Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial

• Main Authors: David A. Andrae (Author), Rachael F. Grace (Author), Adrian Jewett (Author), Brandon Foster (Author), Robert J. Klaassen (Author), Sam Salek (Author), Junlong Li (Author), Feng Tai (Author), Audra N. Boscoe (Author), Erin Zagadailov (Author)
• Format: Book
• Published: SpringerOpen, 2023-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Pyruvate kinase (PK) deficiency is a rare hereditary disorder characterized by chronic hemolytic anemia and serious sequalae which negatively affect patient quality of life. This study aimed to psychometrically validate the first disease-specific patient-reported outcome (PRO) in...