Publications by authors named "Madeleine King"

219 Publications

Improving the patient-reported outcome sections of clinical trial protocols: a mixed methods evaluation of educational workshops.

Qual Life Res 2022 May 12. Epub 2022 May 12.

NHMRC Clinical Trial Centre, University of Sydney, Sydney, Australia.

Introduction: Failure to incorporate key patient-reported outcome (PRO) content in trial protocols affects the quality and interpretability of the collected data, contributing to research waste. Our group developed evidence-based training specifically addressing PRO components of protocols. We aimed to assess whether 2-day educational workshops improved the PRO completeness of protocols against consensus-based minimum standards provided in the SPIRIT-PRO Extension in 2018.

Method: Annual workshops were conducted 2011-2017. Participants were investigators/trialists from cancer clinical trials groups. Although developed before 2018, workshops covered 15/16 SPIRIT-PRO items. Participant feedback immediately post-workshop and, retrospectively, in November 2017 was summarised descriptively. Protocols were evaluated against SPIRIT-PRO by two independent raters for workshop protocols (developed post-workshop by participants) and control protocols (contemporaneous non-workshop protocols). SPIRIT-PRO items were assessed for completeness (0 = not addressed, 10 = fully addressed). Mann-Whitney U tests assessed whether workshop protocols scored higher than controls by item and overall.

Results: Participants (n = 107) evaluated the workshop positively. In 2017, 16/41 survey responders (39%) reported never applying in practice; barriers included role restrictions (14/41, 34%) and lack of time (5/41, 12%). SPIRIT-PRO overall scores did not differ between workshop (n = 13, median = 3.81/10, interquartile range = 3.24) and control protocols (n = 9, 3.51/10 (2.14)), (p = 0.35). Workshop protocols scored higher than controls on two items: 'specify PRO concepts/domains' (p = 0.05); 'methods for handling missing data' (p = 0.044).

Conclusion: Although participants were highly satisfied with these workshops, the completeness of PRO protocol content generally did not improve. Additional knowledge translation efforts are needed to assist protocol writers address SPIRIT-PRO guidance and avoid research waste that may eventuate from sub-optimal PRO protocol content.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-022-03127-wDOI Listing
May 2022

The EORTC QLU-C10D discrete choice experiment for cancer patients: a first step towards patient utility weights.

J Patient Rep Outcomes 2022 May 4;6(1):42. Epub 2022 May 4.

Department of Psychiatry, Psychotherapy and Psychosomatics, Psychiatry I, Medical University of Innsbruck, Innsbruck, Austria.

Background: The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 Dimensions (QLU-C10D) is a novel cancer-specific preference-based measure (PBM) for which value sets are being developed for an increasing number of countries. This is done by obtaining health preferences from the respective general population. There is an ongoing discussion if instead patients suffering from the disease in question should be asked for their preferences. We used the QLU-C10D valuation survey, originally designed for use in the general population, in a sample of cancer patients in Austria to assess the methodology's acceptability and applicability in this target group before obtaining QLU-C10D patient preferences.

Methods: The core of the QLU-C10D valuation survey is a discrete choice experiment in which respondents are asked to give preferences for certain health states (described by a relatively large number of 10 quality of life domains) and an associated survival time. They therewith are asked to trade off quality of life against life time. As this might be a very burdensome task for cancer patients undergoing treatment, a cognitive interview was conducted in a pilot sample to assess burden and potential additional needs for explanation in order to be able to use the DCE for the development of QLU-C10D patient preferences. In addition, responses to general feedback questions on the survey were compared against responses from a matched control group from the already completed Austrian general population valuation survey.

Results: We included 48 patients (mean age 59.9 years; 46% female). In the cognitive interview, the majority indicated that their experience with the survey was positive (85%) and overall clarity as good (90%). In response to the general feedback questions, patients rated the presentation of the health states less clear than matched controls (p = 0.008). There was no difference between patients and the general population concerning the difficulty in choosing between the health states (p = 0.344).

Conclusion: Despite the relatively large number of DCE domains the survey was manageable for patients and allows going on with the QLU-C10D patient valuation study.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s41687-022-00430-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9068836PMC
May 2022

Minimally important differences of EORTC QLQ-C30 scales in patients with lung cancer or malignant pleural mesothelioma - Interpretation guidance derived from two randomized EORTC trials.

Lung Cancer 2022 May 29;167:65-72. Epub 2022 Mar 29.

European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium.

Objectives: A minimally important difference (MID) is the smallest difference in quality of life (QoL) perceived as relevant by patients or clinicians. MIDs aid interpretation of QOL data in research and clinical practice. We aimed to determine MIDs for the EORTC QLQ-C30 for patients with lung cancer or malignant pleural mesothelioma.

Materials And Methods: Data were drawn from two EORTC-sponsored randomized clinical trials (RCTs): a three-arm RCT of two cisplatin-based treatments and paclitaxel plus gemcitabine in advanced non-small-cell lung cancer, and an RCT comparing cisplatin with or without raltitrexed in patients with malignant pleural mesothelioma. MIDs for interpreting within-group change and between-group differences in change over time were computed using anchor-based approaches, for improvements and deteriorations separately. Distribution-based approaches provided corroborative evidence.

Results: The combined data from the trials comprised 730 patients. Available data allowed us to determine 8/14 anchor-based MIDs for EORTC scales for improvements, and 9/14 MIDs for deterioration. Furthermore, we provided distribution-based estimates for all 14 QLQ-C30 scales. Most MIDs for improvements ranged between 5 and 10, for both within-group and between-group differences. Outliers were appetite loss and constipation, with MIDs up to 15 score points. MIDs were slightly larger for within-group deterioration, ranging from -5 to - 15, with the largest for Nausea/vomiting (-1 to 4) and Appetite loss (-1 to 5). MIDs for between-group differences in deterioration ranged from - 4 (Physical, Role, and Social functioning, and Global quality of life) to -9 (Nausea/vomiting, Appetite loss and Constipation).

Conclusions: MIDs vary over scales and for between- versus within-group comparisons; this must be taken into account when interpreting changes. Nevertheless, the majority of MIDs range between 5 and 10 score points, in line with previously used thresholds for QLQ-C30. These findings and those from other tumor-specific MID analyses will inform a planned consensus process identifying commonalities and differences across tumor sites.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.lungcan.2022.03.018DOI Listing
May 2022

Knowledge translation concerns for the CONSORT-PRO extension reporting guidance: a review of reviews.

