International cross‐cultural validation study of the Canadian Haemophilia Outcomes: Kids’ Life Assessment Tool

Abstract

Health-related quality of life (HRQoL) assessment is recognized as an important outcome in the evaluation of different therapeutic regimens for persons with haemophilia. The Canadian Haemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) is a disease-specific measure of HRQoL for 4 to 18-year-old boys with haemophilia. The purpose of this study was to extend this disease-specific, child-centric, outcome measure for use in international clinical trials. We adapted the North American English CHO-KLAT version for use in five countries: France, Germany, the Netherlands, Spain and the United Kingdom (UK). The process included four stages: (i) translation; (ii) cognitive debriefing; (iii) validity assessment relative to the PedsQL (generic) and the Haemo-QoL (disease-specific) and (iv) assessment of inter and intra-rater reliability. Cognitive debriefing was performed in 57 boys (mean age 11.4 years), validation was performed in 144 boys (mean age 11.0 years) and reliability was assessed for a subgroup of 64 boys (mean age 12.0 years). Parents also participated. The mean scores reported by the boys were high: CHO-KLAT 77.0 (SD = 11.2); PedsQL 83.8 (SD = 11.9) and Haemo-QoL 79.6 (SD = 11.5). Correlations between the CHO-KLAT and PedsQL ranged from 0.63 in Germany to 0.39 in the Netherlands and Spain. Test-retest reliability (concordance) for child self-report was 0.67. Child-parent concordance was slightly lower at 0.57. The CHO-KLAT has been fully culturally adapted and validated for use in five different languages and cultures (in England, the Netherlands, France, Germany and Spain) where treatment is readily available either on demand or as prophylaxis.

The Jamaican Haemophilia Registry: Describing the burden of disease

“…The mean scores of the generic HRQoL scores were significantly lower than the reported population normal values in various countries, indicating that haemophilia has a large impact on HRQoL in Jamaica. The mean CHO‐KLAT and Haemo‐QoL‐A scores of 58.5 and 62.5, respectively, are significantly lower than those from countries where prophylaxis is the standard of care: mean CHO‐KLAT score of 75.4 in Canada and 77.0 in European countries; and mean Haemo‐QoL‐A score of 85.6 in the USA . This indicates that there is a large and significant gap in HRQoL between PWH in countries with different access to clotting factor concentrates.…”

Relevance of the Aboriginal Children’s Health and Well-being Measure Beyond Wiikwemkoong

“…A similar process had previously been used by members of this team (NLY, TAB) in other contexts 18,19,24 . One important alteration in the process was implemented: there was no requirement to come to consensus; rather, each community was permitted to modify the measure to fit the needs of their children.…”

Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era

“…The HaemoQoL-A (a self-report questionnaire for adults) and the HaemoQoL (a self-report questionnaire for children) have both demonstrated good internal consistency, but only the adult version meets the US Food and Drug Administration (FDA) criterion of using direct input from patients to generate item content [3,34]. The CHO-KLAT [35] is validated for use in five different languages and cultures [36], but also does not meet the FDA criterion for direct patient input during item development [37]. Inconsistencies have been observed in the use of some response options [3,32] and, as commonly observed in a paediatric population, parent-proxy results were more reproducible than the children's results [36].…”

“…The CHO-KLAT [35] is validated for use in five different languages and cultures [36], but also does not meet the FDA criterion for direct patient input during item development [37]. Inconsistencies have been observed in the use of some response options [3,32] and, as commonly observed in a paediatric population, parent-proxy results were more reproducible than the children's results [36]. In addition, there is also the Hemofilia-QoL, a 36-item questionnaire that has demonstrated acceptable reliability and adequate convergent validity with the SF-36 Health Survey [31].…”