Hemophilia A (Factor VIII Deficiency).

Factor VIII / genetics Hemarthrosis Hemophilia A / diagnosis Hemorrhage Humans Quality of Life
Gene therapy Hemarthrosis Inhibitors Nonfactor therapy Ultrasound prophylaxis

Journal

Hematology/oncology clinics of North America
ISSN: 1558-1977
Titre abrégé: Hematol Oncol Clin North Am
Pays: United States
ID NLM: 8709473

Informations de publication

Date de publication:
12 2021
Historique:
pubmed: 15 8 2021
medline: 3 3 2022
entrez: 14 8 2021
Statut: ppublish

Résumé

Remarkable changes are occurring in the diagnosis and management of individuals with hemophilia A. Genetic testing, including next-generation sequencing, enables family planning, carrier testing, and prenatal diagnosis. Musculoskeletal ultrasound examination facilitates the early detection of acute bleeds and joint disease in clinic, enabling more rapid bleed resolution and treatment planning. Novel therapies offer simpler weekly or monthly administration, some by subcutaneous injection, with better compliance and quality of life, as well as fewer bleeds. Gene therapy provides a 1-time phenotypic "cure" that is cost effective, but may be complicated by waning levels, vector immune responses, and hepatotoxicity.

Identifiants

DOI: 10.1016/j.hoc.2021.07.006 PMID: 34389199
pubmed: 34389199
pii: S0889-8588(21)00085-X
doi: 10.1016/j.hoc.2021.07.006
pii:
doi:

Substances chimiques

Factor VIII 9001-27-8

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1117-1129

Informations de copyright

Copyright © 2021 Elsevier Inc. All rights reserved.

Auteurs

Craig D Seaman (CD)

Department of Medicine, Division Hematology/Oncology, University of Pittsburgh Medical Center, 3636 Boulevard of the Allies, Pittsburgh, PA 15213-4306, USA.

Frederico Xavier (F)

Department of Pediatrics, Division Hematology/Oncology, University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, PA 15224, USA.

Margaret V Ragni (MV)

Medicine and Clinical Translational Research, Department of Medicine, Division Hematology/Oncology, University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, 3636 Boulevard of the Allies, Pittsburgh, PA 15213-4306, USA. Electronic address: ragni@pitt.edu.

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Classifications MeSH

questionsmedicales.fr › Facteurs biologiques › Facteurs de la coagulation sanguine › Facteur VIII › Hemophilia A (Factor VIII Deficiency).
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Hémorragie › Hémarthrose › Hemophilia A (Factor VIII Deficiency).
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Troubles héréditaires de la coagulation sanguine › Hémophilie A › Hemophilia A (Factor VIII Deficiency).
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Hémorragie › Hemophilia A (Factor VIII Deficiency).
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Hemophilia A (Factor VIII Deficiency).
questionsmedicales.fr › Environnement et santé publique › Santé publique › Mesures épidémiologiques › Démographie › État de santé › Qualité de vie › Hemophilia A (Factor VIII Deficiency).