Prophylactic treatment with rurioctocog alfa pegol, a recombinant factor VIII (FVIII), leads to dose-dependent normalization of joint health biomarkers in patients with severe hemophilia A, according to results from the PROPEL study.

These findings were presented as a poster at the 63rd American Society for Hematology Annual Meeting and Exposition.

“This novel biomarker data is the first to demonstrate the importance of FVIII prophylaxis for joint health in patients with hemophilia A from a randomized clinical trial,” the authors wrote. It is important to monitor joint deterioration in patients with hemophilia A to guide the intensity of prophylactic treatment with FVIII, which patients need regularly.


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The relationship between biomarkers of joint health and factor replacement therapy in patients with severe hemophilia A using data from the PROPEL study was examined by a team of researchers led by Morten Karsdal, PhD, mMBA, chief executive officer and head of research for Nordic Bioscience.

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The researchers reported that biomarkers that reflected the remodeling of the basement membrane (collagen type 4 formation PRO-C4 and degradation C4M) significantly decreased after 3 months in patients treated with rurioctocog alfa pegol. 

The same was observed in biomarkers of interstitial matrix remodeling (collagen type 5 formation PRO-C5 and collagen type 3 degradation C3M).

The cartilage formation marker collagen type 2 formation PRO-C2 was significantly increased after 6 months but there was no significant difference in the cartilage degradation biomarker C2M. These observations were consistent across 2 different FVIII trough levels (1%-3% or 8%-12%).

When the researchers analyzed the data according to whether or not patients had received prior treatment, they found that there were significant changes in C4M and C3M between FVIII 1%-3% and FVIII 8%-12% in on-demand patients after 6 months. However, there were no differences in patients who were being treated with FVIII prophylactically before entering the study.

“Together, the data suggest that FVIII prophylaxis leads to a normalization of joint remodeling,” the researchers concluded.

Reference

Manon-Jensen T, Tangada S, Bager C, et al. Prophylactic treatment with rurioctocog alfa pegol results in a dose-dependent normalization of biomarkers of joint health in severe hemophilia A: an exploratory analysis from the Propel study. Poster presented at: 63rd American Society of Hematology Annual Meeting & Exposition: December 12, 2021; Atlanta, Georgia.