Outcome and response to different management regimens in pediatric patients with immune thrombocytopenia (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.