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

Vaht, Krista; Göransson, Magnus; Carlson, Kristina; Isaksson, Cecilia; Lenhoff, Stig LU ; Sandstedt, Anna; Uggla, Bertil; Winiarski, Jacek; Ljungman, Per and Brune, Mats, et al. (2018) In European Journal of Haematology 100(6). p.613-620
Abstract

Objectives: 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. Methods: 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... (More)

Objectives: 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. Methods: 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 < .001); and non-severe 88.5% (P < .001). A logistic regression-based predictive model indicated that VSAA patients with an absolute reticulocyte count <25 × 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. Conclusions: 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.

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published
subject
keywords
Age, Antithymocyte globulin, Aplastic anaemia, Real-world data, Response rate
in
European Journal of Haematology
volume
100
issue
6
pages
613 - 620
publisher
Wiley-Blackwell
external identifiers
  • scopus:85045037330
ISSN
0902-4441
DOI
10.1111/ejh.13057
language
English
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yes
id
0197ce5e-dd8c-43a2-92cd-0b0c969fd8a6
date added to LUP
2018-04-17 10:04:36
date last changed
2019-03-19 03:53:12
@article{0197ce5e-dd8c-43a2-92cd-0b0c969fd8a6,
  abstract     = {<p>Objectives: 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. Methods: 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 &lt; .001); and non-severe 88.5% (P &lt; .001). A logistic regression-based predictive model indicated that VSAA patients with an absolute reticulocyte count &lt;25 × 10<sup>9</sup>/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. Conclusions: 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.</p>},
  author       = {Vaht, Krista and Göransson, Magnus and Carlson, Kristina and Isaksson, Cecilia and Lenhoff, Stig and Sandstedt, Anna and Uggla, Bertil and Winiarski, Jacek and Ljungman, Per and Brune, Mats and Andersson, Per Ola},
  issn         = {0902-4441},
  keyword      = {Age,Antithymocyte globulin,Aplastic anaemia,Real-world data,Response rate},
  language     = {eng},
  month        = {04},
  number       = {6},
  pages        = {613--620},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia - A Swedish nationwide cohort study},
  url          = {http://dx.doi.org/10.1111/ejh.13057},
  volume       = {100},
  year         = {2018},
}