ASCAT 2023 | GBT_HOPE trial: patient experiences of voxelotor as a treatment for SCD

I’m going to be talking about the… so I’m going to deliver two talks. One of them will be on the patient experience of the [GBT_HOPE] trial, which was the trial which led to insight, clinical applicability, of voxelotor in sickle cell patients. So this data has been presented many times before, both at ASCAT as well as at other conferences or published data. So I’m going to recap what the whole trial data of voxelotor has shown, and within this session we’re going to talk about, we’re going to put a lot of emphasis on the patient experience with voxelotor, so there will be patients talking about this.

And then the other trial will be based on the early access to medicine scheme data, which is related to the use of voxelotor in the UK through this early access to medicine scheme. So again this is data that has been presented before, so there’s not going to be anything particularly new on this, but it’s a very important talk because it just supports the fact that this drug will hopefully have a role to play in the management of patients with sickle cell disease, if you fill certain criteria and cannot be treated in other ways.

You’ll hear everything about it on Friday, but overall, the experience has been positive, from the patient access through this early access to medicine scheme. So by and large, patients would have been on voxelotor through this scheme have tolerated it very well and the results that we saw in terms of improvement of the hemoglobin and reduction of the hemolysis, have been in keeping with what the whole trial has shown and what we have expected to see in terms of adverse events. We haven’t seen, again, anything beyond what we expected to see based on the [GBT_HOPE] trial safety data. And the feedback from the patients, by and large, is positive, with a few exceptions whereby the drug has not caused any particularly significant positive impact or a few patients have had some intolerance issues as you would expect with any treatment one can be on. But overall, positive results and positive observations when it comes to patients’ overall well-being feedback as well as increase of the level of hemoglobin and reduction of hemolysis. But I think we need more experience and more data to show us correlation of what we see to painful crises and other interference with reducing risk of end organ damage complications. So, you know, the more experience we collect, the more data we collect nationally but also internationally, the more prepared we will be as clinicians to identify suitable patients for this drug.