Transforming Hemophilia A Care: Insights into New Therapeutic Options.

Life (Basel)

Genetics Department, Carol Davila University of Medicine and Pharmacy, Dionisie Lupu 37 Street, 020021 Bucharest, Romania.

Published: November 2024

Hemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and advancements in treatment strategies. A literature review was conducted using various available databases. Relevant studies on hemophilia A, covering genetics and treatment options, were selected and summarized. Recent developments in gene therapy are discussed, showcasing their potential to offer long-term solutions and reduce the burden of treatment. Additionally, the review addresses global disparities in care and policy implications, emphasizing the need for comprehensive healthcare frameworks to improve outcomes for individuals living with hemophilia A worldwide. By synthesizing recent findings and insights, this review aims to inform clinicians and policymakers about the evolving landscape of hemophilia A management and the necessity for equitable access to care.

Download full-text PDF

Source
http://dx.doi.org/10.3390/life14121568DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11677678PMC

Publication Analysis

Top Keywords

hemophilia
5
transforming hemophilia
4
hemophilia care
4
care insights
4
insights therapeutic
4
therapeutic options
4
options hemophilia
4
hemophilia hereditary
4
hereditary bleeding
4
bleeding disorder
4

Similar Publications

Special Issue: "Post-COVID-19 Syndrome".

Viruses

December 2024

Institute of Transplantation Diagnostics and Cell Therapy, Division of Hemostasis, Hemotherapy, and Transfusion Medicine, Blood and Hemophilia Comprehensive Care Center, Heinrich Heine University Medical Center, D-40225 Düsseldorf, Germany.

On 30 January 2020, the World Health Organization declared COVID-19 a Public Health Emergency of International Concern (PHEIC)-the highest WHO warning level [...

View Article and Find Full Text PDF

: This systematic review evaluates the effectiveness of various hematological treatment protocols and local hemostatic measures in preventing oral bleeding and other complications during and after dental treatments in children with inherited bleeding disorders and inhibitors. : This study was registered in the PROSPERO database. The comprehensive search strategy for this systematic review was conducted across five databases, namely, PubMed, Google Scholar, Web of Science, Scopus, and Cochrane Library.

View Article and Find Full Text PDF

CRISPR/Cas9 Edition of the Gene in Human Mesenchymal Stem Cells for Hemophilia B Therapy.

Life (Basel)

December 2024

División de Genética, Centro de Investigación Biomédica de Occidente, Instituto Mexicano del Seguro Social, Guadalajara 44340, Jalisco, Mexico.

Hemophilia B is a genetic disorder characterized by clotting factor IX deficiency and bleeding in joints and muscles. Current treatments involve intravenous infusion of plasma-derived products or recombinant proteins, which have limited efficacy due to the short half-life of infused proteins. Recently, gene therapy for bleeding disorders has offered a potential solution.

View Article and Find Full Text PDF

Hemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and advancements in treatment strategies. A literature review was conducted using various available databases.

View Article and Find Full Text PDF

Background: Although more than four years have passed since the pandemic began, SARS-CoV-2 continues to be of concern. Therefore, research into the underlying mechanisms that contribute to the development of the disease, especially in more severe forms, remains a priority. Sustained activation of the complement (CS), contact (CAS), and fibrinolytic and kinin-kallikrein systems (KKS) has been shown to play a central role in the pathogenesis of the disease.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!