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

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...

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Ngā kaituhi matua:	David A. Andrae, Rachael F. Grace, Adrian Jewett, Brandon Foster, Robert J. Klaassen, Sam Salek, Junlong Li, Feng Tai, Audra N. Boscoe, Erin Zagadailov
Hōputu:	Artigo
Reo:	Inglês
I whakaputaina:	SpringerOpen 2023-11-01
Rangatū:	Journal of Patient-Reported Outcomes
Ngā marau:	Health-related quality of life Psychometric validation Pyruvate kinase deficiency Pyruvate Kinase Deficiency Diary (PKDD) Pyruvate Kinase Deficiency Impact Assessment (PKDIA)
Urunga tuihono:	https://doi.org/10.1186/s41687-023-00650-3
Ngā Tūtohu:	Tāpirihia he Tūtohu Kāore He Tūtohu, Me noho koe te mea tuatahi ki te tūtohu i tēnei pūkete!