Although many advances have been achieved in the understanding of ITP, critical issues regarding the pathophysiology and biology of the disease remain to be elucidated. The recent characterization of the human genome along with new sophisticated molecular biology techniques will allow basic researchers to study genes that may affect the presentation and clinical course of the disease. Different patterns of gene expression in this population can be studied, leading to the identification of subsets of patients with ITP at higher risk of bleeding. The multigene patterns of expression might also provide clues about regulatory mechanisms and broader cellular functions. In order to answer essential clinical questions, like the incidence of ICH in relation to drug treatment or observation alone, clinical trials should be appropriately designed. More studies are necessary to better define the optimal treatment approach for each child with ITP. Even though the incidence of intracranial hemorrhage cannot be used as the primary outcome measure because of its rarity, numerous other outcomes, such as rate of rise in platelet count, cost and side effects of therapy, health related quality of life of the patient and family, and severity of hemorrhage can be measured and compared between treatment groups. Future investigators should find it attractive to conduct trials in children with this common hematological disease so that decision making can be based more on scientific evidence than on anecdote and opinion.

Original languageEnglish
Pages (from-to)911-928
Number of pages18
JournalPediatric Clinics of North America
Issue number5
StatePublished - Oct 2002


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