Trial reportHaemophilia : the official journal of the World Federation of Hemophilia
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.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04161495 (A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein), which is not on this map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A
Who cites it
1 citing paper in PubMed.
- 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.Haemophilia : the official journal of the World Federation of HemophiliaTrial
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
introductionBleeding 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.
aimTo evaluate psychometric properties of Haem-A-QoL PH and estimate clinically meaningful change thresholds using XTEND-1 data (NCT04161495).
methodsPreviously 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.
resultsThe 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).
conclusionsThe Haem-A-QoL PH is a reliable, valid, and responsive measure to assess the impact of interventions in the treatment of haemophilia A.
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