# Hepcidin-based control as first-line treatment in low-risk polycythaemia vera

Source: https://onco.cc/ideas/idea-pv-hepcidin-first/  
OnCo record `idea-pv-hepcidin-first` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Rusfertide replaced phlebotomy in patients who needed it often. The open question is whether hepcidin control from diagnosis, in low-risk patients who today get phlebotomy alone, prevents the iron deficiency, the count swings and perhaps the clots that phlebotomy leaves behind.

## Summary

Phlebotomy has been first-line PV treatment for a century, but it makes patients iron deficient, lets the haematocrit swing between sessions and is time consuming. Rusfertide and the TMPRSS6-silencing RNA drugs in early trials hold the haematocrit steady by limiting iron to the marrow. VERIFY tested rusfertide only as an add-on in phlebotomy-dependent disease. The idea is a first-line trial in low-risk patients comparing hepcidin control with standard phlebotomy on haematocrit time-in-target, symptoms and thrombosis, with cost and injection burden weighed against the benefit.

## Fields

- Kind: Idea
- Last checked: 2026-09-16
- Tags: polycythaemia-vera; mpn
- Hypothesis: In newly diagnosed low-risk PV, a hepcidin mimetic will keep patients within the haematocrit target for a larger share of time than phlebotomy, with fewer symptoms of iron deficiency and no increase in thrombosis.
- Rationale: Haematocrit variability, not only the average, is linked to clot risk; VERIFY showed sustained control and better patient-reported outcomes; iron deficiency symptoms are common and under-recognised in phlebotomised patients.
- Proposed test: A randomised trial of about 300 low-risk patients, hepcidin mimetic versus phlebotomy plus aspirin, with time within haematocrit target over two years as the primary endpoint and thrombosis, fatigue scores and cost per patient-year as secondary endpoints.
- Maturity: being-tested-at-scale
- Actor: industry

## Connected records

- cancers: [Polycythaemia vera (PV)](https://onco.cc/cancers/polycythaemia-vera/)
- drugs: [Rusfertide](https://onco.cc/drugs/rusfertide/)
- trials: [Low-PV](https://onco.cc/trials/low-pv/), [VERIFY](https://onco.cc/trials/verify/)

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