Best Practice & Research Clinical Haematology
Volume 21, Issue 4 , Pages 639-646 , December 2008

Are new agents really making a difference in MDS?

  • Richard M. Stone, MD (Associate Professor of Medicine, Harvard Medical School Director, Adult Leukemia Program)

      Affiliations

    • Corresponding Author InformationAdult Leukemia Program, Dana-Farber Cancer Institute, 44 Binney Street, Boston, MA 02115, USA. Tel.: +1 617 632 2214; Fax: +1 617 632 2933.

References 

  1. Ebert BL, Pretz J, Bosco J, et al. Identification of RPS14 as a 5q- syndrome gene by RNA interference screen. Nature. 2008;451:335–339
  2. Magnusson MK, Meade KE, Nakamura R, et al. Activity of STI571 in chronic myelomonocytic leukemia with a platelet-derived growth factor beta receptor fusion oncogene. Blood. 2002;100:1088–1091
  3. Braun T, Fenaux P. Farnesyltransferase inhibitors and their potential role in therapy for myelodysplastic syndromes and acute myeloid leukaemia. British Journal of Haematology. 2008;
  4. Schiller GJ, Slack J, Hainsworth JD, et al. Phase II multicenter study of arsenic trioxide in patients with myelodysplastic syndromes. Journal of Clinical Oncology. 2006;24:2456–2464
  5. Galanopoulos A, Kritikou-Griva E, Gligori J, et al. Treatment of patients with myelodysplastic syndrome with amifostine. Leukemia Research. 2001;25:665–671
  6. Burnett AK, Milligan D, Prentice AG, et al. A comparison of low-dose cytarabine and hydroxyurea with or without all-trans retinoic acid for acute myeloid leukemia and high-risk myelodysplastic syndrome in patients not considered fit for intensive treatment. Cancer. 2007;109:1114–1124
  7. Visani G, Malagola M, Piccaluga PP, et al. Low dose Ara-C for myelodysplastic syndromes: is it still a current therapy?. Leukemia & Lymphoma. 2004;45:1531–1538
  8. Bennett CL, Silver SM, Djulbegovic B, et al. Venous thromboembolism and mortality associated with recombinant erythropoietin and darbepoetin administration for the treatment of cancer-associated anemia. JAMA: The Journal of The American Medical Association. 2008;299:914–924
  9. Hellstrom-Lindberg E, Malcovati L. Supportive care and use of hematopoietic growth factors in myelodysplastic syndromes. Seminars in Hematology. 2008;45:14–22
  10. Kurzrock R, Kantarjian HM, Blascovich MA, et al. Phase I study of alternate-week administration of tipifarnib in patients with myelodysplastic syndrome. Clinical Cancer Research. 2008;14:509–514
  11. Fenaux P, Raza A, Mufti GJ, et al. A multicenter phase 2 study of the farnesyltransferase inhibitor tipifarnib in intermediate- to high-risk myelodysplastic syndrome. Blood. 2007;109:4158–4163
  12. Raponi M, Lancet JE, Fan H, et al. A 2-gene classifier for predicting response to the farnesyltransferase inhibitor tipifarnib in acute myeloid leukemia. Blood. 2008;111:2589–2596
  13. Raponi M, Harousseau JL, Lancet JE, et al. Identification of molecular predictors of response in a study of tipifarnib treatment in relapsed and refractory acute myelogenous leukemia. Clinical Cancer Research. 2007;13:2254–2260
  14. Callander N, Ochoa-Bayona JL, Piro L, et al. Hematologic improvement following treatment with TLK199 (TelintraTM), a novel glutathione analog inhibitor of GST P1-1, in myelodysplastic syndrome (MDS): interim results of a dose-ranging phase 2a study. Blood. 2004;104:400a;[abstract 1428]
  15. Laport GG, Sandmaier BM, Storer BE, et al. Reduced-intensity conditioning followed by allogeneic hematopoietic cell transplantation for adult patients with myelodysplastic syndrome and myeloproliferative disorders. Biology of Blood Marrow Transplantation. 2008;14:246–255
  16. Garcia-Manero G, Yang H, Bueso-Ramos C, et al. Phase 1 study of the histone deacetylase inhibitor vorinostat (suberoylanilide hydroxamic acid [SAHA]) in patients with advanced leukemias and myelodysplastic syndromes. Blood. 2008;111:1060–1066
  17. Sloand EM, Wu CO, Greenberg P, et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. Journal of Clinical Oncology. 2008;
  18. Lim ZY, Killick S, Germing U, et al. Low IPSS score and bone marrow hypocellularity in MDS patients predict hematological responses to antithymocyte globulin. Leukemia. 2007;21:1436–1441
  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. 2002;100:1570–1574
  20. Jonasova A, Neuwirtova R, Cermak J, et al. Cyclosporin A therapy in hypoplastic MDS patients and certain refractory anaemias without hypoplastic bone marrow. British Journal of Haematology. 1998;100:304–309
  21. Armand P, Kim HT, Cutler CS, et al. Prognostic impact of elevated pretransplantation serum ferritin in patients undergoing myeloablative stem cell transplantation. Blood. 2007;109:4586–4588
  22. Takatoku M, Uchiyama T, Okamoto S, et al. Retrospective nationwide survey of Japanese patients with transfusion-dependent MDS and aplastic anemia highlights the negative impact of iron overload on morbidity/mortality. European Journal of Haematology. 2007;78:487–494
  23. Porter J, Galanello R, Saglio G, et al. Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. European Journal of Haematology. 2008;80:168–176
  24. List A, Dewald G, Bennett J, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. The New England Journal of Medicine. 2006;355:1456–1465
  25. Raza A, Reeves JA, Feldman EJ, et al. Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1 risk myelodysplastic syndromes with karyotypes other than deletion 5q. Blood. 2008;111:86–93
  26. Ebert BL, Galili N, Tamayo P, et al. An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome. PLoS Medicine. 2008;5:e35
  27. 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. Journal of Clinical Oncology. 2002;20:2429–2440
  28. Kornblith AB, Herndon JE, Silverman LR, et al. Impact of azacytidine on the quality of life of patients with myelodysplastic syndrome treated in a randomized phase III trial: a Cancer and Leukemia Group B study. Journal of Clinical Oncology. 2002;20:2441–2452
  29. Fenaux P, Mufti GJ, Santini V, et al. Azacitidine (AZA) treatment prolongs overall survival (OS) in higher-risk MDS patients compared with conventional care regimens (CCR): Results of the AZA-001 phase III study. Blood. 2007;110:250a;[abstract 817]
  30. Kantarjian H, Issa JP, Rosenfeld CS, et al. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006;106:1794–1803
  31. 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. 2007;109:52–57
  32. Steensma DP, Baer MR, Slack JL, et al. Preliminary results of a phase II study of decitabine administered daily for 5 days every 4 weeks to adults with myelodysplastic syndrome (MDS). Blood. 2007;110:434a;[abstract 1450]
  33. Soriano AO, Yang H, Faderl S, et al. Safety and clinical activity of the combination of 5-azacytidine, valproic acid, and all-trans retinoic acid in acute myeloid leukemia and myelodysplastic syndrome. Blood. 2007;110:2302–2308

PII: S1521-6926(08)00055-8

doi: 10.1016/j.beha.2008.06.004

Best Practice & Research Clinical Haematology
Volume 21, Issue 4 , Pages 639-646 , December 2008