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Intravenous immunoglobulin versus 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

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.

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 × 109/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 × 109/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 × 109/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.

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