Health-related quality of life in neuroendocrine neoplasia: a critical review

Abstract

Incidence of neuroendocrine neoplasia (NEN) is increasing, as is use of health-related quality of life (HRQoL) measurement in clinical trials. Following development of validated questionnaires, HRQoL is widely used to assess outcomes. This review is intended for healthcare professionals and is based on a selection of data published in the last decade. HRQoL is on par with other clinical endpoints such as performance status. Assessments in clinical trials have been particularly useful for monitoring the symptom burden of NEN, for the effects of treatments on patients’ lives, and have provided new data allied to the usual clinical endpoints. QoL expressed as quality-adjusted life years (QALYs) have become the most important primary outcome to establish cost-effectiveness in health economic evaluation. From looking at clinical trials over the last 10 years, we see that the quality of HRQoL evidence reported in published studies has improved and, in general, recent studies are likely to be more methodologically robust. Assessment of HRQoL in clinical trials is likely to become a standard part of clinical practice in NEN, as in other cancers. However, clear methods for calculating the clinical meaningfulness of changes in scores are needed. Other limitations of HRQoL measurement include lack of specificity to certain symptom sets and ease of completion and administration. An international group taking a lead on developing HRQoL research specifically in NEN patients is needed to address limitations of the evidence base. In order for greater weight to be placed on HRQoL data, agreement on optimal, validated scoring systems is needed.

Neuroendocrine neoplasms: Consensus on a patient care pathway

“…For example, for people with stage II–III NENs, follow‐up CT scans can be biannual for up to 5 years; for stage IV disease, radiological monitoring can last for many more years 7 . In addition to the physiological and practical challenges, the psychological burden of living with the uncertainty of an NEN diagnosis, both short‐ and long‐term, can be significant 35,39,55,115–117 …”

“…7 In addition to the physiological and practical challenges, the psychological burden of living with the uncertainty of an NEN diagnosis, both short-and longterm, can be significant. 35,39,55,[115][116][117] Barriers to care Inconsistent and inappropriate care: Follow-up care and ongoing treatment are directly affected by barriers to care at earlier stages in the care pathway. As a result, people with NENs often experience a different frequency and quality of follow-up care, largely due to differences in resource availability, funding and numbers of adequately trained personnel.…”

Physician-perceived utility of the EORTC QLQ-GINET21 questionnaire in the treatment of patients with gastrointestinal neuroendocrine tumours: a multicentre, cross-sectional survey (QUALINETS)

“…There is a large and growing body of literature [18][19][20][21] on the role and benefits of condition-specific PROMs, showing that the routine use of such instruments can improve communication between doctors and their patients and help with clinical decision-making. The findings of the present study confirm these benefits for the QLQ-GINET21.…”

Evaluating cost-effectiveness in the management of neuroendocrine neoplasms

“…If the aim is to compare across conditions, health-related quality of life in a clinical setting can be measured using a generic preference-based multi-attribute utility instrument, such as the EQ-5D, and should be valued using an appropriate tariff (e.g., from a time trade-off or standard gamble study). However disease-specific instruments are often more sensitive and responsive for certain health states [63]. Areas where inaccuracy was introduced in the economic evaluations reviewed included making certain assumptions without justification [55], using other disease areas as proxies [55], use of vignette studies which value hypothetical disease states [27] and referencing unpublished data [27].…”