Registries in Immune Thrombocytopenia: The History of the Intercontinental Cooperative ITP Study Group
Primary immune thrombocytopenia (ITP) is an autoimmune-mediated bleeding disorder with unknown etiology and without a homogenous pathomechanism occurring in children and adults. Premature platelet destruction, inadequate platelet production, and platelet dysfunction of various degrees contribute to bleeding. The methods with which ITP was investigated evolved from descriptions of patients and series at a time when platelets were not yet identified to clinical randomized trials and sophisticated basic research. ITP is an orphan disease and therefore the clinical and pathophysiological investigations are difficult and expensive. Registries are ideal tools to overcome some of these problems and are appropriate preparers of clinical trials, as they allow the establishment of powerful international networks and represent structured databases with the potential to collect demographic, clinical, and laboratory data from a big number of patients. Registries are suited to follow-up patients and investigate the natural history of diseases. However there are also limitations, which are discussed in this review article. The Intercontinental Cooperative ITP Study Group (ICIS) started its registries in 1997 and studied the natural history of ITP in children and adults, including anamnestic, clinical, and laboratory data, but also efficacy and side effects of drugs and splenectomy. Although many successes have occurred during the last centuries, many problems await solutions, such as the predictability of severe bleeding and of the form of ITP.
KeywordsImmune thrombocytopenia Platelets Registry
The author thanks the numerous ICIS investigators and their patients for providing their data and for their ongoing support. Many thanks go to Paul Imbach for his invitation to write this article and for his help. I thank Caroline Martin Asal, Verena Stahel, and Monika Imbach of the ICIS office in Basel, Switzerland, for their patience and help and all members of the ICIS Board for their continuous support of our group.
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