Qual Life Res 2022 Mar 26. Epub 2022 Mar 26.

Centre for Patient Reported Outcomes Research, and Birmingham Health Partners Centre for Regulatory Science and Innovation, Institute of Applied Health Research, University of Birmingham, and University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.

This review of reviews aimed to appraise the use of the CONSORT-PRO Extension as an evaluation tool for assessing the reporting of patient-reported outcome (PROs) in publications, and to describe the reporting of PRO research across reviews. We also outlined how variation in such evaluations impacts knowledge translation and may lead to potential misuse of the CONSORT-PRO Extension. We systematically searched Medline, Pubmed and CINAHL from 2013 to 2025 March 2021 for reviews of the completeness of reporting of PRO endpoints according to CONSORT-PRO criteria. Two reviewers extracted details of each review, the percentage of included studies that addressed each CONSORT-PRO item, and key recommendations from each review. Fourteen reviews met inclusion criteria, and only six of these used the full CONSORT-PRO checklist with minimal justified modifications. The remaining eight studies made significant or unjustified adjustments to the CONSORT-PRO Extension. Review studies also varied in how they scored multi-component CONSORT-PRO items. CONSORT-PRO items were often unreported in trial reports, and certain CONSORT-PRO items were reported less often than others. The reporting of statistical approaches to dealing with missing PRO data were poor in RCTs included in all 14 review articles. Studies reviewing PRO publications often omitted recommended CONSORT-PRO items from their evaluations, which may cause confusion among readers regarding how best to report their PRO research according to the CONSORT-PRO extension. Many trials published since CONSORT-PRO's release did not report recommended CONSORT-PRO items, which may lead to misinterpretation and consequently to research waste.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-022-03119-wDOI Listing
March 2022

The PROTEUS-Trials Consortium: Optimizing the use of patient-reported outcomes in clinical trials.

Clin Trials 2022 Jan 31:17407745221077691. Epub 2022 Jan 31.

Queen's Cancer Research Institute, Queen's University, Kingston, ON, Canada.

Background: The assessment of patient-reported outcomes in clinical trials has enormous potential to promote patient-centred care, but for this potential to be realized, the patient-reported outcomes must be captured effectively and communicated clearly. Over the past decade, methodologic tools have been developed to inform the design, analysis, reporting, and interpretation of patient-reported outcome data from clinical trials. We formed the PROTEUS-Trials Consortium (Patient-Reported Outcomes Tools: Engaging Users and Stakeholders) to disseminate and implement these methodologic tools.

Methods: PROTEUS-Trials are engaging with patient, clinician, research, and regulatory stakeholders from 27 organizations in the United States, Canada, Australia, the United Kingdom, and Europe to develop both organization-specific and cross-cutting strategies for implementing and disseminating the methodologic tools. Guided by the Knowledge-to-Action framework, we conducted consortium-wide webinars and meetings, as well as individual calls with participating organizations, to develop a workplan, which we are currently executing.

Results: Six methodologic tools serve as the foundation for PROTEUS-Trials dissemination and implementation efforts: the Standard Protocol Items: Recommendations for Interventional Trials-patient-reported outcome extension for writing protocols with patient-reported outcomes, the International Society for Quality of Life Research Minimum Standards for selecting a patient-reported outcome measure, Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium recommendations for patient-reported outcome data analysis, the Consolidated Standards for Reporting of Trials-patient-reported outcome extension for reporting clinical trials with patient-reported outcomes, recommendations for the graphic display of patient-reported outcome data, and a Clinician's Checklist for reading and using an article about patient-reported outcomes. The PROTEUS-Trials website (www.TheProteusConsortium.org) serves as a central repository for the methodologic tools and associated resources. To date, we have developed (1) a roadmap to visually display where each of the six methodologic tools applies along the clinical trial trajectory, (2) web tutorials that provide guidance on the methodologic tools at different levels of detail, (3) checklists to provide brief summaries of each tool's recommendations, (4) a handbook to provide a self-guided approach to learning about the tools and recommendations, and (5) publications that address key topics related to patient-reported outcomes in clinical trials. We are also conducting organization-specific activities, including meetings, presentations, workshops, and webinars to publicize the existence of the methodologic tools and the PROTEUS-Trials resources. Work to develop communications strategies to ensure that PROTEUS-Trials reach key audiences with relevant information about patient-reported outcomes in clinical trials and PROTEUS-Trials is ongoing.

Discussion: The PROTEUS-Trials Consortium aims to help researchers generate patient-reported outcome data from clinical trials to (1) enable investigators, regulators, and policy-makers to take the patient perspective into account when conducting research and making decisions; (2) help patients understand treatment options and make treatment decisions; and (3) inform clinicians' discussions with patients regarding treatment options. In these ways, the PROTEUS Consortium promotes patient-centred research and care.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/17407745221077691DOI Listing
January 2022

Symptom burden and quality of life with chemotherapy for recurrent ovarian cancer: the Gynecologic Cancer InterGroup-Symptom Benefit Study.

Int J Gynecol Cancer 2022 Jan 27. Epub 2022 Jan 27.

Australia New Zealand Gynaecological Oncology Group (ANZGOG), Camperdown, New South Wales, Australia

Objective: The Gynecologic Cancer InterGroup (GCIG)-Symptom Benefit Study was designed to evaluate the effects of chemotherapy on symptoms and health-related quality of life (HRQL) in women having chemotherapy for platinum resistant/refractory recurrent ovarian cancer (PRR-ROC) and potentially platinum sensitive with ≥3 lines of chemotherapy (PPS-ROC ≥3).

Methods: Participants completed the Measure of Ovarian Cancer Symptoms and Treatment (MOST) and European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire QLQ-C30 questionnaires at baseline and every 3-4 weeks until progression. Participants were classified symptomatic if they rated ≥4 of 10 in at least one-third of symptoms in the MOST index. Improvement in MOST was defined as two consecutive scores of ≤3 in at least half of the symptomatic items at baseline. Improvement in HRQL was defined as two consecutive scores ≥10 points above baseline in the QLQ-C30 summary score scale (range 0-100).

