AI Article Synopsis
- The narrative review aims to enhance understanding of hemophilia A, a genetic disease that significantly affects the quality of life and is costly for health systems, particularly noted as one of the top economic burdens in Colombia.
- The review highlights the shift towards precision medicine in treating hemophilia, emphasizing the importance of genetic, pharmacokinetic, and environmental factors that affect treatment efficacy.
- Building robust scientific evidence with strong statistical backing is essential to personalize treatment approaches in a cost-effective manner.
Article Abstract
The purpose of this narrative review was to provide an overview that allows readers to improve their understanding of hemophilia A, which is considered a genetic disease with a high impact on the quality of life of people who suffer from it is considered one of the diseases with the highest cost for health systems (In Colombia it is part of the five diseases with the greatest economic impact). After this exhaustive review, we can see that the treatment of hemophilia is on the way to precision medicine, which involves genetic variables specific to each race and ethnicity, pharmacokinetics (PK), as well as environmental factors and lifestyle. Knowing the impact of each of these variables and their relationship with the efficacy of treatment (prophylaxis: regular infusion of the missing clotting factor VIII in order to prevent spontaneous bleeding) will allow for individualizing the medical behavior in a cost-effective way. For this is required to build more strong scientific evidence with statistical power that allows us to infer.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9944491 | PMC |
| http://dx.doi.org/10.3390/hematolrep15010014 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
HINODE study: haemophilia A in infancy and newborns - protocol for a prospective, multicentre, observational study evaluating the coagulation potential and safety of emicizumab prophylaxis.
BMJ Open
December 2024
Department of Paediatrics, Nara Medical University, Kashihara, Japan.
Introduction: Emicizumab prophylaxis is approved for people of all ages with haemophilia A (HA) including infants and children. Although previous studies have demonstrated the efficacy and tolerability of emicizumab in infants with HA, real-world data on emicizumab use in infants are limited. The Haemophilia A in Infancy and NewbOrns: multi-instituional prospective observational study to assess the efficacy anD safety of Emicizumab (HINODE) study aims to evaluate the coagulation potential and safety of emicizumab prophylaxis in infants with congenital HA from birth to <12 months of age.
View Article and Find Full Text PDFAdvances in biopharmaceutical products for hemophilia.
iScience
December 2024
Chengdu Rongsheng Pharmaceuticals Co., Ltd, Chengdu 610041, China.
Hemophilia is caused by the deficiency of clotting factors due to a single genetic abnormality. Replacement therapies have evolved from plasma-derived to recombinant coagulation factor concentrates but continue to have certain limitations. Monoclonal antibodies are clinical prophylactic treatment options unaffected by inhibitors and have better compliance than coagulation factor concentrates for patients with hemophilia.
View Article and Find Full Text PDFCross-Cultural Translation of the Adolescent Menstrual Bleeding Questionnaire (AMBQ).
Haemophilia
December 2024
Division of Hematology/Oncology IWK Health, Department of Pediatrics Dalhousie University, Halifax, Nova Scotia, Canada.
Aim: Heavy menstrual bleeding (HMB) affects up to 37% of adolescents. Given the paucity of available tools to assess health-related quality of life (HRQoL) in adolescents with HMB, we developed the adolescent menstrual bleeding questionnaire (aMBQ), a valid and reliable measure of bleeding-related quality of life. The aim of this study was cross-cultural translation and adaptation of the English aMBQ into French to ensure accessibility for more Canadian adolescents who menstruate.
View Article and Find Full Text PDFEffects of physical therapy on health-related quality of life in patients with haemophilia: A systematic review and meta-analysis.
Haemophilia
December 2024
Department of Physical Medicine and Rehabilitation, Chang Gung Memorial Hospital, Chiayi, Taiwan.
Introduction: Physical therapy benefits patients with haemophilia (PWH), but its impact on the health-related quality of life (HRQOL) remains unclear.
Aim: This systematic review and meta-analysis investigated the association of physical therapy, including therapeutic exercise, manual therapy, and physical agent modality, with HRQOL improvement in PWH.
Methods: Databases PubMed, Embase, MEDLINE, and Scopus were searched from inception until April 2024.
Marstacimab: First Approval.
Drugs
December 2024
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Marstacimab (marstacimab-hncq; HYMPAVZI™) is a subcutaneously administered human monoclonal immunoglobulin G1 antibody against tissue factor pathway inhibitor (TFPI) that is being developed by Pfizer for the treatment of hemophilia A and B. Marstacimab received its first approval on 11 October 2024 in the USA. It was approved for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors.
View Article and Find Full Text PDFWant 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!