Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura

Scully, Marie; Cataland, Spero R.; Peyvandi, Flora; Coppo, Paul; Knöbl, Paul; Hovinga, Johanna A. Kremer; Metjian, Ara; Rubia, Javier de la; Pavenski, Katerina; Callewaert, Filip; Biswas, Debjit; Winter, Hilde De; Zeldin, Robert K.; Investigators, Hercules

doi:10.1056/nejmoa1806311

Cited by 741 publications

(873 citation statements)

References 24 publications

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“…This medication is then used post-TPE treatment until immunological evidence of disease is controlled to prevent relapse. 305,307,308 This medication was FDA approved for use in TTP in 2019.…”

Section: Coagulopathy and Other Benign Hematologic Diseasesmentioning

confidence: 99%

Passive Monoclonal and Polyclonal Antibody Therapies

Pelletier¹,

Mukhtar²

2020

Immunologic Concepts in Transfusion Medicine

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Section: Coagulopathy and Other Benign Hematologic Diseasesmentioning

confidence: 99%

Passive Monoclonal and Polyclonal Antibody Therapies

Pelletier¹,

Mukhtar²

2020

Immunologic Concepts in Transfusion Medicine

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“…Unlike conventional antibodies which have flat or concave antigen-binding sites, a wide variety of loop lengths and structures presented in VHH and VNAR have made them capable to access more cryptic epitopes and catalytic clefts of enzymes [12][13][14]. While the first VHH-based drug (anti-yWF caplacizumab) has already been approved [15,16], the current progress of VNARs may substantially render new perceptions and attitudes towards these molecules in the medical and scientific communities. Hence, this article aims to review the unique characteristics of shark VNAR, and to discuss the potential uses of shark VNAR in diagnostic as well as in therapeutic platform.…”

Section: Introductionmentioning

confidence: 99%

Diagnostic and therapeutic potential of shark variable new antigen receptor (VNAR) single domain antibody

Cheong

Leow

Majeed

2020

International Journal of Biological Macromolecules

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Conventional monoclonal antibodies (mAbs) have been widely used in research and diagnostic applications due to their high affinity and specificity. However, multiple limitations, such as large size, complex structure and sensitivity to extreme ambient temperature potentially weaken the performance of mAbs in certain applications. To address this problem, the exploration of new antigen binders is extensively required in relation to improve the quality of current diagnostic platforms. In recent years, a new immunoglobulin-based protein, namely variable domain of new antigen receptor (VNAR) was discovered in sharks. Unlike conventional mAbs, several advantages of VNARs, include small size, better thermostability and peculiar paratope structure have attracted interest of researchers to further explore on it. This article aims to first present an overview of the shark VNARs and outline the characteristics as an outstanding new reagent for diagnostic and therapeutic applications.

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“…Caplacizumab is a humanized bivalent nanobody that inhibits interaction between vWF and platelets by blocking the A1 domain of vWF, thus reducing ischemia due to platelet aggregation by the ultra‐large vWF . Studies with caplacizumab have demonstrated significant reduction in the number of days of PEX required for platelet recovery . This is the first published report for the use of caplacizumab in a pediatric patient.…”

mentioning

confidence: 99%

“…

LETTER TO THE EDITOR ultra-large vWF. 3,4 Studies with caplacizumab have demonstrated significant reduction in the number of days of PEX required for platelet recovery. 3,4 This is the first published report for the use of caplacizumab in a pediatric patient.

…”

mentioning

confidence: 99%