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High Graft-versus-Host Disease-Free, Relapse/Rejection-Free Survival and Similar Outcome of Related and Unrelated Allogeneic Stem Cell Transplantation for Aplastic Anemia: A Nationwide Swedish Cohort Study

Journal article
Authors Krista Vaht
M. Göransson
K. Carlson
C. Isaksson
S. Lenhoff
A. Sandstedt
B. Uggla
J. Winiarski
P. Ljungman
Per-Ola Andersson
Mats Brune
Published in Biology of Blood and Marrow Transplantation
ISSN 1083-8791
Publication year 2019
Published at Institute of Medicine
Language en
Keywords Allogenic stem cell transplantation, Aplastic anemia, Graft-versus-host disease-free, Real-world data, Relapse/rejection-free survival
Subject categories Hematology


Allogeneic stem cell transplantation (SCT) as primary treatment for aplastic anemia (AA) is being increasingly used. Yet, age, stem cell source, and donor type are important outcome factors. We have recently performed a nationwide cohort study of all patients with AA in Sweden diagnosed from 2000 to 2011 and now present outcome data on SCT patients. In total, 68 patients underwent SCT, and 63% of them had failed immunosuppressive therapy. We found that, with a median follow-up of 109 months (range, 35 to 192 months), 5-year overall survival (OS) for all patients was 86.8%, whereas graft-versus-host disease-free, relapse/rejection-free survival (GRFS) at 5 years was 69.1%. There was no survival impact regarding the donor type or stem cell source. Patients aged ≥40 years had a higher transplant-related mortality (29.4% versus 7.8%; P = .023), which translated into a lower 5-year OS: 70.6% versus 92.2% (P = .022) and a trend of lower GRFS (52.9% versus 74.5%; P = .069). In conclusion, we found in this real-world setting that both OS and GRFS were high, but SCT for patients with AA aged ≥40 years is problematic, and clinical trials addressing this issue are warranted. © 2019 American Society for Blood and Marrow Transplantation

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