Clinical Spectrum and Early Treatment Response in Pediatric Immune Thrombocytopenia: A Prospective Observational Study

Open Access

Abstract

Background: Immune thrombocytopenia (ITP) is the most common acquired autoimmune bleeding disorder in children and is characterized by isolated thrombocytopenia resulting from immune-mediated platelet destruction and impaired platelet production. Although most pediatric patients experience spontaneous recovery or respond favorably to first-line therapy, considerable heterogeneity exists in clinical presentation and early treatment outcomes. This study aimed to evaluate the clinical spectrum of pediatric ITP and identify factors associated with early treatment response. Methods: This prospective observational study was conducted in the Departments of Pediatrics at Mymensingh Medical College Hospital (MMCH) and Community Based Medical College Hospital Bangladesh (CBMCHB), from June 2025 to July 2026. A total of 125 consecutive children (<18 years) with newly diagnosed primary ITP were enrolled. Demographic characteristics, clinical manifestations, laboratory parameters, treatment modalities, and early treatment outcomes were recorded using a structured data collection form. Associations between treatment modality and treatment response were assessed using the Fisher-Freeman-Halton exact test, while independent predictors of complete early response were identified using multivariable binary logistic regression. Statistical analyses were performed using IBM SPSS Statistics version 26.0, with a two-sided p-value <0.05 considered statistically significant. Results: The mean age of the participants was 5.8 ± 3.9 years, and 58.4% were male. Petechiae (87.2%) and purpura (64.8%) were the most frequent presenting manifestations, whereas intracranial hemorrhage occurred in only one patient (0.8%). The mean baseline platelet count was 13.4 ± 8.2 × 10⁹/L. Corticosteroids were the most commonly administered first-line therapy (57.6%), followed by IVIG (19.2%). Early treatment response differed significantly across treatment modalities (p=0.031), with complete response rates of 70.8% for corticosteroids, 87.5% for IVIG, and 100.0% for combined corticosteroid plus IVIG therapy. Multivariable logistic regression demonstrated that age <5 years (adjusted odds ratio [AOR] 1.82, 95% confidence interval [CI] 1.04-3.20; p=0.036), baseline platelet count ≥10 × 10⁹/L (AOR 2.74, 95% CI 1.39-5.41; p=0.004), and initial IVIG therapy (AOR 3.18, 95% CI 1.28-7.89; p=0.012) were independent predictors of complete early treatment response. Conclusions: Pediatric ITP in this cohort predominantly presented with mild-to-moderate mucocutaneous bleeding and demonstrated favorable early treatment outcomes. Corticosteroids remained the most frequently used first-line therapy, whereas IVIG achieved superior early response rates. Younger age, higher baseline platelet count, and initial IVIG therapy independently predicted complete early treatment response. These findings provide clinically relevant evidence for individualized risk stratification and therapeutic decision-making in children with newly diagnosed ITP and may contribute to optimizing pediatric hematology practice in resource-limited settings.