Pediatric Immune Thrombocytopenic Purpura (ITP): a 12 yearRetrospective Study at a Single Center. Public

Abstract

ABSTRACT

ITP is a common acquired bleeding disorder of childhood that usually resolves spontaneously and has a low risk of serious bleeding. About 20-30% of children have chronic ITP which lasts beyond 6 months. Chronic ITP is a more serious disease due to the higher bleeding risk and need for ongoing therapy. Few studies have analyzed outcomes in large numbers of children with chronic ITP. No risk factors have been defined which predict chronic disease, bleeding risk, response to therapy or other outcomes. The objective of this study is to review the characteristics of a large cohort of pediatric patients with ITP at a single center. Four hundred and ninety-eight patients with ITP were evaluated. The incidence of serious bleeding was low (7.8%). There were no intracranial hemorrhages or ITP- related deaths. Most patients (85.5%) received initial therapy with an overall response rate of 80.3%. The most common therapies were corticosteroids (35.3%), IVIG (25.1%) and anti-D (22.7%). Only 14.5% had no reported therapy. Fifty one patients underwent splenectomy (10.2%) with 84% response. Chronic ITP was observed in 176 patients (35.3%). Acute ITP patients had lower platelet counts (mean 12.8 vs. 18.2 x 109/L) and were younger (5.8 vs. 9.6 years) than chronic ITP patients. Platelet count over 10 X 109/L and age over 10 years at presentation were associated with chronic disease. While the risk of bleeding in patients with chronic ITP was low, it was higher than that of acute ITP. Gender and response to therapy were not risk factors for chronic ITP. Twenty four patients (4.8%) had Evans syndrome; chronic ITP with autoimmune hemolytic anemia. There were no significant differences between Evans syndrome and chronic ITP patients. Evans syndrome patients were no more likely to bleed or be refractory to therapy. In general, children with chronic ITP have good outcomes. The risk of serious bleeding and death is low. Therefore, treatment should be individualized and focused on bleeding symptoms and prevention of treatment toxicity. Severe and refractory disease remains a challenging clinical problem. Studies are needed to define the biology of ITP and guide in the development of new targeted therapy.

Table of Contents

TABLE OF CONTENTS

Page INTRODUCTION.............................................................................1 BACKGROUND................................................................................2 METHODS.....................................................................................6 RESULTS.....................................................................................10 DISCUSSION................................................................................14 REFERENCES................................................................................16 TABLES.......................................................................................18

TABLES Page Table 1. Patient Characteristics............................................................18 Table 2. Characteristics by ITP Diagnosis................................................19 Table 3.Univariate Analysis for the Association Between Chronic ITP

and Five Risk Factors..........................................................................20 Table 4 Multivariate Analysis of Chronic ITP with Significant Risk Factors.......21 Table 5 Characteristics of Splenectomy Patients.......................................22

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School	Laney Graduate School
Department	Clinical Research Curriculum Program
Degree	MS
Submission	Dissertation
Language	English
Research Field	Health Sciences, Medicine and Surgery
Mot-clé	immune thrombocytopenic purpura Evans ITP
Committee Chair / Thesis Advisor	Abshire, Thomas, Emory University
Committee Members	Boring, John R, Emory University McGowan Jr., John E, Emory University Blumberg, Henry Michael, Emory University

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