Patient-reported Outcome Instruments Research Articles

Application of Patient-Reported Outcome Measurements in Adult Tumor Clinical Trials in China: Cross-Sectional Study.

International health policies and researchers have emphasized the value of evaluating patient-reported outcomes (PROs) in clinical studies. However, the characteristics of PROs in adult tumor clinical trials in China remain insufficiently elucidated. This study aims to assess the application and characteristics of PRO instruments as primary or secondary outcomes in adult randomized clinical trials related to tumors in China. This cross-sectional study identified tumor-focused randomized clinical trials conducted in China between January 1, 2010, and June 30, 2022. The ClinicalTrials.gov database and the Chinese Clinical Trial Registry were selected as the databases. Trials were classified into four groups based on the use of PRO instruments: (1) trials listing PRO instruments as primary outcomes, (2) trials listing PRO instruments as secondary outcomes, (3) trials listing PRO instruments as coprimary outcomes, and (4) trials without any mention of PRO instruments. Pertinent data, including study phase, settings, geographic regions, centers, participant demographics (age and sex), funding sources, intervention types, target diseases, and the names of PRO instruments, were extracted from these trials. The target diseases involved in the trials were grouped according to the American Joint Committee on Cancer Staging Manual, 8th Edition. Among the 6445 trials examined, 2390 (37.08%) incorporated PRO instruments as part of their outcomes. Within this subset, 26.82% (641/2390) listed PRO instruments as primary outcomes, 52.72% (1260/2390) as secondary outcomes, and 20.46% (489/2390) as coprimary outcomes. Among the 2,155,306 participants included in these trials, PRO instruments were used to collect data from 613,648 (28.47%) patients as primary or secondary outcomes and from 74,287 (3.45%) patients as coprimary outcomes. The most common conditions explicitly using specified PRO instruments included thorax tumors (217/1280, 16.95%), breast tumors (176/1280, 13.75%), and lower gastrointestinal tract tumors (173/1280, 13.52%). Frequently used PRO instruments included the European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire-30, the visual analog scale, the numeric rating scale, the Traditional Chinese Medicine Symptom Scale, and the Pittsburgh Sleep Quality Index. Over recent years, the incorporation of PROs has demonstrated an upward trajectory in adult randomized clinical trials on tumors in China. Nonetheless, the infrequent measurement of the patient's voice remains noteworthy. Disease-specific PRO instruments should be more effectively incorporated into various tumor disease categories in clinical trials, and there is room for improvement in the inclusion of PRO instruments as clinical trial end points.

CAHQL: A patient reported outcome instrument to assess health-related quality of life in congenital adrenal hyperplasia.

Measuring health-related quality of life (HRQoL) is a crucial aspect of evaluating health care outcomes. Patients with congenital adrenal hyperplasia (CAH) often self-report deficiencies in HRQoL. The aim of our study was to develop a disease-specific patient reported outcome (PRO) instrument to evaluate the HRQoL of patients >16 years old with classic congenital adrenal hyperplasia (CAH). Following the FDA guidelines for developing PRO instruments, we developed a conceptual framework for the instrument. A preliminary instrument was created after interviewing a representative sample of 12 patients with CAH between 16 to 68 years old and 3 parents, and obtaining expert feedback from 4 endocrinologists. The instrument was edited after cognitive interviews with 6 patients. Internal consistency of the instrument was evaluated using Cronbach's alpha. Validity was assessed by comparing the scores of our instrument with scores from widely used validated instruments for HRQoL and PRO not specific to CAH. Sixty-nine patients 16 to 75 years old participated in validating our preliminary instrument. The final questionnaire consists of 44 questions within 7 domains: General Health, Adrenal Insufficiency, Glucocorticoid Excess, Physical Functioning, Mental Health and Cognition, Social Functioning, and Sexual Functioning, with acceptable internal consistency (Chronbach's alpha≥0.6) and validity (r = -0.350 to 0.866). CAHQL is the first validated PRO instrument to capture disease specific HRQoL outcomes in CAH. In addition to its anticipated use in the clinical setting, the instrument could be used to assess the efficacy of novel treatments in development.

