P0043symptoms in Patients With Autosomal Dominant Polycystic Kidney Disease: Findings From Qualitative Patient Interviews

Abstract

Background and Aims Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) experience various symptoms due to increasing kidney volume and its associated complications. To understand symptom burden, qualitative interviews were conducted to characterize the ADPKD patient experience pertaining to the most common and disturbing symptoms. Method Combined concept elicitation and cognitive debriefing interviews were performed by trained interviewers following a preliminary conceptual model based on a targeted literature review. We currently report on the concept elicitation component, which explored relevant and bothersome symptoms in ADPKD patients, due to either the disease or associated treatments. Nineteen telephone interviews were conducted in four waves of 5, 4, 6, and 4 patients, respectively. Results The mean age of patients was 47 years (SD: 9 years); 68% (n=13) were female, and the mean estimated glomerular filtration rate (eGFR) was 41 ml/min/1.73m2 (SD: 25 ml/min/1.73m2), with the majority 47% (n=9) in stage 3 chronic kidney disease (CKD). The classes of treatments included antihypertensives (74%, n=14), vasopressin receptor antagonists (21%, n=4); diuretics (16%, n=3), analgesics (16%, n=3) and erythropoietin (5%, n=1). The most frequently mentioned symptoms were fatigue (89%, n=17), pain (84%, n=16), and nocturia (74%, n=14); all were considered at least moderately disturbing to patients’ lives. Most patients experienced constant fatigue (74%, n=14) that worsened over time. Patients’ descriptions of pain were variable, including back and side pain, severe cystic pain, and muscle pain; a few patients also mentioned shoulder pain, or pain due to kidney stones. Among patients reporting nocturia (n=14), about half (57%, n=8) rated nocturia on the upper end of the disturbance scale, regardless of number of voids per night, often accompanied by sleep disruption. While less frequently occurring, itchy skin, muscle cramping, abdominal swelling, and inability to eat were spontaneously mentioned among the most disturbing symptoms. Conclusion The novel component of this study is the significant disruption of nocturia in patients with ADPKD even in the absence of vasopressin receptor antagonist administration. In addition, it confirms the findings of the literature review and highlights the patient experience in ADPKD, most notably fatigue, nocturia, and pain as the most frequent and bothersome symptoms experienced by patients. Subjective evaluation of the ADPKD patient experience is critical to understand symptom severity and the burden of disease, to potentially quantify disease progression, and to assess the impact of emerging therapies.