Clinical Trial Endpoints and Molecular Personalization in PV care

In this section, the panel examines how clinical trials and emerging molecular insights may shape the future of polycythemia vera management. Faculty discuss the challenge of defining meaningful endpoints in a chronic disease where patients often live for decades. Ideal outcomes such as reduced thrombosis, bleeding events, and disease progression require long follow up periods and large patient populations, making them difficult to measure in most trials. As a result, surrogate endpoints such as hematocrit control, phlebotomy independence, and symptom improvement have become necessary tools for evaluating therapeutic benefit.

The experts acknowledge that while these endpoints are imperfect, improvements in quality of life and functional status are highly relevant to patients and clinicians. The discussion then turns to molecular markers, including JAK2 allele burden and additional mutations, which are increasingly measured in practice. Although these markers provide insight into disease biology, clinicians often lack clear guidance on how to translate this information into treatment decisions.

Faculty highlight the potential role of advanced analytics and artificial intelligence in integrating molecular, clinical, and laboratory data to better stratify risk and personalize therapy. While these tools are still evolving, there is optimism that they may eventually enable more precise treatment selection and monitoring. Until then, the panel emphasizes that molecular data should complement, not replace, careful clinical assessment and patient centered decision making.