Christian Buske discusses Waldenström’s macroglobulinemia and the aims and findings of the pan-European data platform.
FILMED AT THE EUROPEAN HEMATOLOGY ASSOCIATION (EHA) ANNUAL MEETING, JUNE 2016
WHAT IS WALDENSTRÖM’S MACROGLOBULINEMIA (WM)?
00:10 – Waldenström’s Macroglobulinemia belongs to the indolent B non-Hodgkin lymphomas. What is very important to know is that Waldenström’s Macroglobulinemia is a rare disease so the incidence is just one or two cases per million per year. And what is also important to know is that Waldenström’s Macroglobulinemia is in some ways special compared to other nodal non-Hodgkin lymphoma such as follicular lymphoma, because these patients characteristically have high monoclonal IgM levels in their blood, and this means that actually they have a lot of problems caused by this high IgM serum levels, which can be hyper viscosity or neuropathy or even dizziness, brain symptoms and so on.
WHAT ARE THE CURRENT TREATMENT OPTIONS FOR WM?
00:59 – One very important treatment option still, is the combination of an anti-cd20 antibody, rituximab, with chemotherapy. So what is very popular, actually, is the combination of rituximab bendamustine. But we have as another indolent B non-Hodgkin lymphomas, also here the wish to get rid of chemo. So there’s this strong tendency to develop chemo-free treatment approaches. And in this sense ibrutinib is a very important new drug which is now approved for the treatment of relapsed patients with this disease, and also for patients first line who are not eligible for classical chemotherapy. And there’s also one…actually, also one abstract here which was in major aspects driven by the so-called European Consortium for Waldenström’s Macroglobulinemia, which is a pan-European network which commits itself to drive clinical research and patient care in this rare disease. And this shows actually that ibrutinib is also highly effective in patients who are rituximab refractory, which is a very important patient population because all the patients get in some way rituximab, and when they don’t respond, what do you do then? Then ibrutinib shown here at the EHA is a very valuable treatment option.
WHAT ARE THE AIMS OF THE PAN-EUROPEAN DATA PLATFORM?
02:21 – This data platform was actually developed because, as I said, Waldenström’s disease is a very rare disease so there are actually more or less no real-world data on how these patients are suffering, how these patients are actually treated outside of clinical trials. This is important. So what is actually the situation in the real world for these patients? And this is why, actually, this platform was developed, which actually includes patients who started their treatment between the years 2000 and 2011. And we included over 350 patients and have now really very nice data on how patient care looks like in daily life in this rare disease.
WHAT ARE THE FINDINGS TO DATE AND HOW WILL THESE DATA BE USED?
03:13 – So with regard to the Waldenström’s registry, what we do is actually that we now look very carefully at these data to get a sense, actually, what the patient and the treating physicians are doing because for us it’s very important to know that for instance when we are developing clinical trials that we are not developing these clinical trials, let’s say, in a wrong direction, so against the wish of the community. So for us it’s very important, as I said, to get a feeling what is happening outside of clinical trials, what is working well with these patients in daily clinical life, where there is room for improvement. And we incorporate all these data into the design of novel clinical trials which are continuously developed by the European Consortium for Waldenström’s Macroglobulinemia. So, for instance as an example, we now know that elderly patients with Waldenström’s Macroglobulinemia based on the registry data, are not treated in a dose-intense way. So I think to set up a clinical trial which now tests those intense approaches in these elderly, quite often…patients, morbid patients, I think is the wrong way. So this is why we think that this registry data are actually a platform or bases for developing the right clinical trials to at the end improve clinical care for these patients.