A randomized controlled trial comparing darbepoetin alfa doses in red blood cell transfusion-dependent patients with low- or intermediate-1 risk myelodysplastic syndromes

作者：Jang Jun Ho; Harada Hironori; Shibayama Hirohiko; Shimazaki Ryutaro; Kim Hyeoung Joon; Sawada Kenichi; Mitani Kinuko^*

来源：International Journal of Hematology, 2015, 102(4): 401-412.

DOI：10.1007/s12185-015-1862-5

• Myelodysplastic syndromes
• MDS
• Darbepoetin alfa
• Erythropoiesis-stimulating agent
• EPO

Darbepoetin alfa (DA) is a standard treatment for anemia in lower-risk MDS. However, to date there has been no comparative study to investigate the initial dosage. We, thus, conducted a randomized controlled trial to elucidate the optimal initial dosage of DA. International Prognostic Scoring System low or intermediate-1 risk MDS patients with hemoglobin levels a parts per thousand currency sign9.0 g/dL, serum erythropoietin levels a parts per thousand currency sign500 mIU/mL, and red blood cell transfusion dependency were enrolled. Patients were randomized to receive DA either at 60, 120, or 240 mu g/week for 16 weeks followed by continuous administration with dose adjustment up to 48 weeks. Of 17, 18, and 15 patients in the 60, 120, and 240 mu g DA groups included in the efficacy analysis, 64.7, 44.4, and 66.7 %, respectively, achieved the primary endpoint (major or minor erythroid response), while 17.6, 16.7, and 33.3 % achieved major erythroid responses in the initial 16-week period. No clinically significant safety concerns were identified. DA reduced the transfusion requirements effectively and safely in transfusion-dependent, lower-risk MDS patients. Given the highest achievement rate of the major erythroid response in the 240 mu g group and the absence of dose-dependent adverse events, 240 mu g weekly is the optimal initial dosage.

• 出版日期2015-10