Scientific report 2023 of the Severe Aplastic Anaemia Working Party (SAAWP)

Continuation of RACE trial scientific activities and investigation on key questions related to transplant for Severe Aplastic Anaemia

The prospective, randomised phase 3 RACE trial set immunosuppressive therapy (ATG+CsA) plus Eltrombopag (also referred as “triple therapy”, or “ACE therapy”) as the new standard of care for adult SAA patients not eligible for a low-risk allogeneic hematopoietic stem cell transplantation. This was a seminal study conducted by the EBMT Clinical Trial Office, showing that this experimental treatment resulted in faster and better haematological response as compared with previous standard of care (is, same treatment without eltrombopag). The SAAWP is still generating results and data from this study, which remains a milestone for the SAAWP. In 2023 the efforts focused on:

• Generation of the final study report (all patients evaluated at the last study visit, at 24 months from treatment starting)
• Analysis of biological data from patients samples collected during the study (at baseline, 6 and 24 months from treatment
• Continuation of the RACE trial within its extension, retrospective “RACE-2” study, which aims to investigate the long-term outcome of patients initially enrolled in the RACE trial. 

In addition to RACE-related activities, the SAAWP is conducting a number of studies aiming to address key questions in the field of transplantation for acquired and inherited bone marrow failures. In 2023 the efforts focused on:

• Outcome of patients transplanted for Fanconi Anaemia
• Outcome of patients transplanted after clonal evolution (ie, transformation to either MDS or AML) of an initial SAA
• Outcome of SAA patients transplanted from haplo-identical donors (vs unrelated and related matched donors)

• SAAWP Business Meeting (Virtual) - November 14, 2023.