New therapies for von Willebrand disease

Research output: Contribution to journalReview article

Abstract

The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C), due to the loss of binding and stabilization by VWF of this intrinsic coagulation factor in flowing blood. The traditional therapeutic weapons (the synthetic derivative of the antidiuretic hormone desmopressin and plasma-derived VWF/FVIII concentrates) are able to transiently correct both the defects. With the goal of tackling the primary deficiency in the disease, that is, VWF, but at the same time exploiting the normal capacity of patients to produce FVIII, the novel approach of replacing only VWF was implemented in the last 10 years. Following the manufacturing of a concentrate fractionated from human plasma and of one obtained by recombinant DNA technology, clinical studies have shown that VWF-only products correct not only the primary VWF deficiency but also the secondary FVIII:C deficiency. The demonstrated efficacy of these products in various clinical situations and, ultimately, in such a hemostasis-challenging context as surgery testifies to the effectiveness and safety of this approach. It remains to be seen whether VWF-only products are efficacious and safe in still-unexplored situations, such as use in children; the long-term use for prophylaxis; and in recurrent gastrointestinal (GI) bleeding due to angiodysplasia, a major therapeutic problem in VWD.

Original languageEnglish
Pages (from-to)3481-3487
Number of pages7
JournalBlood advances
Volume3
Issue number21
DOIs
Publication statusPublished - Nov 12 2019

Fingerprint

von Willebrand Diseases
von Willebrand Factor
Coagulants
Factor VIII
Hemostasis
Therapeutics
Angiodysplasia
Deficiency Diseases
Deamino Arginine Vasopressin
Intrinsic Factor
Weapons
Blood Coagulation Factors
Recombinant DNA
Vasopressins
Hemorrhage
Technology
Safety

ASJC Scopus subject areas

  • Hematology

Cite this

New therapies for von Willebrand disease. / Mannucci, Pier Mannuccio.

In: Blood advances, Vol. 3, No. 21, 12.11.2019, p. 3481-3487.

Research output: Contribution to journalReview article

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