Scientific report 2025 of the Severe Aplastic Anaemia Working Party (SAAWP)

The SAAWP is extending its international leadership in the field of bone marrow failures continuing top-level investigation in transplant and non-transplant treatment for Aplastic Anaemia and other related conditions. This was achieved through research through the different main research programs.

1. RACE trial, with its ancillary studies

The prospective, randomized 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 hematolopoietic 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 hematological 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 2025 the efforts focused on:

• Final manuscript reporting the long-term follow up of the study, looking at 2-year follow up data (manuscript under revision)
• Final manuscript reporting biological data on somatic mutations detected by NGS (manuscript accepted for publication in the New England Journal of Medicine Evidence)
• Manuscript reporting biological data on immune abnormalities detected by CyTOFF (abstract selected as oral publication at ASH, manuscript in preparation)
• Analysis of Patient Reported Outcomes and comparison with novel PRO instruments (to be presented at EBMT 2026)
• Investigation of infectious risk in the natural history of AA (in collaboration with the IDWP)

The RACE trial also generated some new studies: 

• 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
• Retrospective study: comparison of available data from RACE (non-transplant treatment) vs registry data (transplant treatment), in a matched-paired analysis 
   

2. Retrospective studies from the EBMT Registry

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 2025 the efforts focused on the following key projects:

• Outcome of patients transplanted for AA, looking at the impact of age (by donor type) and other possible factors (manuscript in preparation)
• Outcome of patients transplanted after clonal evolution (ie, transformation to either MDS or AML) of an initial SAA
• Outcome of patients transplanted for PNH, looking at the impact of possible factors
• Outcome of patients transplanted for DKC, looking at the impact of possible factors (selected as poster presentation at ASH)
• Impact of TBI (in the context of conditioning regimen for allo-HCT) on fertility of female patients transplanted for SAA

Click here to see the full list of SAAWP studies.

3. Future projects.

The SAAWP is planning to specifically investigate more in detail the differential outcomes of transplant vs non-transplant as (initial) treatment of AA. 

• A Prospective Observational Study describing outcomes of Immunosuppressive treatment and Transplantation as first-line treatment for adult severe aplastic anaemia

• SAAWP Business Meeting - 1 April 2025 - Florence, Italy
• SAAWP Business Meeting - 9 September 2025 - Basel, Switzerland