The myelodysplastic syndromes

Expert Opinion on Biological Therapy

Volumn 7, Issue 3, 2007, Pages 369-377

  Sekeres, Mikkael A ^a  

^a TAUSSIG CANCER INSTITUTE   (United States)

Author keywords

Myelodysplastic syndromes; Outcome; Treatment

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; AMG 531; AZACITIDINE; CYTOKINE; ENZYME INHIBITOR; FG 2216; GROWTH FACTOR; LENALIDOMIDE; RECOMBINANT ERYTHROPOIETIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; SCIO 469; THALIDOMIDE; UNCLASSIFIED DRUG; VALPROIC ACID;

ACUTE GRANULOCYTIC LEUKEMIA; ANEMIA; APOPTOSIS; BLEEDING; BONE MARROW SUPPRESSION; BONE MARROW TRANSPLANTATION; CELL PROLIFERATION; CHRONIC MYELOMONOCYTIC LEUKEMIA; CLINICAL TRIAL; CONSTIPATION; CONTROLLED CLINICAL TRIAL; CYTOPENIA; DISEASE CLASSIFICATION; DRUG DOSE ESCALATION; DRUG MECHANISM; DYSPNEA; ERYTHROCYTE TRANSFUSION; FATIGUE; HEMATOPOIESIS; HUMAN; INFECTION; INJECTION SITE REACTION; MYELODYSPLASTIC SYNDROME; NAUSEA; NEUTROPENIA; PATHOGENESIS; PERIPHERAL NEUROPATHY; RANDOMIZED CONTROLLED TRIAL; REVIEW; RISK ASSESSMENT; RISK FACTOR; THROMBOCYTOPENIA; TREATMENT RESPONSE;

HUMANS; MYELODYSPLASTIC SYNDROMES;

EID: 33847723311     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.7.3.369     Document Type: Review

References (50)