Development and initial validation of a disease-specific instrument to measure health-related quality of life in hypersensitivity pneumonitis

Rationale and ObjectiveDisease-specific health-related quality of life (HRQOL) instruments enable us to capture domains that are most relevant to specific patient populations and are useful when a more individualized approach to patient assessment is desired. In this study, we assessed the validity and reliability of the first instrument specifically developed to measure HRQOL in Hypersensitivity Pneumonitis (HP).MethodsA 39-item HP-HRQOL instrument and several anchors were collected from a cohort of patients with HP. Exploratory factor analysis and item reduction were utilized to construct a shortened version of the instrument. Several validity and reliability analyses were conducted on this version of the HP-HRQOL.Measurements and Main Results59 patients with HP completed the study. The revised HP-HRQOL instrument comprises 15 items composing 2 factors (domains): 1. Impacts on daily life; 2. Mental well-being. Internal consistency reliability was strong for factor 1 (Cronbach's alpha=0.94, 95% CI {0.92–0.96}) and factor 2 (Cronbach's alpha= 0.89, 95% CI {0.85–0.94}). Test re-test reliability was strong (ICC 0.94 95% CI {0.89–0.97}). The HP-HRQOL strongly correlated with other validated patient reported outcome measures and moderately correlated with %predicted forced vital capacity. The HP-HRQOL distinguished between those with different severities of HP as determined by lung function and supplemental oxygen use.ConclusionsThe HP-HRQOL, the first patient reported outcome instrument specific to adults with HP, possesses strong validity and reliability characteristics for measuring disease-specific HRQOL and distinguishes among patients with different severities of disease.

Challenges and benefits of using the HeartDiet food frequency questionnaire in cardiac rehabilitation practice

Background and aimsA heart-healthy diet is an important component of secondary prevention in ischemic heart disease. The Danish Health Authority recommends using the validated 19-item food frequency questionnaire HeartDiet in cardiac rehabilitation practice to assess patients’ need for dietary interventions, and HeartDiet has been included in national electronic patient-reported outcome instruments for cardiac rehabilitation. This study aims to evaluate challenges and benefits of its use. The objectives are to: 1) describe HeartDiet responses of patients with ischemic heart disease and discuss HeartDiet’s suitability as a screening tool, 2) discuss whether an abridged version should replace HeartDiet. Methods and resultsA cross-sectional study using data from a national feasibility test. HeartDiet was sent electronically to 223 patients with ischemic heart disease prior to cardiac rehabilitation. Data were summarised with descriptive statistics, and Spearman’s rank correlations, explorative factor analysis, and Cohen’s kappa coefficient were used to derive and evaluate abridged versions.The response rate was 68 % (n=151). Evaluated with HeartDiet, no respondents had a heart-healthy diet. There was substantial agreement between HeartDiet and an abridged 9-item version (kappa = 0.6926 for Fat Score, 0.6625 for FishFruitVegetable Score), but the abridged version omits information on milk products, wholegrain, nuts, and sugary snacks. ConclusionWith the predefined cut-offs, HeartDiet’s suitability as a screening tool to assess needs for dietary interventions was limited, since no respondents were categorised as having a heart-healthy diet. An abridged version can replace HeartDiet, but the tool’s educational potential will be compromised, since important items will be omitted.

How Do Quality of Life (QoL) and Symptom Burden Evolve in Inpatient Palliative Care (PC) Patients following One Week of Care in a Specialized Palliative Care Unit (PCU)? A Comparison of Two Groups, with One Receiving Specialized Outpatient Palliative Care Prior to Admission.

This study sought to investigate changes in quality of life (QoL) and symptom burden among palliative care patients undergoing one week of inpatient care in a specialized palliative care unit (PCU). The patient population was stratified into two groups, with one group pretreated from pre-admission palliative care (PC) provided by an outpatient multidisciplinary PC team, while the other group did not receive such support prior to admission. Although the average duration of treatment at a PCU in Germany is 1-2 weeks, the question also arises as to whether a significant improvement in symptom burden and QoL can be expected after just one week of PC in a PCU. PC patients with various cancer entities were prospectively included in a non-randomized study. Patients in group 1 received outpatient specialized PC prior to admission, while patients in group 2 did not. Over an 8-month period, we gathered data from one academic cancer center, utilizing the EORTC QLQ-C30, one of the most widely used patient-reported outcome (PRO) instruments to assess health-related QoL in cancer patients. Patients completed the QLQ-C30 at T0 (admission or one day later) and T1 (one week later), enabling the assessment of potential changes in their QoL and symptom burden over time. A total of 103 patients (51.5% male) were enrolled (group 1: 42%, group 2: 58%). At T0, there were no significant differences regarding QLQ-C30 scores between groups 1 and 2, except from global health/QoL (group 1 mean 20.7, group 2 mean 25.6, p = 0.026). Over the course of one week several significant and clinically relevant changes were found: Emotional functioning demonstrated an uplift in both groups (group 1: mean 41.5 IQR 33 vs. 53.1 IQR 50, p = 0.014, group 2: mean 48.2 IQR 46 vs. 56.8 IQR 58, p = 0.029), as did the global health status (group 1: M 20.7 IQR 17 vs. 36.2 IQR 33, p < 0.001, group 2: M 25.6 IQR 25 vs. 35.3 IQR 33, p < 0.001). Nausea and vomiting showed a reduction (group 1: M 29.9 IQR 17 vs. 6.8 IQR 0, p < 0.001, group 2: M 22.6 IQR 17 vs. 8.2 IQR 0, p < 0.001), along with a notable decline in pain (group 1: M 67.4 IQR 67 vs. 25.3 IQR 17, p < 0.001, group 2: M 73.1 IQR 83 vs. 29.7 IQR 17, p < 0.001). A decrease was observed in insomnia (group 1: M 63.6 IQR 67 vs. 27.6 IQR 33, p < 0.001, group 2: M 60.1 IQR 67 vs. 27.6 IQR 33, p < 0.001). There were no significant differences between groups 1 and 2 in the extent of improvement in the various symptom scales from T0 to T1. The findings of our study demonstrate that QoL and several symptoms prevalent in cancer patients cared for in the PCU experienced significant enhancement over the span of just one week. Both groups, patients receiving specialized outpatient PC prior to admission and those without, equally benefited from inpatient PC. All mentioned changes from T0 to T1 are considered not only significant but clinically relevant.

