Danielle Townsley, Bogdan Dumitriu, Phillip Scheinberg, Ronan Desmond, Xingmin Feng, Olga Rios, Barbara Weinstein,Janet Valdez, Thomas Winkler, Keyvan Keyvanfar, Stephanie Sellers, Marie Desierto, Sanaz Soltani, Harshraj Leuva, Swetha Valluri,Colin Wu, Katherine Calvo, Andre Larochelle, Cynthia Dunbar, Neal Young
The standard first treatment for aplastic anemia is immunosuppressive treatment. This treatment improves blood cell counts in about 60% to 65% of patients with severe aplastic anemia. But blood counts return to normal (known as a complete response) in only about 10% of these patients.
Eltrombopag (Promacta®) is a treatment that increases platelet counts and decreases bleeding risk. This phase 2 clinical trial tested the efficacy of adding eltrombopag to immunosuppressive therapy with horse antithymocyte globulin and cyclosporine in two groups of patients. The first cohort included 30 patients (median age 39 years) with aplastic anemia. The second cohort included 23 patients.
- Patients in the first cohort tolerated the combination of eltrombopag and cyclosporine well, and only three patients stopped the treatment before six months.
- One third of cohort-1 patients (33%) had a complete response and 80% had some response at six months.
- 22% of cohort-2 patients had a complete response and 87% had some response at six months.
- Cohort 1 patients stopped needing platelet transfusions after a median 32 days of treatment. They stopped needing red blood cell transfusions after a median 39 days of treatment.
- Three cohort 1 patients who had responded to the treatment and one in cohort 2 had higher numbers of abnormal blood cells at follow-up.
Adding eltrombopag to immunosuppressive therapy increases overall and complete response rates.