{"entity":{"id":"idea-pv-hepcidin-first","kind":"idea","name":"Hepcidin-based control as first-line treatment in low-risk polycythaemia vera","aka":[],"tldr":"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.","asOf":"2026-09-16","links":[],"tags":["polycythaemia-vera","mpn"],"related":[],"cancers":["polycythaemia-vera"],"sections":[],"technologies":[],"targets":[],"drugs":["rusfertide"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["verify","low-pv"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"notes":[],"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.","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","cost":"medium","horizonYears":5},"route":"/ideas/idea-pv-hepcidin-first/","neighbours":{"cancer":[{"id":"polycythaemia-vera","kind":"cancer","name":"Polycythaemia vera (PV)","route":"/cancers/polycythaemia-vera/"}],"drug":[{"id":"rusfertide","kind":"drug","name":"Rusfertide","route":"/drugs/rusfertide/"}],"trial":[{"id":"low-pv","kind":"trial","name":"Low-PV","route":"/trials/low-pv/"},{"id":"verify","kind":"trial","name":"VERIFY","route":"/trials/verify/"}]}}