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

Research output: Contribution to journalArticle


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.


  • Krista Vaht
  • Magnus Göransson
  • Kristina Carlson
  • Cecilia Isaksson
  • Stig Lenhoff
  • Anna Sandstedt
  • Bertil Uggla
  • Jacek Winiarski
  • Per Ljungman
  • Mats Brune
  • Per Ola Andersson
External organisations
  • Queen Silvia Children’s Hospital
  • Uppsala University Hospital
  • Norrland University Hospital
  • Skåne University Hospital
  • Linköping University Hospital
  • Örebro University
  • Karolinska University Hospital
  • Karolinska Institutet
  • University of Gothenburg
  • Sahlgrenska University Hospital
  • South Elfsborg Hospital
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology


  • Age, Antithymocyte globulin, Aplastic anaemia, Real-world data, Response rate
Original languageEnglish
Pages (from-to)613-620
JournalEuropean Journal of Haematology
Issue number6
Early online date2018 Apr 10
Publication statusPublished - 2018
Publication categoryResearch