Elsevier

The Lancet

Volume 358, Issue 9299, 22–29 December 2001, Pages 2122-2125
The Lancet

Articles
Newly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study

https://doi.org/10.1016/S0140-6736(01)07219-1Get rights and content

Summary

Background

Diagnosis and management of idiopathic thrombocytopenic purpura (ITP) have been based primarily on expert opinion and practice guidelines rather than on evidence. We have used a registry to prospectively survey the presenting features and the diagnostic evaluation and management practices used for children with ITP worldwide.

Methods

We used the Intercontinental Childhood ITP Registry which had been widely advertised. 209 physicians from 136 institutions in 38 countries participated by submitting data for each of their newly diagnosed patients. Data from 2031 children with ITP was registered between June, 1997, and May, 2000, and we analysed 6-month follow-up data from 1496 children.

Findings

There was a peak in occurrence of childhood ITP during spring and a nadir in the autumn. Mean initial platelet count was 15·4×109/L (SD 19·7). 1447 (73%) of 1976 children were admitted to hospital. Initial management consisted of no drug treatment in 612 (31%), intravenous immunoglobulin in 576 (29%), corticosteroids in 651 (33%), or both in 137 (7%) patients. Intracranial haemorrhage was reported in two patients.

Interpretation

The variable approaches to management of childhood ITP demonstrate the need for prospective clinical trials, which should be feasible within such a study group.

Introduction

Management of childhood idiopathic thrombocytopenic purpura (ITP) is based more on opinion and local practices than on evidence and has been the subject of fierce debate for decades. Although practice guidelines have been developed and published,1, 2 compliance with the guidelines is poor 3, 4 and the validity of one set of guidelines has been openly challenged.5 A network of shared consensus protocols and trials, such as is in place for malignant diseases, does not exist for this benign condition. The lack of an evidence-based approach is a disadvantage for patients, with diverse medical and socioeconomic consequences, reflected by the array of simple and sophisticated diagnostic procedures and treatments, from no therapy to multiple expensive platelet-enhancing agents with various adverse effects.6, 7 The natural history of the disease has been described in several retrospective studies.2 However, prospective data have only been obtained from randomised clinical trials with short follow-up and fairly small numbers of patients. We aimed to establish an international network of physicians involved in the diagnosis and management of children with ITP, to prospectively collect clinical data regarding the natural history and management of childhood ITP, and to compare results with previously published reports.

Section snippets

Patients and methods

The Intercontinental Childhood ITP Registry was established in June 1997 by the Intercontinental Childhood ITP Study Group to prospectively investigate the pathophysiology, clinical course, management, and outcome of children with ITP. The group advertised the Registry on its web-page (www.unibas.ch/itpbasel, accessed Dec 7, 2001), at national and international haematology meetings, in haematology journals, and by regularly mailed newsletters to prospective participants.

Each patient older than

Results

From June 1, 1997, to May 31, 2000, 2190 children were enrolled by 209 physicians from 136 institutions in 38 countries. We included data from 2031 children with newly diagnosed ITP. The remaining 159 (7%) children were ineligible for analysis because they were aged less than 4 months or 16 years old or older. Of eligible patients, 6-month follow-up data on 1510 (74%) children were received. Of those 14 (0·9%) were excluded because of incomplete data, thus we analysed follow-up data from 1496

Discussion

The Intercontinental Childhood ITP Registry serves as a network for facilitating collaborative research in ITP with the advantage of its prospective nature involving investigators in many countries, making it a unique investigative resource. The small numbers of patients recruited by 74% of institutions may be due to inconsistent registration, but is more likely caused by the relative rarity of the disease, as ITP occurs only in about one in 25 000 children yearly.8

Several features of childhood

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