Results: Of 948 participants enrolled, 910 (96%) completed baseline questionnaires: 546 with PRR-ROC and 364 with PPS-ROC ≥3. The proportions of participants symptomatic at baseline as per MOST indexes were: abdominal 54%, psychological 53%, and disease- or treatment-related 35%. Improvement was reported in MOST indexes: abdominal 40%, psychological 35%, and disease- or treatment-related 38%. Median time to improvement in abdominal symptoms occurred earlier for PRR-ROC than for PPS-ROC ≥3 (4 vs 6 weeks, p=0.044); median duration of improvement was also similar (9.0 vs 11.7 weeks, p=0.65). Progression-free survival was longer among those with improvement in abdominal symptoms than in those without (median 7.2 vs 2.5 months, p<0.0001). Improvements in HRQL were reported by 77/448 (17%) with PRR-ROC and 61/301 (20%) with PPS-ROC ≥3 (p=0.29), and 102/481 (21%) of those with abdominal symptoms at baseline.

Conclusion: Over 50% of participants reported abdominal and psychological symptoms at baseline. Of those, 40% reported an improvement within 2 months of starting chemotherapy. Approximately one in six participants reported an improvement in HRQL. Symptom monitoring and supportive care is important as chemotherapy palliated less than half of symptomatic participants.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1136/ijgc-2021-003142DOI Listing
January 2022

Incorporation of novel therapies for the management of sickle cell disease: A pharmacist's perspective.

J Oncol Pharm Pract 2022 Apr 21;28(3):646-663. Epub 2022 Jan 21.

Rogel Cancer Center, 1259University of Michigan, Ann Arbor, Michigan, USA.

Patients with sickle cell disease (SCD) experience significant disease-related morbidity including multiorgan damage, chronic anemia, and debilitating pain crises. While hydroxyurea has been the primary disease modifying modality in SCD, novel therapies with unique mechanism of action have recently been approved. This review article examines the evidence surrounding the available SCD therapies to guide pharmacists on potential treatment selection and management strategies for patients with SCD. A systematic search of online databases was performed to identify literature on the management of SCD. While the newly approved novel agents have demonstrated clinical benefit it remains unclear how these agents fit into the treatment paradigm. Pharmacists should be aware of the data supporting the use of these novel agents to optimize use on a patient-specific basis.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/10781552211072468DOI Listing
April 2022

Evaluating patient-reported symptoms and late adverse effects following completion of first-line chemotherapy for ovarian cancer using the MOST (Measure of Ovarian Symptoms and Treatment concerns).

Gynecol Oncol 2022 02 24;164(2):437-445. Epub 2021 Dec 24.

Prince of Wales Clinical School, University of New South Wales, Sydney, Australia; Department of Medical Oncology, Prince of Wales Hospital, Sydney, Australia.

Objectives: Knowledge on the course of symptoms patients with ovarian cancer experience is limited. We documented the prevalence and trajectories of symptoms after first-line chemotherapy using the Measure of Ovarian Symptoms and Treatment concerns (MOST).

Methods: A total of 726 patients who received platinum-based chemotherapy for ovarian cancer were asked to complete the MOST every 3 months, beginning 6 months post-diagnosis and continuing for up to 4 years. We used descriptive statistics to examine temporal changes in MOST-S26 index scores for disease or treatment-related (MOST-DorT), neurotoxicity (MOST-NTx), abdominal (MOST-Abdo), and psychological (MOST-Psych) symptoms, and wellbeing (MOST-Wellbeing) and selected individual symptoms. We used group-based trajectory models to identify groups with persistently poor symptoms.

Results: The median MOST-Abdo, MOST-DorT and MOST-Wellbeing score were worst at chemotherapy-end but improved and stabilised by 1, 3 and 12 months after treatment, respectively. The median MOST-NTx score peaked at 1 month after treatment before improving, while the median MOST-Psych score did not change substantially over time. Long-term moderate-to-severe fatigue (32%), trouble sleeping (31%), sore hands and feet (21%), pins and needles (20%) and anxiety (18%) were common. Trajectory models revealed groups of patients with persistent symptoms had MOST-DorT scores above 30 and MOST-NTx scores above 40 at treatment-end.

Conclusions: Although many patients report improvements in symptoms by 3 months after first-line chemotherapy for ovarian cancer, patients who score > 30/100 on MOST-S26-DorT or > 40/100 on MOST-S26-NTx at the end of chemotherapy are likely to have persistent symptoms. The MOST could triage this at-risk subset for early intervention.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ygyno.2021.12.006DOI Listing
February 2022

Providing Psychological Support to Parents of Childhood Cancer Survivors: '' Intervention Trial Results and Lessons for the Future.

Cancers (Basel) 2021 Nov 9;13(22). Epub 2021 Nov 9.

School of Women's and Children's Health, UNSW Medicine and Health, UNSW Sydney, Kensington, NSW 2052, Australia.

We conducted a three-armed trial to assess Cascade, a four-module group videoconferencing cognitive behavior therapy (CBT) intervention for parents of childhood cancer survivors currently aged <18 years. We allocated parents to Cascade, an attention control (peer-support group), or a waitlist. The primary outcome was parents' health-related quality of life () six months post-intervention. Parents also reported their anxiety/depression, parenting self-agency, fear of recurrence, health service and psychotropic medication use, engagement in productive activities, confidence to use, and actual use of, CBT skills, and their child's quality of life. Seventy-six parents opted in; 56 commenced the trial. Cascade achieved good parent engagement and most Cascade parents were satisfied and reported benefits. Some parents expressed concerns about the time burden and the group format. Most outcomes did not differ across trial arms. Cascade parents felt more confident to use more CBT skills than peer-support and waitlisted parents, but this did not lead to more use of CBT. Cascade parents reported lower psychosocial health scores for their child than waitlisted parents. Cascade parents' health service use, psychotropic medication use, and days engaged in productive activities did not improve, despite some improvements in waitlisted parents. Our trial was difficult to implement, but participants were largely satisfied. Cascade did not improve most outcomes, possibly because many parents were functioning well pre-enrolment. We used these findings to improve Cascade and will trial the new version in future.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3390/cancers13225597DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8615912PMC
November 2021

A systematic survey identified methodological issues in studies estimating anchor-based minimal important differences in patient-reported outcomes.

