A1 Refereed original research article in a scientific journal
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
Authors: Devillier Raynier, Eikema Dirk-Jan, Dufour Carlo, Aljurf Mahmoud, Wu Depei, Maschan Alexei, Kulagin Alexander, Halkes Constantijn J. M., Collin Matthew, Snowden John, Renard Cecile, Ganser Arnold, Sykora Karl-Walter, Gibson Brenda E., Maertens Johan, Itala-Remes Maija, Corti Paola, Cornelissen Jan, Bornhaeuser Martin, Colorado Araujo Mercedes, Ozdogu Hakan, Risitano Antonio, Socie Gerard, de latour Regis Peffault
Publication year: 2023
Journal: Haematologica
Journal name in source: HAEMATOLOGICA
Volume: 108
Issue: 9
ISSN: 0390-6078
eISSN: 1592-8721
DOI: https://doi.org/10.3324/haematol.2022.281876
Web address : https://haematologica.org/article/view/haematol.2022.281876
Self-archived copy’s web address: https://research.utu.fi/converis/portal/detail/Publication/182345762
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.
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