Established and novel agents for myelodysplastic syndromes

Hematology (United States)

Volumn 2014, Issue 1, 2014, Pages 82-89

  Sekeres, Mikkael A ^a   Gerds, Aaron T ^a  

^a TAUSSIG CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT;

HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; MYELODYSPLASTIC SYNDROMES; RISK FACTOR;

ANTINEOPLASTIC AGENTS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MYELODYSPLASTIC SYNDROMES; RISK FACTORS;

EID: 84930363104     PISSN: 15204391     EISSN: 15204383     Source Type: Journal    
DOI: 10.1182/asheducation-2014.1.82     Document Type: Article

References (48)

1. SEER Cancer Statistics Review 1975-2008: Myelodysplastic syndromes (MDS), chronic myeloproliferative disorders (CMD), and chronic myelomonocytic leukemia (CMML). Available from: http://seer.cancer.gov/csr/1975-2008/results-merged/sect-30-mds.pdf. Accessed August 2, 2014.
2. Cogle CR, Craig BM, Rollison DE, List AF. Incidence of the myelodysplastic syndromes using a novel claims-based algorithm: high number of uncaptured cases by cancer registries. Blood. 2011;117(26): 7121-7125.
3. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997; 89(6):2079-2088.
4. Kantarjian H, O'Brien S, Ravandi F, et al. Proposal for a new risk model in myelodysplastic syndrome that accounts for events not considered in the original International Prognostic Scoring System. Cancer. 2008; 113(6):1351-1361.
5. 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(30): 7594-7603.
6. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454-2465.
7. Vardiman J, Thiele J, Arber D, et al. The 2008 revisions of the WHO classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114(5):937-951.
8. Howlader N NA, Krapcho M, Garshell J, et al, eds. SEER Cancer Statistics Review, 1975-2010. Bethesda: National Cancer Institute; 2013.
9. Putila J, Remick SC, Guo NL. Combining clinical, pathological, and demographic factors refines prognosis of lung cancer: a populationbased study. PLoS One. 2011;6(2):e17493.
10. Sekeres MA, Maciejewski JP, List AF, et al. Perceptions of Disease State, Treatment Outcomes, and Prognosis Among Patients with Myelodysplastic Syndromes: Results From an Internet-Based Survey. Oncologist. 2011;16(6):904-911.
11. Steensma DP, Komrokji RS, Stone RM, et al. Disparity in perceptions of disease characteristics, treatment effectiveness, and factors influencing treatment adherence between physicians and patients with myelodysplastic syndromes. Cancer. 2014;120(11):1670-1676.
12. Golshayan AR, Jin T, Maciejewski J, et al. Efficacy of growth factors compared to other therapies for low-risk myelodysplastic syndromes. Br J Haematol. 2007;137(2):125-132.
13. Jadersten M, Montgomery SM, Dybedal I, Porwit-MacDonald A, Hellstrom-Lindberg E. Long-term outcome of treatment of anemia in MDS with erythropoietin and G-CSF. Blood. 2005;106(3):803-811.
14. Hellstrom-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. 2003;120(6):1037-1046.
15. Sekeres MA, Fu AZ, Maciejewski JP, Golshayan AR, Kalaycio ME, Kattan MW. A Decision analysis to determine the appropriate treatment for low-risk myelodysplastic syndromes. Cancer. 2007;109(6):1125-1132.
16. Santini V, Schemenau J, Levis A, et al. Can the revised IPSS predict response to erythropoietic-stimulating agents in patients with classical IPSS low or intermediate-1 MDS? Blood. 2013;122(13):2286-2288.
17. Giagounidis A, Mufti GJ, Fenaux P, et al. Results of a randomized, double-blind study of romiplostim versus placebo in patients with low/intermediate-1-risk myelodysplastic syndrome and thrombocytopenia. Cancer. 2014;120(12):1838-1846.
18. Ebert BL, Pretz J, Bosco J, et al. Identification of RPS14 as a 5qsyndrome gene by RNA interference screen. Nature. 2008;451(7176): 335-339.
19. Wei S, Chen X, McGraw K, et al. Lenalidomide promotes p53 degradation by inhibiting MDM2 auto-ubiquitination in myelodysplastic syndrome with chromosome 5q deletion. Oncogene. 2013;32(9):1110-1120.
20. List A, Dewald G, Bennett J, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006;355(14): 1456-1465.
21. Fenaux P, Giagounidis A, Selleslag D, et al. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011;118(14):3765-3776.
22. 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(1):86-93.
23. Germing U, Lauseker M, Hildebrandt B, et al. Survival, prognostic factors, and rates of leukemic transformation in a multicenter study of 303 untreated patients with MDS and del(5q) [abstract]. Blood (ASH Annual Meeting Abstracts). 2009;114(22):945.