J Clin Epidemiol 2022 02 6;142:144-151. Epub 2021 Nov 6.

Department of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Hamilton, Canada.

Objective: To systematically survey the literature addressing the reporting of studies estimating anchor-based minimal important differences (MIDs) and choice of optimal MIDs.

Study Design And Setting: We searched Medline, Embase and PsycINFO from 1987 to March 2020. Teams of two reviewers independently identified eligible publications and extracted quotations addressing relevant issues for reporting and/or selecting anchor-based MIDs. Using a coding list, we assigned the same code to quotations capturing similar or related issues. For each code, we generated an 'item', i.e., a specific phrase or sentence capturing the underlying concept. When multiple concepts existed under a single code, the team created multiple items for that code. We clustered codes addressing a broader methodological issue into a 'category' and classified items as relevant for reporting, relevant for selecting an anchor-based MID, or both.

Results: We identified 136 eligible publications that provided 6 categories (MID definition, anchors, patient-reported outcome measures, generalizability and statistics) and 24 codes. These codes contained 34 items related to reporting MID studies, of which 29 were also related to selecting MIDs.

Conclusion: The systematic survey identified items related to reporting of anchor-based MID studies and selecting optimal MIDs. These provide a conceptual framework to inform the design of studies related to MIDs, and a basis for developing a reporting standard and a selection approach for MIDs.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jclinepi.2021.10.028DOI Listing
February 2022

A Comparison of Generic and Condition-Specific Preference-Based Measures Using Data From Nivolumab Trials: EQ-5D-3L, Mapping to the EQ-5D-5L, and European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions.

Value Health 2021 11 26;24(11):1651-1659. Epub 2021 Aug 26.

Patient-Centered Outcomes, Adelphi Values Ltd, Bollington, England, UK.

Objectives: There is growing interest in condition-specific preference measures, including the European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (QLU-C10D). This research assessed the implications of using utility indices on the basis of the EQ-5D-3L, a mapping of EQ-5D-3L to the EQ-5D-5L, and the QLU-C10D, and compared their psychometric properties.

Methods: Data were taken from 8 phase 3 randomized controlled trials of nivolumab with or without ipilimumab for the treatment of solid tumors. Utilities for progression-related states were calculated using the UK and English value sets and incremental quality-adjusted life-years (QALYs) derived from established UK cost-effectiveness models. The psychometric properties of the utility indices were assessed using pooled trial data.

Results: Compared with the EQ-5D-3L index, the mapped EQ-5D-5L index yielded an average of 6% more and the QLU-C10D index an average of 2% fewer incremental QALYs for nivolumab versus comparators. All indices could differentiate between groups defined by performance status, cancer stage, or self-reported health status at baseline and detect meaningful changes in performance status, tumor response, health status, and quality of life over approximately 12 weeks of treatment.

Conclusions: The lower QALY yield of the QLU-C10D was balanced by evidence of greater validity and responsiveness. Benefits gained from using the QLU-C10D may be apparent when treatments affect targeted symptoms and functional aspects, including sleep, bowel function, appetite, nausea, and fatigue. The observed differences in QALYs may not be sufficiently large to affect health technology assessment decisions.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jval.2021.05.022DOI Listing
November 2021

Minimally important differences for the EORTC QLQ-C30 in prostate cancer clinical trials.

BMC Cancer 2021 Oct 7;21(1):1083. Epub 2021 Oct 7.

European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium.

Background: The aim of the study was to estimate the minimally important difference (MID) for interpreting group-level change over time, both within a group and between groups, for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scores in patients with prostate cancer.

Methods: We used data from two published EORTC trials. Clinical anchors were selected by strength of correlations with QLQ-C30 scales. In addition, clinicians' input was obtained with regard to plausibility of the selected anchors. The mean change method was applied for interpreting change over time within a group of patients and linear regression models were fitted to estimate MIDs for between-group differences in change over time. Distribution-based estimates were also evaluated.

Results: Two clinical anchors were eligible for MID estimation; performance status and the CTCAE diarrhoea domain. MIDs were developed for 7 scales (physical functioning, role functioning, social functioning, pain, fatigue, global quality of life, diarrhoea) and varied by scale and direction (improvement vs deterioration). Within-group MIDs ranged from 4 to 14 points for improvement and - 13 to - 5 points for deterioration and MIDs for between-group differences in change scores ranged from 3 to 13 for improvement and - 10 to - 5 for deterioration.

Conclusions: Our findings aid the meaningful interpretation of changes on a set of EORTC QLQ-C30 scale scores over time, both within and between groups, and for performing more accurate sample size calculations for clinical trials in prostate cancer.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12885-021-08609-7DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8496068PMC
October 2021

Perceived benefits and limitations of using patient-reported outcome measures in clinical practice with individual patients: a systematic review of qualitative studies.

Qual Life Res 2022 Jun 27;31(6):1597-1620. Epub 2021 Sep 27.

The University of Sydney, Faculty of Science, School of Psychology, Sydney Quality of Life Office, Sydney, Australia.

Purpose: Patient-reported outcome measures (PROMs) are increasingly used in clinical settings to inform individual patient care. In-depth understanding of end-users' experiences may help identify factors that promote or hinder their use in clinical decision-making. We aimed to examine stakeholder perceptions of the utility of using PROMs in clinical practice based on real-life experience.

Methods: Systematic review searching Medline, Embase and PsychINFO from inception to May 2021. Qualitative studies examining patients' and/or clinicians' experiences of using PROMs in clinical settings were included. Study screening and data extraction were performed by two independent reviewers. Qualitative data from included studies was analysed thematically.

Results: Of 2388 abstracts retrieved, 52 articles reporting 50 studies met eligibility. Five key benefits were identified: (1) promotes active patient involvement (enables goal setting and discussion of sensitive topics); (2) enhances the focus of consultations (prioritizes patient needs); (3) improves quality of care (enables tailored, holistic care and prompts action); (4) enables standardized monitoring of patient outcomes; and (5) enhances the patient-clinician relationship (provides reassurance). Perceived limitations included the capacity of PROMs to shift the focus of consultations; inaccurately estimate problems; raise unrealistic expectations for care; inhibit patient-clinician interaction; lack clinically meaningful information; and not be suitable for all patients.

