Immunomodulatory Therapy for Myelodysplastic Syndromes


Thalidomide and lenalidomide belong to the proprietary group of immunomodulatory drugs (IMiDs) that display broad biologic and pharmacologic properties. Encouraging results of clinical studies that evaluated the efficacy of thalidomide in patients with myelodysplastic syndromes (MDSs) led to the investigation of its structural analogue, lenalidomide, in patients with lower-risk MDS. The cumulative results of studies that tested lenalidomide in patients with interstitial deletion of chromosome 5q, ie, del(5q), showed a high frequency of both erythroid and cytogenetic responses (approximately 75% of patients), which led to US Food and Drug Administration approval of this agent for this cytogenetically defined MDS subset. A multicenter phase III study (MDS-002) that investigated the frequency of transfusion response in lower-risk non-del(5q) MDS patients showed that lenalidomide had significant erythropoietic activity, albeit less robust in lower-risk MDS without del(5q). These studies established lenalidomide as an active erythropoietic-remitting agent with novel cytogenetic-remitting activity in lower-risk MDS patients who would not otherwise benefit from therapy with erythropoietic growth factors. The National Comprehensive Cancer Network Clinical Practice Guidelines recently added lenalidomide to the therapeutic algorithm for MDS as front-line therapy for lower-risk MDS patients with del(5q) and transfusion-dependent anemia.

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  1. 1.

    Vardiman JW, Harris NL, Brunning RD. The World Health Organization (WHO) classification of the myeloid neoplasms.Blood. 2002;100:2292–2302.

    Article  CAS  Google Scholar 

  2. 2.

    Hellstrom-Lindberg E. Efficacy of erythropoietin in the myelodysplastic syndromes: a meta-analysis of 205 patients from 17 studies.Br J Haematol. 1995;89:67–71.

    Article  CAS  Google Scholar 

  3. 3.

    Naing A, Sokol L, List AF. Developmental therapeutics for myelodysplastic syndromes. JNatl Compr Canc Netw. 2006;4:78–82.

    Article  CAS  Google Scholar 

  4. 4.

    Bellamy WT, Richter L, Sirjani D, et al. Vascular endothelial cell growth factor is an autocrine promoter of abnormal localized immature myeloid precursors and leukemia progenitor formation in myelodysplastic syndromes.Blood. 2001;97:1427–1434.

    Article  CAS  Google Scholar 

  5. 5.

    Bennett JM, Catovsky D, Daniel MT, et al. Proposals for the classification of the myelodysplastic syndromes.Br J Haematol. 1982;51:189–199.

    Article  CAS  Google Scholar 

  6. 6.

    Greenberg P, Cox C, LeBeau M, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes.Blood. 1997;89:2079–2088.

    PubMed  CAS  Google Scholar 

  7. 7.

    Cheson BD, Bennett JM, Kantarjian H, et al. Report of an international working group to standardize response criteria for myelodysplastic syndromes.Blood. 2000;96:3671–3674.

    PubMed  CAS  Google Scholar 

  8. 8.

    Giagounidis AA, Germing U, Aul C. Biological and prognostic significance of chromosome 5q deletions in myeloid malignancies.Clin Cancer Res. 2006;12:5–10.

    Article  CAS  Google Scholar 

  9. 9.

    Van den Berghe H, Cassiman JJ, David G, Fryns JP, Michaux JL, Sokal G. Distinct haematological disorder with deletion of long arm no. 5 chromosome.Nature. 1974;251:437–438.

    Article  Google Scholar 

  10. 10.

    Giagounidis AA, Germing U, Haase S, et al. Clinical, morphological, cytogenetic, and prognostic features of patients with myelodysplastic syndromes and del(5q) including band q31.Leukemia. 2004;18:113–119.

    Article  CAS  Google Scholar 

  11. 11.

    Raza A, Meyer P, Dutt D, et al. Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes.Blood. 2001;98:958–965.

    Article  CAS  PubMed  Google Scholar 

  12. 12.

    Revlimid [package insert]. Summit, NJ: Celgene Corp; July 2006.

  13. 13.

    Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents.Nat Rev Cancer. 2004;4:314–322.

    Article  CAS  Google Scholar 

  14. 14.

    Zorat F, Shetty V, Dutt D, et al. The clinical and biological effects of thalidomide in patients with myelodysplastic syndromes.BrJ Haematol. 2001;115:881–894.

    Article  CAS  Google Scholar 

  15. 15.

    Moreno-Aspitia A, Geyer S, Li C, et al. N998B: multicenter phase II trial of thalidomide (Thal) in adult patients with myelodysplastic syndromes (MDS) [abstract].Blood. 2002;100:96a.

    Article  Google Scholar 

  16. 16.

    Tamburini J, Elie C, Vassilief D, et al. Low doses of thalidomide (Thal) in low risk MDS with transfusion-dependant anemia: the GFM THAL-SMD-200 trial [abstract].Blood. 2006;108:756a. Abstract 2673.

    Article  CAS  Google Scholar 

  17. 17.

    List A, Kurtin S, Roe DJ, et al. Efficacy of lenalidomide in myelodysplastic syndromes.N Engl J Med. 2005;352:549–557.

    Article  CAS  Google Scholar 

  18. 18.

    List AF, Baker AF, Green S, Bellamy W. Lenalidomide: targeted anemia therapy for myelodysplastic syndromes.Cancer Control. 2006;13(Suppl):4–11.

    Article  Google Scholar 

  19. 19.

    List A, Dewald G, Bennett J, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion.N Engl J Med. 2006;355:1456–1465.

    Article  CAS  Google Scholar 

  20. 20.

    Raza A, Reeves JE, Feldman EJ, et al. Long term clinical benefit of lenalidomide (Revlimid) treatment in patients with myelodysplastic syndrome without del 5q cytogenetic abnormalities [abstract].Blood. 2006;108:78a. Abstract 250.

    Article  Google Scholar 

  21. 21.

    National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology V.1.2007. Myelodysplastic syndromes. Available at: Accessed October 16, 2007.

  22. 22.

    Kurtin S, Sokol L. Practical considerations in the use of lenalidomide therapy for myelodysplastic syndromes.Cancer Control. 2006;13(Suppl):26–31.

    Article  Google Scholar 

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Correspondence to Lubomir Sokol or Alan F. List.

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Sokol, L., List, A.F. Immunomodulatory Therapy for Myelodysplastic Syndromes. Int J Hematol 86, 301–305 (2007).

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Key words

  • Myelodysplastic syndromes
  • Thalidomide
  • Lenalidomide
  • Immunomodulatory therapy
  • del(5q)