Importance of responder criteria for reporting health-related quality-of-life data in clinical trials for advanced cancer: recommendations of Common Sense Oncology and the European Organisation for Research and Treatment of Cancer.

The goals of treatment for people with advanced cancer are to prolong survival and improve symptoms and health-related quality of life (HRQOL). Although many phase 3 randomised clinical trials seek to evaluate HRQOL during treatment, informing individual patients about expected HRQOL outcomes is challenging, as the common method of analysis and reporting compares averages for randomised groups, and clinicians find these data difficult to apply in clinical practice. Symptomatic patients with advanced cancer would like to know the probability that a proposed treatment might improve their survival or their dominant symptoms, and the probability of having treatment-related side-effects.

The EORTC QLQ-F17 as a shortened version of the EORTC QLQ-C30 to assess self-reported functioning in cancer patients: investigating equivalence and psychometric properties in a randomized cross-over trial.

Background: The EORTC QLQ-F17, a shortened version of the EORTC QLQ-C30, solely contains the items related to functioning and omits symptom scales. The QLQ-F17 is conceived an equivalent to the functional part of the QLQ-C30, but to date there is no empirical evidence to support this rationale.

Methods: This randomized, cross-over, multi-national, questionnaire-based study investigates the equivalence and psychometric properties of the QLQ-F17 compared to the functional scales of the QLQ-C30.

Adherence to daily electronic symptom screening in pediatric cancer: associations with time, location and pain.

Purpose: Regularly collected patient-reported outcome measures (PROMs) can facilitate early symptom detection and improve health outcomes. This explorative analysis aimed to investigate PROM adherence and factors associated with daily PROM completion among pediatric cancer patients.

Methods: We analyzed data from a prospective study at the Medical University of Innsbruck in which pediatric patients with cancer treated with chemotherapy completed daily PROMs via a web-based portal (ePROtect).

Assessing frailty in patients with relapsed/refractory multiple myeloma: A comparison between the patient-reported frailty phenotype and the International Myeloma Working Group frailty index.

Introduction: The International Myeloma Working Group Frailty Index (IMWG FI) is one of the most used frailty assessment tools in patients with multiple myeloma (MM). A patient-centered frailty tool based on patient-reported outcomes (PROs) has been recently proposed for patients with relapsed/refractory MM (RRMM): the Patient-Reported Frailty Phenotype (PRFP). This cross-sectional analysis aimed to replicate the PRFP within a real-world setting and to describe health-related quality of life (HRQoL) profiles based on this new patient-centered frailty classification.

The current use and application of thresholds for clinical importance of the EORTC QLQ-C30, the EORTC CAT core and the EORTC QLQ-C15-PAL- a systematic scoping review.

Background: Thresholds for clinical importance (TCIs) were previously established for the cancer-specific patient reported outcome (PRO) measures EORTC QLQ-C30, EORTC QLQ CAT Core, and EORTC QLQ-C15-PAL. TCIs aim to aid the interpretation of scores for individual patients at a single point in time. They intend to indicate whether a symptom or functional health limitation is of clinical relevance, i.

Development and validation of crosswalks between the EORTC QLQ-C30 physical, role, social and emotional functioning, fatigue and global health status/quality of life scales and their corresponding PROMIS® scales.

Objectives: Across patient-reported outcome measures (PROMs), the European Organization of Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) is one of the most widely used among patients with cancer. Patients may also complete other PROMs such as forms of the Patient-Reported Outcomes Measurement Information System (PROMIS®). The objective of this study was to develop crosswalks between the EORTC QLQ-C30 physical, role, social and emotional functioning, fatigue and global health status/quality of life (QoL) scales and their corresponding PROMIS® scales.

Cancer-Specificity of Sex Differences and Sex-Specific Impact of Sociodemographic Characteristics in Cancer Patients' Health-Related Quality of Life: A Cross-Sectional Study.

Background: Health-related quality of life (HRQoL) is a multi-dimensional concept commonly assessed in patient-centered research on the impact of cancer and its treatment. Apart from disease and treatment characteristics, HRQoL is also influenced by sociodemographic variables. However, detailed evaluations of the impact of sociodemographic variables on HRQoL are scarce.

Flexibility in patient-reported outcome and health-related quality of life measurement: The EORTC Quality of Life Group measurement strategy.

