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Evaluation and Treatment of Thrombotic Thrombocytopenic Purpura

  • Satheesh Chonat
Chapter

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder, characterized by microangiopathic hemolytic anemia, thrombocytopenia, and varying degrees of organ ischemia from microvascular occlusion. This is an aggressive form of thrombotic microangiopathy (TMA), resulting from severe deficiency of ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin motifs 13) activity. The deficiency is either congenital (cTTP) in 5% of cases from biallelic (homozygous or compound heterozygous) mutations involving ADAMTS13 or acquired (immune-mediated TTP) in 95% of subjects due to autoantibodies against ADAMTS13. Though it is a rare disease, mortality during acute presentation remains very high. Therefore, TTP requires prompt diagnosis and immediate institution of lifesaving treatment modalities. This complex multisystem disorder is reviewed here with a focus on the diagnosis and clinical management of immune-mediated TTP, including emerging disease-modifying therapies.

Keywords

Thrombotic thrombocytopenic purpura TTP TMA Microangiopathic hemolytic anemia ADAMTS13 Evaluation Treatment 

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© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Pediatric Hematology, Department of PediatricsEmory University School of Medicine and Aflac Cancer and Blood Disorders Center, Children’s Healthcare of AtlantaAtlantaUSA

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