Health‐related quality of life assessment in adult haemophilia patients: a systematic review and evaluation of instruments

Abstract

The objective of this study was to review and evaluate the performance of health-related quality of life (HRQL) and other health status measures used in studies of adult haemophilia and provide recommendations for future research. A systematic literature review was performed to identify HRQL and health status measures used in haemophilia. Literature was identified using medical databases, Internet and manual searches. The search was restricted to articles published in English since 1986. Ninety-six abstracts were located; 19 relevant articles were selected for detailed review. Three main types of HRQL measures were identified: generic psychometric-based HRQL (SF-36 and SF-12), utility-based HRQL [EQ-5D and Health Utilities Index (HUI)], and musculoskeletal-specific HRQL (Arthritis Impact Measurement Scale 2, AIMS 2) instruments. No patient-rated haemophilia-specific HRQL instrument was found. The SF-36, the EQ-5D, and the HUI were able to discriminate haemophilia patient subgroups with respect to disease severity and HIV comorbidity status. Sixteen additional scales were identified that were used to measure the different aspects of physical, psychological, and social functioning of patients. There were no clinical studies of haemophilia carried out that employed HRQL instruments, thus responsiveness of these instruments could not be evaluated. The variety of instruments used in haemophilia studies highlights the need for a tool that can capture the full impact of haemophilia and its treatment on patients' HRQL. Developing such a tool poses the unique challenge of accounting for common comorbidities, such as HIV and chronic hepatitis that may have a greater HRQL impact than the underlying disease.

Cross‐cultural development and psychometric evaluation of a patient‐reported health‐related quality of life questionnaire for adults with haemophilia

“…The development of the HAEMO‐QoL‐A was conducted systematically [16] based on a thorough review of published medical literature [17], focus groups with patients, and a focus group and meetings with experts in haematology and outcomes research. The objective was to identify HRQL and symptom domains affected by haemophilia that are relevant to the countries in which the HAEMO‐QoL‐A was evaluated and to develop an instrument covering the main psycho‐social domains in haemophilia for application in clinical trials and other studies.…”

Randomized trial of a DVD intervention to improve readiness to self-manage joint pain

“…The SF-36 was recommended for use in chronic pain samples [3], and has good reliability and validity in painful conditions like rheumatoid arthritis [39]. It is frequently used in hemophilia [16], and has good internal consistency and discriminates well between patients with differing severity of hemophilia [40]. In the present sample (n=108), internal reliabilities at baseline and follow-up respectively were 0.85 and 0.86 for PCS, and 0.85 and 0.86 for MCS.…”

An assessment of the relative influence of pain coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia

“…A two-factor structure (PCS and MCS), internal consistency, and discriminant validity were supported in general population samples [31,40], and the reliability, validity and responsiveness of the PCS and MCS scales were supported in painful conditions like rheumatoid arthritis [35]. The SF-36 was recommended for use in chronic pain samples [2] and is a frequently used measure in hemophilia [10], with good internal consistency and good discrimination between patients with differing hemophilia severity [41].…”