Open AccessFive‐day regimen of azacitidine for lower‐risk myelodysplastic syndromes (refractory anemia or refractory anemia with ringed sideroblasts): A prospective single‐arm phase 2 trial
Author(s) -
Morita Yasuyoshi,
Maeda Yasuhiro,
Yamaguchi Terufumi,
Urase Fumiaki,
Kawata Shuhei,
Hanamoto Hitoshi,
Tsubaki Kazuo,
Ishikawa Jun,
Shibayama Hirohiko,
Matsumura Itaru,
Matsuda Mitsuhiro
Publication year - 2018
Publication title -
cancer science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.035
H-Index - 141
eISSN - 1349-7006
pISSN - 1347-9032
DOI - 10.1111/cas.13739
Subject(s) - medicine , azacitidine , myelodysplastic syndromes , neutropenia , regimen , anemia , refractory (planetary science) , gastroenterology , international prognostic scoring system , adverse effect , clinical endpoint , phases of clinical research , febrile neutropenia , surgery , chemotherapy , clinical trial , bone marrow , biochemistry , gene expression , chemistry , physics , astrobiology , dna methylation , gene
Although azacitidine is the first‐line drug for higher‐risk myelodysplastic syndrome ( MDS ) patients, its efficacy for lower‐risk MDS remains unestablished. Therefore, we conducted a prospective study to examine the efficacy and safety of a 5‐day regimen of azacitidine ( AZA ‐5) for lower‐risk MDS . The primary endpoint was hematological improvement ( HI ) after 4 courses of therapy. A total of 51 patients with lower‐risk MDS based on the French‐American‐British ( FAB ) classification (44 patients with refractory anemia [ RA ] and 7 patients with refractory anemia with ringed sideroblasts [ RARS ]) were enrolled from 6 centers in Japan. The median age was 75 years (range: 51‐88). These patients received AZA ‐5 (75 mg/m 2 ; once daily for 5 sequential days). The median number of AZA ‐5 courses was 8 (range: 1‐57), and 45 patients (88.2%) received more than 4 courses. HI and transfusion independency were seen in 24 patients (47.1%) and 11 patients (39.2%), respectively. A total of 11 patients (21.6%) achieved complete remission or marrow remission. WT 1 mRNA levels were not significantly correlated with therapy response. Grade 3 or 4 neutropenia and thrombocytopenia occurred in 26 (51.0%) and 11 (21.5%) patients, respectively. Nonhematological grade 3 or 4 adverse events were observed in 9 patients (17.6%). Together, these results indicate that AZA ‐5 is feasible and effective for lower‐risk MDS patients as well as for higher‐risk MDS patients.