Skip to content
My WebMD Sign In, Sign Up

Cancer Health Center

Font Size

Myelodysplastic Syndromes Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment for Myelodysplastic Syndromes


Combinations of azacitidine with lenalidomide [29] and vorinostat [30] are currently being compared with single-agent azacitidine in a national randomized phase II trial (S1117 [NCT01522976]).

AML induction-type chemotherapy

Induction chemotherapy typically used to treat AML may be used to treat patients with higher-risk MDS with excess blasts.[31] Low-dose cytarabine has benefitted some patients; however, this treatment was associated with a higher infection rate when compared with observation in a randomized trial. No difference in time to progression or OS was observed for patients treated with low-dose cytarabine or supportive care.

Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Allogeneic HSCT is the only potentially curative treatment for MDS. Retrospective data suggest cure rates in selected patients ranging from 30% to 60%; outcomes varied with IPSS score at time of transplant, with inferior survival in patients with higher IPSS scores.[32][Level of evidence: 3iiiDiv] The role of cytoreductive therapy in reducing the blast percentage before HSCT remains uncertain. Outcomes may not be as good for patients with treatment-related MDS (5-year disease-free survival of 8% to 30%).[33]

Although HSCT represents the only treatment modality with curative potential, the relatively high morbidity and mortality of this approach limits its use. A decision analysis predating approval of azacitidine, in patients with a median age younger than 50 years, suggested optimal survival when transplant was delayed until disease progression for lower-risk patients but implemented at diagnosis for higher-risk patients.[34]

Allogeneic stem cell transplantation with reduced-intensity conditioning (RIC) has extended transplantation as a possible modality for treatment of older patients.[35] In a retrospective analysis of 1,333 patients aged 50 years or older (median, 56 years) who underwent allogeneic transplants for MDS using HLA-matched sibling and unrelated donors, 62% of the patients received RIC HSCT, and the others received standard-dose HSCT. On multivariate analysis, use of RIC and advanced disease stage at transplantation were associated with increased relapse (hazard ratio [HR] of 1.44 and 1.51, respectively).[35][Level of evidence: 3iiiDiv] The predictors of non-relapse mortality included advanced disease stage (HR, 1.43), use of an unrelated donor, and standard dose HSCT (HR, 1.27). The 4-year OS was similar in both groups (30% after myeloablative conditioning vs. 32% in RIC.[35]

Therapy-Related Myeloid Neoplasms

In the absence of prospective data, therapy-related myeloid neoplasms are treated similarly to de novo MDS.

Current Clinical Trials

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with adult myelodysplastic syndromes. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.


