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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
Authors:Krista Vaht  Magnus Göransson  Kristina Carlson  Cecilia Isaksson  Stig Lenhoff  Anna Sandstedt  Bertil Uggla  Jacek Winiarski  Per Ljungman  Per-Ola Andersson  Mats Brune
Affiliation:1. Section of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden;2. Institute of Medicine, Sahlgrenska Academy at Gothenburg University, Gothenburg, Sweden;3. Department of Pediatrics, The Queen Silvia Children''s Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden;4. Department of Hematology, Uppsala University Hospital, Uppsala, Sweden;5. Department of Hematology, Cancer Centre, University Hospital, Umeå, Sweden;6. Department of Hematology, Skåne University Hospital, Lund University, Lund, Sweden;7. Department of Hematology, Linköping University Hospital, Linköping, Sweden;8. Section of Hematology Department of Medicine, Faculty of Medicine and Health, Örebro University, Örebro, Sweden;9. Astrid Lindgren Children''s Hospital, Karolinska University Hospital, Huddinge and CLINTEC, Karolinska Institutet, Stockholm, Sweden;10. Centre of Allogeneic Stem Cell Transplantation Unit (CAST), Karolinska University Hospital, Stockholm, Sweden;11. Department of Medicine, Huddinge, Karolinska Institutet, Stockholm, Sweden;12. Department of Medicine, Södra Älvsborg Hospital Borås, Borås, Sweden
Abstract:
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.
Keywords:Correspondence and reprint requests: Krista Vaht, MD, Section of Hematology and Coagulation, Department of Medicine, Sahlgrenska University Hospital, 413 45 Gothenburg, Sweden.  Aplastic anemia  Allogenic stem cell transplantation  Graft-versus-host disease-free  Relapse/rejection-free survival  Real-world data
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