APMAT, A Multi-Centre observational study of patients with microangiopathic thrombocytopenia

Le résumé fourni par la source

Microangiopathic Thrombocytopenia (MAT) is a rare but often fatal collection of disorders. The disorder can be categorised by distinct disease states including thrombotic thrombocytopenia purpura (TTP) and atypical haemolytic uremic syndrome. Clinical presentations of MAT, however, often represent di_erent but related aetiologies from overlapping syndromes. TTP has an incidence rate of 2-15 cases per million people-years. When left untreated, it has a mortality rate of ~90%; with treatment the mortality rate remains relatively high (~20%). Moreover, TTP patients often su_er a high rate of recurrent relapses. Aetiologically, TTP is a result of insu_cient cleavage of high molecular weight von Willebrand Factor multimers due to abrogated ADAMTS13 activity. Current diagnosis lacks disease _delity and temporal resolution, making clinical management di_cult. There exists, therefore, a need for a faster and standardised clinical management for TTP. The APMAT project is a retrospective study instigated to _ll this information gap. The study aims to collect a total of 150 patient samples from various study centres in the Asia-Paci_c (AP) region, with WACTH Murdoch University acting as a repository, co-ordinating and R&D centre. Five key outcomes of APMAT will be: 1. Develop the APMAT research network protocol, 2. Formation of an AP medical advisory panel of APMAT experts for individual clinical advice, 3. Establish a clinical adjudication committee for independent classi_cation of MAT patients, 4. Standardise laboratory testing for ADAMTS13 and any other novel assays in the AP region, 5. Facilitate basic science and translational clinical research into MAT.

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