What are the treatment protocols for very-low-, low-, or intermediate-risk myelodysplastic syndromes (MDS)?

Updated: Nov 04, 2019
  • Author: Matthew C Foster, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Answer

These patients should receive supportive care. Selected patients who have not required transfusion and have modest, asymptomatic cytopenias may be observed initially.

Patients with symptomatic anemia; < 2 units (U) of red blood cell (RBC) transfusion required per month; and 5q31 deletion, with or without other cytogenetic abnormalities:

  • Lenalidomide 10 mg PO daily, either continuous dosing or on days 1-21 every 28 d

  • If no response or intolerance, treat as for patients without del5q

Patients with symptomatic anemia, < 2 units (U) of RBC transfusion required per month, no del5q, ring sideroblasts < 15%, and low (≤500 mU/mL) serum erythropoietin levels:

Patients with symptomatic anemia, < 2 units (U) of RBC transfusion required per month, no del5q, ring sideroblasts ≥15%, and low (≤500 mU/mL) serum erythropoietin levels:

  • Epoetin alfa 40,000-60,000 U SC 1-3 times weekly, or

  • Darbepoetin alfa 150-300 µg SC weekly [3, 4]  plus

  • Filgrastim 300 µg SC 3 times weekly, doses adjusted to hemoglobin (Hb) level of 11-13 g/dL, and total white blood cell (WBC) count ≤10 × 109/L [5]

Patients with symptomatic anemia, < 2 units (U) of RBC transfusion required per month, no del5q, and serum erythropoietin levels > 500 mU/mL:\

  • Evaluate likelihood of response to immunosuppressive therapy

  • Patients who are < 60y and have ≤5% marrow blasts or those with hypocellular marrows, are human leukocyte antigen (HLA)–DR15 positive, paroxysmal nocturnal hemoglobinuria (PNH) clone positive, or have STAT-3 mutant cytotoxic T cell clones are likely to respond to equine antithymocyte globulin (ATG) 40 mg/kg/day IV over 4-6h for 4d plus cyclosporine starting at 5-12 mg/kg/day IV beginning on day 14, dosed to maintain therapeutic levels of 200-400 ng/mL. [6]

  • Patients without those characteristics are unlikely to respond to immunosuppressive therapy and should be considered for  azacitidine/decitabine or lenalidomide. [6]

  • Recommended azacitidine dose is 75 mg/m2 SC or IV on days 1-7; every 28 d [7, 8, 9]  or

  • Alternative azacitidine schedule, associated with similar response rates: 75 mg/m2 SC or IV on days 1-5, 8, and 9; every 28 d [10]  or

  • Decitabine on FDA-approved schedule: 15 mg/m 2 IV over 3 h every 8 h (or 45 mg/m 2/day) for 3 d; every 6 wk [11]   or
  • Decitabine on outpatient schedule, now widely adopted: 20 mg/m 2 IV over 1 h daily on days 1-5; every 28 d [11, 12, 13]   or
  • Lenalidomide , 10 mg PO daily, either continuous dosing or on days 1-21 every 28 d

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