Development of a value-based healthcare burns core set for adult burn care

BackgroundValue-based healthcare (VBHC) is increasingly implemented in healthcare worldwide. Transparent measurement of outcomes most important and relevant to patients is essential in VBHC, which is supported by a core set of most important quality indicators and outcomes. Therefore, the aim of this study was to develop a VBHC-burns core set for adult burn patients. MethodsA three-round modified national Delphi study, including 44 outcomes and 24 quality indicators, was conducted to reach consensus among Dutch patients, burn care professionals and researchers. Items were rated on a nine-point Likert scale and selected if ≥70% in each group considered an item ‘important’. Subsequently, instruments quantifying selected outcomes were identified based on a literature review and chosen in a consensus meeting using recommendations from the Dutch consensus-based standard set and the Dutch Centre of Expertise on Health Disparities. Time assessment points were chosen to reflect the burn care and patient recovery process. Finally, the initial core set was evaluated in practice, leading to the adapted VBHC-burns core set. ResultsTwenty-seven patients, 63 burn care professionals and 23 researchers participated. Ten outcomes and four quality indicators were selected in the Delphi study, including the outcomes pain, wound healing, physical activity, self-care, independence, return to work, depression, itching, scar flexibility and return to school. Quality indicators included shared decision-making (SDM), the number of patients receiving aftercare, determination of burn depth, and assessment of active range of motion. After evaluation of its use in clinical practice, the core set included all items except SDM, which are assessed by nine patient-reported outcome instruments or measured in clinical care. Assessment time points included are at discharge, 2 weeks, 3 months, 12 months after discharge and annually afterwards. ConclusionA VBHC-burns core set was developed, consisting of outcomes and quality indicators that are important to burn patients and burn care professionals. The VBHC-burns core set is now systemically monitored and analysed in Dutch burn care to improve care and patient relevant outcomes. As improving burn care and patient relevant outcomes is important worldwide, the developed VBHC-burns core set could be inspiring for other countries.

A Prospective Investigation of Patient Satisfaction and Psychosocial Status Following Facial Bone Contouring Surgery using the Face-Q.

The objective of this study was to assess satisfaction and psychosocial status before and after facial bone contouring surgery using the Face-Q. The Face-Q, a multimodular patient-reported outcome (PRO) instrument, comprises independently functioning scales and checklists designed to assess outcomes in facial aesthetic patients. A prospective cohort study was conducted from November 2020 to May 2022. Participants undergoing facial bone contouring surgery (reduction mandibuloplasty and/or malarplasty) were asked to complete the Face-Q preoperatively and 12months postoperatively. Comparative analyses were conducted using normative Face-Q data from 534 matched normal individuals. Face-Q scores were evaluated for each domain on a scale of 0 to 100, with higher scores indicating greater satisfaction with appearance or a superior quality of life. A total of 284 patients (274 female and 10 male) completed the Face-Q preoperatively and 12 months postoperatively. Of these, 146 underwent reduction mandibuloplasty, 18 underwent malarplasty, and 120 underwent both procedures. Post-surgery, patients experienced significant improvements in overall appearance, features altered by surgery, and quality of life, excluding the patient-perceived age. Preoperatively, patients demonstrated significantly lower scores compared to normative data, with scores significantly increasing postoperatively to levels representative of the general population. Satisfaction with outcome was significantly correlated with postoperative Face-Q measurements but not preoperatively. Facial bone contouring surgery significantly improves the satisfaction and quality of life in patients with square faces, reaching a level at least equivalent to the normative population. The use of Face-Q should be highlighted in the clinic practice. This journal requires that authors assign a level of evidence to each article. For a full description of these Evidence-Based Medicine ratings, please refer to the Table of Contents or the online Instructions to Authors www.springer.com/00266.

