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Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia A Swedish nationwide cohort study
Sahlgrens Univ Hosp, Sweden; Gothenburg Univ, Sweden.
Sahlgrens Univ Hosp, Sweden.
Uppsala Univ Hosp, Sweden.
Univ Hosp, 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]

ObjectivesAntithymocyte 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. MethodsWe 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 gradeespecially at time of treatment initiation: very severe (VSAA) 22.7%; severe (SAA) 54.5% (Pamp;lt;.001); and non-severe 88.5% (Pamp;lt;.001). A logistic regression-based predictive model indicated that VSAA patients with an absolute reticulocyte count amp;lt;25x10(9)/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. ConclusionsReal-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
WILEY , 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:liu:diva-148637DOI: 10.1111/ejh.13057ISI: 000434100400011PubMedID: 29532518OAI: oai:DiVA.org:liu-148637DiVA, id: diva2:1220036
Note

Funding Agencies|Goteborgs Lakaresallskap; ALF Vastra Gotaland

Available from: 2018-06-18 Created: 2018-06-18 Last updated: 2018-06-18

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Sandstedt, Anna
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Department of Social and Welfare StudiesDepartment of HaematologyFaculty of Medicine and Health Sciences
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CiteExportLink to record
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