Conclusion: Both patients and clinicians reported benefits of using PROMs across diverse health conditions and clinical settings, but also highlighted several limitations. These limitations shed some light on why PROM use may not always improve patient outcomes and provide considerations for the design and implementation of future PROM initiatives.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-021-03003-zDOI Listing
June 2022

Getting the MOST out of follow-up: a randomized controlled trial comparing 3 monthly nurse led follow-up via telehealth, including monitoring CA125 and patient reported outcomes using the MOST (Measure of Ovarian Symptoms and Treatment concerns) with routine clinic based or telehealth follow-up, after completion of first line chemotherapy in patients with epithelial ovarian cancer.

Int J Gynecol Cancer 2022 Apr 4;32(4):560-565. Epub 2022 Apr 4.

Prince of Wales Clinical School, University of New South Wales, Randwick, New South Wales, Australia.

Background: Physical symptoms, anxiety, depression, fear of recurrence, sexual dysfunction, and social withdrawal are common in women after treatment for ovarian cancer. Most patients would like and need help dealing with these symptoms. The traditional model of follow-up care is unstructured and largely focused on diagnosing recurrent disease, and most oncologists lack skills to identify and manage psychosocial issues. No high quality prospective clinical trials have been conducted to determine the optimal follow-up regimen or the cost effectiveness of ovarian cancer surveillance strategies.

Primary Objectives: To assess emotional wellbeing, acceptability, safety, and cost effectiveness of nurse led follow-up via telehealth for women with ovarian cancer following completion of primary treatment.

Study Hypothesis: We hypothesize that compared with routine clinic based follow-up, nurse led follow-up via telehealth, including serum CA125 monitoring and completion of a patient reported outcome instrument, the Measure of Ovarian Symptoms and Treatment concerns-Surveillance (MOST-S26), will improve emotional wellbeing in women with ovarian cancer; be feasible, safe, acceptable, and not delay the time to diagnosis of recurrent disease; will result in greater patient satisfaction; will identify more patients with psychological distress, lead to better care, and improved psychological outcomes; and be cost-effective.

Trial Design: Phase II multicenter randomized trial comparing 3 monthly nurse led telehealth consultations that include serum CA125 monitoring and completion of the MOST-S26, with routine clinic based follow-up. The allocation ratio will be 1:1.

Major Inclusion/exclusion Criteria: Eligible patients will be women with high grade epithelial ovarian cancer who have normalized serum CA125 (to <35 kU/L) at completion of first line chemotherapy.

Primary Endpoints: Emotional wellbeing at 12 months.

Sample Size: 150 patients.

Estimated Dates For Completing Accrual And Presenting Results: July 2023. Results expected in 2025, 24 months after the last participant is enrolled.

Trial Registration: ACTRN12620000332921.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1136/ijgc-2021-002999DOI Listing
April 2022

The role of breast reconstruction choice on body image patient-reported outcomes at four years post-mastectomy for breast cancer: A longitudinal prospective cohort study.

Psychooncology 2022 01 8;31(1):54-61. Epub 2021 Sep 8.

Breast & Surgical Oncology at the Poche Centre, North Sydney, New South Wales, Australia.

Objectives: To examine the impact of breast reconstruction on women's perceptions of body image over time and to assess the influence of sociodemographic variables on body image.

Methods: A prospective, longitudinal cohort study, using validated breast cancer-specific questionnaires, to compare patient-reported outcomes in women choosing immediate (n = 61), delayed (n = 16) or no (n = 23) breast reconstruction.

Results: One hundred women completed baseline questionnaires that included items on body image; 30 women completed all four annual follow-up sets, while 20 women completed baseline only. The three groups were well matched at baseline and similar trajectories in body image measures were identified over 48 months in all groups. At 12 months post-mastectomy, significant changes were seen in eight of the 10 subscales; this reduced to seven subscales at 24 months and four at 36 months. By 48 months, only three subscales remained significantly different to baseline scores: women remained less vulnerable and had fewer limitations (improved outcomes); the one worse outcome was persistently higher levels of arm concern. Three of the sociodemographic variables (health insurance, age and employment status) showed significant inter-group differences at some time points.

Conclusion: These findings suggest women recover from the negative impact of mastectomy on body image within four years of surgery, whether they have immediate, delayed or no reconstruction. Our results provide some indirect evidence that having a choice of BR options is important, regardless of the choice made. Four years appears to be a suitable follow-up period for future studies in this area.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/pon.5776DOI Listing
January 2022

Development and validation of the measure of ovarian symptoms and treatment concerns for surveillance (MOST-S26): An instrument to complement the clinical follow-up of women with ovarian cancer after completion of first-line treatment.

Gynecol Oncol 2021 11 1;163(2):398-407. Epub 2021 Sep 1.

Gynaecological Cancers Group, QIMR Berghofer Medical Research Institute, Brisbane, Australia; School of Public Health, The University of Queensland, Brisbane, Australia.

Objective: The Measure of Ovarian Symptoms and Treatment (MOST-T35) is a patient-reported symptom index, developed and validated in the context of palliative chemotherapy for recurrent ovarian cancer (OC). We aimed to develop and validate a version suitable for surveillance of symptoms following first-line treatment for OC to support clinical follow-up.

Methods: In a prospective study of women following completion of first-line chemotherapy for OC, patients completed MOST-T35 every 3 months for up to 3.5 years and other patient-reported outcome measures. Construct validity (Spearman's correlations), discriminative validity (t-tests/ANOVAs assessing differences between clinically distinct groups), ability to detect clinically important symptoms (receiver operating characteristic analysis), and responsiveness (t-tests examining change) were assessed.

Results: Data from 726 women who received ≥3 cycles of chemotherapy, did not progress within 3 months, and completed ≥one MOST-T35 were analysed. The revised version, MOST-S26, has 26 items and 5 multi-item indexes: peripheral neuropathy (MOST-NTx), disease or treatment-related (MOST-DorT), abdominal (MOST-Abdo), and psychological symptoms (MOST-Psych), and MOST-Wellbeing, plus 9 individual items. Construct validity was confirmed (r range = 0.43-0.88). Discriminative validity confirmed expected differences between groups. MOST-NTx and MOST-Psych detected improvements in peripheral neuropathy and psychological symptoms respectively, whereas MOST-Abdo detected worsening of abdominal symptoms pre-recurrence.

