RWD25 Patient Characteristics and Healthcare Resource Utilization of Patients with Pyruvate Kinase Deficiency in a United States Electronic Health Record Database

Abstract

Pyruvate kinase (PK) deficiency is a rare, inherited disorder caused by autosomal recessive mutations in the PKLR gene. There are no approved pharmacotherapies for PK deficiency. This study used data from a a large US electronic health record (EHR) database to summarize the clinical characteristics and healthcare resource use (HCRU) of patients with PK deficiency.