  1. Tricot GJ, Lauer RC, Appelbaum FR, et al.: Management of the myelodysplastic syndromes. Semin Oncol 14 (4): 444-53, 1987.
  2. Boogaerts MA: Progress in the therapy of myelodysplastic syndromes. Blut 58 (6): 265-70, 1989.
  3. Hellström-Lindberg E: Efficacy of erythropoietin in the myelodysplastic syndromes: a meta-analysis of 205 patients from 17 studies. Br J Haematol 89 (1): 67-71, 1995.
  4. Park S, Grabar S, Kelaidi C, et al.: Predictive factors of response and survival in myelodysplastic syndrome treated with erythropoietin and G-CSF: the GFM experience. Blood 111 (2): 574-82, 2008.
  5. Mannone L, Gardin C, Quarre MC, et al.: High-dose darbepoetin alpha in the treatment of anaemia of lower risk myelodysplastic syndrome results of a phase II study. Br J Haematol 133 (5): 513-9, 2006.
  6. Gabrilove J, Paquette R, Lyons RM, et al.: Phase 2, single-arm trial to evaluate the effectiveness of darbepoetin alfa for correcting anaemia in patients with myelodysplastic syndromes. Br J Haematol 142 (3): 379-93, 2008.
  7. Jädersten M, Montgomery SM, Dybedal I, et al.: Long-term outcome of treatment of anemia in MDS with erythropoietin and G-CSF. Blood 106 (3): 803-11, 2005.
  8. Hellström-Lindberg E, Gulbrandsen N, Lindberg G, et al.: A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony-stimulating factor: significant effects on quality of life. Br J Haematol 120 (6): 1037-46, 2003.
  9. Hellström-Lindberg E, Ahlgren T, Beguin Y, et al.: Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients. Blood 92 (1): 68-75, 1998.
  10. Hellström-Lindberg E, Kanter-Lewensohn L, Ost A: Morphological changes and apoptosis in bone marrow from patients with myelodysplastic syndromes treated with granulocyte-CSF and erythropoietin. Leuk Res 21 (5): 415-25, 1997.
  11. Negrin RS, Stein R, Doherty K, et al.: Maintenance treatment of the anemia of myelodysplastic syndromes with recombinant human granulocyte colony-stimulating factor and erythropoietin: evidence for in vivo synergy. Blood 87 (10): 4076-81, 1996.
  12. Greenberg PL, Rigsby CK, Stone RM, et al.: NCCN Task Force: Transfusion and iron overload in patients with myelodysplastic syndromes. J Natl Compr Canc Netw 7 (Suppl 9): S1-16, 2009.
  13. List A, Dewald G, Bennett J, et al.: Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med 355 (14): 1456-65, 2006.
  14. List A, Kurtin S, Roe DJ, et al.: Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med 352 (6): 549-57, 2005.
  15. Sekeres MA, Maciejewski JP, Giagounidis AA, et al.: Relationship of treatment-related cytopenias and response to lenalidomide in patients with lower-risk myelodysplastic syndromes. J Clin Oncol 26 (36): 5943-9, 2008.
  16. Fenaux P, Giagounidis A, Selleslag D, et al.: RBC transfusion independence and safety profile of lenalidomide 5 or 10 mg in pts with low- or int-1-risk MDS with Del5q: results from a randomized phase III trial (MDS-004). [Abstract] Blood 114 (22): A-944, 2009.
  17. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al.: Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol 10 (3): 223-32, 2009.
  18. Molldrem JJ, Caples M, Mavroudis D, et al.: Antithymocyte globulin for patients with myelodysplastic syndrome. Br J Haematol 99 (3): 699-705, 1997.
  19. Saunthararajah Y, Nakamura R, Nam JM, et al.: HLA-DR15 (DR2) is overrepresented in myelodysplastic syndrome and aplastic anemia and predicts a response to immunosuppression in myelodysplastic syndrome. Blood 100 (5): 1570-4, 2002.
  20. Sloand EM, Olnes MJ, Shenoy A, et al.: Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions. J Clin Oncol 28 (35): 5166-73, 2010.
  21. Kantarjian H, Issa JP, Rosenfeld CS, et al.: Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer 106 (8): 1794-803, 2006.
  22. Silverman LR, Demakos EP, Peterson BL, et al.: Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol 20 (10): 2429-40, 2002.
  23. Lyons RM, Cosgriff TM, Modi SS, et al.: Hematologic response to three alternative dosing schedules of azacitidine in patients with myelodysplastic syndromes. J Clin Oncol 27 (11): 1850-6, 2009.
  24. Wijermans P, Lübbert M, Verhoef G, et al.: Low-dose 5-aza-2'-deoxycytidine, a DNA hypomethylating agent, for the treatment of high-risk myelodysplastic syndrome: a multicenter phase II study in elderly patients. J Clin Oncol 18 (5): 956-62, 2000.
  25. Lübbert M, Suciu S, Baila L, et al.: Low-dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European Organisation for Research and Treatment of Cancer Leukemia Group and the German MDS Study Group. J Clin Oncol 29 (15): 1987-96, 2011.
  26. Issa JP, Garcia-Manero G, Giles FJ, et al.: Phase 1 study of low-dose prolonged exposure schedules of the hypomethylating agent 5-aza-2'-deoxycytidine (decitabine) in hematopoietic malignancies. Blood 103 (5): 1635-40, 2004.
  27. Kantarjian H, Oki Y, Garcia-Manero G, et al.: Results of a randomized study of 3 schedules of low-dose decitabine in higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia. Blood 109 (1): 52-7, 2007.
  28. Kaminskas E, Farrell A, Abraham S, et al.: Approval summary: azacitidine for treatment of myelodysplastic syndrome subtypes. Clin Cancer Res 11 (10): 3604-8, 2005.
  29. Sekeres MA, List AF, Cuthbertson D, et al.: Phase I combination trial of lenalidomide and azacitidine in patients with higher-risk myelodysplastic syndromes. J Clin Oncol 28 (13): 2253-8, 2010.
  30. Garcia-Manero G, Estey EH, Jabbour E, et al.: Final report of a phase II study of 5-azacitidine and vorinostat in patients with newly diagnosed myelodysplastic syndrome or acute myelogenous leukemia not eligible for clinical trials because poor performance and presence of other comorbidities. [Abstract] Blood 118 (21): A-608, 2011.
  31. de Witte T, Suciu S, Verhoef G, et al.: Intensive chemotherapy followed by allogeneic or autologous stem cell transplantation for patients with myelodysplastic syndromes (MDSs) and acute myeloid leukemia following MDS. Blood 98 (8): 2326-31, 2001.
  32. Deeg HJ, Storer B, Slattery JT, et al.: Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome. Blood 100 (4): 1201-7, 2002.
  33. Witherspoon RP, Deeg HJ, Storer B, et al.: Hematopoietic stem-cell transplantation for treatment-related leukemia or myelodysplasia. J Clin Oncol 19 (8): 2134-41, 2001.
  34. Cutler CS, Lee SJ, Greenberg P, et al.: A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood 104 (2): 579-85, 2004.
  35. Schetelig J, van Biezen A, Brand R, et al.: Allogeneic hematopoietic stem-cell transplantation for chronic lymphocytic leukemia with 17p deletion: a retrospective European Group for Blood and Marrow Transplantation analysis. J Clin Oncol 26 (31): 5094-100, 2008.

WebMD Public Information from the National Cancer Institute

Last Updated: February 25, 2014
This information is not intended to replace the advice of a doctor. Healthwise disclaims any liability for the decisions you make based on this information.
Next Article:

Today on WebMD

Colorectal cancer cells
A common one in both men and women.
Lung cancer xray
See it in pictures, plus read the facts.
sauteed cherry tomatoes
Fight cancer one plate at a time.
Ovarian cancer illustration
Do you know the symptoms?
Jennifer Goodman Linn self-portrait
what is your cancer risk
colorectal cancer treatment advances
breast cancer overview slideshow
prostate cancer overview
lung cancer overview slideshow
ovarian cancer overview slideshow
Actor Michael Douglas