Conclusions: This study developed and validated the MOST-S26, for surveillance of women in follow-up after first-line chemotherapy for OC. MOST-S26 reliably detected improvement in symptoms of peripheral neuropathy, psychological distress and may detect symptoms of relapse. Administration of MOST-S26 in follow-up consultations could identify concerning symptoms and facilitate timely and appropriate intervention.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ygyno.2021.08.022DOI Listing
November 2021

The use of proxies and proxy-reported measures: a report of the international society for quality of life research (ISOQOL) proxy task force.

Qual Life Res 2022 Feb 12;31(2):317-327. Epub 2021 Jul 12.

Faculty of Medicine, Sydney Medical School, Central Clinical School, The University of Sydney, Sydney, NSW, Australia.

Aims: Proxy reports are often used when patients are unable to self-report. It is unclear how proxy measures are currently in use in adult health care and research settings. We aimed to describe how proxy reports are used in these settings, including the use of measures developed specifically for proxy reporting in adult health populations.

Methods: We systematically searched Medline, PsycINFO, PsycTESTS, CINAHL and EMBASE from database inception to February 2018. Search terms included a combination of terms for quality of life and health outcomes, proxy-reporters, and health condition terms. The data extracted included clinical context, the name of the proxy measure(s) used and other descriptive data. We determined whether the measures were developed specifically for proxy use or were existing measures adapted for proxy use.

Results: The database search identified 17,677 possible articles, from which 14,098 abstracts were reviewed. Of these, 11,763 were excluded and 2335 articles were reviewed in full, with 880 included for data extraction. The most common clinical settings were dementia (30%), geriatrics (15%) and cancer (13%). A majority of articles (51%) were paired studies with proxy and patient responses for the same person on the same measure. Most paired studies (77%) were concordance studies comparing patient and proxy responses on these measures.

Discussion: Most published research using proxies has focused on proxy-patient concordance. Relatively few measures used in research with proxies were specifically developed for proxy use. Future work is needed to examine the performance of measures specifically developed for proxies.

Systematic Review Registration: PROSPERO No. CRD42018103179.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-021-02937-8DOI Listing
February 2022

International perspectives on suboptimal patient-reported outcome trial design and reporting in cancer clinical trials: A qualitative study.

Cancer Med 2021 08 5;10(16):5475-5487. Epub 2021 Jul 5.

Centre for Patient-Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK.

Purpose: Evidence suggests that the patient-reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non-reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions.

Methods: Semi-structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame.

Results: Forty-four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists' reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified.

Conclusion: Misconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/cam4.4111DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8366078PMC
August 2021

Estimation of an EORTC QLU-C10 Value Set for Spain Using a Discrete Choice Experiment.

Pharmacoeconomics 2021 09 3;39(9):1085-1098. Epub 2021 Jul 3.

Division of Psychiatry II, Department of Psychiatry, Psychotherapy and Psychosomatics, Medical University of Innsbruck, Innsbruck, Austria.

Background: The EORTC QLU-C10D is a preference-based measure derived from the EORTC QLQ-C30. For use in economic evaluations, country-specific value sets are needed. This study aimed to generate an EORTC QLU-C10 value set for Spain.

Methods: A sample of the Spanish general population completed an online discrete choice experiment. An attribute-balanced incomplete block design was used to select 960 choice tasks, with a total of 1920 health states. Each participant was randomly assigned 16 choice sets without replacement. Data were modelled using generalized estimating equations and mixed logistic regressions.

Results: A total of 1625 panel members were invited to participate, 1010 of whom were included in the study. Dimension decrements were generally monotonic with larger disutilities at increased severity levels. Dimensions associated with larger decrements were physical functioning and pain, while the dimension with the smallest decrement was sleep disturbances. The PITS state (i.e. worst attainable health) for the Spanish population is - 0.043.

Conclusions: This study generated the first Spanish value set for the QLU-C10D. This can facilitate cost-utility analyses when applied to data collected with the EORTC QLQ-C30.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s40273-021-01058-xDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8352836PMC
September 2021

SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials.

BMJ Open 2021 06 30;11(6):e045105. Epub 2021 Jun 30.

Value Evidence and Outcomes-Patient Centered Outcomes, GSK, Collegeville, Pennsylvania, USA.

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients' symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item's importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1136/bmjopen-2020-045105DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8246371PMC
June 2021

The Functional Assessment of Cancer Therapy Eight Dimension (FACT-8D), a Multi-Attribute Utility Instrument Derived From the Cancer-Specific FACT-General (FACT-G) Quality of Life Questionnaire: Development and Australian Value Set.

Value Health 2021 06 7;24(6):862-873. Epub 2021 May 7.

Northwestern University Feinberg School of Medicine, Department of Medical Social Sciences, Chicago, IL, USA.

Objectives: To develop a cancer-specific multi-attribute utility instrument derived from the Functional Assessment of Cancer Therapy - General (FACT-G) health-related quality of life (HRQL) questionnaire.

Methods: We derived a descriptive system based on a subset of the 27-item FACT-G. Item selection was informed by psychometric analyses of existing FACT-G data (n = 6912) and by patient input (n = 82). We then conducted an online valuation survey, with participants recruited via an Australian general population online panel. A discrete choice experiment (DCE) was used, with attributes being the HRQL dimensions of the descriptive system and survival duration, and 16 choice-pairs per participant. Utility decrements were estimated with conditional logit and mixed logit modeling.

Results: Eight HRQL dimensions were included in the descriptive system: pain, fatigue, nausea, sleep, work, social support, sadness, and future health worry; each with 5 levels. Of 1737 panel members who accessed the valuation survey, 1644 (95%) completed 1 or more DCE choice-pairs and were included in analyses. Utility decrements were generally monotonic; within each dimension, poorer HRQL levels generally had larger utility decrements. The largest utility decrements were for the highest levels of pain (-0.40) and nausea (-0.28). The worst health state had a utility of -0.54, considerably worse than dead.