Postnasal Drip

Postnasal drip (PND) is a common symptom encountered when evaluating subjects with sinonasal disease; it may be present without sinonasal disease or as a symptom of laryngopharyngeal reflux (LPR). It is postulated that PND stems from dysfunction of normal mucus clearance mechanisms, however, there is no definitive evidence elucidating an exact pathophysiology. Compounding the uncertainties surrounding PND is the lack of an objective assessment tool. Instead, treating physicians must rely on subjective complaints and investigators utilize validated patient reported outcome instruments, such as the sino-nasal outcome test (SNOT), to determine treatment efficacy. This review seeks to discuss the current understanding of PND and evidence regarding effective treatment strategies.

Prospective trial of Functional Lung Avoidance Radiation Therapy for Lung Cancer: Quality of Life Report

PurposeA novel form of lung function imaging has been developed that uses 4DCT data to generate lung ventilation images (4DCT-ventilation). Functional avoidance uses 4DCT-ventilation to reduce doses to functional lung with the aim of reducing pulmonary side-effects. A 4DCT-ventilation functional avoidance, phase II, multi-center clinical trial was completed. The purpose of this work is to quantify patient reported outcomes (PRO) changes for patients treated with functional avoidance and to determine which metrics are predictive of PRO changes. Materials and MethodsPatients with locally advanced lung cancer receiving curative intent radiotherapy were accrued. Each patient had a 4DCT-ventilation image generated using 4DCT data and image processing. PRO instruments included the Functional Assessment of Cancer Therapy-Lung (FACT-L) questionnaire, administered pre-treatment, 3, 6, and 12 months post-treatment. FACT-TOI (Trial Outcome Index) and the FACT-LCS (Lung Cancer Subscale) percentage of clinically meaningful declines (CMD) were determined. A linear mixed-effects model was used to determine which patient, clinical, dose, and dose-function metrics were predictive of PRO decline. Results59 patients completed baseline PRO surveys. 83% of patients had non-small-cell lung cancer, with 75% having stage III disease. The median dose was 60 Gy in 30 fractions. CMD FACT-TOI decline was 46.3%, 38.5%, and 26.8%, at 3, 6, and 12 months, respectively. CMD FACT-LCS decline was 33.3%, 33.3%, and 29.3%, at 3, 6, and 12 months, respectively. While an increase in most dose and dose-function parameters was associated with a modest decline in PROs, none of the results were significant (all p>0.053). ConclusionThe current work provides an innovative combination of functional avoidance and PROs and is the first report of PROs for patients treated with prospective 4DCT-ventilation functional avoidance. Approximately 30% of patients had clinically significant decline in PROs at 12 months. The study provides additional data on outcomes with 4DCT-ventilation functional avoidance.

Translation and validation of the German version of the FACE-Q paralysis module in adult patients with unilateral peripheral facial palsy

The aim was to develop and validate a German version of the FACE-Q paralysis module, a patient-reported outcome measure to assess health-related quality of life in adult patients with unilateral facial palsy. The FACE-Q craniofacial questionnaire, which includes the paralysis module, was translated. 213 patients with facial palsy completed the German FACE-Q paralysis along with the established FDI and FaCE questionnaires. Regression analyses were performed to examine the relationships between the different FACE-Q domains and patient and therapy characteristics. The FACE-Q scales had high internal consistency (Cronbach’s alpha all > 0.6). High correlations were found between the FACE-Q and the FDI and FaCE (mean rho = 0.5), as well as within the FACE-Q (mean rho = 0.522). Unifactorial influences were found for all domains except Breathing (all p < 0.05). Multivariate independent predictors were found for some FACE-Q domains. Most influential predictors (> 8 subdomains): Patients who received physical therapy scored lower in ten subdomains than those who did not (all p < 0.05). Patients who had surgery scored lower in nine subdomains than patients without surgery (all p < 0.05). The German version of the FACE-Q Paralysis Module can now be used as a patient-reported outcome instrument in adult patients with facial nerve palsy.

Understanding the Symptoms and Impacts Experienced by People with Relapsing-Remitting MS: A Qualitative Investigation Using Semi-Structured Interviews.

