PTGX - Quick Take - EHA26 notable updates and addressing some key clinical questions in Polycythemia Vera (PV)

Research evidence excerpt

PTGX - Quick Take - EHA26 notable updates and addressing some key clinical questions in Polycythemia Vera (PV)

ble therapeutic option for managing PV

without the burden of frequent therapeutic phlebotomy.

5 Page Document Clinical questions and our take:

• Is hematocrit control correlated with any biomarker changes or baseline

Reasons for this report characteristics? After CYTO-PV established <45% hematocrit (Hct) level as a meaningful threshold where a 3% difference above 45% can lead to a 4x increase in CV

events and thrombosis, we are comfortable associating Hct control correlation with other ✓ Industry Conference Takeaways

biomarkers, although associations are hard to find. Besides known correlations between

biomarkers (e.g., Hct roughly corresponds with 3x Hb), associations to Hct specifically are

hard to find although independently age, gender, and thrombotic history have been found

to increase thrombotic event risk (Waggoner et al 2023). Separately, although Hct control

did not appear to affect symptom burden, this may have been affected by aggressive

treatments that increase symptom burden with prolonged therapy (Scherber et al 2016).

• Does raising hepcidin actually translate into clinically meaningful effects? Hepcidin

does not appear to have a significant correlation with iron profiles (serum hemoglobin, iron

and ferritin levels) (Semercioglu et al 2020, Hsu et al 2006, Locatelli et al 2006) in anemic

and hemodialysis patient populations, although the nature of the disease could have

confounded the effects and treatments such as iron supplementation could have further

confounded results. However, these studies did find that hematocrit may be causally

linked to prohepcidin levels. Because prohepcidin is more stable than hepcidin, we think