From the Guidelines
The treatment of myelodysplastic syndrome (MDS) should be individualized based on the patient's specific disease characteristics, with a focus on improving quality of life, reducing morbidity, and delaying disease progression, as recommended by the most recent guidelines 1.
Overview of MDS Treatment
The treatment approach for MDS depends on the disease subtype, risk category, patient age, and overall health.
- For lower-risk MDS, treatment often begins with supportive care, including red blood cell or platelet transfusions to manage cytopenias, along with erythropoiesis-stimulating agents like epoetin alfa or darbepoetin to improve anemia.
- Lenalidomide (10mg daily for 21 days of a 28-day cycle) is particularly effective for patients with deletion 5q, as noted in earlier guidelines 2.
Higher-Risk MDS Treatment
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 outlined in recent clinical practice guidelines 1.
- Luspatercept (1mg/kg subcutaneously every 3 weeks) may be used for ring sideroblast-positive MDS with transfusion-dependent anemia.
- Allogeneic hematopoietic stem cell transplantation remains the only potentially curative option but is limited to younger, healthier patients due to transplant-related risks.
Supportive Care and Iron Chelation
Supportive care, including red blood cell transfusions, is crucial for managing cytopenias and improving quality of life in MDS patients, with the goal of transfusing at a sufficiently high hemoglobin threshold, as recommended in recent guidelines 1.
- Iron chelation therapy with deferasirox (starting at 10-20mg/kg/day) may be necessary for transfusion-dependent patients to prevent iron overload, particularly in those with a relatively favorable prognosis or significant iron overload, as suggested by recent studies 1.
Treatment Selection and Monitoring
Treatment selection should be individualized based on the patient's specific disease characteristics, with regular monitoring of blood counts and bone marrow evaluations to assess response and disease progression, emphasizing the importance of personalized care in MDS management 1.
From the FDA Drug Label
Decitabine for injection is a nucleoside metabolic inhibitor 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.
Three Day Regimen 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.
Five Day Regimen 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 of myelodysplastic syndrome is decitabine for injection, which can be administered through two different regimens:
- Three Day Regimen: 15 mg/m^2 by continuous intravenous infusion over 3 hours repeated every 8 hours for 3 days, repeated every 6 weeks.
- Five Day Regimen: 20 mg/m^2 by continuous intravenous infusion over 1 hour repeated daily for 5 days, repeated every 4 weeks 3.
From the Research
Treatment Overview
- The treatment of myelodysplastic syndrome (MDS) is tailored to the patient's disease characteristics and comorbidities 4.
- Therapy for lower-risk MDS focuses on treating symptoms and reducing the number of required transfusions, while higher-risk MDS is treated with hypomethylating agents or hematopoietic cell transplantation 4, 5.
Lower-Risk MDS Treatment
- Erythropoiesis stimulating agents, such as recombinant humanized erythropoietin or darbepoetin alfa, can improve anemia in 15% to 40% of patients with lower-risk MDS 4.
- Luspatercept has recently been approved for transfusion-dependent anemic lower-risk MDS patients 5.
- Supportive care with red blood cell transfusions, erythropoiesis-stimulating agents, and iron chelation remains the mainstay of therapy for lower-risk MDS patients 5.
Higher-Risk MDS Treatment
- Hypomethylating agents, such as azacitidine, decitabine, or decitabine/cedazuridine, are first-line therapy for higher-risk MDS 4, 6.
- Azacitidine is the only agent that has shown an overall survival benefit in randomized trials 5.
- Hematopoietic cell transplantation is considered for higher-risk patients and represents the only potential cure 4, 5.
Transfusions and Iron Overload
- Red blood cell transfusions are the mainstay of treatment of anemia in MDS, but can lead to iron overload 7, 8.
- Iron chelation therapy is recommended to maintain serum ferritin levels of <1000 ng/mL and prevent clinical consequences of iron overload 8.
- Prophylactic antigen matching for RhCE and K must be used to reduce the risk of red blood cell alloimmunization 7.