Multiple sclerosis (MS) is a disabling disease with unpredictable clinical manifestations. As clinical assessments may not fully capture the impact of MS on quality of life, they can be complemented by patient-reported outcome (PRO) measures to provide a more comprehensive picture of MS disease state and impact. The objectives of this study were to explore the experiences of people with relapsing-remitting MS, including symptoms and impacts on daily life, and to provide a conceptual model of MS outcomes. A literature review of studies that evaluated the experiences of people with MS was completed and combined with semi-structured concept elicitation interviews conducted with 14 people with relapsing-remitting MS in the USA. The average age of the 14 participants was 43.9 (range 25-64) years, most were White (78.6%) and female (78.6%), and the mean duration since diagnosis was 6.6 (2-10) years. The most bothersome symptoms identified included fatigue (n = 9), cognitive dysfunction (n = 5), mobility/difficulty with walking (n = 3), and vision problems (n = 3). The most commonly reported impacts on daily life were balance problems/instability (n = 13), work life/productivity (n = 12), difficulty walking (n = 11), daily activities/household chores (n = 11), and leisure activities (n = 10). There was a high frequency of concepts associated with physical function, fatigue, and sensory-motor actions. A conceptual model was developed that captures the disease symptoms, impairments, and impacts identified in the interviews as well as known processes and symptoms identified in the literature search. This model underpins the appropriateness of PRO instruments, such as the PROMIS Fatigue (MS) 8a and PROMIS Physical Function (MS) 15a, which evaluate symptoms and impacts that matter most to people with MS.

Estimating meaningful change thresholds for Skin Pain-Numeric Rating Scale, Sleep-Numeric Rating Scale and Dermatology Life Quality Index in patients with prurigo nodularis.

Prurigo nodularis (PN) is characterized by intensely itchy nodules/lesions and skin pain, which can have a substantial impact on health-related quality of life (HRQoL). Treatment benefits on such symptoms and impacts are best assessed in trials using patient-reported outcome (PROs) instruments such as Skin Pain Numerical Rating Scale (NRS), Sleep-NRS and Dermatology Life Quality Index (DLQI). However, no guidance exists for interpreting meaningful changes in scores using these PROs in patients with PN. The main objective was to derive within-patient (responder definition) and between-group improvement thresholds for interpreting Skin Pain-NRS, Sleep-NRS and DLQI total scores in patients with PN. The measurement properties of the three PROs were also evaluated. Intention-to-treat (ITT), blinded and pooled data were used from the Phase 3 PRIME (NCT04183335) and PRIME2 (NCT04202679) studies evaluating the efficacy of dupilumab in adult patients with PN. Anchor- and distribution-based methods were applied to derive responder definition and between-group thresholds for Skin Pain-NRS, Sleep-NRS and DLQI. Data were additionally used to examine the instrument measurement properties, including reliability, validity and responsiveness. A total of 311 patients (mean age 49.5 years, 65.3% female) were included in the pooled ITT population. The within-patient improvement threshold for Skin Pain-NRS was estimated as 4.0 points, 2.0 points for Sleep-NRS and 9.0 points for DLQI total score. A 1.5-point improvement in Skin Pain-NRS scores, 1.0-point in Sleep-NRS and 4.0-point in DLQI indicated a between-group meaningful change. Adequate to good psychometric properties were demonstrated for all three instruments. The results of this study can aid interpretation of Skin Pain-NRS, Sleep-NRS and DLQI scores in patients with PN in both clinical trials and clinical practice to better understand and treat PN-related skin pain and the impact of PN on sleep quality and HRQoL.

Development of the Japanese version of the Ego-Dissolution Inventory (EDI).

Psychedelics have recently gained attention as potential therapeutic agents for various psychiatric disorders. Previous research has highlighted that a diminished sense of self, commonly termed "ego-dissolution" is a pivotal feature of the psychedelic-induced state. While the Ego-Dissolution Inventory (EDI) is a widely acknowledged instrument for measuring this phenomenon, no Japanese version has been available. This study aimed to develop a Japanese version of the EDI. We adhered to the "Guidelines for Best Practices in the Translation and Cultural Modification Process for Patient-Reported Outcomes Instruments: Document from the ISPOR Committee on Translation and Cultural Modification" during our translation approach. Two Japanese psychiatrists independently conducted initial translations, and a consolidated version was achieved via mutual agreement. This version was then back-translated to English and assessed by the original authors for consistency. The repetitive modification process was conducted in continuous dialogues with the original authors until they accepted the concluding back-translated version. The finalized, approved back-translated version of the EDI is presented in the accompanying figure. In addition, the authorized Japanese version of the EDI is included in the Appendix. In this study, we successfully developed the Japanese version of the EDI. This instrument will assist in assessing ego-dissolution experiences associated with psychedelic-assisted therapy among Japanese speakers. Additional studies are necessary to evaluate the reliability and validity of this newly translated instrument.

