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

Katja M. J. Heitink-Pollé, Cuno S. P. M. Uiterwaal, Leendert Porcelijn, Rienk Y. J. Tamminga, Frans J. Smiers, Nicole L. van Woerden, Judit Wesseling, Gestur Vidarsson, Annemieke G. Laarhoven, Masja de Haas and Marrie C. A. Bruin for the TIKI Investigators

Key Points

  • In children with newly diagnosed ITP, IVIg treatment at diagnosis does not result in a lower rate of chronic ITP.

  • Upfront treatment with IVIg led to faster recovery and less severe bleeding events.

Publisher's Note: There is a Blood Commentary on this article in this issue.

Abstract

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 www.trialregister.nl as NTR 1563.

  • Submitted February 14, 2018.
  • Accepted June 13, 2018.
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