The development of the first European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group (QLG) health-related quality of life (HRQoL) questionnaires contributed to the systematic uptake of HRQoL as an endpoint in cancer clinical trials, and to the measurement of HRQoL for individual assessment in routine care. Following a modular approach, these patient-reported outcome (PRO) measures (PROMs) ensure that both generic and disease-specific issues are assessed, enabling comparison of PROs across groups and studies. The application of a comprehensive and continually refined methodology for developing and updating these PROMs has been crucial in supporting their psychometric and cross-cultural validity, and their continued implementation in clinical research.

Using prior information to individualize start item selection when assessing physical functioning with the EORTC CAT Core.

Background: Computerized adaptive test (CAT) provides individualized measurement, using the patient's previous responses to select the next most informative item. However, the first item, the start item, is usually not individualized as no score estimate is available a priori. The European Organisation for Research and Treatment of Cancer (EORTC) CAT Core covers 15 health-related quality of life domains.

Patient-reported outcomes in randomized controlled trials evaluating BRAF inhibitors in patients with cutaneous melanoma: a systematic scoping review of quality of reporting and trial results.

The objective of this study was to provide an overview of the current practice of patient-reported outcome (PRO) assessments in trials investigating treatment with BRAF inhibitors in patients with advanced melanomas. In addition, we extracted information on symptomatic adverse events (AEs) reported by clinicians to inform future PRO measurement strategies. For our systematic scoping review, we investigated randomized controlled trials (RCTs) evaluating treatment with BRAF inhibitors that had a primary, secondary or exploratory PRO endpoint and were indexed on PubMed.

Patient-reported outcome domains in multiple myeloma randomized controlled trials and association with survival outcomes.

Patient-reported outcomes (PROs) are crucial endpoints in multiple myeloma (MM) randomized controlled trials (RCTs), yet there is significant variability in their methodology and reporting. Our study aimed to (a) identify the most commonly pre-specified PRO domains in MM RCTs and those most responsive to modern therapies, and (b) examine the association between PROs and progression-free survival (PFS)/overall survival (OS). We performed a systematic review of MM RCTs that used EORTC QLQ-C30 and published between 01/2014-06/2023.

Comparing the contents of patient-reported outcome measures for fatigue: EORTC CAT Core, EORTC QLQ-C30, EORTC QLQ-FA12, FACIT, PRO-CTCAE, PROMIS, Brief Fatigue Inventory, Multidimensional Fatigue Inventory, and Piper Fatigue Scale.

Background: To assess fatigue in cancer patients, several patient-reported outcome measures (PROMs) are available that differ in content. To support the selection of suitable measures for specific applications and to evaluate possibilities of quantitative linking, the present study provides a content comparison of common fatigue measures, scales, and item banks. We included the EORTC CAT Core, EORTC QLQ-FA12, EORTC QLQ-C30, FACIT-F, PROMIS Fatigue (Cancer item bank v1.

Data collection methods for patient-reported outcome measures in cancer randomised controlled trials: a protocol for a rapid scoping review.

Background: There are different modes and ways to assess patient-reported outcomes (PROs) in clinical trials. However, there is little systematic information on how often different modes of assessment (MOA) are used in cancer clinical trials and how exactly assessments are conducted. The goal of this scoping review is to gain an understanding of the MOA and data management of PROs in cancer randomised controlled trials (RCTs) and the reporting quality thereof.

Toward a more patient-centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS).

Notable treatment advances have been made in recent years for patients with myelodysplastic syndromes/neoplasms (MDS), and several new drugs are under development. For example, the emerging availability of oral MDS therapies holds the promise of improving patients' health-related quality of life (HRQoL). Within this rapidly evolving landscape, the inclusion of HRQoL and other patient-reported outcomes (PROs) is critical to inform the benefit/risk assessment of new therapies or to assess whether patients live longer and better, for what will likely remain a largely incurable disease.

Financial toxicity and health-related quality of life in long-term survivors of acute promyelocytic leukaemia.

Objectives: We aimed to investigate the association between financial toxicity (FT) and the health-related quality of life profile of long-term survivors of acute promyelocytic leukaemia (APL) treated within a universal healthcare system.

Methods: We evaluated FT using the financial difficulties item of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). We also compared the prevalence of clinically important problems and symptoms between the survivors of APL with or without FT, using evidence-based thresholds for the EORTC QLQ-C30.

General population normative values for the EORTC QLQ-C30 by age, sex, and health condition for the French general population.

