Emicizumab for the treatment of haemophilia A: a narrative review.


Journal

Blood transfusion = Trasfusione del sangue
ISSN: 2385-2070
Titre abrégé: Blood Transfus
Pays: Italy
ID NLM: 101237479

Informations de publication

Date de publication:
05 2019
Historique:
received: 03 02 2019
accepted: 15 04 2019
entrez: 28 6 2019
pubmed: 28 6 2019
medline: 26 12 2019
Statut: ppublish

Résumé

One of the most serious complications of the treatment of severe haemophilia A is the development of alloantibodies against exogenous factor VIII (FVIII). Inhibitors render factor replacement therapy ineffective, exposing patients to a remarkably high risk of morbidity and mortality. Besides the well-known bypassing agents (i.e. activated prothrombin complex concentrate and recombinant activated factor VII) used to treat or prevent bleeding in haemophilia patients with inhibitors, there is growing interest in newer haemostatic therapies that are not based on the replacement of the deficient FVIII. This review will focus on the most interesting among these innovative therapies, emicizumab, and will provide an update on its current stage of clinical development.

Identifiants

pubmed: 31246563
pii: 2019.0026-19
doi: 10.2450/2019.0026-19
pmc: PMC6596376
doi:

Substances chimiques

Antibodies, Bispecific 0
Antibodies, Monoclonal, Humanized 0
Blood Coagulation Factor Inhibitors 0
Isoantibodies 0
Recombinant Proteins 0
emicizumab 7NL2E3F6K3
F8 protein, human 839MOZ74GK
Factor VIII 9001-27-8
recombinant FVIIa AC71R787OV
Factor VIIa EC 3.4.21.21

Types de publication

Journal Article Systematic Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

223-228

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Auteurs

Massimo Franchini (M)

Department of Haematology and Transfusion Medicine, "Carlo Poma" Hospital, Mantua, Italy.
Italian National Blood Centre, National Institute of Health, Rome, Italy.

Giuseppe Marano (G)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Ilaria Pati (I)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Fabio Candura (F)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Samantha Profili (S)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Eva Veropalumbo (E)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Francesca Masiello (F)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Liviana Catalano (L)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Vanessa Piccinini (V)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Stefania Vaglio (S)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Simonetta Pupella (S)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

Giancarlo M Liumbruno (GM)

Italian National Blood Centre, National Institute of Health, Rome, Italy.

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