What type of study is this?

This is a Pre-Registered Trial study.

What question did this study set out to answer?

This research aims to evaluate the effects of mitapivat on red blood cell integrity and ubiquitin accumulation in sickle cell disease.

December 8, 2025BloodOpen Access

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Ex vivo findings show mitapivat treatment increased ATP and restored function of p97, decreasing oxidative stress in sickle RBCs.

This research aims to evaluate the effects of mitapivat on red blood cell integrity and ubiquitin accumulation in sickle cell disease.
RBC membranes were isolated from whole blood samples of individuals with HbAA, HbAS, and HbSS.
K48-Ub and p97 levels were analyzed through Western blotting and densitometry analysis.
RBCs from HbSS treated with mitapivat were evaluated at varying concentrations and time points for K48-Ub and ATP levels.
Statistical significance was assessed using a one-sample, two-sided t-test and Spearman rank correlation analysis.
Higher levels of K48-Ub and p97 were found on HbSS RBC membranes compared to HbAA and HbAS groups.
Mitapivat treatment improved ATP production and reduced K48-Ub and p97 levels in a dose- and time-dependent manner.
A significant negative correlation was observed between K48-Ub and ATP/Hct levels, indicating improved RBC integrity.

Gao et al. (2025) studied this question.

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