Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan

Objective In Japan, immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG), and cyclosporine A (CsA) is the standard of care in patients aplastic anemia (AA) who are not indicated for stem-cell transplantation, although some may experience relapse. This study assessed efficacy safety eltrombopag combination rabbit-ATG/CsA IST-naive non-severe or severe AA Japan. Methods this non-randomized, open-label, single-arm, phase II study, were initiated on Days 1 15 (±3 days), respectively, continued ?26 weeks; rabbit-ATG was given 5 days (Days to 5). The primary endpoint overall response rate (ORR) at Week 26. Patients ?70 years old between 71 75 based recommendation investigator enrolled Results Of 11 patients, 10 started treatment eltrombopag. ORRs Weeks 26 52 70.0% 60.0%, respectively. ORR 100% (all 3 patients) 57.1% (4/7) AA. Among transfusion-dependent 66.7% (4/6) 62.5% (5/8) became red blood cell- platelet-transfusion independent, most common adverse events nausea headache. No deaths hematologic malignancies reported. cytogenetic abnormality reported one patient. Conclusion confirmed clinical benefit plus