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
Year: 2023
Type: article
Study design: Observational Study
Abstract: 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.
Source: Haematologica
Authors: Raynier Devillier, Dirk-Jan Eikema, Carlo Dufour, Mahmoud Aljurf, Depei Wu, Alexei Maschan, Alexander Kulagin, Constantijn J.M. Halkes, Matthew Collin, John Snowden, Cécile Renard, Arnold Ganser, Karl-Walter Sykora, Brenda E Gibson, Johan Maertens, Maija Itäla-Remes, Paola Corti, Jan Cornelissen, Martin Bornhäuser, Mercedes Colorado Araujo, Hakan Ozdogu, Antonio Risitano, Gerard Socie, Regis Peffault De Latour
Institutions: Institut Paoli-Calmettes, European Society for Blood and Marrow Transplantation, Istituto Giannina Gaslini, King Faisal Specialist Hospital & Research Centre, First Affiliated Hospital of Soochow University, First Pavlov State Medical University of St. Petersburg, Leiden University Medical Center, Sheffield Teaching Hospitals NHS Foundation Trust, Institut d’Hématologie et d’Oncologie Pédiatrique, Medizinische Hochschule Hannover, Royal Hospital for Children, Universitair Ziekenhuis Leuven, Turku University Hospital, Erasmus MC Cancer Institute, Marqués de Valdecilla University Hospital, Başkent University, University of Naples Federico II, Hôpital Saint-Louis
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,157
OpenAlex ID: W4360600298