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Scientific report 2021 of the Severe Aplastic Anaemia Working Party (SAAWP)

Major achievements

Top level publication and initiation of the RACE-2 trial:

  1. RACE trial comparing standard immunosuppressive therapy (ATG+CsA) plus Eltrombopag vs Standard IST alone in patients with severe aplastic anemia (SAA) has been accepted for publication in the NEJM on October 14th 2021. This EBMT study, supported financially by Novartis, Pfizer and Alexion, demonstrated that Eltrombopag at the top of standard hATG+CSA was associated with a better and quicker response at 3 and 6 months. At 2 years, event free survival was significantly higher for patients receiving first line the experimental treatment. Horse ATG+CSA+Eltrombopag is now the standard of care for adult patients with SAA. We acknowledge the Clinical Trial Office (CTO) of the EBMT, together with all RACE investigators and nurses for their outstanding work, as well as patients who have accepted to participate to this trial.
  2. The RACE-2 trial is ready in 2021. This retrospective study will assess the long-term follow-up of patients included in RACE which is of outstanding importance to evaluate the long-term safety of the use of Eltrombopag first line in patients with SAA. The trial will begin early 2022.
  2019 2020 2021
Oral presentations 8 4 8
Poster presentations 1 6 0
Educational events 2 4 3