Symptom Burden at the End of Life for Neuroendocrine Tumors: An Analysis of 2579 Prospectively Collected Patient-Reported Outcomes.

BACKGROUND: Given a slow course of disease, end-of-life issues are understudied in neuroendocrine tumors (NETs). To date, there are no data regarding symptoms at the end of life. This study examined symptom trajectories and factors associated with high symptom burden in NETs at the end of life. METHODS: We conducted a retrospective cohort study of NET patients diagnosed from 2004 to 2015 and who died between 2007 and 2016, in Ontario, Canada. Prospectively collected patient-reported Edmonton Symptom Assessment System scores were linked to administrative healthcare datasets. Moderate-to-severe symptom scores (≥ 4 out of 10) in the 6 months before death were analyzed, with multivariable modified Poisson regression identifying factors associated with moderate-to-severe symptoms scores. RESULTS: Among 677 NET decedents, 2579 symptom assessments were recorded. Overall, moderate-to-severe scores were most common for tiredness (86%), wellbeing (81%), lack of appetite (75%), and drowsiness (68%), with these proportions increasing as death approached. For symptoms of lack of appetite, drowsiness, and shortness of breath, the increase was steepest in the 8 weeks before death. On multivariable analyses, the risk of moderate-to-severe symptoms was significantly higher in the last 2 months before death and for patients with shorter survival (< 6 months). Women had higher risks of anxiety, nausea, and pain. CONCLUSION: A high prevalence of moderate-to-severe symptoms was observed for NETs at the end of life, not previously described. The proportion of moderate-to-severe symptoms increases steeply as death nears, highlighting an opportunity for improved management. Combined with identified factors associated with moderate-to-severe symptoms, this information is important to improve patient-centred and personalized supportive care for NETs at the end of life.

What Outcomes are Important for Gout Patients? In-Depth Qualitative Research into the Gout Patient Experience to Determine Optimal Endpoints for Evaluating Therapeutic Interventions.

BACKGROUND AND OBJECTIVES: Characterized by sudden onset of severe joint pain, swelling, redness, and tenderness to touch, gout 'flare ups' have a substantial impact on quality of life (QoL). This research employed a patient-centered approach to explore the symptoms and impacts of gout, and assess the content validity of existing patient-reported outcomes (PROs). METHODS: Qualitative interviews were conducted with 30 US gout patients (non-tophaceous: n = 20, tophaceous: n = 10) and five expert rheumatologists. Each interview included both concept elicitation (CE) questioning to learn about the patient experience and cognitive debriefing to assess the content validity of three PRO instruments (HAQ-DI, GAQ, and TIQ-20). Nine of the patients provided further real-time qualitative data through a smart phone application. All qualitative data were subject to thematic analysis using Atlas.ti. Two patient advisors and three expert clinicians were engaged as advisors at key stages throughout the research. RESULTS: Interview and real-time data identified the same core symptoms and proximal impact concepts. Severe pain (typically in joints of extremities) was described as the cardinal symptom, often accompanied by swelling, redness, heat, sensitivity to touch, and stiffness. Domains of QoL impacted included physical functioning, sleep, daily activities, and work. The PRO instruments were generally well-understood by patients, but each included items with questionable relevance to at least some of the sample, dependent on the specific joints affected. CONCLUSIONS: Gout patients experience severe pain in affected joints, resulting in substantial limitations in physical functioning. Both the HAQ-DI and the TIQ-20 are useful for specific research purposes in the gout population, although modifications are recommended.

A Longitudinal Approach to Understanding the Relationship Between Symptom Status and QOL.

This study demonstrates how a variant of growth curve modeling known as longitudinal parallel-process modeling can yield an understanding of the effect of symptoms on quality of life (QOL). A two-level hierarchical linear model with random intercepts and slopes was implemented within a structural equation modeling approach. The data (N = 367) comes from a large database of persons with HIV-associated illness. Twenty-three symptoms based on the Sign and Symptom Checklist for Persons with HIV disease and items measuring QOL from the general health status scales were used. Each respondent completed from 1 to 11 questionnaires. The number of reported symptoms had a significant association with patient QOL over time. These findings suggest that appropriate symptom management has the potential to improve patient QOL. This study demonstrates how a state-of-the-art longitudinal modeling technique evaluates the relationship between concurrent rates of change in measurements of two relevant variables.