The thrombopoietin receptor agonists (TPO-RAs) have continued to demonstrate efficacy in the frontline management of patients with severe aplastic anaemia.
Associate Professor Antonio Risitano told the ASH 2024 Annual Meeting that the final analysis of the RACE study confirmed the benefit of adding eltrombopag to standard immunosuppressive therapy (IST) in this patient group.
The European RACE study delivered early results in 2022 [link to NEJM here].
Associate Professor Risitano, chair of the EBMT Severe Aplastic Anaemia Working Party, presented the final 24-months analysis which found significantly higher haematologic response rates, event-free survival and disease-free survival with eltrombopag.
Overall survival was borderline superior with eltrombopag and IST compared to IST alone.
“I think we can conclude and confirm that eltrombopag therapy should be the first line treatment for the severe aplastic anemia patients not eligible for transplantation,” he said.
Second generation TPO-RA
Professor Zoe McQuilten
Speaking in the same session, Professor Zoe McQuilten from Monash Health, presented the results of the DIAAMOND-Ava-FIRST study of the second generation TPO-RA avatrombopag in treatment-naive severe aplastic anaemia.
While approved for other indications including chronic ITP, avatrombopag has not previously been evaluated for use in severe aplastic anaemia.
Professor McQuilten said the potential advantages of avatrombopag over eltrombopag included that its pharmacokinetics were not altered by age, sex, race or hepatic impairment.
The single arm study enrolled 56 patients from 11 sites across Australia, with 49 evaluable for the primary endpoint of complete response (CR) at 6 months. The study protocol was aligned with that of the larger RACE study.
She said the overall response with avatrombopag was 65% with 35% of participants achieving a CR and nearly 31% achieving a partial response. Response rates were comparable to that of the RACE study.
Regarding safety, 5% of DIAAMOND-Ava-FIRST participants demonstrated acquired clonal evolution at 6 months.
The efficacy and safety of avatrombopag is also being evaluated in relapsed/refractory severe aplastic anaemia in the DIAAMOND-Ava-NEXT study.
Similar efficacy, fewer boundaries:
Avatrombopag + IST delivers comparable results to RACE RCT (35% CR, 5% ACE at 6 months).
A new chapter in sAA treatment! #ASH24 pic.twitter.com/t2u3Sq3GRV
— Dr.Rachit Khandelwal, MD (@dr_khandelwal99) December 8, 2024