This is a substudy of Study AG348-C-008, an observational, longitudinal, multicenter, global registry that aims to better understand the longitudinal clinical implications of pyruvate kinase deficiency (PK deficiency). The primary purpose of this study is to describe the cognition of participants with PK deficiency, as objectively measured by the Cogstate Brief Battery (CBB).
Study Type
OBSERVATIONAL
Enrollment
13
Massachusetts General Hospital
Boston, Massachusetts, United States
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Change Over Time in Cogstate Brief Battery (CBB) Scores
CBB is a series of brief computerized assessments used to evaluate 4 major cognitive domains: psychomotor speed, attention/vigilance, visual learning, and working memory. If the CBB score falls between -10 to +10, it is considered normal. In Cogstate scoring 0 could be considered as performing in the average range, a negative score means below-average performance, and a positive score means above-average performance. A positive change in scores indicates slightly better performance.
Time frame: Day 0, Day 90
CBB Scores of Participants as Compared to the Scores of Age-Matched Controls
CBB is a series of brief computerized assessments used to evaluate 4 major cognitive domains: psychomotor speed, attention/vigilance, visual learning, and working memory. An age-standardized score of ≤-1 on a test will be defined as abnormal. Cognitive impairment on the day of testing will be defined as ≥2 tests with abnormal performance.
Time frame: Day 0, Day 90
Percentage of Participants With Blood Transfusions Affecting Cognition, as Assessed by the Investigator
Time frame: Day 90
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