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Biological stratification of clinical disease courses in childhood immune thrombocytopenia

Sanquin Research, Department of Experimental Immunohematology, Amsterdam, The Netherlands. Landsteiner Laboratory, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands. Department of Pediatric Hematology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands. Department of Medical Statistics, Leiden University Medical Center, Leiden, The Netherlands. Department of Immunohematology Diagnostics, Sanquin Diagnostic Services, Amsterdam, The Netherlands. Sanquin Research, Center for Clinical Transfusion Research, Leiden, The Netherlands. Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands. Princess Maxima Pediatric Oncology Center, Utrecht, Netherlands. Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.

Journal of thrombosis and haemostasis : JTH. 2021
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Abstract
BACKGROUND In childhood immune thrombocytopenia (ITP), an autoimmune bleeding disorder, there is a need for better prediction of individual disease courses and treatment outcomes. OBJECTIVE To predict the response to intravenous Immunoglobulins (IVIg) and ITP disease course using genetic and immune markers. METHODS Children aged below seven years with newly diagnosed ITP (N = 147) from the TIKI study were included, which randomized children to an IVIg or observation group. A total of 46 variables were available: clinical characteristics, targeted genotyping, lymphocyte immune phenotyping, and platelet autoantibodies. RESULTS In the treatment arm, 48/80 children (60%) showed a complete response (platelets ≥100 x 10(9) /L) that lasted for at least one month (complete sustained response; CSR) and 32 exhibited no or a temporary response (absence of a sustained response; ASR). For a biological risk score, five variables were selected by regularized logistic regression that predicted ASR vs CSR: 1) hemoglobin; 2) platelet count; 3) genetic polymorphisms of FcγRIIc; 4) the presence of IgG anti-platelet antibodies; and 5) preceding vaccination. The ASR sensitivity was 0.91 (95% CI, 0.80 - 1.00) and specificity was 0.67 (95% CI, 0.53 - 0.80). In the 67 patients of the observation arm, this biological score was also associated with recovery during one-year follow-up. The addition of the biological score to a predefined clinical score further improved the discrimination of favorable ITP disease courses. CONCLUSIONS The prediction of disease courses and IVIg treatment responses in ITP is improved by using both clinical and biological stratification.
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Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine