We were fortunate that ASH really wanted to highlight ways that we could optimize treatment for people with sickle cell disease beyond just pursuing curative and transformative options. So, my part of our session was actually to discuss the disease-modifying medicines that have been approved for sickle cell disease, and then really go into how you select those and providing a practical guide to clinicians about how we can go about selecting them based on the available clinical evidence, patients’ and families’ preferences, and then just availability and access...
We were fortunate that ASH really wanted to highlight ways that we could optimize treatment for people with sickle cell disease beyond just pursuing curative and transformative options. So, my part of our session was actually to discuss the disease-modifying medicines that have been approved for sickle cell disease, and then really go into how you select those and providing a practical guide to clinicians about how we can go about selecting them based on the available clinical evidence, patients’ and families’ preferences, and then just availability and access. And then we also talked about ways that we could optimize them after we’d started those therapies. So we wanted to know what are the factors that might contribute to a patient having a suboptimal response. So things like not consistently taking it or non-adherence. We wanted to know about whether or not patients were optimally dosed on the medication that they were receiving and whether or not the treatment outcomes were as expected with what the patients initially had planned for their expectations with that treatment. And then finally, we went on to discuss that there are limited resources available to guide clinicians and to guide patients about how to use these medications, how to use them in combination. But as the treatment landscape for sickle cell disease changes, we need better, more patient-guided and evidence-based guided management tools to help us select the right therapy for the right patient.
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