Abstract
Summary
Researchers looked at the latest research studies about plasma-derived human factor X concentrate (pdFX for short) for people living with hereditary factor X deficiency (HFXD for short). These people’s health and daily lives were impacted in different ways by HFXD, and their condition was classified as mild, moderate, or severe. The researchers assessed information from 5 clinical trials and 1 case series that described the bleeding symptoms and treatment that a group of people living with HFXD had.
Researchers found that pdFX has been successfully used in adults and children with HFXD to treat bleeds when they happen, to prevent bleeds, and to manage bleeding around having surgery. Researchers found that people living with HFXD had minimal side effects when they received pdFX.
Research studies to date have shown that pdFX is safe and effective as a treatment for people living with mild, moderate, or severe HFXD. This information will help healthcare experts to develop recommendations based on research results about pdFX treatment for people living with HFXD.
The purpose of this plain language summary is to help you to understand the findings from recent research.
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Acknowledgements
The authors would like to thank the people living with HFXD, healthcare professionals, and study site staff who took part in these studies. Medical writing assistance for this summary was provided by Alison Halliday, PhD, Lauri Arnstein Williams, MA MBBS, and Simon Stones, PhD, ISMPP CMPP™, at Amica Scientific, and funded by Kedrion Biopharma Inc. This summary was also reviewed by an expert patient.
Disclaimers
Researchers must look at the results of many types of studies to understand whether a study treatment works, how it works, and whether it is safe to prescribe to people. This summary reports information from a review article. The findings might be different from other review articles.
Author contributions
All authors were involved in the writing, reviewing, and editing of the summary, and approved the final version for publication.
Funding
This summary was sponsored by Kedrion Biopharma Inc.
Ethical disclosure
As described in the original article, the studies involving human participants were reviewed and approved by the institutional review board/research ethics committee at each study center. The 3 studies each involved multiple study sites. Participants in the studies agreed to have their results published. Written informed consent to participate in this study was provided by each participant or the participants’ legal guardian/next of kin as applicable and appropriate.
Financial and competing interest disclosure
Miguel A. Escobar received honoraria for participation in advisory boards and consultation from Bayer, Biomarin, CSL Behring, Genentech/Roche, LFB/Hemobiologics, UniQure, Novo Nordisk, Pfizer, Sanofi, Kedrion, Takeda, and the National Hemophilia Foundation. Kaan Kavakli received honoraria for participation in advisory boards or scientific speeches in meetings from Novo Nordisk, Bayer, Pfizer, Roche, Sanofi, and Takeda.
Consent for publication
Not applicable.
