What are the treatment options for myelodysplastic syndromes (MDS)?

From the Guidelines

Treatment options for myelodysplastic syndromes (MDS) should be guided by disease risk, patient age, and overall health status, with a focus on improving morbidity, mortality, and quality of life. For lower-risk MDS, supportive care with red blood cell or platelet transfusions is often used to manage cytopenias, while erythropoiesis-stimulating agents like epoetin alfa or darbepoetin can improve anemia, as recommended by the ESMO clinical practice guidelines 1.

• Lenalidomide (Revlimid) is particularly effective for patients with deletion 5q abnormalities, typically given at 10mg daily for 21 days of a 28-day cycle, with a reported response rate of 60-70% 1.
• Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine may benefit younger patients with hypocellular bone marrow, with a response rate of 25-40% 1. For higher-risk MDS, hypomethylating agents such as azacitidine (75mg/m² for 7 days every 28 days) or decitabine (20mg/m² for 5 days every 28 days) are standard treatments that can improve blood counts and delay progression to acute myeloid leukemia, as recommended by the ESMO clinical practice guidelines 1.
• Allogeneic hematopoietic stem cell transplantation remains the only potentially curative option but is limited to younger, healthier patients due to significant risks, and should be proposed to all higher-risk MDS patients <70 years old without major comorbidities and with a donor 1.
• Luspatercept has recently been approved for treating anemia in MDS with ring sideroblasts, and is recommended after ESA failure in RBC transfusion-dependent MDS-RS patients 1. Treatment selection depends on careful risk stratification using systems like IPSS-R, which considers cytogenetics, blast percentage, and cytopenia severity to guide appropriate therapy and optimize outcomes, as recommended by the NCCN clinical practice guidelines 1.

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.

Treatment options for myelodysplastic syndromes (MDS) include:

• Decitabine for injection, a nucleoside metabolic inhibitor
• Two dosing regimens:
  • 15 mg/m^2 by continuous intravenous infusion over 3 hours repeated every 8 hours for 3 days, repeated every 6 weeks
  • 20 mg/m^2 by continuous intravenous infusion over 1 hour repeated daily for 5 days, repeated every 4 weeks 2

From the Research

Treatment Options for Myelodysplastic Syndromes (MDS)

The treatment options for MDS can be categorized into several approaches, including:

• Supportive care measures, such as hematopoietic growth factors, red blood cell or platelet transfusions, and antimicrobial agents 3
• Allogeneic stem cell transplantation, which can be curative, but is only suitable for a small subset of patients 3, 4
• DNA methyltransferase inhibitors, such as azacitidine and decitabine, which have been approved by the US Food and Drug Administration (FDA) for the treatment of MDS 3
• Immunomodulatory agents, such as lenalidomide, which can be used to treat patients with MDS 3
• Erythropoiesis-stimulating agents, such as recombinant humanized erythropoietin or darbepoetin alfa, which can improve anemia in patients with lower-risk MDS 5
• Hypomethylating agents, such as azacitidine, decitabine, or decitabine/cedazuridine, which are first-line therapy for patients with higher-risk MDS 5
• Hematopoietic cell transplantation, which is considered for higher-risk patients and represents the only potential cure 5, 6
• Experimental therapies, such as venetoclax, myeloid cell leukemia 1 (MCL-1) inhibitors, and immunotherapies, which are being investigated for the treatment of MDS 6

Risk-Based Management Approach

A risk-based management approach is recommended for MDS, which takes into account the patient's disease characteristics, comorbidities, and risk factors 3, 6. This approach can help clinicians choose the most appropriate treatment for an individual patient.

Supportive Care

Supportive care measures are essential for patients with MDS, particularly those with lower-risk disease 7. These measures include:

• Transfusion support
• Management of iron overload
• Antimicrobial prophylaxis
• Routine immunizations
• Palliative care

Individualized Treatment

Individualized treatment is crucial for patients with MDS, as each patient's disease characteristics and comorbidities are unique 6. Clinicians should consider a patient's risk factors, disease subtype, and overall health when selecting a treatment approach.