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