24. Sekeres MA, Giagounidis A, List AF, et al. Predictive factors for overall survival (OS) and AML progression in a large cohort of patients with low-/int-1-risk MDS with del(5Q) treated with lenalidomide (LEN). Haematologica. 2011;96(S2):481.
25. Raza A, Galili N, Smith SE, et al. A phase 2 randomized multicenter study of 2 extended dosing schedules of oral ezatiostat in low to intermediate-1 risk myelodysplastic syndrome. Cancer. 2012;118(8): 2138-2147.
26. Sokol L, Cripe L, Kantarjian H, et al. Randomized, dose-escalation study of the p38alpha MAPK inhibitor SCIO-469 in patients with myelodysplastic syndrome. Leukemia. 2013;27(4):977-980.
27. Passweg JR, Giagounidis AA, Simcock M, et al. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care-SAKK 33/99. J Clin Oncol. 2011;29(3):303-309.
28. Komrokji RS, Mailloux AW, Chen DT, et al. A phase 2 multicenter rabbit anti-thymocyte globulin trial in patients withmyelodysplastic syndromes identifying a novel model for responseprediction. Haematologica. 2014;99(7):1176-1183.
29. Garcia-Manero G, Jabbour E, Borthakur G, et al. Randomized open-label phase II study of decitabine in patients with low- or intermediate-risk myelodysplastic syndromes. J Clin Oncol. 2013; 31(20):2548-2553.
30. Mahfouz RZ, Rickki E, Juersivich JA, et al. Non-cytotoxic differentiation therapy based on mechanism of disease produces complete remission in myelodysplastic syndromes (MDS) with high risk cytogenetics [abstract]. Blood (ASH Annual Meeting Abstracts). 2012;120(21): 1696.
31. Garcia-Manero G, Gore SD, Cogle C, et al. Phase I study of oral azacitidine in myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia. J Clin Oncol. 2011;29(18):2521-2527.
32. Jiang Y, Dunbar A, Gondek LP, et al. Aberrant DNA methylation is a dominant mechanism in MDS progression to AML. Blood. 2009;113(6): 1315-1325.
33. Shen L, Kantarjian H, Guo Y, et al. DNA methylation predicts survival and response to therapy in patients with myelodysplastic syndromes. J Clin Oncol. 2010;28(4):605-613.
34. Traina F, Visconte V, Elson P, et al. Impact of molecular mutations on treatment response to DNMT inhibitors in myelodysplasia and related neoplasms. Leukemia. 2014;28(1):78-87.
35. 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(10): 2429-2440.
36. 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(3):223-232.
37. 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(8):1794-1803.
38. Lubbert 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. 2011;29(15):1987-1996.
39. Sekeres MA, Tiu RV, Komrokji R, et al. Phase 2 study of the lenalidomide and azacitidine combination in patients with higher-risk myelodysplastic syndromes. Blood. 2012;120(25):4945-4951.
40. Silverman LR, Verma A, Odchimar-Reissig R, et al. A phase II trial of epigenetic modulators vorinostat in combination with azacitidine (azaC) in patients with the myelodysplastic syndrome (MDS): initial results of Study 6898 Of The New York Cancer Consortium [abstract]. Blood (ASH Annual Meeting Abstracts). 2013;122(21):386.
41. Quintas-Cardama A, Kantarjian HM, Ravandi F, et al. Very high rates of clinical and cytogenetic response with the combination of the histone deacetylase inhibitor pracinostat (SB939) and 5-azacitidine in high-risk myelodysplastic syndrome [abstract]. Blood (ASH Annual Meeting Abstracts). 2012;120(21):3821.
42. Prebet T, Sun Z, Figueroa ME, et al. Prolonged administration of azacitidine with or without entinostat for myelodysplastic syndrome and acute myeloid leukemia with myelodysplasia-related changes: results of the US Leukemia Intergroup Trial E1905. J Clin Oncol. 2014;32(12): 1242-1248.
43. Gerds AT, Gooley TA, Estey EH, Appelbaum FR, Deeg HJ, Scott BL. Pretransplantation therapy with azacitidine vs induction chemotherapy and posttransplantation outcome in patients with MDS. Biol Blood Marrow Transplant. 2012;18(8):1211-1218.
44. Damaj G, Duhamel A, Robin M, et al. Impact of azacitidine before allogeneic stem-cell transplantation for myelodysplastic syndromes: a study by the Societe Francaise de Greffe de Moelle et de Therapie-Cellulaire and the Groupe-Francophone des Myelodysplasies. J Clin Oncol. 2012;30(36):4533-4540.
45. Prebet T, Gore SD, Esterni B, et al. Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. J Clin Oncol. 2011; 29(24):3322-3327.
46. Yang H, Bueso-Ramos C, Dinardo C, et al. Expression of PD-L1, PD-L2, PD-1 and CTLA4 in myelodysplastic syndromes is enhanced by treatment with hypomethylating agents. Leukemia. 2014;28(6):1280-1288.
47. Sekeres MA, Cutler C. How we treat higher-risk MDS. Blood. 2014;123(6):829-836.
48. Gerds AT, Scott BL. Last marrow standing: bone marrow transplantation for acquired bone marrow failure conditions. Curr Hematol Malig Rep. 2012;7(4):292-299.