How targeted therapy disrupts the treatment paradigm for acquired TTP: The risks, benefits, and unknowns

Marshall A. Mazepa; Camila Masias; Shruti Chaturvedi

doi:10.1182/blood.2019000954

How targeted therapy disrupts the treatment paradigm for acquired TTP: The risks, benefits, and unknowns

Marshall A. Mazepa
, Camila Masias
, Shruti Chaturvedi

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Article › peer-review

45 Scopus citations

Abstract

Insights into immune-mediated thrombotic thrombocytopenic purpura (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies: rituximab is effective in inducing responses in refractory/relapsed TTP and increasing relapse-free survival; caplacizumab targets the von Willebrand factor–platelet interaction to hasten platelet count recovery and reduce mortality and TTP-related ischemic events. Bortezomib and recombinant ADAMTS13 are under investigation. This review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.

Original language	English (US)
Pages (from-to)	415-420
Number of pages	6
Journal	Blood
Volume	134
Issue number	5
DOIs	https://doi.org/10.1182/blood.2019000954
State	Published - Aug 1 2019

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Publisher Copyright:
© 2019 by The American Society of Hematology.

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abstract = "Insights into immune-mediated thrombotic thrombocytopenic purpura (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies: rituximab is effective in inducing responses in refractory/relapsed TTP and increasing relapse-free survival; caplacizumab targets the von Willebrand factor–platelet interaction to hasten platelet count recovery and reduce mortality and TTP-related ischemic events. Bortezomib and recombinant ADAMTS13 are under investigation. This review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.",

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AU - Masias, Camila

AU - Chaturvedi, Shruti

PY - 2019/8/1

Y1 - 2019/8/1

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AB - Insights into immune-mediated thrombotic thrombocytopenic purpura (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies: rituximab is effective in inducing responses in refractory/relapsed TTP and increasing relapse-free survival; caplacizumab targets the von Willebrand factor–platelet interaction to hasten platelet count recovery and reduce mortality and TTP-related ischemic events. Bortezomib and recombinant ADAMTS13 are under investigation. This review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.

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JO - Blood

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ER -

How targeted therapy disrupts the treatment paradigm for acquired TTP: The risks, benefits, and unknowns

Abstract

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