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

Updated: Nov 04, 2019
  • Author: Matthew C Foster, MD; Chief Editor: Emmanuel C Besa, MD  more...
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R-IPSS high-risk or very-high-risk patients regardless of transfusion frequency or intermediate-risk patients with high transfusion needs (> 2 U RBCs per month)

Patients who are good candidates for high-intensity therapy (ie, young patients with few or no comorbidities, good performance status, and adequate psychosocial support):

  • Acute myelogenous leukemia (AML)–type induction therapy, such as 7+3 (idarubicin 12 mg/m2 IV push on days 1-3 plus cytarabine 100-200 mg/m2/day continuous IV infusion on days 1-7) [14] or azacitidine or decitabine, as outlined above

  • Allogeneic stem cell transplantation consultation should also be recommended

  • Allogeneic stem cell transplantation may be performed as initial therapy or following cytoreduction with any of the other therapies for MDS

Patients who are not good candidates for high-intensity therapy:

  • Azacitidine has been associated with improvements in transfusion dependence, quality of life (QoL), and overall survival (OS) in phase III trials

  • Azacitidine 75 mg/m2 SC or IV on days 1-7; every 28 d; this regimen has been associated with improvements in transfusion dependence, QoL and OS in phase III trials [7, 8, 9]  or

  • Alternative schedule of azacitidine, 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/m2 IV over 3 h every 8h (or 45 mg/m2/day) for 3 d; every 6 wk [11] or

  • Decitabine on outpatient schedule, now widely adopted: 20 mg/m2 IV over 1 h daily on days 1-5; every 28 d [11, 12, 13]

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