-
- Han-Mou Tsai.
- Division of Hematology/Oncology, State University of New York Downstate Medical Center, Brooklyn. Electronic address: hmtsai@gmail.com.
- Am. J. Med. 2019 Sep 1; 132 (9): 1032-1037.
AbstractFor many years after its first description in 1924, thrombotic thrombocytopenic purpura was an intriguing puzzle for clinicians and researchers, not only for its unique pathology, perplexing changes in von Willebrand factor multimers, and high rate of rapid fatality but also for its dramatic response to plasma infusion or exchange. The discovery of ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 repeats member-13) and its deficiency in patients with thrombotic thrombocytopenic purpura, due to inhibitory autoantibodies or genetic mutations, provides a mechanistic scheme for understanding its pathogenesis. This new knowledge quickly led to the use of rituximab to promote its remission and prevent recurrence. Recombinant ADAMTS13 is also under development to replace plasma infusion as the therapy for hereditary thrombotic thrombocytopenic purpura. Recently, caplacizumab, a bivalent nanobody targeting the glycoprotein 1b binding epitope of von Willebrand factor A1 domain, was approved as an addition to the current regimen of plasma exchange and immunomodulation for adult patients of acquired thrombotic thrombocytopenic purpura. This review discusses how the new treatment may improve patient outcomes and its potential pitfalls.Copyright © 2019 Elsevier Inc. All rights reserved.
Notes
Knowledge, pearl, summary or comment to share?You can also include formatting, links, images and footnotes in your notes
- Simple formatting can be added to notes, such as
*italics*
,_underline_
or**bold**
. - Superscript can be denoted by
<sup>text</sup>
and subscript<sub>text</sub>
. - Numbered or bulleted lists can be created using either numbered lines
1. 2. 3.
, hyphens-
or asterisks*
. - Links can be included with:
[my link to pubmed](http://pubmed.com)
- Images can be included with:
![alt text](https://bestmedicaljournal.com/study_graph.jpg "Image Title Text")
- For footnotes use
[^1](This is a footnote.)
inline. - Or use an inline reference
[^1]
to refer to a longer footnote elseweher in the document[^1]: This is a long footnote.
.