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Scientific report 2020 of the Severe Aplastic Anaemia Working Party (SAAWP)

Major achievements

Progression of two prospective randomised clinical trials:

  1. RACE trial compares standard immunosuppressive therapy (ATG+CsA) plus Eltrombopag vs Standard IST alone in patients with severe aplastic anemia. This is an EBMT study supported financially by Novartis, Pfizer and Alexion. Accrual is finished since April 2019. Manuscript preparation is on going.
  2. MAA study compares CsA plus Placebo vs CsA plus Eltrombopag in patients with moderate aplastic anaemia. This study is sponsored by the University of Ulm and supported financially by Novartis. 16 EBMT centres from 6 countries (Germany, Italy, France, The Netherlands, Switzerland and the UK) will recruit patients. Up to now, 49 patients have been enrolled and treated according to the study plan. The trial is still open for enrolment.

Van Bekkum Award

2020’s EBMT van Bekkum award has been awarded jointly to Régis Peffault de Latour, Carlo Dufour and Antonio Risitano for RACE: “Results of the EBMT Severe Aplastic Anemia Working Party (SAAWP) phase 3 prospective randomised multicentre study of horse ATG and ciclosporin with or without eltrombopag in naïve SAA patients”. We acknowledge the Clinical Trial Office 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. Read our press release “EBMT trial shows improvements in treatment of Severe Aplastic Anaemia”.

Principal research studies

  1. Haploidentical stem cell transplantation for congenital bone marrow failure: PI: Stefano Giardino
  2. Clonal evolution in AA: immune escape and somatic genomic alterations PI: Pedro Prata de Lima
  3. Danazol / Oxymethalone in SAA PI: Simona Pagliuca & Austin Kulasekararaj
  4. Fertility after a conditioning regimen containing 2-4 Gy TBI PI: Andrea Bacigalupo
  5. Comprehensive analysis of modified GRFS for severe aplastic anemia: PI: Raynier Devillier

Key publications

Impact factor_(Aplastic Aenemia)

  2015 2016 2017 2018 2019 2020
Oral presentations 23 20 13 17 8 4
Poster presentations 5 1 9 3 1 10
International educational events 0 1 1 1 2 1

Major educational courses

  1. SAAWP Business Meeting – June 10, 2020 / [Virtual].
  2. Mid-term focus meeting on non-malignant disease – July 8-10, 2020 / [Virtual].
  3. Joint EBMT-ESH Educational Meeting – Translational research conference on bone marrow failure – Nov 13-15, 2020 / [Virtual].
  4. SAAWP Business Meeting – Nov 27, 2020 / [Virtual]