Background: General population normative values for the widely used health-related quality of life (HRQoL) measure EORTC QLQ-C30 support the interpretation of trial results and HRQoL of patients in clinical practice. Here, we provide sex-, age- and health condition-specific normative values for the EORTC QLQ-C30 in the French general population.

Methods: French general population data was collected in an international EORTC project.

Development of standard computerised adaptive test (CAT) settings for the EORTC CAT Core.

Aims: Computerised adaptive test (CAT) provides individualised patient reported outcome measurement while retaining direct comparability of scores across patients and studies. Optimal CAT measurement requires an appropriate CAT-setting, the set of criteria defining the CAT including start item, item selection criterion, and stop criterion. The European Organisation for Research and Treatment of Cancer (EORTC) CAT Core allows for assessing the 14 functional and symptom domains covered by the EORTC QLQ-C30 questionnaire.

Financial Toxicity and Health-Related Quality of Life Profile of Patients With Hematologic Malignancies Treated in a Universal Health Care System.

Purpose: We investigated the association of financial toxicity (FT) with the health-related quality of life (HRQoL) profile of patients with hematologic malignancies treated in a universal health care system.

Methods: We did a secondary analysis of six multicenter studies enrolling patients with hematologic malignancies. FT was evaluated using the financial difficulties item of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30).

Equipercentile equating of scores from common patient-reported outcome measures of physical function in patients with cancer.

Objective: To provide equipercentile equating of physical function (PF) scores from frequently used patient-reported outcome measures (PROMs) in cancer patients to facilitate data pooling and comparisons.

Study Design And Setting: Adult cancer patients from five European countries completed the European Organization for Research and Treatment of Cancer (EORTC) computer adaptive test (CAT) Core, EORTC Quality of Life Questionnaire Version 3.0 (QLQ-C30), Functional Assessment of Cancer Therapy - General (FACT-G), 36-item Short Form Health Survey (SF-36), and the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function 20a short form.

Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation.

Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-TM). However, little data are available regarding patients and their parents' health-related quality of life (HRQoL) after the procedure. As such, we investigated the HRQoL of pediatric patients with β-TM after HSCT compared to that of patients treated with blood transfusions and iron chelation.

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA.

Health-related quality of life (HRQoL) is an important goal of therapy for patients with myelodysplastic syndromes (MDS); however, little is known about HRQoL of these patients at clinical presentation. We report HRQoL profile of newly diagnosed patients with MDS across both the the International Prognostic Scoring System (IPSS) and IPSS-Revised (IPSS-R) classifications, stratified by sex and age group categories, aiming to also establish European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core30 (EORTC QLQ-C30) reference values for these patients. Analysis was based on 927 patients with a median age of 73.

The estimand framework had implications in time to patient-reported outcomes deterioration analyses in cancer clinical trials.

Objectives: To apply the estimand framework in time to deterioration (TTD) analysis of patient-reported outcomes (PROs), and identify the appropriate statistical methods to deal with intercurrent event (IEs) such as death.

Study Design And Setting: Data from phase II randomized trial were used. We estimated TTD using European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 questionnaire with death as the IE, by applying Kaplan-Meier (K.

Adherence to Patient-Reported Symptom Monitoring and Subsequent Clinical Interventions for Patients With Multiple Myeloma in Outpatient Care: Longitudinal Observational Study.

Background: The use of software to monitor patient-reported outcome measures (PROMs) can improve outcomes for patients with cancer receiving anticancer therapy; however, evidence from applications used in routine clinical practice is lacking.

Objective: We aimed to investigate adherence to and patient perceptions of a weekly, web-based PROM symptom monitoring program in routine clinical practice for patients with Multiple Myeloma. Moreover, we aimed to capture how clinical alerts prompted by the system influenced clinical care.

Information about missing patient-reported outcome data in breast cancer trials is frequently not documented: a scoping review.

Objectives: This review addresses the common problem of missing patient-reported outcome (PRO) data in clinical trials by assessing the current practice of their statistical handling as reported in publications of randomized controlled trials (RCTs) in patients with breast cancer.

Study Design And Setting: We searched PubMed to identify RCTs evaluating biomedical treatments in breast cancer patients with at least one PRO endpoint published between January 2019 and February 2022. Two reviewers independently assessed the eligibility of the publications for this scoping review and extracted prespecified information on missing PRO data and related statistical practices.