependence, improve quality of life, delay disease progression, and potentially achieve cure in appropriate candidates. Key considerations in treatment selection include:
- Disease risk, with lower-risk MDS managed differently than higher-risk MDS
- Patient age and overall health status
- Presence of specific cytogenetic abnormalities, such as deletion 5q
- Need for iron chelation therapy to prevent iron overload and its complications, such as heart failure Some treatment options for MDS include:
- Supportive care with red blood cell or platelet transfusions
- Erythropoiesis-stimulating agents like epoetin alfa or darbepoetin
- Lenalidomide, particularly for patients with deletion 5q
- Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine for younger patients with hypoplastic MDS
- Hypomethylating agents, such as azacitidine or decitabine, for higher-risk MDS
- Allogeneic hematopoietic stem cell transplantation, which remains the only potentially curative option but is limited to younger, fit patients
- Newer approved therapies, including luspatercept for anemia in ring sideroblast MDS and venetoclax combinations for higher-risk disease, as noted in earlier guidelines 1. However, the most recent and highest quality study 1 provides the most up-to-date recommendations for iron chelation therapy and supportive care, which should be prioritized in treatment decisions.
From the FDA Drug Label
Decitabine for injection is indicated for treatment of adult patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.
Administer decitabine for injection at a dose of 15 mg/m 2 by continuous intravenous infusion over 3 hours repeated every 8 hours for 3 days. Repeat cycle every 6 weeks.
Administer decitabine for injection at a dose of 20 mg/m 2 by continuous intravenous infusion over 1 hour repeated daily for 5 days. Repeat cycle every 4 weeks.
The treatment options for myelodysplastic syndrome (MDS) include:
- Decitabine (IV): a nucleoside metabolic inhibitor, which can be administered through two different regimens:
- Three Day Regimen: 15 mg/m² every 8 hours for 3 days, repeated every 6 weeks.
- Five Day Regimen: 20 mg/m² daily for 5 days, repeated every 4 weeks. 2
From the Research
Treatment Options for Myelodysplastic Syndrome (MDS)
The treatment options for MDS can be categorized into several approaches, including:
- Supportive care measures, such as red blood cell transfusions and erythroid colony stimulating factors, to manage anemia and other cytopenias 3, 4
- Iron chelation therapy to reduce iron overload, which can be achieved through oral agents like deferasirox and deferiprone, or parenteral administration of deferoxamine 3, 5
- Immunomodulatory therapies, such as lenalidomide, to improve blood cell counts and reduce transfusion dependence 6
- Hypomethylating agents, like azacitidine, to modify the disease course and improve survival 6, 7
- Stem cell transplantation, which may be considered for patients with lower-risk MDS who have a high risk of progression to acute myeloid leukemia 7
Management of Lower-Risk MDS
For patients with lower-risk MDS, the goals of care focus on symptom control and quality of life, rather than changing the natural history of the disease 6. Treatment options for lower-risk MDS include:
- Erythropoiesis stimulating agents (ESAs) to improve anemia 6, 7
- Immunosuppressive therapy (IST) to reduce immune-mediated suppression of blood cell production 6
- Lenalidomide to improve blood cell counts and reduce transfusion dependence 6
- Iron chelation therapy to reduce iron overload 3, 5
Predictors of Response to Treatment
Several factors can predict response to treatment in MDS patients, including:
- Demographic and clinical parameters, such as age, gender, and performance status 7
- Laboratory parameters, such as degree of anemia, absolute neutrophil count, and platelet count 7
- Cellular and molecular parameters, such as immunophenotype, clonal granulocytes, and genetic mutations 7
- Presence of poor prognostic features (PPF), such as high serum ferritin, bone marrow fibrosis, and multi-lineage dysplasia 7