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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| Main Authors: | , , , , , , , , , |
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| Format: | Book |
| Published: |
SpringerOpen,
2023-11-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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