An examination of rectal prolapse surgery patients’ quality of life and symptoms using patient-reported outcome instruments: A prospective cohort study

BackgroundWe measured changes in self-reported health and symptoms attributable to rectal prolapse surgery using patient-reported outcome (PRO) measures. MethodsA prospectively recruited cohort of patients scheduled for rectal prolapse repair in Vancouver, Canada between 2013 and 2021 were surveyed before and 6-months after surgery using seven PROs: the EuroQol Five-Dimension Instrument (EQ-5D-5L), Generalized Anxiety Disorder Scale (GAD-7), Pain Intensity, Interference with Enjoyment of Life and General Activity (PEG), Patient Health Questionnaire (PHQ-9), Fecal Incontinence Severity Index (FISI), Gastrointestinal Quality of Life Index (GIQLI), and the Fecal Incontinence Quality of Life Scale (FIQL). ResultsWe included 46 participants who reported improvements in health status (EQ-5D-5L; p ​< ​0.01), pain interference (PEG; p ​< ​0.01), depressive symptoms (PHQ-9; p ​= ​0.01), fecal incontinence severity (FISI; p ​< ​0.01), gastrointestinal quality of life (GIQLI; p ​< ​0.01), and fecal incontinence quality of life (FIQL) related to lifestyle (p ​= ​0.02), coping and behaviour (p ​= ​0.02) and depression and self-perception (p ​= ​0.01). ConclusionSurgical repair of rectal prolapse improved patients’ quality of life with meaningful improvements in fecal incontinence severity and pain, and symptom interference with daily activities.

Patient-Reported Outcomes After Vestibular Implantation for Bilateral Vestibular Hypofunction

Standard-of-care treatment proves inadequate for many patients with bilateral vestibular hypofunction (BVH). Vestibular implantation is an emerging alternative. To examine patient-reported outcomes from prosthetic vestibular stimulation. The Multichannel Vestibular Implant (MVI) Early Feasibility Study is an ongoing prospective, nonrandomized, single-group, single-center cohort study conducted at Johns Hopkins Hospital that has been active since 2016 in which participants serve as their own controls. The study includes adults with severe or profound adult-onset BVH for at least 1 year and inadequate compensation despite standard-of-care treatment. As of March 2023, 12 candidates completed the eligibility screening process. The MVI system electrically stimulates semicircular canal branches of the vestibular nerve to convey head rotation. Patient-reported outcome instruments assessing dizziness (Dizziness Handicap Inventory [DHI]) and vestibular-related disability (Vestibular Disorders-Activities of Daily Living [VADL]). Health-related quality of life (HRQOL) assessed using the Short Form-36 Utility (SF36U) and Health Utilities Index Mark 3 (HUI3), from which quality-adjusted life-years were computed. Ten individuals (5 female [50%]; mean [SD] age, 58.5 [5.0] years; range, 51-66 years) underwent unilateral implantation. A control group of 10 trial applicants (5 female [50%]; mean [SD] age, 55.1 [8.5] years; range, 42-73 years) completed 6-month follow-up surveys after the initial application. After 0.5 years of continuous MVI use, a pooled mean (95% CI) of within-participant changes showed improvements in dizziness (DHI, -36; 95% CI, -55 to -18), vestibular disability (VADL, -1.7; 95% CI, -2.6 to -0.7), and HRQOL by SF36U (0.12; 95% CI, 0.07-0.17) but not HUI3 (0.02; 95% CI, -0.22 to 0.27). Improvements exceeded minimally important differences in the direction of benefit (exceeding 18, 0.65, and 0.03, respectively, for DHI, VADL, and SF36U). The control group reported no mean change in dizziness (DHI, -4; 95% CI, -10 to 2), vestibular disability (VADL, 0.1; 95% CI, -0.9 to 1.1) or HRQOL per SF36U (0; 95% CI, -0.06 to 0.05) but an increase in HRQOL per HUI3 (0.10; 95% CI, 0.04-0.16). Lifetime HRQOL gain for MVI users was estimated to be 1.7 quality-adjusted life-years (95% CI, 0.6-2.8) using SF36U and 1.4 (95% CI, -1.2 to 4.0) using HUI3. This cohort study found that vestibular implant recipients report vestibular symptom improvements not reported by a control group. These patient-reported benefits support the use of vestibular implantation as a treatment for bilateral vestibular hypofunction.

