Haemophilia : the official journal of the World Federation of Hemophilia · 2026
A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND-1 Trial in Patients With Severe Haemophilia A.
Клиническое исследование III фазыСтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Msihid Jérôme, Iorio Alfonso, Wilson Amanda, Kragh Nana, Nelson Lauren, Qin Shanshan, Willemze Annemieke
Аннотация
INTRODUCTION: Bleeding episodes can impair physical functioning and cause chronic pain in haemophilia A. The Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) was administered in XTEND-1; the Physical Health (PH) subscale evaluated Physical Health/Functioning.
AIM: To evaluate psychometric properties of Haem-A-QoL PH and estimate clinically meaningful change thresholds using XTEND-1 data (NCT04161495).
METHODS: Previously treated patients (aged ≥12 years) with severe haemophilia A received once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) for 52 weeks (Arm A; pre-study prophylaxis), or on-demand efanesoctocog alfa (50 IU/kg) for 26 weeks followed by 26 weeks once-weekly prophylaxis (Arm B; pre-study on-demand). Psychometric analyses used pooled data (Arms A and B) to confirm test-retest reliability, construct validity, and responsiveness of the Haem-A-QoL PH and total scores. Clinically meaningful change thresholds were estimated using data from Arm A.
RESULTS: The Haem-A-QoL PH items were completed by all patients in the psychometric sample (N = 126) at baseline and 120 patients (95%) at Week 52. Haem-A-QoL PH and Haem-A-QoL total scores demonstrated high internal consistency reliability, construct validity, acceptable test-retest reliability, responsiveness, and discriminated between known groups at baseline and Week 52. The meaningful within-patient change (MWPC) threshold estimate was -10.0 (range -15.0 to -8.7) for Haem-A-QoL PH and -8.5 (-8.5 to -4.6) for Haem-A-QoL total scores (using PGIS-Joint). Respective proposed meaningful within-group improvement thresholds were -6.8 (range -4.8 to -6.8) and -5.8 (-5.8 to -3.5).
CONCLUSIONS: The Haem-A-QoL PH is a reliable, valid, and responsive measure to assess the impact of interventions in the treatment of haemophilia A.
AIM: To evaluate psychometric properties of Haem-A-QoL PH and estimate clinically meaningful change thresholds using XTEND-1 data (NCT04161495).
METHODS: Previously treated patients (aged ≥12 years) with severe haemophilia A received once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) for 52 weeks (Arm A; pre-study prophylaxis), or on-demand efanesoctocog alfa (50 IU/kg) for 26 weeks followed by 26 weeks once-weekly prophylaxis (Arm B; pre-study on-demand). Psychometric analyses used pooled data (Arms A and B) to confirm test-retest reliability, construct validity, and responsiveness of the Haem-A-QoL PH and total scores. Clinically meaningful change thresholds were estimated using data from Arm A.
RESULTS: The Haem-A-QoL PH items were completed by all patients in the psychometric sample (N = 126) at baseline and 120 patients (95%) at Week 52. Haem-A-QoL PH and Haem-A-QoL total scores demonstrated high internal consistency reliability, construct validity, acceptable test-retest reliability, responsiveness, and discriminated between known groups at baseline and Week 52. The meaningful within-patient change (MWPC) threshold estimate was -10.0 (range -15.0 to -8.7) for Haem-A-QoL PH and -8.5 (-8.5 to -4.6) for Haem-A-QoL total scores (using PGIS-Joint). Respective proposed meaningful within-group improvement thresholds were -6.8 (range -4.8 to -6.8) and -5.8 (-5.8 to -3.5).
CONCLUSIONS: The Haem-A-QoL PH is a reliable, valid, and responsive measure to assess the impact of interventions in the treatment of haemophilia A.