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Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000-2011

Journal article
Authors Krista Vaht
M. Goransson
K. Carlson
C. Isaksson
S. Lenhoff
A. Sandstedt
B. Uggla
J. Winiarski
P. Ljungman
Mats Brune
Per-Ola Andersson
Published in Haematologica
Volume 102
Issue 10
Pages 1683-1690
ISSN 0390-6078
Publication year 2017
Published at Institute of Medicine
Pages 1683-1690
Language en
Links doi.org/10.3324/haematol.2017.16986...
Keywords bone-marrow-transplantation, rabbit antithymocyte globulin, immunosuppressive therapy, working party, antilymphocyte globulin, prospective multicenter, donor transplants, european group, older, patients, cyclosporine, Hematology
Subject categories Hematology

Abstract

A plastic anemia is a rare life-threatening disease. However, since the introduction of immunosuppressive therapy and allogeneic stem cell transplantation, the outcome has improved considerably, and the 5-year survival is reported to be 70-80% in selected patient cohorts. Yet, contemporary population-based data on incidence and survival are lacking. We performed a national retrospective study to determine the incidence, treatment, and survival of patients with aplastic anemia diagnosed in Sweden from 2000-2011. Patients were included via the National Patient Registry, and diagnosed according to the Camitta criteria. In total, 257 confirmed cases were identified, with an overall incidence of 2.35 (95% CI: 2.06-2.64) cases per million inhabitants per year. Median age was 60 years (range: 2-92), and median follow up was 76 (0-193) months. Primary treatments included immunosuppressive therapy (63%), allogenic stem cell transplantation (10%), or single-agent cyclosporine/no specific therapy (27%). The 5-year survival was 90.7% in patients aged 0-18 years, 90.5% in patients aged 19-39 years, 70.7% in patients aged 40-59 years, and 38.1% in patients aged >= 60 years. Multivariate analysis showed that age (both 40-59 and >= 60 age groups), very severe aplastic anemia and single-agent cyclosporine/no specific therapy were independent risk factors for inferior survival. In conclusion, younger aplastic anemia patients experience a very good long-term survival, while that of patients >= 60 years in particular remains poor. Apparently, the challenge today is to improve the management of older aplastic anemia patients, and prospective studies to address this medical need are warranted.

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