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Review
. 2022 May;13(Suppl 3):7-20.
doi: 10.6004/jadpro.2022.13.4.8. Epub 2022 Jun 21.

Hemophilia A: Strategies for Improving Long-Term Holistic Management, Adherence, and Quality of Life

Miguel A Escobar  1 , Cindy Leissinger  2 , Guy Young  3
Affiliations
Review

Hemophilia A: Strategies for Improving Long-Term Holistic Management, Adherence, and Quality of Life

Miguel A Escobar et al. J Adv Pract Oncol. 2022 May.

Abstract

Hemophilia A is a rare inherited bleeding disorder characterized by a deficiency in factor VIII. The evolution of currently approved prophylaxis therapy in hemophilia A will be reviewed, including the clinical value of prophylaxis, real-world experience with prophylaxis, and patient quality-of-life factors that must be considered when choosing treatment options for these patients.

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Conflict of interest statement

The faculty reported the following relevant financial relationships or relationships they have with ineligible companies of any amount during the past 24 months: Miguel A. Escobar, MD, reported a financial interest/relationship or affiliation in the form of Consultant: Biomarin; Kedrion Biopharma; Rani Therapeutics; Magellan Pharmaceuticals; Takeda Pharmaceutical Co, LTD; sanofi; uniQure; CSL Bering; Genentech, Inc; Novo Nordisk; and Pfizer, Inc. Serve(d) as a speaker or a member of a speakers bureau for: CSL Bering; Novo Nordisk; Pfizer, Inc; Kedrion Biopharma; Roche; and Bayer HealthCare, Inc. Contracted research: Takeda Pharmaceutical Co, LTD; Novo Nordisk; uniQure; sanofi; Pfizer, Inc; Opto Biologics; and Genentech, Inc. Cindy Leissinger, MD, reported a financial interest/relationship or affiliation in the form of Advisory board: Bayer HealthCare, Inc; CSL Bering; Catalyst Pharmaceuticals; Genentech, Inc; uniQure; sanofi; and Takeda Oncology. Contracted research: BioMarin Pharmaceutical, Inc. Guy Young, MD, reported a financial interest/relationship or affiliation in the form of Serve(d) as a speaker or a member of a speakers bureau for: Genentech, Inc; BioMarin Pharmaceutical, Inc; sanofi; Takeda Oncology; and Grifols. Consultant: BioMarin Pharmaceutical, Inc; Genentech, Inc; Spark Therapeutics; sanofi; Novo Nordisk; and Takeda Oncology.

Figures

Figure 1
Figure 1
Personalized prophylaxis. Information from Carcao & Iorio (2015).
Figure 2
Figure 2
Emicizumab-kxwh.
Figure 3
Figure 3
HAVEN 1–4: Number of treated bleeds.
Figure 4
Figure 4
HAVEN 1–4: Mean ABR over time.
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See this image and copyright information in PMC

References

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