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Blood. 2018 Aug 30;132(9):883-891. doi: 10.1182/blood-2018-02-830844. Epub 2018 Jun 26.

Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial.

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Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Pediatrics, Flevo Hospital, Almere, The Netherlands.
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Immunohematology Diagnostics, Sanquin Diagnostic Services, Amsterdam, The Netherlands.
Department of Pediatric Hematology and Oncology, University Medical Center Groningen, Groningen, The Netherlands.
Department of Pediatric Hematology, University Medical Center Leiden, Leiden, The Netherlands.
Department of Pediatrics, Meander Medical Center, Amersfoort, The Netherlands.
Department of Pediatrics, Rijnstate Hospital, Arnhem, The Netherlands.
Department of Experimental Immunohematology, Sanquin Research, and.
Landsteiner Laboratory, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, The Netherlands; and.
Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands.


Management of children with newly diagnosed immune thrombocytopenia (ITP) consists of careful observation or immunomodulatory treatment. Observational studies suggest a lower risk for chronic ITP in children after intravenous immunoglobulin (IVIg) treatment. In this multicenter randomized trial, children aged 3 months to 16 years with newly diagnosed ITP, platelet counts 20 × 109/L or less, and mild to moderate bleeding were randomly assigned to receive either a single infusion of 0.8 g/kg IVIg or careful observation. Primary outcome was development of chronic ITP, which at the time of study initiation was defined as a platelet count lower than 150 × 109/L after 6 months. Two hundred six children were allocated to receive IVIg (n = 102) or careful observation (n = 104). Chronic ITP occurred in 18.6% of the patients in the IVIg group and 28.9% in the observation group (relative risk [RR], 0.64; 95% confidence interval [CI], 0.38-1.08). Platelet counts lower than 100 × 109/L at 12 months (current definition of chronic ITP) were observed in 10% of children in the IVIg group and 12% in the observation group (RR, 0.83; 95% CI, 0.38-1.84). Complete response rates in the first 3 months were significantly higher in the IVIg group. Immunoglobulin G Fc receptor IIb genetic variations were associated with early complete response in both groups. Grade 4 to 5 bleeding occurred in 9% of the patients in the observation group vs 1% in the IVIg group. This trial was registered at as NTR 1563.

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