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Azacitidine Azacitidine (Vidaza) 75 mg/m2/d sc qd x 7 days q4w
Silverman LR 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; 24:3895 (link to the article). List AF et al. Effect of azacitidine (AZA) on overall survival in higher-risk myelodysplastic syndromes (MDS) without complete remission. 2008 ASCO annual meeting. Abstract 7006 (link to the abstract).
Silverman LR et al. Randomized controlled trial of azacitidine in patients with myelodysplastic syndrome: a study of the Cancer and Leukemia Group B. J Clin Oncol 2002; 20:2429 (link to the article).
Decitabine (Dacogen) Regimen 1 20 mg/m2/d iv over 1 h d1-5 q4w Kantarijian H et al. Results of a randomized study of three schedules of low-dose decitabine in higher risk myelodysplastic syndrome and chronic myelomonocytic leukemia. Blood 2007; 109:52 (link to the article). Regimen 2 15 mg/m2 iv over 3 hrs q8h x 3 days q6w Kantarjian H et al. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer 2006; 106:1794 (link to the article). Ruter B et al. Superiority of prolonged low-dose azanucleoside administration? Results of 5-aza-2'-deoxycytidine retreatment in high-risk myelodysplasia patients. Cancer 2006; 106:1744 (link to the article). Approved by FDA on 5/2/2006
Lenalidomide Lenalidomide (Revlimid) 10 mg po qd Approved by FDA on 12/27/2005 Raza A 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 (link to the article). List A et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Eng J Med 2006; 355:1456 (link to the article). Tefferi A et al. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood 2006; 108:1158 (link to the article). List A et al. Efficacy of lenalidomide in myelodysplastic syndromes. N Eng J Med 2005; 352:549 (link to the article).
Imatinib (for MDS/MPD associated with PDGFR gene re-arrangements) Imatinib (Gleevec) 400 mg po qd Approved by FDA on 10/19/06 (link to FDA site). David M et al. Durable responses to imatinib in patients with PDGFRB fusion gene-positive and BCR-ABL-negative chronic myeloproliferative disorders. Blood 2007; 109:61 (link to the article).
Antithymocyte globulin (ATG) 40 mg/kg/d iv d1-4 Sloand EM et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol 2008; 26:2505 (link to the article). Molldrem JJ et al. Antithymocyte globulin for treatment of bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med 2002; 137:156 (link to the article). Steensma DP et al. Antithymocyte globulin has limited efficacy and substantial toxicities in unselected anemic patients with myelodysplastic syndrome. Blood 2003; 101:2156 (link to the article).
Cyclosporine Cyclosporine 5-6 mg/kg/d po bid, adjust for blood levels between 100 to 300 ng/mL Sloand EM et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol 2008; 26:2505 (link to the article).
Jonasova A et al. Cyclosporine A therapy in hypoplastic MDS patients and certain refractory anemias without hypoplastic bone marrow. Br J Haematol 1998; 100:304 (link to the article).
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