Meaningful score changes for SF-36v2, FACIT-fatigue, and RASIQ in rheumatoid arthritis

BackgroundInterpretation thresholds for patient-reported outcome (PRO) scores are of crucial importance, particularly when interpreting treatment benefit. This study was designed to determine the within-patient meaningful improvement (WPMI) thresholds for the Short-Form 36 Health Survey version 2 (SF-36v2), the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and the novel Rheumatoid Arthritis Symptoms and Impact Questionnaire (RASIQ) among patients with rheumatoid arthritis (RA).MethodsIn this post-hoc analysis, anchor-based and supportive distribution-based methods were used to derive WPMI based on blinded data from all treatment arms in two Phase 2 RA trials with otilimab. Patient’s Global Assessment of Disease Activity (PtGA) was the general anchor for all SF-36v2 scales. SF-36 Patient’s Global Impression of Status (PGIS), PtGA, and VT03 (an SF-36v2 item) were used as anchors for FACIT-Fatigue. SF-36 PGIS, PtGA, and Patient’s Assessment of Arthritis Pain (PAIN) were anchors for RASIQ. Mean change was calculated for the anchor category associated with minimal meaningful improvement from baseline to Week 24 for SF-36v2 and FACIT-Fatigue, and to Week 12 for RASIQ. Sensitivity and specificity were used to evaluate the accuracy of estimated WPMI values.ResultsFor the SF-36v2 physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health domains, anchor-based estimates of WPMI based on 0–100 scores were 24.5, 24.5, 25.4, 13.6, 21.5, 20.5, 16.9, and 14.3, respectively. Anchor-based WPMI estimates were 9.7 for the Physical Component Summary score and 7.6 for the Mental Component Summary score (using norm-based T-score metric). For FACIT-Fatigue (range 0–52), WPMI estimates ranged from 9.7 to 11.3 points. For RASIQ (range 0–100), anchor-based WPMI was determined as a change between -32.7 and -21.7 points for the Joint Pain scale, -26.7 to -23.7 for the Joint Stiffness scale, and -21.1 to -17.4 for the Impact scale.ConclusionsThis study derived WPMI thresholds for SF-36v2, FACIT-Fatigue, and RASIQ among patients with RA, using multiple anchors. Derivation of WPMI thresholds for these PRO instruments will enable their broader use in evaluating and interpreting treatment benefit in future RA studies.

Exploring health preference heterogeneity in the UK: Using the online elicitation of personal utility functions approach to construct EQ-5D-5L value functions on societal, group and individual level.

A new method has recently been developed for valuing health states, called 'Online elicitation of Personal Utility Functions' (OPUF). In contrast to established methods, such as time trade-off or discrete choice experiments, OPUF does not require hundreds of respondents, but allows estimating utility functions for small groups and even at the individual level. In this study, we used OPUF to elicit EQ-5D-5L health state preferences from a (not representative) sample of the UK general population, and then compared utility functions on the societal-, group-, and individual level. A demo version of the survey is available at: https://eq5d5l.me. Data from 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. These personal value sets predicted respondents' choices in three hold-out discrete choice tasks with an accuracy of 78%. Overall, preferences varied greatly between individuals. However, PERMANOVA analysis showed that demographic characteristics explained only a small proportion of the variability between subgroups. While OPUF is still under development, it has important strengths: it can be used to construct value sets for patient reported outcome instruments such as EQ-5D-5L, while also allowing examination of underlying preferences in an unprecedented level of detail. In the future, OPUF could be used to complement existing methods, allowing valuation studies in smaller samples, and providing more detailed insights into the heterogeneity of preferences across subgroups.

Health-related quality of life (HRQoL) in patients with metastatic colorectal cancer (mCRC) treated with sotorasib and panitumumab (pmab) versus trifluridine/tipiracil (T/T) or regorafenib (rego) in CodeBreaK 300.

10 Background: CodeBreaK 300 (NCT05198934) is a phase 3, multicenter, randomized, open-label study, evaluating sotorasib 960 mg (soto960) + pmab, sotorasib 240 mg (soto240) + pmab against investigator’s choice T/T or rego in patients with chemorefractory KRAS G12C-mutated mCRC. The study met its primary endpoint and demonstrated statistically significant improvement in progression-free survival with both doses of sotorasib. This analysis evaluated patient-reported outcomes (PROs) as secondary and exploratory endpoints in CodeBreaK 300. Methods: Change in PRO scores from study baseline through week 8 was compared for soto960+pmab and soto240+pmab vs the control group using a mixed effect model for repeated measures. PRO instruments included the Brief Fatigue Inventory, the Brief Pain Inventory, and the European Organisation for Research and Treatment of Cancer Core 30-item Quality of Life questionnaire. Time to deterioration (TTD) were assessed using Kaplan-Meier plots and Cox proportional hazard models. All analyses were conducted on patients with baseline and at least one post-baseline PRO score. Results: 160 pts were randomized 1:1:1 to soto960+pmab (n=53), soto240+pmab (n=53), or T/T or rego (n=54). Compliance rates for PRO assessments were high (&gt;83%) and similar across the three treatment groups. Least square (LS) mean changes from baseline in fatigue at its worst, pain at its worst, physical functioning (PF), and global health status (GHS)/QoL favored the two sotorasib dose groups (Table). The 95% confidence intervals (CI) suggested improvement in pain at its worst and PF for both sotorasib dose groups, and improvement in GHS/QoL for the soto960+pmab group versus the control group. TTD hazard ratios &lt; 1 indicated a trend in delayed deterioration for patients treated in both sotorasib dose groups. The 95% CIsuggested delays with respect to fatigue at its worst and PF for the soto240+pmab group. Conclusions: In CodeBreaK 300, both doses of sotorasib resulted in better HRQoL and a trend to decreased risk of deterioration vs the control group. In addition to improved clinical outcomes, these findings provide further evidence on the benefit of soto+pmab in patients with chemorefractory mCRC. Clinical trial information: NCT05198934 . [Table: see text]

