Myelodysplastic syndromes - Many new drugs, little therapeutic progress

Mayo Clinic Proceedings

Volumn 85, Issue 11, 2010, Pages 1042-1045

  Tefferi, Ayalew ^a  

^a MAYO CLINIC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; ALKYLATING AGENT; AZACITIDINE; CYTARABINE; DEFERASIROX; DEPSIPEPTIDE; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; GYRASE INHIBITOR; HYDROXYUREA; INTERLEUKIN 3; LENALIDOMIDE; PANOBINOSTAT; VALPROIC ACID; VORINOSTAT;

ACUTE GRANULOCYTIC LEUKEMIA; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ANEMIA; CANCER SURVIVAL; CELL STRUCTURE; CYTOPENIA; DNA METHYLATION; ERYTHROCYTE TRANSFUSION; HUMAN; LOW DRUG DOSE; MYELODYSPLASTIC SYNDROME; MYELOID LEUKEMIA; NOTE; RISK ASSESSMENT; TREATMENT INDICATION;

EID: 78149306054     PISSN: 00256196     EISSN: None     Source Type: Journal    
DOI: 10.4065/mcp.2010.0502     Document Type: Note

References (48)

1. Tefferi A, Vardiman JW. Myelodysplastic syndromes. N Engl J Med. 2009;361:1872-1885.
2. Vardiman JW, Thiele J, Arber DA, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114:937-951.
3. Daley GQ, Van Etten RA, Baltimore D. Induction of chronic myelogenous leukemia in mice by the P210bcr/abl gene of the Philadelphia chromosome. Science. 1990;247:824-830. (Pubitemid 20078558)
4. Pozdnyakova O, Miron PM, Tang G, et al. Cytogenetic abnormalities in a series of 1,029 patients with primary myelodysplastic syndromes: a report from the US with a focus on some undefined single chromosomal abnormalities. Cancer. 2008;113:3331-3340.
5. Rollison DE, Howlader N, Smith MT, et al. Epidemiology of myelodysplastic syndromes and chronic myeloproliferative disorders in the United States, 2001-2004, using data from the NAACCR and SEER programs. Blood. 2008;112:45-52.
6. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89:2079-2088.
7. Germing U, Hildebrandt B, Pfeilstocker M, et al. Refinement of the international prognostic scoring system (IPSS) by including LDH as an additional prognostic variable to improve risk assessment in patients with primary myelodysplastic syndromes (MDS). Leukemia. 2005;19:2223-2231.
8. List A. Deciphering the path ahead in myelodysplastic syndrome [editorial]. Am J Hematol. 2010;85:157-158.
9. Graubert T. Therapy-related myelodysplastic syndrome: models and genetics. Biol Blood Marrow Transplant. 2010;16(1, suppl):S45-S47.
10. Chang C, Storer BE, Scott BL, et al. Hematopoietic cell transplantation in patients with myelodysplastic syndrome or acute myeloid leukemia arising from myelodysplastic syndrome: similar outcomes in patients with de novo disease and disease following prior therapy or antecedent hematologic disorders. Blood. 2007;110:1379-1387.
11. Warlick ED, Cioc A, Defor T, Dolan M, Weisdorf D. Allogeneic stem cell transplantation for adults with myelodysplastic syndromes: importance of pretransplant disease burden. Biol Blood Marrow Transplant. 2009;15:30-38.
12. Martino R, Iacobelli S, Brand R, et al. Retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic hematopoietic stem cell transplantation using HLA-identical sibling donors in myelodysplastic syndromes. Blood. 2006;108:836-846.
13. Tefferi A. Is "cure" essential in the treatment of cancer [letter]? Mayo Clin Proc. 2008;83:1413-1414.
14. Rajkumar SV. Treatment of myeloma: cure vs control. Mayo Clin Proc. 2008;83:1142-1145.
15. Figueroa ME, Skrabanek L, Li Y, et al. MDS and secondary AML display unique patterns and abundance of aberrant DNA methylation. Blood. 2009;114:3448-3458.
16. Flotho C, Claus R, Batz C, et al. The DNA methyltransferase inhibitors azacitidine, decitabine and zebularine exert differential effects on cancer gene expression in acute myeloid leukemia cells. Leukemia. 2009;23:1019-1028.
17. Fandy TE, Herman JG, Kerns P, et al. Early epigenetic changes and DNA damage do not predict clinical response in an overlapping schedule of 5-azacytidine and entinostat in patients with myeloid malignancies. Blood. 2009;114:2764-2773.
18. 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. 2002;20:2429-2440.
19. 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.
20. Zwierzina H, Suciu S, Loeffler-Ragg J, et al. Low-dose cytosine arabinoside (LD-AraC) vs LD-AraC plus granulocyte/macrophage colony stimulating factor vs LD-AraC plus Interleukin-3 for myelodysplastic syndrome patients with a high risk of developing acute leukemia: final results of a randomized phase III study (06903) of the EORTC Leukemia Cooperative Group. Leukemia. 2005;19:1929-1933.
21. 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. 2009;10:223-232.
