| Autor: |
Egan, Shayna, Zagadailov, Erin, Jewett, Adrian, Li, Junlong, Tai, Feng, Boscoe, Audra, Andrae, David A. |
| Zdroj: |
Blood; November 2021, Vol. 138 Issue: 1, Number 1 Supplement 1 p4144-4144, 1p |
| Abstrakt: |
Introduction:Pyruvate kinase (PK) deficiency is a rare hereditary disorder characterized by chronic hemolytic anemia and long-term complications such as iron overload, osteoporosis, and pulmonary hypertension. There are no patient-reported outcome (PRO) instruments that are specific for this condition. The PK Deficiency Diary (PKDD), a disease-specific PRO, was designed as an evening diary to assess signs and symptoms for patients with PK deficiency. The seven-item instrument assesses tiredness, jaundice, bone pain, shortness of breath, and energy levels. Six of the seven items employ a numeric rating scale (NRS) ranging from 0-10 and one item uses a 5-point verbal descriptor scale. The instrument underwent psychometric validation using blinded data from the eighty patients participating in the ACTIVATE trial, a randomized, multicenter, double-blind, phase 3 trial of mitapivat, an allosteric activator of PK, in non-regularly transfused adults with PK deficiency (NCT03548220). |
| Databáze: |
Supplemental Index |
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