IPIG 2025 | The impacts of PNH and symptoms that clinicians should be aware of

Yes, well, so there’s different aspects to that. So about a third of our patients are undiagnosed for more than 12 months in the UK. They feel much worse than they look. They’ve got a chronic anemia, intermittent bouts where they’ve got abdominal pain, difficulty swallowing, erectile dysfunction in men, their urine can just go black at the drop of a hat, and they may have seen non-haematology clinicians and had cystoscopies looking for hematuria, gastroscopies, colonoscopies looking for GI bleeding, and it’s not until some bright spark thinks let’s test for PNH that they get referred to a specialist at one of the two national centres. And I think that’s very frustrating to patients with a rare condition, access to the right diagnosis and then to seeing a specialist who knows about their disease. 

I think on treatment it’s slightly different. On treatment it’s more about… all patients feel better on treatment, I’ve not come across anyone not feeling better. So we do see some, what I would say, suboptimal responders, those who’ve got clinically significant extravascular hemolysis, and the main symptoms that they present with is fatigue and tiredness. They’re protected against intravascular hemolysis, they’re not going to die from their disease, their risk of thrombosis is dramatically reduced, their abdominal pain, their erectile dysfunction, the hemoglobinuria, that’s gone, but they can have a residual anemia and it’s fatigue and tiredness and those are the patients we should look to switch treatments.

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