Phm5 Assessment of Health-Related Quality of Life in Patients With Haemophilia With the Newly Developed Haemophilia-Specific Instrument Haem-a-Qol

Measurement properties of the Haem‐A‐QoL in haemophilia clinical trials

“…These baseline results compare favourably with those of the original Haem‐A‐QoL validation that was conducted in the Italian COCHE study, an observational study of 233 adult subjects with haemophilia A and B . In the COCHE study, the only other published validation study of the Haem‐A‐QoL, internal consistency reliability estimates using Cronbach's alpha ranged from 0.74 to 0.88 for the Haem‐A‐QoL domains, and α = 0.96 for the ‘Total Score’.…”

“…Known-group validity: comparison of Haem-A-QoL mean (SD) baseline score by response levels in EQ-5D domains in the A-LONG study. [8,9]. The 6-month A-LONG change score correlations with the EQ-5D were moderate for the Haem-A-QoL 'Total Score,' 'Physical Health' and 'Feelings' domains, demonstrating the sensitivity to change for these outcome measures in the A-LONG data.…”

“…Known‐groups validity was investigated using comparisons of haemophilia severity groups in the original 2005 validation study; however, all subjects in the A‐LONG and B‐LONG studies had severe haemophilia. The Haem‐A‐QoL's responsiveness, or the sensitivity to change over time, was not reported in the COCHE study .…”

“…Outcome measures included patient-reported outcomes for the assessment of HRQoL (Haem-A-QoL) [8,9] and health utility measures [EuroQoL-5 Dimension (EQ-5D)] [11][12][13], as well as the Modified Hemophilia Joint Health Score (mHJHS; modified for use in adults) [14], change in physical activity questionnaire and bleeding assessments (see Data S1 for additional details). In this analysis, once unidirectional item score values were obtained with the Haem-A-QoL, a raw score for each domain of the Haem-A-QoL was calculated by summing up all of the item values, provided that the minimum number of questions had been answered for that domain: 'Physical Health' (5), 'Feelings' (4), 'View of Self' (5), 'Sports and Leisure' (5), 'Work and School' (4), 'Dealing With Haemophilia' (3), 'Treatment' (8), 'Future' (5), 'Family Planning' (4) or 'Partnership and Sexuality' (3). The raw scores were transformed to a 0-100 scale, with higher scores representing higher impairments in HRQoL.…”

Quality of Life in Hemophilia

“…Italian adult hemophilia patients on continuous replacement therapy reported worse QoL compared to patients on on-demand treatment, which can be explained by the deteriorated clinical situation of these patients [105]. Up to 64% of the variance of HRQoL in German adult patients assessed with the generic SF-36 and the hemophilia-specific Haem-A-QoL could be explained by physical performance measured with the HEP-Test-Q [106] indicating that physical activity and sports is beneficial for HRQoL [107].…”