Journal of Patient-Reported Outcomes最新文献

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.

Objective: To translate and validate Hebrew versions of two patient-reported outcome measure questionnaires: the Self-Reported Mini Olfactory Questionnaire (Self-MOQ) and the Brief Questionnaire of Olfactory Disorders (Brief-QOD).

Methods: A forward-backward translation process was conducted for both questionnaires. All participants rated their sense of smell using a general Visual Analog Scale (VAS) ranging from 0 (no dysfunction) to 10 (severe dysfunction). The patient group completed the questionnaires and the SNOT-22 questionnaire once. A control group of healthy participants completed the questionnaires twice to evaluate test-retest reliability. Subsets of both groups took the Sniffin' Sticks test.

Results: The translation process resulted in Hebrew versions deemed clear and culturally appropriate. A total of 91 individuals were enrolled in the control group and 62 in the patient group. The Hebrew versions of the Self-MOQ and Brief-QOD demonstrated high internal consistency (Cronbach's α of 0.79-0.94 in the full sample) and overall test-retest reliability. The patient group had higher scores than the control group across all measures (p < 0.001). Logistic regression indicated that the Self-MOQ was the strongest predictor of group membership, while the Brief-QOD QOL and Visual Analog Scale components also significantly contributed. Receiver operating characteristic curve analysis identified an optimal Self-MOQ cutoff score of ≥ 2 for distinguishing patients from controls, with excellent accuracy (AUC = 0.97).

Conclusions: The Hebrew Self-MOQ and Brief-QOD are reliable and valid tools for assessing olfactory dysfunction in the Hebrew-speaking population. Future research should evaluate questionnaire results after clinical interventions.

Background: Comprehensive research on the specific factors affecting the quality of life (QOL) of patients with colorectal cancer (CRC) is lacking. We investigated patient factors affecting QOL in this population.

Methodology: We conducted a cross-sectional study at Dr. Suliman Fakeeh Hospital, a private tertiary center in Jeddah City, KSA. We used the validated Arabic versions of the Cancer Quality of Life Questionnaire-30 (QLQ-30) and Colorectal Cancer Quality of Life Questionnaire-29 (QLQ-CR29) in patients with CRC. We used a generalized linear model to analyze the data. Significant linear correlations between functional and symptom scores and 11 factors, including age, sex, tumor site and stage, comorbidities, surgery type/approach, leaks, stoma, and neoadjuvant or adjuvant therapy, were detected.

Results: A total of 104 participants answered the survey. The mean global health score was 72.43 (95% confidence interval [CI] 68.18-76.52). Cognitive and physical function scored highest and lowest on the QLQ-30, with a mean of 76.92 (95% CI 71.47-81.73) and 24.92 (95% CI 65.57-74.61), respectively. The worst symptom was insomnia, with a mean of 42.30 (95% CI 36.21-48.71). The most distressing symptoms on the QLQ-CR29 were stoma embarrassment and bloating, with a mean of 41.34 (95% CI 24.67-58.01). Tumor stage and stoma were the two most significant factors for predicting poor QOL. The factors influencing impotence and male libido included age, tumor stage, comorbidities, and the use of chemoradiotherapy.

Conclusions: A one-unit increase in tumor stage was correlated with a 4.88-unit decrease in the global health status score, whereas a stoma was linked to a 22-fold reduction in social function scores. Advanced tumor stage and the presence of a stoma significantly affected patients' functional status and symptoms.