22. WijerMans P. Suciu S, Baila L, et al. Low dose decitabine versus best supportive care in elderly patients with intermediate or high risk MDS not eligible for intensive chemotherapy: final results of the randomized phase III study (06011) of the EORTC Leukemia and German MDS Study Groups [abstract 226]. ASH Annual Meeting Abstracts 2008;112(11):226.
23. Oosterveld M, Muus P, Suciu S, et al. Chemotherapy only compared to chemotherapy followed by transplantation in high risk myelodysplastic syndrome and secondary acute myeloid leukemia; two parallel studies adjusted for various prognostic factors. Leukemia. 2002;16:1615-1621.
24. Beran M, Shen Y, Kantarjian H, et al. High-dose chemotherapy in high-risk myelodysplastic syndrome: covariate-adjusted comparison of five regimens. Cancer. 2001;92:1999-2015.
25. 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.
26. Schafer PH, Gandhi AK, Loveland MA, et al. Enhancement of cytokine production and AP-1 transcriptional activity in T cells by thalidomide-related immunomodulatory drugs. J Pharmacol Exp Ther. 2003;305:1222-1232.
27. Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents. Nat Rev Cancer. 2004;4:314-322.
28. 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.
29. 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.
30. Ades L, Boehrer S, Prebet T, et al. Efficacy and safety of lenalidomide in intermediate-2 or high-risk myelodysplastic syndromes with 5q deletion: results of a phase 2 study. Blood. 2009;113:3947-3952.
31. Ma X, Does M, Raza A, Mayne ST. Myelodysplastic syndromes: incidence and survival in the United States. Cancer. 2007;109:1536-1542. (Pubitemid 46595689)
32. Malcovati L, Porta MG, Pascutto C, et al. Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol. 2005;23:7594-7603.
33. Chee CE, Steensma DP, Wu W, Hanson CA, Tefferi A. Neither serum ferritin nor the number of red blood cell transfusions affect overall survival in refractory anemia with ringed sideroblasts. Am J Hematol. 2008;83:611-613.
34. Patnaik MM, Lasho TL, Finke CM, et al. WHO-defined 'myelodysplastic syndrome with isolated del(5q)' in 88 consecutive patients: survival data, leukemic transformation rates and prevalence of JAK2, MPL and IDH mutations. Leukemia. 2010;24:1283-1289.
35. Tefferi A, Siragusa S, Hussein K, et al. Transfusion-dependency at presentation and its acquisition in the first year of diagnosis are both equally detrimental for survival in primary myelofibrosis - prognostic relevance is independent of IPSS or karyotype. Am J Hematol. 2010;85(1):14-17.
36. Tefferi A, Mesa RA, Pardanani A, et al. Red blood cell transfusion need at diagnosis adversely affects survival in primary myelofibrosis - increased serum ferritin or transfusion load does not. Am J Hematol. 2009;84:265-267.
37. Gattermann N, Finelli C, Porta MD, et al. Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: results from the large 1-year EPIC study. Leuk Res. 2010;34(9):1143-1150.
38. Rose C, Brechignac S, Vassilief D, et al. Does iron chelation therapy improve survival in regularly transfused lower risk MDS patients? A multicenter study by the GFM (Groupe Francophone des Myelodysplasies). Leuk Res. 2010;34:864-870.
39. Raptis A, Duh MS, Wang ST, et al. Treatment of transfusional iron overload in patients with myelodysplastic syndrome or severe anemia: data from multicenter clinical practices. Transfusion. 2010;50:190-199.
40. Metzgeroth G, Dinter D, Schultheis B, et al. Deferasirox in MDS patients with transfusion-caused iron overload - a phase-II study. Ann Hematol. 2009;88:301-310.
41. Leitch HA. Improving clinical outcome in patients with myelodysplastic syndrome and iron overload using iron chelation therapy. Leuk Res. 2007;31(suppl 3):S7-S9.
42. 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. Eur J Haematol. 2007;78:487-494.
43. Tefferi A, Stone RM. Iron chelation therapy in myelodysplastic syndrome - cui bono [editorial]? Leukemia. 2009;23(8):1373.
44. Steensma DP. Myelodysplasia paranoia: iron as the new radon. Leuk Res. 2009;33:1158-1163.
45. DeLoughery TG. Iron: the fifth horseman of the apocalypse [editorial]? Am J Hematol. 2009;84(5):263-264.
46. Camp CL, Smoot RL, Kolettis TN, Groenewald CB, Greenlee SM, Farley DR. Patient records at Mayo Clinic: lessons learned from the first 100 patients in Dr Henry S. Plummer's dossier model. Mayo Clin Proc. 2008;83:1396-1399.
47. Christmas C, Kravet SJ, Durso SC, Wright SM. Clinical excellence in academia: perspectives from masterful academic clinicians. Mayo Clin Proc. 2008;83:989-994.
48. Lanier WL. Bidirectional conflicts of interest involving industry and medical journals: who will champion integrity? [editorial]? Mayo Clin Proc. 2009;84(9):771-775.