Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy.
Jennifer QuinnKathleen A DelaneyWing Yen WongWolfgang A MiesbachMonika BullingerPublished in: Patient related outcome measures (2022)
Our preliminary results suggest that the Haemo-QOL-A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.