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

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) instrument...

Descripción completa

Detalles Bibliográficos
Autores principales: Andrae, David A., Grace, Rachael F., Jewett, Adrian, Foster, Brandon, Klaassen, Robert J., Salek, Sam, Li, Junlong, Tai, Feng, Boscoe, Audra N., Zagadailov, Erin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10636000/
https://www.ncbi.nlm.nih.gov/pubmed/37943362
http://dx.doi.org/10.1186/s41687-023-00650-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10636000/
https://www.ncbi.nlm.nih.gov/pubmed/37943362
http://dx.doi.org/10.1186/s41687-023-00650-3

Ejemplares similares