Graft-versus-host disease and relapse/rejection-free survival after allogeneic transplantation for idiopathic severe aplastic anemia: a comprehensive analysis from the SAAWP of the EBMT

Research output: Contribution to journalResearch articleContributedpeer-review


  • Raynier Devillier - , Paoli Calmettes Institute (Author)
  • Dirk-Jan Eikema - , EBMT Statistical Unit (Author)
  • Carlo Dufour - , IRCCS Gaslini Children's Research Hospital (Author)
  • Mahmoud Aljurf - , King Faisal Specialist Hospital and Research Centre (Author)
  • Depei Wu - , Soochow University (Author)
  • Alexei Maschan - , Federal Research Center for Pediatric Hematology (Author)
  • Alexander Kulagin - , Pavlov First State Medical University of St. Petersburg (Author)
  • Constantijn J M Halkes - , Leiden University Medical Centre (LUMC) (Author)
  • Matthew Collin - , Adult HSCT Unit (Author)
  • John Snowden - , Sheffield Teaching Hospitals NHS Foundation Trust (Author)
  • Cécile Renard - , Institut d`Hematologie et d'Oncologie Pediatrique (Author)
  • Arnold Ganser - , Hannover Medical School (MHH) (Author)
  • Karl-Walter Sykora - , Hannover Medical School (MHH) (Author)
  • Brenda E Gibson - , University Hospitals Bristol and Weston NHS Foundation Trust (Author)
  • Johan Maertens - , University Hospitals Leuven (Author)
  • Maija Itäla-Remes - , Turku University Hospital (Author)
  • Paola Corti - , U.O. Ematologia E Centro Trapianti Midollo Osseo (Author)
  • Jan Cornelissen - , Children's Cancer Institute Australia (Author)
  • Martin Bornhäuser - , University Cancer Centre, Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Mercedes Colorado Araujo - , Hospital Universitario Marques de Valdecilla (Author)
  • Hakan Ozdogu - , Baskent University Ankara Hospital (Author)
  • Antonio Risitano - , Universita' di Napoli Federico II (Author)
  • Gerard Socie - , Hopital Louis-Mourier (Author)
  • Regis Peffault De Latour - , Hopital Louis-Mourier (Author)


Survival after allogeneic hematopoietic stem cell transplantation (allo-HSCT) for severe idiopathic aplastic anemia (SAA) has improved in recent years, approaching 75% at 5 years. However, an SAA-adapted composite endpoint, graft-versus-host disease (GvHD) and relapse/rejection-free survival (GRFS), may more accurately assess patient outcomes beyond survival. We analyzed GRFS to identify risk factors and specific causes of GRFS failure. Our retrospective analysis from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation included 479 patients with idiopathic SAA who underwent allo-HSCT in two conventional situations: i) upfront allo-HSCT from a matched related donor (MRD) (upfront cohort), and ii) allo-HSCT for relapsed or refractory SAA (rel/ref cohort). Relevant events for GRFS calculation included graft failure, grade 3-4 acute GvHD, extensive chronic GvHD, and death. In the upfront cohort (n=209), 5-year GRFS was 77%. Late allo-HSCT (i.e., >6 months after SAA diagnosis) was the main poor prognostic factor, specifically increasing the risk of death as the cause of GRFS failure (hazard ratio [HR]=4.08; 95% confidence interval [CI]: 1.41-11.83; P=0.010). In the rel/ref cohort (n=270), 5-year GRFS was 61%. Age was the main factor significantly increasing the risk of death (HR=1.04; 95% CI: 1.02-1.06; P<0.001), acute GvHD (HR=1.03; 95% CI: 1.00-1.07; P=0.041), and chronic GvHD (HR=1.04; 95% CI: 1.01-1.08; P=0.032) as the cause of GRFS failure. GRFS after upfront MRD allo-HSCT was very good, notably with early allo-HSCT, confirming that younger patients with an MRD should be transplanted immediately. GRFS was worse in cases of salvage allo-HSCT, most notably in older patients, questioning the utility of allo-HSCT earlier in the disease course.


Original languageEnglish
Pages (from-to)2305-2315
Number of pages11
Issue number9
Publication statusPublished - 1 Sept 2023

External IDs

PubMedCentral PMC10483355
Scopus 85170111699



  • Humans, Aged, Anemia, Aplastic/complications, Retrospective Studies, Disease-Free Survival, Graft vs Host Disease/diagnosis, Transplantation, Homologous/adverse effects, Hematopoietic Stem Cell Transplantation/adverse effects, Bronchiolitis Obliterans Syndrome

Library keywords