Cross‐cultural adaptation and linguistic validation of age‐group‐specific haemophilia patient‐reported outcome (PRO) instruments for patients and parents

Abstract

Currently, haemophilia care aims to provide the best possible quality of life for individuals living with this chronic disease. Many factors are known to influence treatment adherence, including treatment satisfaction. Health-related quality of life (HRQoL) and treatment satisfaction are, therefore, important outcomes in clinical trials and clinical practice. As individuals' perception of their well-being often differs from that of their physician, it is recommended that self-report instruments are used to assess patient-reported outcomes (PROs). The way that the impact of haemophilia is perceived by the patient and their family can be different, so it is important to assess how parents perceive the impact on their children. A series of PRO instruments have been developed, adapted to different age groups and parents of patients with haemophilia. To allow the instruments to be used internationally, culturally adapted and linguistically validated translations have been developed; some instruments have been translated into 61 languages. Here, we report the process used for cultural adaptation of the Haemo-QoL, Haem-A-QoL and Hemo-Sat into 28 languages. Equivalent concepts for 22 items that were difficult to adapt culturally for particular languages were identified and classed as semantic/conceptual (17 items), cultural (three items), idiomatic (one item), and grammatical (one item) problems. This has resulted in linguistically validated versions of these instruments, which can be used to assess HRQoL and treatment satisfaction in clinical trials and clinical practice. They will provide new insights into areas of haemophilia that remain poorly understood today.

Estimates of utility weights in hemophilia: implications for cost-utility analysis of clotting factor prophylaxis

“…As seen in Table 4, researchers from Germany developed hemophilia-specific HRQL questionnaires for children and adolescents [85] (Haemo-QoL) and for adults (Haem-A-QoL) [86]. Culturally adapted and linguistically validated translations were developed so that these instruments could be used internationally [86].…”

“…Culturally adapted and linguistically validated translations were developed so that these instruments could be used internationally [86]. Another disease-specific instrument, the Haemo-QoL-A, has been shown to discriminate significantly between adults with hemophilia by severity and HIV status, and the physical functioning subscale to discriminate between patients receiving prophylactic or on-demand therapy [87].…”

Quality of Life in Hemophilia

“…The Haemo-QoL is available as a self-administered questionnaire for children of three age groups (4-7, 8-12, and 13-16 years of age) as well as respective proxy versions for their parents. The Haemo-QoL has been translated into several languages [44,45] and has been linguistically validated in 34 languages [46], which can be downloaded from the Haemo-QoL website (www.haemoqol.org). The psychometric structure of the questionnaire showed acceptable psychometric properties for the three age group versions as well as the accompanying parent forms.…”

Adolescent’s subjective perceptions of chronic disease and related psychosocial factors: highlights from an outpatient context study

“…This choice is in agreement with previous studies pointing out the need to address limitations in ordinary activities of chronically ill adolescents [38], the need to focus less on diagnostic categories (where more variability exists) and more on psychosocial dimensions and on the effects that the chronic disease might have on the social activities/socialization and emotional health [1]. In addition, considering that QoL/HRQoL and psychosocial dimensions are related to the individual’s perceptions on the impact of diseases, the literature recommends to focus on self-reported instruments for a better measurement [38, 39] and the WHO guidelines [40] also emphasize its use whenever possible.…”