Journal of Patient-Reported Outcomes最新文献

Background: Lived experience involvement in measure development is recognised as best practice. This article is a reflective account, co-written between researchers and PPIE (Patient and Public Involvement and Engagement) consultants with lived experience of psychosis, about the impact of lived experience involvement in developing the self-Dehumanisation in Psychosis Scale (DiPS). Reporting is guided by the Guidance for Reporting Involvement of Patients and the Public 2 - Short Form (GRIPP2-SF).

Methodology: In developing the DiPS, people with lived experience of psychosis contributed both as research participants and as PPIE consultants across multiple stages of development. This included in study document review; identification of domains; item generation, refinement, and amendment; psychometric validation; and dissemination.

Results: Contributions made by people with lived experience were significant across all stages of developing the DiPS, including developing and shaping conceptual understanding of self-dehumanisation; item generation and selection; and improving the comprehensibility and acceptability of the measure.

Discussion: Working collaboratively with people lived experience in measure development can be of benefit both to the validity of the measure, and to those involved. We reflect on the value and importance of working collaboratively, and offer recommendations based on our experiences for researchers co-developing measures.

Background: Chronic inducible cold urticaria (ColdU) is characterized by itchy wheals (hives) with or without angioedema induced by cold exposure. It severely impacts patients' health-related quality of life (HRQoL). Qualitative research describing the burden of ColdU from direct patients' voice remains sparse. The study aims to explore the patients' ColdU experience by identifying signs, symptoms, and associated impacts, and to develop the first conceptual model for ColdU from the patients' perspective.

Methodology: One-to-one telephone concept elicitation (CE) interviews were conducted with patients with ColdU between 2022 and 2023. Participants were asked open-ended questions about their experience of the signs, symptoms and impacts and the most and least bothersome aspects of their disease.

Results: Eight adults and 5 adolescent participants interviewed reported 22 ColdU-related symptoms/signs and 32 impacts. Six children/caregiver dyads and 1 caregiver of a 2-year-old child with ColdU reported 17 ColdU-related symptoms/signs and 19 impacts. Patient experience of symptoms and impacts was similar across all age groups, with hives and itch reported most frequently as the most bothersome symptoms. Similarly, for impacts, all participants reported impairment in activities of daily living (e.g., not taking part in hobbies, wearing warm clothes). Two conceptual models of patients' experience of ColdU (one for adult/adolescent patients and one for pediatric patients) were developed.

Conclusions: This study is the first to report the ColdU experiences directly from patients. It identifies hives and itch as being the core symptoms and describes the debilitating nature of ColdU and its substantial impact on the HRQoL of all age ranges.

Background: Acromegaly is a rare endocrine disease caused by excessive growth hormone (GH) secretion typically due to a pituitary adenoma. Patients ineligible for or with an inadequate response to surgery and/or radiotherapy often require pharmacotherapy such as depot somatostatin receptor ligand (SRL) injections. This study evaluated the current management of patients with acromegaly and real-world experience of disease burden, treatment burden, and healthcare resource utilization (HCRU).

Methodology: A quantitative study was conducted among symptomatic adults with acromegaly receiving therapy, including depot SRL injections. A web-based survey captured the 3-month disease experience including the presence and severity of acromegaly-associated symptoms, treatment experience, HCRU, and impact on the ability to work (Work Productivity and Impairment Questionnaire [WPAI], assessed over prior 7 days).

Results: Among 58 patients who completed the survey, 36 (62.1%) received a depot SRL injection, either as monotherapy (18 [50%]), or in combination with other agents. All patients experienced ≥ 1 symptom during the previous 3 months, with 67% having ≥ 1 symptom with a severity of ≥ 8 on a scale of 0 to 10. Patients reported that acromegaly had a moderate (59%) or high (22%) level of interference in their life overall. Of 18 patients on depot SRL injection monotherapy, 12 (67%) reported ≥ 1 breakthrough acromegaly symptom at any time prior to the next injection. WPAI scores were 51% for daily activity impairment, 38% for presenteeism, 34% for overall work impairment, and 8% for absenteeism. Patients receiving depot SRL injection (monotherapy/combination therapy) were more likely than those not receiving this injection to report moderate-to-high interference of acromegaly treatment with their life (56%/72% vs. 18%) and that their treatment was moderately to highly burdensome (67%/72% vs. 41%). HCRU due to acromegaly treatment included: a mean of 2.6 office visits, 13.8% with ≥ 1 emergency department visit, and 10.3% with ≥ 1 overnight hospitalization. When asked about treatment preferences, 60% of patients preferred oral therapy and 22% injectable mediation; 81% preferred a therapy that can be taken at home.

Conclusions: Despite current pharmacotherapies, patients reported substantial burden due to acromegaly and its treatment, which extends beyond clinical manifestations to impact activities, productivity, and HCRU.

Purpose: The concept of 'score interpretation threshold' for understanding score differences of clinical outcome assessments (COAs) and terminology around this topic have evolved over several decades. Yet, considerable confusion regarding terminology remains, leading to potentially erroneous interpretation of COA results. This article sought to provide an updated overview of terminology and an assessment of trends to explore opportunities for harmonizing the field.

Methods: A targeted literature review was conducted for review articles published 2016-September 2024 discussing terminology related to COA score interpretation thresholds, followed by a review of guidance by regulatory and reimbursement/payer stakeholders for specific terminology in this context. A targeted review of original research articles that were aimed at deriving interpretation thresholds was undertaken, spanning a five-year period (2016- Apr 2021) to explore potential trends regarding use of terms, acronyms, and definitions.

Results: As expected, vast heterogeneity in terminology and definitions was observed across review articles and regulatory/reimbursement/payer guidelines. Across 318 original research articles, 39 different terms were identified, with 'minimal clinically important difference' (MCID) most frequently used (mentioned in 163 articles), which was more than twice as often as the next term ('minimal important difference'; MID), mentioned in 76 articles, followed by 'minimal important change' (MIC), mentioned in 54 articles. Articles also showed great variation in how thresholds were defined, derived, and applied. Frequently, authors failed to provide sufficient details on methods and application, making it difficult to interpret derived thresholds.

Conclusions: COA score interpretation threshold terminology is far from harmonized. Evidence is insufficient to derive specific recommendations on which terms to use. Instead, we present minimum reporting standards for defining thresholds to ensure that they are comprehensible and reproducible, regardless of the specific terms and acronyms used.