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IPIG 2025 | Treatment options available for patients with PNH in the UK

Richard Kelly, MBChB, PhD, St James’s University Hospital, Leeds, UK, outlines the available treatment options for patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom, highlighting the agents used in treatment-naive patients and in the second-line setting. Dr Kelly notes that ravulizumab is the most commonly used first-line treatment and discusses the importance of individualizing treatment decisions to each patient. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.

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Transcript

Currently, first-line treatment, we can treat patients with crovalimab, we can treat patients with eculizumab or ravulizumab or iptacopan. The majority of patients that have been treated or are being treated as first-line have been with ravulizumab, which is what we’ve had the most experience with, but we equally discuss crovalimab and iptacopan with patients as they present...

Currently, first-line treatment, we can treat patients with crovalimab, we can treat patients with eculizumab or ravulizumab or iptacopan. The majority of patients that have been treated or are being treated as first-line have been with ravulizumab, which is what we’ve had the most experience with, but we equally discuss crovalimab and iptacopan with patients as they present. If someone presents with a thrombosis we would treat them as an emergency and we would still give them ravulizumab because we know that it’s very quick in its action of preventing and stopping that further thrombosis occurring. 

For patients who have a suboptimal response to C5 inhibition, then second-line treatment we discuss all the available options in the UK which is pegcetacoplan, it’s iptacopan or it’s the addition of danicopam to ravulizumab. And it’s really down to an individual patient discussion, so it’s not one drug suitable for all, so there are nuances with each of the patients. So we’re trying to individualize treatment as much as possible to get the right treatment for each patient.

 

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Disclosures

Consultant/Advisory board: Alexion, Novartis, Sobi, Roche, Omeros, Florio, Otsuka, Amgen; Speaker fees: Alexion, Sobi, Novartis.