Conclusions: A descriptive system and preference-based scoring approach were developed for the FACT-8D, a new cancer-specific multi-attribute utility instrument derived from the FACT-G. The Australian value set is the first of a series of country-specific value sets planned that can facilitate cost-utility analyses based on items from the FACT-G and related FACIT questionnaires containing FACT-G items.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jval.2021.01.007DOI Listing
June 2021

Randomized Trial of Radiation Therapy With Weekly Cisplatin or Cetuximab in Low-Risk HPV-Associated Oropharyngeal Cancer (TROG 12.01) - A Trans-Tasman Radiation Oncology Group Study.

Int J Radiat Oncol Biol Phys 2021 11 4;111(4):876-886. Epub 2021 Jun 4.

Genesiscare St Vincent's Hospital, Melbourne, Australia; Department of Medicine, University of Melbourne, Melbourne, Australia.

Purpose: The excellent prognosis of patients with low-risk human papillomavirus (HPV)- associated oropharyngeal squamous cell carcinoma has led to concerns about overtreatment and excessive toxicity with radiation therapy and cisplatin, leading to interest in de-intensification trials. We investigated whether cetuximab, an epidermal growth factor receptor targeting antibody, when combined with radiation therapy would result in a decrease in symptom burden and toxicity with similar efficacy compared with weekly cisplatin.

Methods And Materials: TROG12.01, a randomized, multicenter trial involving 15 sites in Australia and New Zealand enrolled patients with HPV-associated oropharyngeal squamous cell carcinoma, American Joint Committee on Cancer 7th edition stage III (excluding T1-2N1) or stage IV (excluding T4 and/or N3 and/or N2b-c if smoking history >10 pack years and/or distant metastases). Patients were randomized (1:1) to receive radiation therapy (70 Gy in 35 fractions) with either weekly cisplatin, 7 doses of 40 mg/m, or cetuximab, loading dose of 400 mg/m followed by 7 weekly doses of 250 mg/m. The primary outcome was symptom severity assessed by the MD Anderson Symptom Inventory Head and Neck Symptom Severity Scale from baseline to 13 weeks postcompletion of radiation therapy using the area under the curve. Trial was registered on ClinicalTrials.gov: NCT01855451.

Results: Between June 17, 2013, and June 7, 2018, 189 patients were enrolled, with 92 in cisplatin arm and 90 in cetuximab included in the main analysis. There was no difference in the primary endpoint of symptom severity; difference in area under the curve cetuximab-cisplatin was 0.05 (95% confidence interval [CI], -0.19, 0.30), P = .66. The T-score (mean number of ≥grade 3 acute adverse events) was 4.35 (standard deviation 2.48) in the cisplatin arm and 3.82 (standard deviation 1.8) in the cetuximab arm, P = .108. The 3-year failure-free survival rates were 93% (95% CI, 86%-97%) in the cisplatin arm and 80% (95% CI, 70%-87%) in the cetuximab arm (hazard ratio = 3.0 [95% CI, 1.2-7.7]); P = .015.

Conclusions: For patients with low-risk HPV-associated oropharyngeal cancer, radiation therapy and cetuximab had inferior failure-free survival without improvement in symptom burden or toxicity compared with radiation therapy and weekly cisplatin. Radiation therapy and cisplatin remain the standard of care.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ijrobp.2021.04.015DOI Listing
November 2021

Efficacy and Safety of Non-Vitamin K Antagonist Oral Anticoagulants in Pediatric Venous Thromboembolism Treatment and Thromboprophylaxis: A Systematic Review of the Literature.

Semin Thromb Hemost 2021 Sep 10;47(6):643-653. Epub 2021 May 10.

Johns Hopkins All Children's Institute for Clinical and Translational Research, St. Petersburg, Florida.

Venous thromboembolism (VTE) in children can lead to significant morbidity and mortality. Traditionally, treatment for thrombotic events in pediatric patients has been limited mainly to unfractionated heparin, low-molecular-weight heparin (LMWH), or vitamin K antagonists. Since the first non-vitamin K antagonist oral anticoagulant (NOAC) was approved for adult use, these agents have gained popularity for a variety of indications. This is largely due to their ease of administration, favorable pharmacokinetic and pharmacodynamic profile, decreased food interactions, and decreased need for therapeutic drug monitoring. Treating and preventing VTE with traditional anticoagulants in pediatric patients presents many challenges. This systematic review evaluated the current literature regarding pediatric NOAC trials. Additionally, based on an up-to-date query of , we detail current ongoing and as-yet unpublished clinical trials, study outcomes, and projected completion dates. Published pediatric NOAC trials have included 1,007 total children to date and have ranged from phase 1 to 4, with "indications" including both thromboembolism prophylaxis and VTE treatment. Three recent phase 3 trials, specifically involving rivaroxaban and dabigatran, have shown the agents to be at least as effective as traditional anticoagulants for acute and/or extended VTE treatment, with low frequency of recurrent thrombosis and clinically significant bleeding rates. Additionally, specially developed and tested pediatric formulations have allowed for accurate and reliable dosing, oral administration, stable pharmacokinetics and pharmacodynamics, and fewer drug or food interactions. Ongoing trials, anticipated for completion in the next few years, will reveal important information with regard to thromboembolism prophylaxis in special pediatric subpopulations and settings.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1055/s-0041-1725944DOI Listing
September 2021

Patient-reported quality of life and symptom burden measures in human papillomavirus associated oropharyngeal cancer - A review of the literature and PRO methodology.

Oral Oncol 2021 07 29;118:105309. Epub 2021 Apr 29.

The Sir Peter MacCallum Department of Medical Oncology, The University of Melbourne, Melbourne, Australia; Department of Medical Oncology, Peter MacCallum Cancer Centre, Melbourne, Australia.

