The key advance in severe aplastic anaemia has been the addition of eltrombopag, an oral thrombopoietin-receptor agonist, to standard immunosuppressive therapy (horse antithymocyte globulin plus cyclosporine). In the Phase 3 RACE trial, adding eltrombopag improved the complete-response rate at three months (22% vs 10%), raised the six-month overall response rate (about 68% vs 41%), and shortened the time to response, without additional toxicity — establishing eltrombopag-containing triple therapy as first-line for patients not proceeding directly to transplant. Its main considerations are hepatotoxicity and a small risk of clonal evolution (including chromosome 7 abnormalities), warranting monitoring. Allogeneic transplantation remains the preferred option for younger patients with a matched donor.
Further reading: RACE eltrombopag plus immunosuppression (Peffault de Latour et al., NEJM 2022); earlier eltrombopag-IST data (Townsley et al., NEJM 2017).