Initial Treatment Approach for Myelodysplastic Syndrome

Risk Stratification

The International Prognostic Scoring System (IPSS) and IPSS-Revised (IPSS-R) are standard tools for risk stratification, categorizing patients into lower-risk and higher-risk groups 4, 5
Risk assessment should consider cytopenias, bone marrow blast percentage, cytogenetic abnormalities, and molecular mutations 3, 5
Patient factors including age, performance status, comorbidities, and transfusion requirements guide treatment decisions 3, 6

For anemia without del(5q), the European Society for Medical Oncology recommends first-line treatment with erythropoiesis-stimulating agents (ESAs) at weekly doses of 30,000-80,000 units or darbepoetin 150-300 μg 1, 5
For anemia with del(5q), lenalidomide is the most effective first-line treatment with 60-65% response rates and median transfusion independence of 2-2.5 years 1, 7, 3
The American Society of Hematology recommends TPO receptor agonists (romiplostim, eltrombopag) for severe thrombocytopenia, but only in patients with marrow blasts <5% 1, 8

The National Comprehensive Cancer Network recommends first-line therapy with azacitidine 75 mg/m² daily for 7 days for patients not immediately eligible for allogeneic stem cell transplantation (allo-SCT) 1, 3, 7
Allogeneic stem cell transplantation should be proposed to all higher-risk MDS patients <70 years without major comorbidities who have a donor 1, 3, 6

The European Society for Medical Oncology recommends RBC transfusions for symptomatic anemia, generally at hemoglobin thresholds of at least 8 g/dl (higher in patients with comorbidities) 8
Iron chelation therapy is recommended for transplant candidates with iron overload, non-transplant candidates with major iron overload, and patients with relatively favorable prognosis who have received 20-60 RBC concentrates or if serum ferritin rises above 1000-2500 U/l 1, 2, 3