Efficacy and Safety of Mitapivat in Pyruvate Kinase Deficiency: A Systematic Review and Meta-analysis of Clinical Trials.

ABSTRACT:

Background

Pyruvate kinase deficiency (PKD), a rare autosomal recessive disease, often leads to chronic hemolytic complications stemming from mutations in the PKLR gene. Mitapivat, an innovative, allosteric activator of the pyruvate kinase enzyme in red blood cells, has emerged as a potential therapeutic agent. This systematic review aims to meticulously evaluate the efficacy and safety of Mitapivat in treating PKD patients.

Methods

We conducted a comprehensive search across three major databases-PubMed, Web of Science, and Scopus-up to November 2023, utilizing a single-arm meta-analysis methodology.

Results

Our analysis included three clinical trials comprising 94 PKD patients. Hemoglobin (HB) levels improved, with an average increase of 1.05 g/dl (95% Confidence Int

SUBMITTER: Mohammed HE 

PROVIDER: S-EPMC11794918 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature