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Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia: A Swedish nationwide cohort study
Section of Haematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden; Institute of Medicine, Sahlgrenska Academy at Gothenburg University, Gothenburg, Sweden.
Department of Pediatrics, The Queen Silvia Children's Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden.
Department of Haematology, Uppsala University Hospital, Uppsala, Sweden.
Department of Haematology, Cancer Centre, University Hospital, Umeå, Sweden.
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2018 (English)In: European Journal of Haematology, ISSN 0902-4441, E-ISSN 1600-0609, Vol. 100, no 6, p. 613-620Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
Munksgaard Forlag, 2018. Vol. 100, no 6, p. 613-620
Keywords [en]
Age, antithymocyte globulin, aplastic anaemia, real-world data, response rate
National Category
Hematology
Identifiers
URN: urn:nbn:se:oru:diva-67146DOI: 10.1111/ejh.13057ISI: 000434100400011PubMedID: 29532518Scopus ID: 2-s2.0-85045037330OAI: oai:DiVA.org:oru-67146DiVA, id: diva2:1212862
Note

Funding Agencies:

Göteborgs Läkaresällskap  

ALF Västra Götaland 

Available from: 2018-06-04 Created: 2018-06-04 Last updated: 2018-06-20Bibliographically approved

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