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Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia - a Swedish nationwide cohort study.

Journal article
Authors Krista Vaht
Magnus Göransson
Kristina Carlson
Cecilia Isaksson
Stig Lenhoff
Anna Sandstedt
Bertil Uggla
Jacek Winiarski
Per Ljungman
Mats Brune
Per-Ola Andersson
Published in European journal of haematology
Volume 100
Issue 6
Pages 613-620
ISSN 1600-0609
Publication year 2018
Published at Institute of Medicine
Pages 613-620
Language en
Keywords age, antithymocyte globulin, aplastic anaemia, real‐world data, response rate
Subject categories Hematology


Antithymocyte globulin (ATG)-based immunosuppression remains a cornerstone in aplastic anaemia (AA) treatment. However, most ATG studies are not population-based and knowledge about real-world results concerning response and outcome could offer important information for treating physicians.We have recently performed a nationwide retrospective cohort study on all AA patients diagnosed in Sweden in 2000-2011 and now present treatment and outcome data on patients receiving first-line ATG.In total, 158 patients showed a 47.0% response rate which was similar in all age groups (range 41.5%-51.7%) with no difference regarding ATG formulation. The response was significantly associated with severity grade - especially at time of treatment initiation: very severe (VSAA) 22.7%; severe (SAA) 54.5% (P < 0.001); and non-severe 88.5% (P < 0.001). A logistic regression-based predictive model indicated that VSAA patients with an absolute reticulocyte count <25 x 109/l had only a 19% probability of response. In a multivariable analysis, age and VSAA at the time of treatment were the independent factors for inferior survival.Real-world VSAA patients respond poorly to ATG which indicates the need for a different treatment approach. Our findings suggest that age alone should not be a discriminating factor for administering ATG treatment. This article is protected by copyright. All rights reserved.

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