Thrombotic thrombocytopenic purpura treatment in year 2000

Abstract

BACKGROUND AND OBJECTIVE: For several decades clinicians worldwide considered TTP a severe and frustrating therapeutic problem. Fortunately, however, the prognosis of TTP patients has greatly benefited from the use of plasma manipulation techniques, particularly plasma-exchange (PE), so that the overall rate of complete responses currently ranges between 70-85% and may even exceed these figures. Despite this dramatic improvement, a number of questions remain concerning the best treatment for TTP patients. Analyzing acquired data and discussing future perspectives, this review will address the following key issues: is PE really the treatment of choice for TTP and what is the role of PE with cryosupernatant? what is the role of all the drugs which are commonly combined with PE, antiplatelet drugs and steroids in particular? what, if any, is the role of cytotoxic agents, especially vincristine? is there a treatment for PE-resistant patients? does secondary TTP need different treatments? DESIGN AND METHODS: The authors have been involved in the study and treatment of TTP for years; furthermore, they extensively searched the PubMed database of the National Library of Congress through the Internet. INTERPRETATION AND CONCLUSIONS: PE remains the treatment of choice for TTP. A large randomized trial now in progress will assess whether exchange with cryosupernatant plasma can improve treatment efficacy. The administration of antiplatelet drugs in combination with PE was fiercely debated over the past years but seems indicated both in acute TTP and as a prophylactic treatment to prevent relapses. It appears that steroids cannot be avoided, especially in light of the latest findings on TTP pathogenesis, but only specific trials will assess the optimal cortisone type and dose. Presently, different treatments can be suggested only to patients failing to respond to PE, while no specific therapy can be indicated for secondary TTP, which usually has a very poor prognosis. Finally, we would like to stress that only international co-operative (multicenter) trials on large series of patients will be able to shed light on a still obscure, if fascinating, disease. Our hope and wish is that the new century will see TTP among the diseases defeated by man's clever mind and heart.