So I’m predominantly a nonmalignant hematologist, so PNH, aplastic anemia. But also I think, importantly, autoimmune hemolytic anemia. So the EPO poster in autoimmune hemolytic anemia is certainly very interesting.
But I’ve also been involved here at EHA on cold agglutinin disease and really, again, this is another rare disorder like PNH, it’s considered benign, non-malignant, but it has a huge consequence to patients and a huge burden to our healthcare systems.
And I think we in the past have not had very many effective therapies. We also haven’t had any approved therapies, so I think we’re all waiting in anticipation for the first approved therapies in this disease. I’ve been delighted that actually two of the UK patients came to the conference with me and have been presenting their experiences of living with rare diseases. So we have one patient with cold agglutinin disease who showed that it wasn’t just about getting Raynaud type symptoms in the hands.
She struggles with even having a glass of water at home. She has to let it warm up to room temperature. So really very striking how patients are trying to live everyday life with a condition like cold agglutinin disease.
And the second patient I’ve brought as aplastic anemia, and he was a world class athlete, and when you looked at him, you’d think he was well and healthy, but actually he’s not doing what he wants to do. He can’t even walk 30 meters, never mind run a hundred meters in 10 seconds.
So this is again, a huge impact. And this is where I mentioned before about quality of life. It’s so important that we don’t just look at the outward appearance and think they look well. We should be questioning the patients quite deeply. Are they living the lives they want to live or can we do anything to improve that further?