Biological stratification of clinical disease courses in childhood immune thrombocytopenia
© 2021 The Authors. Journal of Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis..
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 younger than 7 years with newly diagnosed ITP (N = 147) from the Treatment With or Without IVIG for Kids with ITP 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 × 109 /L) that lasted for at least 1 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 Fc-receptor (FcγR) IIc; (4) the presence of immunoglobulin G (IgG) anti-platelet antibodies; and (5) preceding vaccination. The ASR sensitivity was 0.91 (95% confidence interval, 0.80-1.00) and specificity was 0.67 (95% confidence interval, 0.53-0.80). In the 67 patients of the observation arm, this biological score was also associated with recovery during 1 year of 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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:19 |
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| Enthalten in: |
Journal of thrombosis and haemostasis : JTH - 19(2021), 4 vom: 01. Apr., Seite 1071-1081 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Schmidt, David E [VerfasserIn] |
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| Links: |
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| Themen: |
Immune thrombocytopenia |
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| Anmerkungen: |
Date Completed 14.05.2021 Date Revised 29.08.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1111/jth.15232 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM319506843 |
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| 100 | 1 | |a Schmidt, David E |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Biological stratification of clinical disease courses in childhood immune thrombocytopenia |
| 264 | 1 | |c 2021 | |
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| 500 | |a Date Revised 29.08.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2021 The Authors. Journal of Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis. | ||
| 520 | |a BACKGROUND: In childhood immune thrombocytopenia (ITP), an autoimmune bleeding disorder, there is a need for better prediction of individual disease courses and treatment outcomes | ||
| 520 | |a OBJECTIVE: To predict the response to intravenous immunoglobulins (IVIg) and ITP disease course using genetic and immune markers | ||
| 520 | |a METHODS: Children aged younger than 7 years with newly diagnosed ITP (N = 147) from the Treatment With or Without IVIG for Kids with ITP 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 | ||
| 520 | |a RESULTS: In the treatment arm, 48/80 children (60%) showed a complete response (platelets ≥100 × 109 /L) that lasted for at least 1 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 Fc-receptor (FcγR) IIc; (4) the presence of immunoglobulin G (IgG) anti-platelet antibodies; and (5) preceding vaccination. The ASR sensitivity was 0.91 (95% confidence interval, 0.80-1.00) and specificity was 0.67 (95% confidence interval, 0.53-0.80). In the 67 patients of the observation arm, this biological score was also associated with recovery during 1 year of follow-up. The addition of the biological score to a predefined clinical score further improved the discrimination of favorable ITP disease courses | ||
| 520 | |a CONCLUSIONS: The prediction of disease courses and IVIg treatment responses in ITP is improved by using both clinical and biological stratification | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a immune thrombocytopenia | |
| 650 | 4 | |a intravenous immunoglobulins | |
| 650 | 4 | |a molecular epidemiology | |
| 650 | 4 | |a pediatrics | |
| 650 | 4 | |a prognosis | |
| 650 | 7 | |a Immunoglobulin G |2 NLM | |
| 650 | 7 | |a Immunoglobulins, Intravenous |2 NLM | |
| 700 | 1 | |a Heitink-Pollé, Katja M J |e verfasserin |4 aut | |
| 700 | 1 | |a Mertens, Bart |e verfasserin |4 aut | |
| 700 | 1 | |a Porcelijn, Leendert |e verfasserin |4 aut | |
| 700 | 1 | |a Kapur, Rick |e verfasserin |4 aut | |
| 700 | 1 | |a van der Schoot, C Ellen |e verfasserin |4 aut | |
| 700 | 1 | |a Vidarsson, Gestur |e verfasserin |4 aut | |
| 700 | 1 | |a van der Bom, Johanna G |e verfasserin |4 aut | |
| 700 | 1 | |a Bruin, Marrie C A |e verfasserin |4 aut | |
| 700 | 1 | |a de Haas, Masja |e verfasserin |4 aut | |
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