Long-term patient-reported outcomes from monarchE: Abemaciclib plus endocrine therapy as adjuvant therapy for HR+, HER2-, node-positive, high-risk, early breast cancer

BackgroundIn monarchE, abemaciclib demonstrated a sustained benefit in invasive disease-free survival and a tolerable safety profile at 42-months median follow-up. With no expected disease-related symptoms, therapies in the adjuvant setting should preserve quality of life (QoL). With all patients off abemaciclib, we report updated patient-reported outcomes (PROs) for the full 2-year treatment period and follow-up. MethodsPatients completed PROs including FACT-B, FACT-ES, and FACIT-Fatigue at baseline, 3, 6, 12, 18, and 24 months during treatment, and 1, 6, and 12 months after treatment discontinuation. Mixed effects repeated measures model estimated changes from baseline within and between arms for QoL scales and individual items. Meaningful changes were prespecified and no statistical testing was performed. Frequencies of responses to items associated with relevant adverse events and treatment bother were summarized. ResultsAt baseline, completion rates for PRO instruments were >96 %. Mean changes from baseline for all QoL scales were numerically similar within and between arms (ie, less than prespecified thresholds). The same was observed for all individual items, except diarrhea. Within abemaciclib arm, meaningful differences for diarrhea were observed at 3 and 6 months (mean increases of 1.19 and 1.03 points on 5-point scale, respectively). During treatment, most patients in both arms (69–78 %) reported being bothered “a little bit” or “not at all” by side effects. Overall, patterns for fatigue were similar between arms. During post-treatment follow-up, PROs in both arms were similar to baseline. ConclusionPRO findings confirm a tolerable and reversible toxicity profile for abemaciclib. QoL was preserved with the addition of adjuvant abemaciclib to endocrine therapy, supporting its use in patients with HR+, HER2-, high-risk early breast cancer.

The Symptom Experience of Patients with Advanced Melanoma Undergoing Immune Checkpoint Inhibitor (ICI) Therapy

ObjectivesThe advent of immune checkpoint inhibitor (ICI) therapy has vastly improved outcomes for patients with advanced melanoma. However, the symptom burden and intensity with their impact on quality-of-life (HRQoL) and functionality are heterogeneous and unpredictable. We used descriptive exploratory content analysis from interviews to capture the patient experience after they had completed quantitative data collection of their symptom burden and interference with the use of two patient-reported outcome (PRO) instruments. Data SourcesParticipants from a single center with advanced melanoma (n = 19) who are undergoing ICI therapy completed the Modified MD Anderson Symptom Inventory and Functional Assessment of Cancer Therapy-Melanoma and recorded semistructured interviews. Interpretive description informed the inductive and iterative analysis approach. ConclusionParticipants had a heterogenous experience of ICI and melanoma-related symptoms: distress (84%), fatigue (68%), rash or skin changes (53%), pain (30%), diarrhea (30%), itching (26%), and shortness of breath (21%), with varying interference within HRQoL domains, mood (47%), relations with other people (26%), and activity (21%). Some noted a lack of physical interference (79%). Uncertainty was a pervasive theme in the interviews (68%) despite the majority having positive thoughts about ICI therapy (58%) and expectations of the success of therapy (53%). The physical and emotional burden of a melanoma diagnosis, undergoing therapy, and the uncertainty of the outcomes are pervasive for patients. Implications for Nursing PracticeCommunication surrounding the diagnosis, prognosis, treatment options, and outcomes need to be clear and acknowledge there are unknowns. Nurses may benefit from using a validated PRO instrument to help document and understand the patient's symptom experience while undergoing ICI therapy.