Treatment Landscape in Pediatric Immune Thrombocytopenia: Addressing Unmet Needs

Abstract

Pediatric immune thrombocytopenia (ITP) is associated with a multifaceted burden on children and their parents/caregivers due to bleeding, fatigue, activity restrictions, and psychological distress. Most children recover within 12 months, but up to 30% develop chronic ITP. While emergent therapies, such as steroids and intravenous immunoglobulin, are effective in many children and transiently raise platelet counts, 38-47% of children require subsequent therapies. The choice of subsequent therapy for individual children with ITP is often complex and the absence of head-to-head comparisons of available therapies and the use of nonstandardized outcomes in randomized clinical trials complicates treatment decisions. Furthermore, medication access varies globally and by age. Additional unmet needs in pediatric ITP include a lack of support and educational resources allowing children and parents/caregivers to effectively participate in treatment decisions, inadequate prediction of treatment response and disease chronicity, heterogeneous approaches to diagnostic evaluation of ITP, scarcity of novel treatments for children unresponsive to current therapies, and the need for a multispecialty approach to support the mental health of children and their families. This review summarizes the known impact of ITP on children and their families, current treatment strategies, and unmet needs in pediatric ITP.

Keywords: health‐related quality of life; immune thrombocytopenia; pediatric; rare disease; subsequent treatments; unmet needs.