The emergence of human papillomavirus-associated oropharyngeal cancer (HPVOPC) has resulted in an explosion of clinical research offering reduced toxicity and improved health-related quality of life (HRQL) through treatment de-escalation. At the heart of this objective are patient-reported outcomes (PROs) which aim to quantify the patient experience, usually through the measurement of HRQL or symptom burden. A number of PRO measures (PROMs) are available to HNC researchers and selection of the optimal instrument relies on a detailed understanding of their content and psychometric properties matched to the clinical endpoint of interest. As PROMs become increasingly favoured as the primary or co-primary endpoints of interest in HNC clinical trials, particularly those focussed on HPVOPC, future treatment paradigms will be determined by these measures and it is imperative that they are applied with sophistication and rigor. This review draws attention to the limitations and challenges our specialty faces in PRO application, analysis and reporting. These shortfalls typically include a reliance on statistical rather than clinically relevant differences, multiple hypothesis testing, a lack of evidence-based minimal clinically important differences for the commonly used tools, as well as variations in PROM selection. The aim of this review is to provide: (1) an overview of PRO/PROM terminology and methodology in the HNC setting; (2) to provide a summary of HRQL and symptom burden reports in the HPVOPC literature; and (3) to draw attention to the unmet research need of refining PROM development, application and interpretation to guide our treatment decisions based on what matters to patients.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.oraloncology.2021.105309DOI Listing
July 2021

United States Utility Algorithm for the EORTC QLU-C10D, a Multiattribute Utility Instrument Based on a Cancer-Specific Quality-of-Life Instrument.

Med Decis Making 2021 05 5;41(4):485-501. Epub 2021 Apr 5.

School of Population Health, Curtin University, Perth, WA, Australia.

Background: The EORTC QLU-C10D is a multiattribute utility measure derived from the cancer-specific quality-of-life questionnaire, the EORTC QLQ-C30. The QLU-C10D contains 10 dimensions (physical, role, social and emotional functioning, pain, fatigue, sleep, appetite, nausea, bowel problems). The objective of this study was to develop a United States value set for the QLU-C10D.

Methods: A US online panel was quota recruited to achieve a representative sample for sex, age (≥18 y), race, and ethnicity. Respondents undertook a discrete choice experiment, each completing 16 choice-pairs, randomly assigned from a total of 960 choice-pairs. Each pair included 2 QLU-C10D health states and duration. Data were analyzed using conditional logistic regression, parameterized to fit the quality-adjusted life-year framework. Utility weights were calculated as the ratio of each dimension-level coefficient to the coefficient for life expectancy.

Results: A total of 2480 panel members opted in, 2333 (94%) completed at least 1 choice-pair, and 2273 (92%) completed all choice-pairs. Within dimensions, weights were generally monotonic. Physical functioning, role functioning, and pain were associated with the largest utility weights. Cancer-specific dimensions, such as nausea and bowel problems, were associated with moderate utility decrements, as were general issues such as problems with emotional functioning and social functioning. Sleep problems and fatigue were associated with smaller utility decrements. The value of the worst health state was 0.032, which was slightly greater than 0 (equivalent to being dead).

Conclusions: This study provides the US-specific value set for the QLU-C10D. These estimated health state scores, based on responses to the EORTC QLQ-C30 questionnaire, can be used to evaluate the cost-utility of oncology treatments.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1177/0272989X211003569DOI Listing
May 2021

How is quality of life defined and assessed in published research?

Qual Life Res 2021 Aug 1;30(8):2109-2121. Epub 2021 Apr 1.

Faculty of Science, School of Psychology, University of Sydney, Sydney, NSW, Australia.

Purpose: To ensure clarity in communication in the field of quality of life research, and meaningful use of 'quality of life' as a research outcome, requires two things: awareness that there is a range of conceptualisations and definitions of 'quality of life', and for any particular study, consistency between the way the term is defined and operationalised in that setting. We aimed to identify how frequently research articles described (HR)QOL as a construct of interest, how frequently they referred to "patient-reported outcome (measures)", which patient-reported outcome measures were used, and how (HR)QOL was defined.

Methods: We reviewed all Quality of Life Research articles published in 2017 and recorded whether they described health-related quality of life or quality of life as constructs of interest, and/or mentioned the term(s) patient-reported outcome (measures). We recorded definitions of (HR)QOL stated and questionnaires used. We classified articles according to constructs assessed and instruments used, and examined whether articles citing the same definition used the same questionnaires.

Results: We reviewed 300 articles; 65% stated that (HR)QOL was a construct of interest, 27% mentioned patient-reported outcome (measures), and 20% mentioned neither. Fifty-one articles provided definitions of (HR)QOL, citing 66 sources, with 11 definitions cited more than once. PROMIS, SF, EQ-5D, and EORTC instruments were the most commonly used. The only definition and questionnaire consistently used together were the WHO definitions/instruments.

Conclusion: These results demonstrate considerable heterogeneity in the definition and operationalisation of (HR)QOL, between and within studies. This limits meaningful interpretation of (HR)QOL scores and complicates literature searches. Investigators should define constructs and select instruments aligned with their definitions.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11136-021-02826-0DOI Listing
August 2021

The EORTC QLU-C10D was more efficient in detecting clinical known group differences in myelodysplastic syndromes than the EQ-5D-3L.

J Clin Epidemiol 2021 09 20;137:31-44. Epub 2021 Mar 20.

Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center & Health Outcomes Research Unit, Rome, Italy.

Background: The aim was to investigate the relative validity of the preference-based measure EORTC QLU-C10D in comparison with the EQ-5D-3L in myelodysplastic syndromes (MDS) patients.

Methods: We used data from an international multicentre, observational cohort study of MDS patients. Baseline EORTC QLU-C10D and EQ-5D-3L scores were used and index scores calculated for Italy, Australia, and the UK. Criterion validity was established by Spearman and intraclass correlations (ICC) and Bland-Altman plots. Construct validity was established by the instruments' ability to discriminate known groups, i.e. groups whose health status is expected to differ.

Results: We analyzed data from 619 MDS patients (61.1% male; median age 73.8 years). Correlations between theoretically corresponding domains were largely higher than between unrelated domains. ICCs and Bland-Altman plots indicated moderate to good criterion validity. Ceiling effects were lower for the QLU-C10D (4.7%) than for the EQ-5D-3L (22.6%). The EQ-5D-3L failed to discriminate known-groups in two and the QLU-C10D in one of the comparisons; the QLU-C10D's efficiency in doing so was higher in clinical known-groups. Results were comparable between the countries.

Conclusions: The QLU-C10D may be suitable to generate health utilities for economic research in MDS. Responsiveness and minimal important differences need yet to be established.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jclinepi.2021.03.015DOI Listing
September 2021
-->