title: Pilot study on combination of azacitidine and low-dose cytarabine for patients with refractory anemia with excess blast.
abstract: This study analyzed the outcomes of the combination of azacitidine and low-dose cytarabine in patients newly diagnosed with refractory anemia with excess blast (RAEB). Patients were treated with azacitidine 75 mg/m(2) for 7 days subcutaneously and cytarabine 20 mg/m(2) intravenously for 7 days every 28 days. The assigned regimen was repeated for two cycles, then the patients treated with azacytidine alone until progression or allogeneic stem cell transplantation (allo-SCT). Eighteen patients with 5 RAEB-1 and 13 RAEB-2 were enrolled in the current study. After two cycles of the combination therapy, responses were achieved in nine patients (50.0%): four complete response (CR) (22.2%), one partial response (5.6%), two marrow-CR (11.1%), and two hematologic improvement (11.1%). Four patients (22.2%) progressed to acute leukemia during two cycles of the combination therapy. The 1-year overall survival (OS) was 87.5% for the early response group (responses at two cycles) and 0% for the late response group (responses at four cycles, p = 0.042). Plus, the median survival time was 476 days (range, 37-718 days) for the early response group and 221 days (range, 193-249 days) for the late response group. The 1-year OS was 100% for the patients who underwent allo-SCT and 73.4% for those without allo-SCT. In summary, the combination therapy showed promising response rate when compared to treatment with azacitidine alone. However, it was limited in terms of preventing leukemic transformation. Allo-SCT would seem to be the only available treatment that can alter disease progression.
authors.affiliation: Department of Hematology/Oncology, Kyungpook National University Hospital, Kyungpook National University School of Medicine, Jung-Gu, Daegu, South Korea.
authors.forename: Joon Ho
authors.initials: JH
authors.lastname: Moon
authors.forename: Soo Jung
authors.initials: SJ
authors.lastname: Lee
authors.forename: Yoo Jin
authors.initials: YJ
authors.lastname: Lee
authors.forename: Byung Woog
authors.initials: BW
authors.lastname: Kang
authors.forename: Yee Soo
authors.initials: YS
authors.lastname: Chae
authors.forename: Jong Gwang
authors.initials: JG
authors.lastname: Kim
authors.forename: Jang Soo
authors.initials: JS
authors.lastname: Suh
authors.forename: Sang Kyun
authors.initials: SK
authors.lastname: Sohn
mesh_terms: D000328:Adult; D000368:Aged; D000754:Anemia, Refractory, with Excess of Blasts / Q000188:drug therapy* / Q000503:physiopathology / Q000601:surgery; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use*; D003561:Cytarabine / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D003561:Cytarabine; D001374:Azacitidine