# An Observational Study of Glycopegylated Extended Half-Life Factor Prophylaxis in Hemophilia A and B in a Tertiary Care Center in India

## TL;DR

This study shows that extended half-life clotting factors reduce bleeding and improve joint health in Indian hemophilia patients.

## Contribution

Demonstrates the efficacy and safety of glycopegylated EHL factors in a real-world Indian setting with long-term outcomes.

## Key findings

- Glycopegylated EHL FVIII reduced annual bleeding rates and improved joint health in hemophilia A patients over 24 months.
- Hemophilia B patients showed reduced bleeding and joint issues within eight months of EHL FIX prophylaxis.
- Prophylaxis with EHL factors improved treatment adherence and is viable in resource-limited settings.

## Abstract

Prophylactic treatment for hemophilia in India remains significantly underutilized compared to developed nations, leading to reliance on episodic therapy with limited long-term benefits. This study evaluated the efficacy and safety of glycopegylated extended half-life (EHL) clotting factors in patients with hemophilia A and B. A retrospective observational design was employed, including 25 patients aged ≥18 years with moderate or severe hemophilia A or B who transitioned to EHL therapy. Hemophilia A patients (n=19) switched from standard half-life (SHL) factor VIII (FVIII) to weekly EHL FVIII prophylaxis, while hemophilia B patients (n=6) transitioned from on-demand SHL factor IX (FIX) to glycopegylated EHL FIX prophylaxis every 21 days. Outcomes were assessed through the annual bleeding rate (ABR), functional independence score in hemophilia (FISH score), and hemophilia joint health score (HJHS). A subset analysis for quality of life (QoL) was also performed on hemophilia A patients using the Haem-A-QoL questionnaire in patients with hemophilia A. Glycopegylated EHL FVIII therapy significantly reduced ABR and improved joint health and functional scores over 24 months (p<0.001). Hemophilia B patients demonstrated a significant reduction in ABR and joint bleeding rates at eight months (p<0.05). The study found that prophylaxis with individualized doses of glycopegylated EHL factors is not only effective in reducing bleeding episodes and improving joint health but also safe with improved treatment adherence, offering a viable prophylactic option in resource-constrained settings.