Valid Patient-reported Outcome Measure Research Articles

Guidelines for the development and validation of patient-reported outcome measures: a scoping review

ObjectiveThe objectives of this scoping review were to provide an overview of existing guidelines for the development and validation of patient-reported outcome measures (PROMs), review them for comprehensiveness and clarity...

Abstract PO2-19-12: Enhancing skin appearance and quality of life in breast cancer survivors on aromatase inhibitor therapy

Abstract Background: Post-menopausal women with estrogen receptor positive breast cancer (BC) are typically prescribed endocrine therapy with an aromatase inhibitor (AI) for 5-10 years following BC diagnosis. AIs have multiple established side effects related to estrogen deprivation including vasomotor symptoms, vaginal dryness, and accelerated bone thinning. Many patients also complain about the impact of AIs on skin quality; however, limited data currently exists, particularly with objective measurements of skin quality. Canfield Sciences VISIA-CA is a device that can be used to objectively measure skin changes using patented comparison to norms analysis with a skin feature database to grade the skin relative to others in the same age and skin type. Herein, we aim to evaluate changes in skin quality with the Canfield Sciences VISIA-CA Device after initiation of AI therapy and investigate potential benefits of skincare intervention. Trial Design: This is a pilot study aiming to describe changes in skin quality after initiation of AI therapy in BC survivors through use of VISIA-CA technology and patient reported outcome measures. This study will allow us to collect data and identify the trend (if any) for a future larger study. Patients who consent for participation are given a baseline survey to assess self-reported quality of life, perception of skin quality, and self-image prior to initiation of AI. Patients also receive baseline skin measurement with the VISIA-CA device and facial skin photography. Baseline skincare regimen is documented. Any patients on prescription tretinoin products at time of baseline assessment are excluded from the study. Patients return 6 months after initiation of AI for follow-up surveys, updated VISIA-CA measurement, and facial skin photography. Patients then meet with a Mayo Clinic Cosmetic Center aesthetician for skincare intervention with recommended topical treatments including daily facial cleanser, exfoliator, toner, hydrating crème, and retinol-based product. Patients return for additional visits with updated survey, VISIA-CA measurement, and facial skin photography at 1 month, 3 months, and 6 months after skincare intervention. Patients are asked to complete skincare compliance diary to document adherence on a weekly basis. Eligibility Criteria: Post-menopausal patients with newly diagnosed ER-positive BC at Mayo Clinic Florida are identified for participation in the study prior to beginning AI therapy. Specific Aims: 1.) Evaluate the incidence and severity of skin changes after initiation of AI therapy in BC survivors through use of Canfield Sciences VISIA-CA technology, facial skin photography, and validated patient reported outcome measures. 2.) Assess for objective improvements in skin quality measurements after skincare intervention including a retinol-based product Statistical Methods: Continuous variables will be summarized as mean (standard deviation) or median (range) and categorical variables will be reported as frequency (percentage). The changes of skin measurement and patient reported outcome measurements will be calculated and summarized for the following time frames: 1) from initiation of AI therapy to 26 weeks after 2) from the initiation of skincare intervention and 4 weeks, 12 weeks, and 26 weeks after. Appropriate statistical plots will be used to illustrate the change. Target Accrual: 50 patients. Schedule of Events Citation Format: Lauren Cornell, Leila Tolaymat, Pooja Advani, Saranya Chumsri, Pincelli Thais. Enhancing skin appearance and quality of life in breast cancer survivors on aromatase inhibitor therapy [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-19-12.

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.

Development and Initial Validation of a Meniere's Disease Quality of Life Instrument: The MenQOL.

To design and validate a disease-specific quality of life instrument for Meniere's disease. We used a sequential process of expert input, patient focus groups, and analyses of responses to draft questionnaires to create a 24-item Meniere's disease quality of life (MenQOL) instrument. The MenQOL and the SF-36v2 were administered to a cohort of 50 patients with Meniere's disease and 60 comparison patients with tinnitus, vertigo, or hearing loss from other causes identified at a tertiary academic center. We performed exploratory factor analysis, Cronbach's α, between group comparisons of total MenQOL scores, and regression analyses between the MenQOL and SF-36v2 to evaluate the instrument's factor structure, internal consistency, face validity, and external validity. Segregation of the instrument into domains was assessed by exploratory factor analysis. Exploratory factor analysis revealed that the MenQOL has a single domain. Cronbach's α = 0.914 indicated high internal consistency for the instrument as a whole. Mean MenQOL scores showing significantly worse quality of life among patients with Meniere's disease than comparison participants (52.5 ± 15.8 vs. 43.2 ± 12.6; p = 0.0051), indicating good construct validity. Significant inverse relationships in bivariate linear regressions between total MenQOL scores and SF-36v2 physical (slope = -0.94, p < 0.0001) and mental (slope = -1.16, p < 0.0001) composite scores showed acceptable concurrent validity. We have described the initial development of the MenQOL, a simple, valid patient-reported outcome measure that, subject to further study, may be used to assess the effects of treatment on disease-specific quality of life in patients with Meniere's disease. 3 Laryngoscope, 2024.

