So my name is Johan Zakwaja. I am a haematologist in the UK at the University College London Hospitals and the National Aminoidosis Centre. Systemic amyloidosis is a rare condition and it's seen in certain patients who have myeloma or other plasma cell disorders. And it's an incurable condition but something that we can treat. Patients with amyloidosis, with light chain disease, AL amyloidosis, are treated with similar treatments to those with myeloma. So they're different diseases but they're treated in a similar manner. Typically those with AL amyloidosis have a small burden of disease so they don't typically have lots of abnormal plasma cells or you know disease when we do a bone marrow test. But these abnormal cells can be really problematic. They can cause problems with the kidneys, the heart, the liver, the spleen and lots of organs and that really can cause the manifestations and problems in amyloidosis. The reason why it's really important for people to think about amyloidosis is it's a rare condition but if it's picked up for patients who've got some of these abnormal symptoms, like with the heart or the kidneys etc and they've got this abnormal monoclonal protein, if this is diagnosed there's some really excellent treatments out there that can potentially treat this disease and give really good outcomes. So we know in recent years there have been changes in treatments for patients with amyloidosis and that's really encouraging I think for the whole myeloma community that outcomes have actually improved. In the UK we've looked at our data over a number of decades and found that in the recent decades outcomes have improved and that's across all stages of disease. So early stage disease always did really excellently but now even more moderate and advanced stage disease has improved in terms of outcomes. A lot of that is related to the developments in anti-myeloma therapies that are more tolerable, that have less toxicity and we're able to use those I think better in patients with amyloidosis. And as well as that there's better I think supportive care so we're a bit more tuned into you know how to to deal with some of the potential side effects with treatments so maybe problems with fluid overload or neuropathy or steroids that we're using we can tolerate treatment modifications better. We did a study which looked at outcomes in Europe so from the UK, from Greece and from the Netherlands in patients with amyloidosis and found that in the most recent years there's been improvements in outcomes particularly in the earlier stages of disease so that's really encouraging. We've also tried to see how do we find out those who do the poorest and we looked at a number of different tests so one of these were kind of blood tests that we traditionally look at your heart markers. We also looked at a marker that we see in scans in the heart so an echocardiogram scan of the heart. We use that with blood tests to see do those pick out those patients who do more poorly and we found actually across patients in all three of these countries we can use these tests to accurately identify patients who have poorest disease and that's relevant because in modern treatment areas where outcomes have improved it's important for us to be able to accurately tell patients well we think you're going to do well because of these factors or we're a bit more guarded in our prognosis because of these factors so I think it's really encouraging that outcomes have improved in general. I think it's encouraging that we've got better tools at giving us an idea of which patients will do well and which patients may do less well.
HealthTree • June 27, 2025