Exciting new immunosuppressive therapy treatment for aplastic anaemia

Dr Townsley talks to ecancertv at ASH 2015 about adding eltrombopag to standard immunosuppressive therapy for aplastic anaemia in previously untreated patients.

Eltrombopag was originally developed to increase platelet counts in patients with chronic immune thrombocytopenia (ITP) but it was found to also increase white and red cell counts. This was logical in hindsight, Dr Townsley observes in the interview.

Although it is licensed for the treatment of refractory aplastic anaemia in Europe, eltrombopag is only approved for use in ITP in the USA and further study data were needed. The study presented by Dr Townsley aimed to provide evidence that eltrombopag could be used in aplastic anaemia setting and included 92 patients with the rare disease.

Results showed a clear benefit of adding eltrombopag to standard immunosuppressive therapy of horse antithymocyte globulin (hATG) and cyclosporine versus the immunosuppressive therapy alone.

Haematological overall response rates were 80% and 85% at 3 and 6 months, respectively, and highest if eltrombopag was given on the first day of immunosuppressive therapy as opposed to 2 weeks after and continued for either 3 or 6 months.

Overall survival is 99%, Dr Townsley highlights, as only one patient has died while in the study to date but follow up is short.