1. LIST AF, VARDIMAN J, ISSA JP, DEWITTE TM: Myelodysplastic syndromes. Hematology (Am. Soc. Hematol. Educ. Program) (2004) 2004:297-317.
2. HARRIS NL, JAFFE ES, DIEBOLD J et al.: The World Health Organization classification of neoplastic diseases of the hematopoietic and lymphoid tissues. Report of the Clinical Advisory Committee meeting, Airlie House, Virginia, November, 1997. Ann. Oncol. (1999) 10(12):1419- 1432. •• The basis for the current pathologic classification system for MDS.
3. AUL C, GATTERMANN N, SCHNEIDER W: Age-related incidence and other epidemiological aspects of myelodysplastic syndromes. Br. J. Haematol. (1992) 82(2):358-367.
4. PAUL B, REID MM, DAVISON EV, ABELA M, HAMILTON PJ: Familial myelodysplasia: progressive disease associated with emergency of monosomy 7. Br. J. Haematol. (1987) 65(3):321-323.
5. PASQUALETTI P, CASALE R, COLANTONIO D, COLLACCIANI A: Occupational risk for hematological malignancies. Am. J. Hematol. (1991) 38(2):147-149.
6. KARP JE, SMITH MA: The molecular pathogenesis of treatment-induced (secondary) leukemias: foundations for treatment and prevention. Semin. Oncol. (1997) 24(1):103-113.
7. 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(5):1427-1434.
8. SCHIPPERUS M, SONNEVELD P, LINDEMANS J et al.: The effects of interleukin-3, GM-CSF, and G-CSF on the growth kinetics of colony-forming cells in myelodysplastic syndromes. Leukemia (1990) 4(4):267-272.
9. SUGIMOTO K, HIRANO N, TOYOSHIMA H et al.: Mutations of the p53 gene in myelodysplastic syndrome (MDS) and MDS-derived leukemia. Blood (1993) 81(11):3022-3026.
10. HAASE D, STEIDL C, SCHANZ J et al.: Correlation of cytogenetic findings with morphology, clinical course and prognosis in 2124 patients with MDS. Blood (2005) 106:232a.
11. GREENBERG P, COX C, LEBEAU MM et al.: International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood (1997) 89(6):2079-2088. •• The most widely accepted prognostic system for MDS.
12. BENNETT JM, CATOVSKY D, DANIEL MT et al.: Proposals for the classification of the myelodysplastic syndromes. Br. J. Haematol. (1982) 51(2):189-199.
13. VARDIMAN JW, HARRIS NL, BRUNNING RD: The World Health Organization (WHO) classification of the myeloid neoplasms. Blood (2002) 100(7):2292-2302. •• The basis for the pathologic classification system for MDS used at present.
14. MALCOVATI L, GERMING U, KUENDGEN A et al.: A WHO classification-based prognostic scoring system (WPSS) for predicting survival in myelodysplastic syndromes. Blood (2005) 106:232a.
15. CHESON BD, GREENBERG PL, BENNETT JM et al.: Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood (2006) 108(2):419-425. •• Article standardizing response criteria in MDS patients.
16. HELLSTROM-LINDBERG E, NEGRIN R, STEIN R et al.: Erythroid response to treatment with G-CSF plus erythropoietin for the anaemia of patients with myelodysplastic syndromes: proposal for a predictive model. Br. J. Haematol. (1997) 99(2):344-351.
17. 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. •• This article includes a tool to predict the probability that MDS patients will respond to growth factors.
18. SEKERES M, FU A, MACIEJEWSKI J, GOLSHAYAN A, KALAYCIO M, KATTAN M: A decision analysis to determine the appropriate treatment for low-risk myelodysplastic syndromes. Blood (2005) 106:711a.
19. CORRAL LG, KAPLAN G: Immunomodulation by thalidomide and thalidomide analogues. Ann. Rheum. Dis. (1999) 58(Suppl. 1):I107-113.
20. DAVIES FE, RAJE N, HIDESHIMA T et al.: Thalidomide and immunomodulatory derivatives augment natural killer cell cytotoxicity in multiple myeloma. Blood (2001) 98(1):210-216.
21. GEITZ H, HANDT S, ZWINGENBERGER K: Thalidomide selectively modulates the density of cell surface molecules involved in the adhesion cascade. Immunopharmacology (1996) 31(2-3):213-221.
22. PARMAN T, WILEY MJ, WELLS PG: Free radical-mediated oxidative DNA damage in the mechanism of thalidomide teratogenicity. Nat. Med. (1999) 5(5):582-585.
23. 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(4):958-965.
24. CHESON BD, BENNETT JM, KANTARJIAN H et al.: Myelodysplastic syndromes standardized response criteria: further definition. Blood (2001) 98(6):1985.
25. MUSTO P, FALCONE A, SANPAOLO G, BISCEGLIA M, MATERA R, CARELLA AM: Thalidomide abolishes transfusion-dependence in selected patients with myelodysplastic syndromes. Haematologica (2002) 87(8):884-886.
26. STRUPP C, GERMING U, AIVADO M, MISGELD E, HAAS R, GATTERMANN N: Thalidomide for the treatment of patients with myelodysplastic syndromes. Leukemia (2002) 16(1):1-6.
27. MORENA-ASPITIA A, GEYER S, LI C-Y et al: N998B: multicenter Phase II trial of thalidomide (thal) in adult patients with myelodysplastic syndromes (MDS). Blood (2002) 100:96a.
