Addition of eltrombopag to immunosuppressive therapy in patients with newly diagnosed aplastic anemia.

ABSTRACT:

Background

The immune-mediated destruction of hematopoietic stem cells is implicated in the pathophysiology of aplastic anemia (AA). Immunosuppressive therapy (IST) using antithymocyte globulin and cyclosporine is successful in this setting. Eltrombopag is active in patients with refractory AA, presumably by increasing the bone marrow progenitors.

Methods

This phase 2 trial initially was designed to evaluate standard IST in newly diagnosed patients with severe AA and later was amended to add eltrombopag to simultaneously address immune destruction and stem cell depletion. The primary outcome was the overall response rate (ORR) at 3 months and 6 months.

Results

A total of 38 patients were enrolled: 17 (45%) received IST alone and 21 (55%) received additional eltrombopa

SUBMITTER: Assi R 

PROVIDER: S-EPMC6800043 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature