Management of Children With Persistent ITP, A Novel Approach

Summary

The goal of this clinical trial is to learn if mini-pool intravenous immunoglobulin (IVIG) is a safe and effective treatment for children with persistent immune thrombocytopenia (ITP). ITP is a condition that causes low platelet levels and increases the risk of bleeding. The main questions this study aims to answer are: Can mini-pool IVIG raise platelet levels in children with persistent ITP? Can it reduce bleeding episodes and hospital visits? What side effects, if any, are seen with this treatment? There is no comparison group in this study. All participants will receive mini-pool IVIG, which is made from small pools of donated plasma using a cost-effective process. Participants will: Receive one dose of mini-pool IVIG through a vein over 6 to 8 hours Receive follow-up doses every 2 to 4 weeks for up to 5 doses, based on their platelet count Have regular blood tests and checkups during the study and for 6 months after treatment Report on bleeding episodes, physical activity, school attendance, and side effects

Timeline

Start

2025-01-01

Primary completion

2025-12-30

Completion

2026-03-01

Publications

• Background 1. Kohli, R. and S. Chaturvedi, Epidemiology and Clinical Manifestations of Immune Thrombocytopenia. Hamostaseologie, 2019. 39(3): p. 238-249. 2. Pietras, N.M., et al., Immune Thrombocytopenia, in StatPearls. 2024, StatPearls Publishing Copyright © 2024, StatPearls Publishing LLC.: Treasure Island (FL). 3. Thakur, Y., R.J. Meshram, and A. Taksande, Diagnosis and Management of Immune Thrombocytopenia in Paediatrics: A Comprehensive Review. Cureus, 2024. 16(9): p. e69635. 4. Wang, Z., et al., Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China. Br J Haematol, 2023. 202(2): p. 422-428. 5. Gotesman, M., et al., Pediatric Immune Thrombocytopenia. Adv Pediatr, 2024. 71(1): p. 229-240. 6. El-Ekiaby, M., et al., Minipool caprylic acid fractionation of plasma using disposable equipment: a practical method to enhance immunoglobulin supply in developing countries. PLoS Negl Trop Dis, 2015. 9(2): p. e0003501. 7. Elalfy, M., et al., A randomized multicenter study: safety and efficacy of mini-pool intravenous immunoglobulin versus standard immunoglobulin in children aged 1-18 years with immune thrombocytopenia. Transfusion, 2017. 57(12): p. 3019-3025. 8. Rodeghiero, F., et al., Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood, 2009. 113(11): p. 2386-93.

Drugs

Indications