28. LIST A, TATE W, GLINSMANN-GIBSON B: The immunomodulatory thalidomide analog, CC-5013, inhibits tropic response in VEGF in AML cells by abolishing cytokine-induced PI3/Akt activation. Blood (2002) 100:139a.
29. LIST A, KURTIN S, ROE D et al.: Efficacy of lenalidomide in myelodysplastic syndromes. N. Engl. J. Med. (2005) 352(6):549-557. •• Article demonstrating the efficacy of lenalidomide in patients with the del(5q) abnormality.
30. LIST A, DEWALD G, BENNETT J et al.: Hematologic and cytogenetic (CTG) response to lenalidomide (CC-5013) in patients with transfusion-dependent (TD) myelodysplastic syndrome (MDS) and chromosome 5q31.1 deletion: Results of the multicenter MDS-003 study. J. Clin. Oncol. (Proc. Am. Soc. Clin. Oncol.) (2005) 23:2s.
31. SCHILLER GJ, SLACK J, HAINSWORTH JD et al.: Phase II multicenter study of arsenic trioxide in patients with myelodysplastic syndromes. J. Clin. Oncol. (2006) 24(16):2456-2464. •• Article reporting US response rates to aresenic trioxide in MDS patients.
32. VEY N, BOSLY A, GUERCIA et al.: Arsenic trioxide in patients with myelodysplastic syndromes: a Phase II multicenter study. J. Clin. Oncol. (2006) 24(16):2465-2471. •• Article reporting EU response rates to aresenic trioxide in MDS patients.
33. SILVERMAN LR: Targering hypomethylation of DNA to achieve cellular differentiation in myelodysplastic syndromes (MDS). Oncologist (2001) 6(Suppl. 5):8-14.
34. 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. •• The registration trial for 5-azacytidine in MDS.
35. SILVERMAN LR, MCKENZIE DR, PETERSON BL et al.: Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J. Clin. Oncol. (2006) 24(24):3895-3903.
36. 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. •• The registration trial for decitabine in MDS.
37. KANTARJIAN H, OKI Y, GARCIA-MANERO G et al.: Results of a randomized study of three schedules of low-dose decitabine in higher risk myelodysplastic syndrome and chronic myelomonocytic leukemia. Blood (2006) (In Press).
38. ESTEY E: Reducing mortality associated with immediate treatment complications of adult leukemias. Semin. Hematol. (2001) 38(4 Suppl. 10):32-37.
39. KANTARJIAN H, BERAN M, CORTES J et al.: Long-term follow-up results of the combination of topotecan and cytarabine and other intensive chemotherapy regimens in myelodysplastic syndrome. Cancer (2006) 106(5):1099-1109.
40. HALLEMEIER CL, GIRGIS MD, BLUM WG et al.: Long-term remissions in patients with myelodysplastic syndrome and secondary acute myelogenous leukemia undergoing allogeneic transplantation following a reduced intensity conditioning regimen of 550 cGy total body irradiation and cyclophosphamide. Biol. Blood Marrow Transplant. (2006) 12(7):749-757.
41. ALYEA EP, KIM HT, HO V et al.: Comparative outcome of nonmyeloablative and myeloablative allogeneic hematopoietic cell transplantation for patients older than 50 years of age. Blood (2005) 105(4):1810-1814.
42. ANDERSON JE, APPELBAUM FR, FISHER LD et al.: Allogeneic bone marrow transplantation for 93 patients with myelodysplastic syndrome. Blood (1993) 82(2):677-681.
43. SIERRA J, RADICH J, HANSEN JA et al.: Marrow transplants from unrelated donors for treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia. Blood'(1997) 90(4):1410-1414.
44. RUNDE V, DE WITTE T, ARNOLD R et al.: Bone marrow transplantation from HLA-identical siblings as first-line treatment in patients with myelodysplastic syndromes: early transplantation is associated with improved outcome. Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. (1998) 21(3):255-261.
45. SUTTON L, CHASTANG C, RIBAUD P et al.: Factors influencing outcome in de novo myelodysplastic syndromes treated by allogeneic bone marrow transplantation: a long-term study of 71 patients Societe Francaise de Greffe de Moelle. Blood (1996) 88(1):358-365.
46. 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 (2004) 104(2):579-585.
47. WANG B, NICHOL JL, SULLIVAN JT: Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin. Pharmacol. Ther. (2004) 76(6):628-638.
48. SALITURO FG, GERMANN UA, WILSON KP, BEMIS GW, FOX T, SU MS: Inhibitors of p38 MAP kinase: therapeutic intervention in cytokine-mediated diseases. Curr. Med. Chem. (1999) 6(9):807-823.
49. WARNECKE C, ZABOROWSKA Z, KURRECK J et al.: Differentiating the functional role of hypoxia-inducible factor (HIF)-1alphaand HIF-2alpha (EPAS-1) by the use of RNA interference: erythropoietin is a HIF-2alpha target gene in Hep3B and Kelly cells. FASEB J. (2004) 18(12):1462-1464.
50. KUENDGEN A, KNIPP S, FOX F et al.: Results of a Phase II study of valproic acid alone or in combination with all-trans retinoic acid in 75 patients with myelodysplastic syndrome and relapsed or refractory acute myeloid leukemia. Ann. Hematol. (2005) 84(Suppl. 13):61-66.