Pediatric Immune Thrombocytopenic Purpura (ITP): a 12 year Retrospective Study at a Single Center. 公开

Bennett, Carolyn Marie (2009)

Permanent URL: https://etd.library.emory.edu/concern/etds/0g354f55x?locale=zh
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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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