The construct validity and internal consistency of QuickDASH in pediatric patients with upper extremity fractures.

Investigation of treatment options in the pediatric population necessitates the use of valid patient-reported outcome measures (PROMs). We aimed to assess the construct validity and internal consistency of the Quick Disabilities of the Arm, Shoulder and Hand (QuickDASH) in the pediatric population with upper extremity fractures treated both operatively and conservatively. QuickDASH, along with several reference PROMs and objective outcome measures, was obtained from 148 5- to 18-year-old patients with a humeral medial epicondyle fracture or a fracture of the distal forearm in a cross-sectional setting with a single follow-up visit. Spearman's rank correlation and linear regression models were used to assess convergent validity, exploratory factor analysis (EFA) to assess structural validity, and Cronbach's alpha to investigate internal consistency. The direction and magnitude of correlation showed by QuickDASH with reference outcome measures was consistent and demonstrated good convergent validity. EFA indicated a 3-factor model with poor fit indices and structural validity remained questionable. Construct validity was considered acceptable overall. QuickDASH demonstrated good internal consistency with an acceptable Cronbach's alpha (α = 0.75). QuickDASH demonstrated acceptable construct validity and good internal consistency and is thus a valid instrument, with some limitations, to assess disability and quality of life in pediatric patients with upper extremity fractures.

A Comparison of PROMIS Scores in Patients Treated with First Metatarsophalangeal Joint Arthrodesis and Polyvinyl Alcohol Hydrogel Implant Hemiarthroplasty for Hallux Rigidus

Introduction/Purpose: Current literature shows similar clinical outcomes between first metatarsophalangeal joint arthrodesis and polyvinyl alcohol hydrogel (synthetic cartilage implant or SCI) hemiarthroplasty in the treatment of hallux rigidus; however, prior studies have not reported validated patient-reported outcome measures. To our knowledge, this is the first study to compare patient-reported outcome measures using the validated Patient Reported Outcomes Measurement Information System (PROMIS) in patients treated for hallux rigidus with metatarsophalangeal joint arthrodesis and SCI hemiarthroplasty. In addition, this novel study provides comparative data of the complication and revision rates for each procedure. Methods: A retrospective review of prospectively collected data within an institutional registry identified 101 patients who underwent metatarsophalangeal joint arthrodesis and 82 patients who underwent SCI hemiarthroplasty for treatment of hallux rigidus between January 2016 and June 2022. Chart review was performed to obtain demographic information, complications, and revision rates. Preoperative, 1-year, and 2-year PROMIS scores were obtained from the registry and confirmed via chart review. Equivalence testing was performed using two one sided t-tests (TOST) and setting a +/-5 unit margin of difference as the clinically meaningful difference to determine if there was a difference in PROMIS scores between groups. Linear regression models were also utilized to compare adjusted postoperative PROMIS scores between the two cohorts. For the equivalence testing, a p-value &gt; 0.05 indicated a statistically significant result. Results: Demographic information and preoperative hallux rigidus severity between the cohorts showed no statistically significant difference (Table 1). The SCI cohort had no intra-operative or post-operative wound complications, but one patient within the MTP fusion cohort was treated for a superficial wound infection. The SCI group had six patients revised to MTP fusions within 3- years due to continued pain and/or implant subsidence. The MTP fusion group had 2 patients revised due to non-union and 13 patients that had symptomatic hardware requiring removal. Equivalence comparison of PROMIS scores showed the SCI cohort had significantly worse pain intensity scores at 2 years and significantly less improvement in pain intensity scores from pre- operative to 1-year post-op (Table 2). There were no differences found between cohorts for other PROMIS domains. Conclusion: For the treatment of hallux rigidus, the SCI hemiarthroplasty and metatarsophalangeal joint arthrodesis have equivalent outcomes for all PROMIS domains except the pain intensity domain. While patients in both cohorts had improvement in pain from pre-operative to post-operative time points, SCI was not as effective as first MTP fusion at relieving pain intensity at a follow-up of 2 years. While SCI is a motion-sparing procedure, patients with a primary goal of improving pain may be better suited for first MTP joint arthrodesis. Tables demonstrating the comparisons of both the preoperative demographic data as well as demonstrating the PROMIS scores across different time periods (preoperative, 1 year and 2 years).

