# Outcome and response to different management regimens in pediatric patients with immune thrombocytopenia (ITP)

## TL;DR

This study examines treatment outcomes for immune thrombocytopenia in children, finding that most respond to initial therapies like corticosteroids or IVIG, with second-line treatments effective for non-responders.

## Contribution

The study provides empirical evidence on treatment efficacy and response rates for pediatric ITP using a real-world cohort.

## Key findings

- 68.9% of patients responded to first-line therapies like corticosteroids or IVIG within three months.
- 32.2% of patients required second-line therapy with TPO-RAs due to lack of response or relapse.
- Corticosteroids remain the primary first-line treatment for newly diagnosed pediatric ITP.

## Abstract

Immune thrombocytopenia (ITP) is the most common acquired bleeding disorder in children and a frequent source of clinical concern. This study aimed to evaluate the clinical outcomes and treatment responses to different therapeutic lines. This observational study included 90 children with newly diagnosed ITP who were registered and followed at Cairo University Children’s Hospital between June 2022 and December 2023. The study cohort consisted of 40 males (44.4%) and 50 females (55.6%), with a mean age of 5.3 years. The mean platelet count at presentation was 9.9 ± 11.9 × 10⁹/L, increasing to 384.6 ± 141.0 × 10⁹/L at six months post-treatment. Six patients (5.8%) experienced spontaneous recovery without treatment, while 84 patients (94.2%) received therapeutic interventions including corticosteroids or intravenous immunoglobulin (IVIG). By three months, 61 patients (68.9%) had responded to treatment, while 29 patients (32.2%) required second-line therapy with thrombopoietin receptor agonists (TPO-RAs). Corticosteroids remain the cornerstone of first-line therapy in newly diagnosed pediatric ITP. Patients who do not respond to initial treatment or experience relapse demonstrate favorable outcomes with TPO-RA therapy.