ArticlesNewly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study
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.
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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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