Plain Language Summary
What is this summary about?
This is a plain language summary of a clinical research study called XTEND-1. The study looked into how safe and effective a medicine called efanesoctocog alfa is for people with severe hemophilia A. Hemophilia A is a genetic condition in which the body does not produce enough or makes dysfunctional clotting factor VIII (eight) – a protein that is essential for blood clotting. People with hemophilia A are prone to dangerous bleeding, particularly internally and into their joints and muscles.
How was the research done?
The XTEND-1 study compared two ways of using the medication to treat bleeding episodes: (1) injecting it once a week for 12 months as a prophylaxis (regular preventative) treatment, or (2) injecting it as needed for 6 months, followed by weekly prophylaxis treatment for a further 6 months.
What did the research find out?
People who had prophylaxis treatment with efanesoctocog alfa injections for one year saw a significant reduction in the amount of bleeding they experienced compared to their pre-study prophylaxis treatment. The effects of the treatment also lasted longer, with higher factor VIII levels for longer, than previous prophylaxis treatment and less frequent injections were needed. There was a significant reduction in the amount of bleeding in people taking once-weekly prophylaxis treatment compared to ‘as needed’ treatment.
Clinical Trial Registration: NCT04161495 (ClinicalTrials.gov)
This is an abstract of the Plain Language Summary of Publication article.
To read the full Plain Language Summary of this article, click here to view the PDF.
Link to original article here
Financial disclosure
The authors review and approved the PLSP and the content of this PLSP is based on their manuscript. Supported by Sanofi and Sobi. Disclosure forms provided by the authors are available with the full text of this article at NEJM.org. Sanofi owns the rights for Sanofi employees who are authors, but the contributors who are external authors also have a share of ownership. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed
Acknowledgments
Sanofi, Sobi, and the authors of this manuscript would like to thank all of the patients who participated in this clinical study, their families and all of the study center staff members who contributed. They also wish to thank Stacey Poloskey for monitoring the safety and welfare of the participants in this study. Medical writing assistance was provided by Danielle Birchall MRes MICR, and Catherine Barras MSc, Simplifying Science® and was supported financially by Sanofi and Sobi. Simplifying Science® is a registered trademark in Great Britain and Northern Ireland