期刊
EXPERT REVIEW OF HEMATOLOGY
卷 13, 期 5, 页码 461-470出版社
TAYLOR & FRANCIS LTD
DOI: 10.1080/17474086.2020.1750361
关键词
Thrombotic Thrombocytopenic Purpura; treatment; therapeutic plasma exchange; rituximab; caplacizumab; N-acetylcysteine; novel agents
类别
Introduction: Thrombotic thrombocytopenic purpura (TTP) is an infrequent but fatal disease. Plasma exchange and corticosteroids continue to be the mainstay of treatment; however, repurposed drugs and novel agents are emerging as efficient treatment options. Areas covered: In this review, new therapeutic developments in immune-mediated TTP including rituximab, bortezomib, N-acetylcysteine, caplacizumab, and recombinant ADAMTS13, among others, are summarized. Expert opinion: Evidence on the use of rituximab in first and second-line settings is accumulating showing promising potential for avoiding relapses in patients in remission but with low circulating levels of ADAMTS13 in a preemptive fashion. Other repurposed drugs such as bortezomib and N-acetylcysteine are increasingly used off-label. Recombinant ADAMTS13 is slowly emerging. Caplacizumab, a humanized anti-von Willebrand factor-directed nanobody that blocks platelet adhesion and avoids microthrombi formation, was approved by regulatory agencies based on the positive results of a phase-III clinical trial, adding a new drug to the therapeutic arsenal in TTP.
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