Methodology and value of measuring symptoms in dialysis patients: case study of chronic kidney disease associated pruritus

People on haemodialysis (HD) experience significant symptom burden and effects on health-related quality of life (HRQoL). Chronic Kidney Disease associated pruritus (CKD-aP) is one of the common symptoms reported by HD patients. In the UK, patient reported outcome measures (PROMs) are not routinely measured in HD populations. Neglecting to assess and manage symptoms in this cohort, notably CKD-aP, has been shown to prolonged suffering, increased healthcare utilisation, and higher healthcare costs, significantly impacting patients' overall HRQoL and healthcare outcomes.
Hence, this thesis examines these aspects through primary data analysis, a mapping study involving established PROMs for CKD-aP, as well as secondary data analysis from an observational longitudinal study, the SHAREHD trial.
The first manuscript highlights the significance of standardising the timing of symptom assessment measures to accurately capture their dynamics and impact on PROMs. The second manuscript explores the symptoms trajectory in prevalent HD patients, emphasising the importance of considering symptom improvements with standard care in clinical trial design. The third study suggests that pruritus instruments should include additional domains beyond severity to align more closely with generic HRQoL measures such as EQ-5D. The fourth manuscript assesses the cost-effectiveness of Difelikefalin, offering its value in addressing the disease burden of CKD-aP among people on HD and the 5th manuscript explores the economic dimensions of CKD-aP, revealing significant healthcare costs and unmet need.
The overall recommendation resulting from this work emphasises the importance for healthcare practitioners and researchers to use standardised measurement methods and comprehensive assessments when dealing with symptoms in people on HD, particularly CKD-aP. Understanding how symptoms evolve over time and how they interact with economic factors and HRQoL is crucial for optimising patient care and resource allocation. Understanding cost effectiveness and unmet needs in terms of symptoms management enables more effective healthcare interventions to enhance HRQoL and allocate resources efficiently among diverse populations.