Key Takeaways From Discussion

Caroline Piatek, MD: An important takeaway is that ITP is a heterogeneous disorder. There are different ways to treat it in the up-front and relapsed setting, so there’s no right way to do it. As we look at new and immersion therapies, we’re finding further evidence of the heterogeneity of this disease, seeing that there are subsets of patients who respond to complement inhibition, etc. We have our work cut out for us in the coming years as we figure out how to best treat our patients with ITP.

Craig Kessler, MD: The most important takeaway message in ITP is that the disease is so heterogeneous that we need robust clinical trials. In my opinion, every patient who has the diagnosis of ITP should be placed into some prospective randomized controlled trial and a registry. This registry should look at biomarkers. It should be a biorepository so we can have a genetic as well as tissue examination. No patient in this country should be diagnosed and treated without being placed or considered to be placed into a clinical trial. We’re so far behind the Europeans and the Chinese in being able to gather data that we also have to convince the patients of the importance to participate in clinical trials. Our fellow hematologists in private practice have to appreciate that this ITP [immune thrombocytopenic purpura] is not the kind they learned about in medical school. This is a much more complicated disease, and patients deserve to be treated in a more innovative and creative manner.

James Bussel, MD: One way we could do that is by better access of large databases and using existing records. If people could agree on standard things that would be evaluated in the way it was recorded, we could get there faster. Even 1 large randomized clinical trial of comparing second-line options would be amazing. I still think the biggest lesion, if you will, is our ability to define what’s in front of us. You talked about that, Craig, with what you said about the repository and genomic examination. It’s become pretty clear that it’s not as if 52% of the patients have a mutation in X and we just never saw that. It’s not impossible, but this is a multigenic disease with a number of contributing features. It will be hard to sort out, but the onus is on us to do that.

Dr Piatek, Dr Kessler, thank you very much for making this an enjoyable and fun discussion. I’ll look forward to seeing you all in the near future.

Transcript Edited for Clarity