One of the effects of the coronavirus health crisis has been that AA patients who would otherwise be candidates for a transplant are unable to have one. This is one of the issues we have been keenly advocating on, to ensure that AA patients are a priority in these difficult times.

The AAT is delighted that, thanks to this patient advocacy work, - the drug eltrombopag will now be rapidly made available for these patients, and for severe AA patients who have previously failed to respond to ATG/ciclosporin.

Eltrombopag is given as a tablet - it helps to improve blood counts and can reduce the urgent requirement for first line stem cell transplant or immunosuppressive therapyduring the COVID-19 pandemic. It reduces a patient’s chances of requiring hospital admission until they can receive definitive treatment (which of course is particularly desirable at times when Covid-19 cases are high.)

Previously, getting access to eltrombopag for patients who hadn't responded to immunosuppressive therapy involved a quite laborious process, involving individual funding requests - requests which were often rejected by individual clinical commissioning groups.

This new policy, approved by the Department for Health, removes these barriers - and recommends eltrombopag as a bridging treatment for patients with severe or very severe aplastic anaemia during the COVID-19 pandemic. The drug will be funded centrally for everywhere in the UK.

Particular congratulations are due to the AAT’s trustee Dr Judith Marsh, who has been working closely with the NHS England to get approval for this drug, and to highlight the importance of protecting aplastic anaemia patients in these challenging times.

Will children with AA be given access to Eltrombotag?

This announcement only affects adult AA patients. In our recent Q&A, pediactic consultant Dr Samarasinghe discussed whether eltrombotag was effective for children with aplastic anaemia and how they can get access to it:

 You can view all the answers to questions from the webinar here