The influence of patient-reported outcome measures on healthcare delivery: A review of methodologies and applications

Patient-Reported Outcome Measures (PROMs) have emerged as integral tools in assessing healthcare outcomes from the patient's perspective. This comprehensive review explores the methodologies employed in the development, administration, and validation of PROMs, shedding light on their applications and influence on healthcare delivery. It examines the various modes of PROMs administration, including the burgeoning role of technology through electronic platforms and mobile applications. Challenges related to patient adherence, engagement, and disparities in technological access are discussed, offering insights into potential barriers. It meticulously outlines the multifaceted applications of PROMs in healthcare. From influencing clinical decision-making and quality of care assessments to fostering patient-centered care and contributing to health policy initiatives, PROMs have permeated diverse aspects of healthcare delivery. The review also explores the role of PROMs in shaping research endeavors, serving as essential components in health policy initiatives and advancing the understanding of patient outcomes and experiences. While emphasizing the positive impact of PROMs, the paper addresses challenges and considerations associated with their use, including standardization issues, interpretation challenges, and technology-related barriers. The future directions and opportunities section examines potential advancements in personalized medicine, the integration of technology, and the empowerment of patients through increased involvement in their healthcare journey. Ethical considerations surrounding privacy, confidentiality, and informed consent in PROMs implementation are also scrutinized. This review consolidates current knowledge on PROMs methodologies and applications, offering a comprehensive understanding of their influence on healthcare delivery. The paper emphasizes the need for ongoing research, standardization efforts, and the ethical use of PROMs as they continue to shape a patient-centric and personalized approach to healthcare.

Comparing patient-reported outcomes of the Oxford Ankle and Foot Questionnaire in children with clubfoot from two different geographic and cultural environments: a cohort study in India and Canada.

The purpose of this study is to compare the results of the Oxford Ankle and Foot Questionnaire (OxAFQ) in children with clubfoot in Canada and India to assess its ability to predict outcomes and capture patient experiences in different cultural contexts. This is a retrospective study of children with clubfoot in India and Canada who completed the OxAFQ. Statistical analyses were implemented on registry-collected data to test for independent predictors of poor outcomes and compare scores between countries, among children and their parents, and in Canada where relapse data was collected and the effect of a relapse on scores. A total of 361 children were included. The mean Indian OxAFQ scores were higher (p < 0.001) in all domains. Pirani score, tenotomy, laterality, and age at presentation were found to be predictive of outcomes between the sites (p < 0.05). OxAFQ scores decreased after relapse for children in Canada (p < 0.05). Canadian children were found to generally have lower OxAFQ scores in all domains compared to their parents (n = 95; Z = -3.178, -3.493, -3.353, and -3.635 for physical, school and play, emotion, and footwear, respectively; p < 0.001). Indian parents and children showed no significant differences in their scores. A difference was observed in scores between both sites, suggesting there may be differences in how these populations assess personal health outcomes. These findings support the need for cultural validity of patient-reported outcome measures.

Screening for Palliative Care Need in Oncology: Validation of Patient-Reported Outcome Measures

ContextLeading oncology societies recommend monitoring symptoms and support needs through patient-reported outcome measures (PROMs), but their use for assessing specialist palliative care (SPC) need has not yet been explored. Research on SPC integration has focused on staff-assessed screening tools, which are time-consuming. ObjectivesThis study aimed to assess the diagnostic validity of the Integrated Palliative Outcome Scale (IPOS) and NCCN Distress Thermometer (NCCN DT) in identifying need for SPC in patients with incurable cancer. MethodsIn a cross-sectional study, patients with incurable cancer (prognosis <2 years) completed PROMs. In an independent process, the palliative care consultation service (PCCS) assessed the need for SPC in each patient through multiprofessional case review, and this was used as the reference standard. ROC analyses were employed to determine diagnostic validity. ResultsOf the 208 participants, 71 (34.1 %) were classified as having SPC need by the PCCS. Aiming for a minimum sensitivity of 80%, a cut-off of ≥2 items with high/very high burden in the IPOS resulted in a 90.2% sensitivity (specificity = 50; AUC = 0.791; CI 95%= 0.724–0.858). A cut-off of ≥5 resulted in a sensitivity of 80 % for NCCN DT (specificity = 49.5 %; AUC = 0.687; CI 95% = 0.596–0.777). ConclusionPROMs are useful for identifying SPC need in cancer patients. Their implementation might facilitate timely integration of SPC. Future research should focus on an integrated assessment approach with PROMs that combines the requirements of the different specialties to save patient and staff resources.

