Emerging Therapies in β-Thalassemia.

Humans beta-Thalassemia / therapy Erythropoiesis Iron Iron Overload / therapy
Advancement of care Clinical trial Novel therapies Quality of life Transfusion burden β-Thalassemia

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:
04 2023
Historique:
entrez: 12 3 2023
pubmed: 13 3 2023
medline: 15 3 2023
Statut: ppublish

Résumé

Advances in understanding the underlying pathophysiology of β-thalassemia have enabled efforts toward the development of novel therapeutic modalities. These can be classified into three major categories based on their ability to target different features of the underlying disease pathophysiology: correction of the α/β globin chain imbalance, targeting ineffective erythropoiesis, and targeting iron dysregulation. This article provides an overview of these different emerging therapies that are currently in development for β-thalassemia.

Identifiants

DOI: 10.1016/j.hoc.2022.12.010 PMID: 36907614
pubmed: 36907614
pii: S0889-8588(22)00151-4
doi: 10.1016/j.hoc.2022.12.010
pii:
doi:

Substances chimiques

Iron E1UOL152H7

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

449-462

Informations de copyright

Copyright © 2022 Elsevier Inc. All rights reserved.

Auteurs

Rayan Bou-Fakhredin (R)

Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy.

Kevin H M Kuo (KHM)

Division of Hematology, University of Toronto, Toronto, ON, Canada.

Ali T Taher (AT)

Division of Hematology-Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon. Electronic address: ataher@aub.edu.lb.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Emerging Therapies in β-Thalassemia.
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Thalassémie › bêta-Thalassémie › Emerging Therapies in β-Thalassemia.
questionsmedicales.fr › Phénomènes physiologiques respiratoires et circulatoires › Phénomènes physiogiques du sang › Hématopoïèse › Érythropoïèse › Emerging Therapies in β-Thalassemia.
questionsmedicales.fr › Produits chimiques inorganiques › Métaux › Métaux lourds › Fer › Emerging Therapies in β-Thalassemia.
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Troubles du métabolisme du fer › Surcharge en fer › Emerging Therapies in β-Thalassemia.