Kevin H M Kuo, MD, University of Toronto, Toronto, ON, Canada, provides insight into the use of mitapivat to improve erythropoiesis in adult patients with alpha- or beta-non-transfusion-dependent thalassemia from a long-term study.

These results were presented at the 2022 ASH Annual Meeting & Exposition in New Orleans, LA.

Transcript:

Hello, my name is Dr. Kevin Kuo. I am from the University of Toronto. Today, I'll be talking to you about my abstract on the use of mitapivat in patients with non-transfusion-dependent alpha or beta thalassemia in the long-term extension study, and specifically we'll be focusing on the erythropoietic activity. As we all know, thalassemia is a devastating complication where even in non-transfusion-dependent patients, they suffer from iron overload. The complications are well-known, which includes complications in the heart, in the liver, as well as in the endocrine tissues.

Previously, we presented the results of the phase 2 study where we enrolled 20 patients from the US, UK, and Canada. In this study, we have a mix of patients with alpha and beta thalassemia. All of them are non-transfusion-dependent with hemoglobin of less than 10 grams per deciliter. After the 24 week of core period, the patients were then transitioned to a long-term extension study.

In this long-term extension study up to week 72, we have shown a clear reduction in feron, a stabilization of erythropoietin as well as hepcidin. We have also demonstrated that there was a reduction in soluble transfer in the receptor activity level. And just like previously shown, there was a reduction in bilirubin and marker of hemolytic activity, as well as a reduction in the reticulocyte-to-erythrocyte ratio. In terms of iron overload, the majority of patients had very little liver iron concentration to begin with, so it was difficult to tell, but the majority of patients either had the same amount of liver iron concentration, or there was a slight reduction.

Taken altogether, this demonstrates that the long-term treatment with mitapivat in patients with non-transfusion-dependent alpha- or beta- thalassemia results in improvement in erythropoiesis in addition to an improvement in both hemoglobin as well as hemolytic activity. Thank you.

Source:

Kuo, K H M, Layton D M, Lal A et al. Mitapivat improves markers of erythropoietic activity in long-term study of adults with alpha- or beta-non-transfusion-dependent thalassemia. Presented at the ASH Annual Meeting & Exposition; December 10-13, 2022; New Orleans, LA, and virtual. Abstract 1030.