Scientific report 2024 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 confirmed by four oral presentations at ASH 2024 (all the 4 studies submitted were selected for oral sessions. See HERE).

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 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 2024 the efforts focused on:

• Final manuscript reporting the long-term follow up of the study, looking at 2-year follow up data
• 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 (see below)
• Investigation of infectious risk in the natural history of AA (in collaboration with the IDWP)
   

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 2024 the efforts focused on the following key projects:

• 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
• Outcome of patients transplanted for AA, looking at the impact of age (by donor type) and other possible factors
• 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 SAA patients transplanted with a PTCY GVHD prophylaxis

For the full list of studies see the section below.

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.

• Retrospective study: comparison of available data from RACE (non-transplant treatment) vs registry data (transplant treatment), in a matched-paired analysis
• Prospective study: A Prospective Observational Study describing outcomes of Immunosuppressive treatment and Transplantation as first-line treatment for adult severe aplastic anaemia
• 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. 

• ESH-EBMT-EHA-IPIG 3rd Translational Research Conference: Bone Marrow Failure and Leukaemia Predisposition Syndromes - 15-17 November 2024 - Paris, France
• SAAWP Business Meeting - 16 April 2024 - Glasgow, UK
• SAAWP Business Meeting - 11 September 2024 - Genova, Italy