PUK37 - USE OF HEALTH-RELATED QUALITY OF LIFE (HRQOL) INSTRUMENTS IN CLINICAL TRIALS OF ANAEMIA OF CHRONIC KIDNEY DISEASE (CKD)

Abstract

Anaemia is common in patients with chronic kidney disease (CKD). Symptoms include fatigue, weakness, dyspnoea, dizziness, headaches, pallor, and decreased mental acuity, which could impact on health-related quality of life (HRQoL). This research identifies instruments used to measure HRQoL in anaemia of CKD. A targeted search of PubMed, Embase, and clinicaltrials.gov was performed to identify key phase III clinical trials (defined as those used as evidence in EMA/FDA submissions, HTAs from the EU5/The Netherlands/Sweden, or clinical guidelines) and phase III trials of treatments in development for anaemia of CKD from January 2003 to March 2018. Thirty-four completed key phase III clinical trials were identified, of which nine collected HRQoL data. Thirty-five phase III trials of treatments in development (all hypoxia-inducible factor [HIF] stabilisers) were identified, of which 20 measure HRQoL. Five generic instruments were identified; the most commonly used were the 36-item Short Form (SF-36; seven key trials; 16 trials of HIF stabilisers) and EuroQol Five-Dimensions questionnaires (EQ-5D; 16 trials of HIF stabilisers). Four disease-relevant instruments were used, among them the Functional Assessment of Cancer Therapy-Anaemia (FACT-An; 11 trials of HIF stabilisers) and Kidney Disease Quality of Life questionnaires (KDQOL; two key trials). One disease-specific instrument, the Chronic Kidney Disease-Anaemia Symptoms questionnaire (CKD-AQ), is being used in four trials of HIF stabilisers but is not publicly available for review. The energy and physical activity domains covered in generic instruments may be particularly relevant to the anaemia symptoms of fatigue, weakness, and dyspnoea. The pain and mental/emotional well-being domains are also of potential interest. Symptoms such as headaches, dizziness, and pallor are only captured by disease-relevant instruments. In the absence of a validated anaemia of CKD-specific instrument, a combination of generic and disease-specific instruments for related conditions may allow the most accurate valuation of HRQoL change.