Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

ABSTRACT:

Background

Acquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.

Methods

We enrolled 92 consecutive patients in a prospective phase 1-2 study of immunosuppressive therapy plus eltrombopag. The three consecutively enrolled cohorts differed with regard to the timing of initiation and the duration of th

SUBMITTER: Townsley DM 

PROVIDER: S-EPMC5548296 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature