Back to Search Results

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

Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, Netherlands; k.m.j.heitink-polle@umcutrecht.nl. Julius Centre for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, Netherlands. Department of Immunohematology Diagnostics, Sanquin Diagnostic Services, Amsterdam, Netherlands. Department of Pediatric Hematology and Oncology, University Medical Center Groningen, Groningen, Netherlands. Department of Pediatric Hematology, University Medical Center Leiden, Leiden, Netherlands. Department of Pediatrics, Meander Medical Center, Amersfoort, Netherlands. Department of Pediatrics, Rijnstate Hospital, Arnhem, Netherlands. Department of Experimental Immunohematology, Sanquin Research, and Landsteiner Laboratory, Academic Medical Center, University of Amsterdam, Amsterdam, Netherlands. Department of Experimental Immunohematology, Sanquin Research, and Landsteiner Laboratory, Academic Medical Center, University of Amsterdam, Amsterdam, Netherlands. Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Netherlands. Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, Netherlands.

Blood. 2018;132((9):):883-891
Full text from:

Other resources

Abstract
Management of children with newly diagnosed immune thrombocytopenia (ITP) consists of careful observation or immunomodulatory treatment. Observational studies suggest a lower risk of chronic ITP in children after intravenous immunoglobulin (IVIg) treatment. In this multicenter randomized trial, children aged 3 months-16 years with newly diagnosed ITP, platelet counts ≤20 x 10(9)/L 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, at time of study initiation defined as a platelet count < 150 x 10(9)/L after 6 months. Two hundred and six children were allocated to receive IVIg (n=102) or careful observation (n=104). Chronic ITP occurred in 18.6% in the IVIg group and in 28.9% in the observation group (relative risk [RR] 0.64; 95% confidence interval [CI] 0.38-1.08). Platelet counts < 100 x 10(9)/L at 12 months (current definition of chronic ITP) were observed in 10% children in the IVIg group and in 12% in the observation group (RR 0.83; 95% CI 0.38-1.84). Complete response rates in the first three months were significantly higher in the IVIg group. IgG- Fc receptor IIb genetic variations were associated with early complete response in both groups. Grade 4-5 bleeding occurred in 9% in the observation group versus 1% in the IVIg group. IVIg treatment at diagnosis in children with ITP did not result in a lower rate of chronic ITP. In the IVIg group higher early complete response rates and less bleeding events were observed. This trial was registered at www.trialregister.nl as NTR 1563.
Study details
Language : English
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine