Journal of Patient-Reported Outcomes最新文献

Background: Health utility values are required for cost-utility analyses in breast cancer, yet EQ-5D-5L-based health utility estimates and validity evidence across clinically relevant health states remain limited. The objectives of this study were to estimate health utility values for pre-defined breast cancer health states using the EQ-5D-5L (EuroQoL 5-level) instrument and to investigate its construct validity in breast cancer.

Methods: This cross-sectional study included women with invasive breast cancer, who completed both EQ-5D-5L and the Edmonton Symptom Assessment System. Participants were classified into five pre-defined health states, considered relevant both to clinical practice and economic modeling. We used the Canadian EQ-5D-5L value set to calculate community-valued health utility scores for each health state. Additionally, we evaluated aspects of construct validity (known-group and convergent validity) of EQ-5D-5L in breast cancer.

Results: 549 women were included; the mean age was 57 (SD 12) years. The mean EQ-5D-5L index score was 0.83 (SD 0.13; range 0.13 to 0.95), with a distribution skewed towards full health and a ceiling effect of 20%. The mean health utility value for early-stage breast cancer was 0.84 (95% CI 0.83-0.86) and for metastatic breast cancer was 0.78 (95% CI 0.76-0.81). This difference was 0.060 (lower 95% CI bound 0.036, slightly lower than the pre-specified minimum important difference of 0.037). Health utility values and ESAS scores met almost all pre-specified criteria for convergent validity.

Conclusion: We generated a list of health utility values for five pre-defined breast cancer health states using EQ-5D-5L. Additional research is required to confirm the validity of EQ-5D-5L as an outcome measure in breast cancer.

Objective: Financial toxicity, characterized by the financial burden and psychological distress associated with cancer treatment, has become a critical issue impacting patient outcomes and quality of life. This study aimed to conduct an updated and comprehensive systematic review to evaluate the methodological quality and measurement properties of financial toxicity measurement instruments for cancer patients, thereby guiding the selection of high-quality tools for future empirical research.

Methods: Literature on financial toxicity measurement instruments for cancer patients was retrieved from CNKI, Wanfang, VIP, SinoMed, PubMed, Web of Science, CINAHL, Scopus, and Embase databases, with a search range from the inception up to April 16, 2025. Two researchers independently screened the literature and extracted data. The included studies were assessed using the Consensus-Based Standards for the Selection of Health Measurement Instruments (COSMIN), and recommendations were formulated based on the quality of evidence and measurement properties.

Results: A total of eighteen studies were included, involving eight financial toxicity measurement instruments for cancer patients. None of the studies reported measurement error. The Comprehensive Score for Financial Toxicity (COST), Financial Index of Toxicity (FIT), and Subjective Financial Distress Questionnaire (SFDQ) were provisionally recommended for use (Category B), while the other five instruments were not recommended due to insufficient evidence (Category C).

Conclusion: The COST is provisionally recommended for measuring financial toxicity in cancer patients. The methodological quality and measurement properties of existing financial toxicity instruments still require further improvement. Future research should adhere strictly to the COSMIN guideline to validate existing tools or develop new, high-quality instruments with comprehensive evaluations of their measurement properties.

Introduction: Acromegaly is a rare disease caused by a pituitary adenoma in most cases. Physical changes, metabolic disturbances, osteoarticular symptoms, as well as psychological and social repercussions reduce the Quality of Life (QoL) of people suffering from the disease.

Objective: To assess both generic and disease-specific instruments employed to capture the influence of acromegaly on patients' QoL.

Methods and analysis: This scoping review assessed the different tools used to evaluate QoL in people with acromegaly. This review followed the methods proposed by the Joanna Briggs Institute and is reported following the Preferred Reporting Items for Systematic Reviews manual and Meta-Analyses extension for Scoping Reviews.

Results: Of a total of 198 studies, 30 questionnaires were identified and categorized by type (specific or generic) and number of domains assessed (physical health, psychological state, autonomy, and social relationships). Among these, 3 were specific-multidomain, 18 generic-multidomain, 3 specific-unidomain, 5 generic-unidomain, and only 1 had no domains. All were patient-reported outcome measures (PROM) used in various contexts. Optimal instruments were disease-specific, multidimensional, brief, easily administered, available in multiple languages, with psychometric validation in acromegaly patients and cross-cultural validation, and assessing treatment impact on QoL.

Conclusions: This review explores the use of PROMs in assessing HRQoL in acromegaly patients, emphasizing the need for specific, validated, and culturally adapted tools addressing symptoms and treatment impact. We propose a novel domain-based classification to overcome existing limitations of instruments and improve their presentation in future studies. This study provides a synthesis that helps advance research and guide clinical practices around HRQoL in acromegaly management.