Development and Pilot testing of a Disease-Specific Quality of Life Questionnaire for Adult Patients with Haemophilia (Haem-A-QoL).

Background: Haemophilia and its treatment influence the every-day-life of patients and impact on their quality of life. For the adequate assessment of quality of life validated instruments are necessary. While haemophilia-specific questionnaires for children are existing (v. Mackensen et al., 2004; Young et al., 2004), up to now no validated instrument for adults is available. Currently some developmental works are on-going in order to develop such an instrument as well for adults. In a multi-centre validation study a health-related quality of life questionnaire for adults with haemophilia was developed and pilot tested in Italy.

Aim: This study was designed to develop and validate a haemophilia-specific quality of life measurement for adults (Haem-A-QoL) in Italy.

Methods: The study consisted of three phases: a) a qualitative study including focus groups (with patients and physicians), item formulation and expert ratings of a catalogue of items by patients and physicians, b) a psychometric study including cognitive debriefing, and feasibility testing and c) data analysis. The qualitative study has been performed by the University of Hamburg in cooperation with the University of Milan.

Results: Focus groups with semi-structured interviews were performed in 32 patients from 6 Italian haemophilia centres (Milan, Perugia, Genova, Bari, Castelfranco, Parma). 14 physicians and 8 nurses were asked the same questions. Patients and physicians/nurses consider similar dimensions important for quality of life. But the order of importance is different for patients (physical health, social interaction, dependence, future, work) and physicians/nurses (dependence, work, social interaction, treatment, physical health). Based on the focus group results a draft version of the questionnaire was formulated and given to patients and physicians in order to evaluate the items concerning importance for haemophilia and comprehensibility. The draft version consisted of 159 items. After expert evaluation the questionnaire was revised: several items could be eliminated and some were reworded. The revised version of the Haem-A-QoL consisting of 50 items and was psychometrically tested in 10 Italian centres. The pilot testing showed excellent psychometric characteristics in terms of reliability and validity (convergent, discriminant).

Conclusions: The development of a disease-specific quality of life instrument requires multiple steps and the involvement of different experts (patients, physicians, nurses) in order to have a standardised and valid questionnaire. The Haem-A-QoL is well-accepted by adult haemophiliacs and proved to be a valid and reliable disease-specific self-report measurement for the assessment of quality of life in haemophiliacs. In a next step the Haem-A-QoL will be applied in clinical trials to evaluate patient perceptions of different treatment regimens in haemophilia.