Eltrombopag (a thrombomimetic agent that stimulates or boosts the production and maturation of blood cells, from the precursor blood making cells within the bone marrow) received FDA approval in 2014 as a drug of use in the setting of refractory aplastic anaemia.


Reports from the extended study of the use of Eltrombopag from the National Institute of Haematology (NIH) by Dunbar and colleagues, have shown that extended use of this drug, for up to 6 months at a fixed dose of 150 mg/day could speed and improve the response rates. Data from this extended study shared at ASH 2017, showed responses in up to a half of these patients, when they were given the larger dose of 150 mg/day and for an extended period of 6 months. Nearly 2/3rd of patients who had a robust response after a median duration of 12 months, could discontinue treatment.

Of the 3 patients who had disease relapse, after discontinuation of the drug, response was restored on re-initiation of treatment. However, clonal evolution remains a concern and is seen in nearly 15% of patients at 6 months interval. No factor can reliably predict at this point in time, as to who is likely to progress with clonal evolution. This remains a story in development, undoubtedly with the promise of response in patients especially who have been refractory to IST previously, but with the caveat of careful monitoring.