The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: Key findings at Enrolment until 2017

Congenital thrombotic thrombocytopenic purpura is an autosomal recessive inherited disease with a clinically heterogeneous course and an incompletely understood genotype–phenotype correlation. In 2006, the Hereditary TTP Registry started recruitment with the purpose of improving the understanding of this ultra-rare disease. The objective of this study is to present characteristics of the cohort until the end of 2017 and to explore the...