1.
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
Vaht, K., Goransson, M., Carlson, K., Isaksson, C., Lenhoff, S., Sandstedt, A., Uggla, B., Winiarski, J., Ljungman, P., Andersson, P. O., et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019
Abstract
Allogeneic stem cell transplantation (SCT) as primary treatment for aplastic anemia (AA) is being increasingly utilized. Yet, age, stem cell source and donor type are important outcome factors. We have recently performed a nationwide cohort study of all AA patients in Sweden diagnosed 2000-2011 and now present outcome data on SCT patients. In total, 68 patients underwent SCT and 63% of them had failed immunosuppressive therapy (IST). We found that, with a median follow-up of 109 months (range 35-192 months), 5-year overall survival (OS) for all patients was 86.8% while 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% vs 7.8%; p=0.023), that translated into a lower 5-year OS: 70.6% vs 92.2% (p=0.022) and a trend of lower GRFS (52.9% vs 74.5%; p=0.069). In conclusion, we found in this real-world setting that both OS and GRFS were high, but SCT for AA patients aged ≥40 years is problematic and clinical trials addressing this issue are warranted.
2.
Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party
Devillier, R., Dalle, J. H., Kulasekararaj, A., D'Aveni, M., Clement, L., Chybicka, A., Vigouroux, S., Chevallier, P., Koh, M., Bertrand, Y., et al
Haematologica. 2016;101(7):884-90
Abstract
Unrelated allogeneic transplantation for severe aplastic anemia is a treatment option after immunosuppressive treatment failure in the absence of a matched sibling donor. Age, delay between disease diagnosis and transplantation, and HLA matching are the key factors in transplantation decisions, but their combined impact on patient outcomes remains unclear. Using the French Society of Bone Marrow Transplantation and Cell Therapies registry, we analyzed all consecutive patients (n=139) who underwent a first allogeneic transplantation for idiopathic severe aplastic anemia from an unrelated donor between 2000 and 2012. In an adjusted multivariate model, age over 30 years (Hazard Ratio=2.39; P=0.011), time from diagnosis to transplantation over 12 months (Hazard Ratio=2.18; P=0.027) and the use of a 9/10 mismatched unrelated donor (Hazard Ratio=2.14; P=0.036) were independent risk factors that significantly worsened overall survival. Accordingly, we built a predictive score using these three parameters, considering patients at low (zero or one risk factors, n=94) or high (two or three risk factors, n=45) risk. High-risk patients had significantly shorter survival (Hazard Ratio=3.04; P<0.001). The score was then confirmed on an independent cohort from the European Group for Blood and Marrow Transplantation database of 296 patients, with shorter survival in patients with at least 2 risk factors (Hazard Ratio=2.13; P=0.005) In conclusion, a simple score using age, transplantation timing and HLA matching would appear useful to help physicians in the daily care of patients with severe aplastic anemia. Copyright© Ferrata Storti Foundation.