IPIG 2025 | Unmet needs in the field of PNH

Yeah, so again we heard a lot this morning about certain unmet needs. One area that’s of interest is identifying individuals who may benefit from proximal complement inhibitors and switching from frontline treatment with C5 or terminal complement inhibitors. We know that individuals can remain anemic or have markers of extravascular hemolysis. We know the clinical trial criteria that have been used but there’s interest in developing new biomarkers or being able to identify patients who may be able to make that switch. This is an area of active research that members of this group are contributing to. 

One area that I think that we should also talk about and is important to recognize is that PNH is a very rare disease. So many general hematologists may not have seen patients with PNH or may have very few cases in their practice. And then in terms of general practitioners or other physicians, it may not even be a disease that’s necessarily at the top of their awareness. And so I think broader education within our health system about this disease, how it may present and how to, especially in this era where we have a lot of very exciting and effective new treatments, it’s very important to be able to diagnose this condition early because we know that it comes with significant complications if untreated, and be able to get these patients on effective therapy. And so I think that’s an area that from a broader medical perspective is really essential.

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