Patient-Reported Outcomes in Venous Thromboembolism: A Systematic Review of the Literature, Current Challenges, and Ways Forward.

Venous thromboembolism (VTE) affects >1.2 million Americans annually. Although the clinical outcomes and economic burdens of VTE have been well described, the impact of VTE on patients' health status has yet to be summarized. This systematic review summarizes how patient-reported outcome measures (PROMs) have been used in VTE to date. PubMed/MEDLINE was queried for literature published through March 2023 using PROMs in a population of patients with VTE. Studies were excluded if the reference was an editorial, review, or case report, or if the study included patients with conditions other than VTE. Qualitative analyses were performed. After screening and exclusion, 136 references were identified; 5 described PROM development, 20 focused on PROM validation, and 111 used PROMs in outcomes research. The most used generic PROMs were the 36-item Short-Form Health Survey and EuroQol 5-dimensional questionnaire, and the most common disease-specific PROMs were the Venous Insufficiency Epidemiological and Economic Study-Quality of Life/Symptoms and the Pulmonary Embolism Quality of Life Questionnaire. PROMs were used to quantify the changes in health status after diagnosis, characterize the trajectory of subsequent improvement, and identify drivers of continued impairments in health status like postthrombotic syndrome and postpulmonary embolism syndrome. PROMs were also used to investigate the impact of novel treatment modalities on quality of life. This review demonstrates the many benefits of PROM use, including quantifying changes in health status with treatment, capturing patients' experiences with the treatment itself, and identifying complications of VTE. Incorporating PROMs into VTE care will be an essential component of evaluating the effectiveness of novel therapies and should lead to improved shared decision-making for patients with VTE.

Quality of patient- and proxy-reported outcomes for children with impairment of the lower extremity: A systematic review using the COnsensus-based Standards for selection of health Measurement INstruments methodology.

Patient-reported outcome measures have become crucial in the clinical evaluation of patients. Appropriate selection, in a young population, of the instrument is vital to providing evidence-based patient-centered healthcare. This systematic review applies the COnsensus-based Standards for selection of health Measurement INstruments methodology to provide a critically appraised overview of patient-reported outcome measures targeted at pediatric orthopedic patients with lower limb impairment. A systematic search of electronic databases was performed to identify original studies reporting the development and/or validation of patient-reported outcome measures evaluating children with impairment of the lower extremity. Data extraction, quality assessment, and risk of bias evaluation were performed following the COnsensus-based Standards for selection of health Measurement INstruments guidelines and Preferred Reporting Items for Systematic reviews and Meta-Analyses statement. A total of 6919 articles were screened. Thirty-three studies were included, reporting evidence on the measurement properties of 13 different patient-reported outcome measures and 20 translations. Four studies reported on content validity and patient-reported outcome measure development. The methodological quality of studies on structural validity, content validity, or patient-reported outcome measure development was mostly rated as "doubtful" or "very good." The quality of evidence on measurement properties varied noticeably, with most studies needing to perform improve their methodological quality to justify their results. This review provides an extensive overview of all available patient-reported outcome measures for patients with lower extremity impairment within pediatric orthopedics. We cautiously advise the use of four patient-reported outcome measures. However, the scarce availability of research on content validity and patient-reported outcome measure development highlights an area for future research endeavors to improve our knowledge on the currently available patient-reported outcome measures. Diagnostic level 1.

The Validity of Patient-Reported Outcome Measures of Quality of Life in Palliative Care: A Systematic Review.

Importance: A recent systematic review and meta-analysis found that palliative care was not associated with improvement in quality of life (QOL) in terminal noncancer illness. Among potential reasons for a null effect, it is unclear if patient-reported outcome measures (PROMs) measuring QOL were derived or validated among populations with advanced life-limiting illness (ALLI). Objective: To systematically review the derivation and validation of QOL PROMs from a recent meta-analysis of randomized controlled trials (RCT) of palliative care interventions in people with terminal noncancer illness. Evidence Review: EMBASE, MEDLINE, and PsycINFO were searched from inception to January 8, 2023 for primary validation studies of QOL PROMs in populations with ALLI, defined as adults with a progressive terminal condition and an estimated median survival of less than or equal to one year. The primary outcome was the proportion of PROMs that were derived or validated in ≥1 ALLI population. Findings: Twenty-one unique studies of derivation (n = 13) and validation (n = 11, 3 studies evaluated both) provided data on 9657 participants (mean age 63 years, 50% female) across 15 unique QOL PROMs and subscales. Among studies of validation, 9 were in people with cancer (n = 2289, n = 5 PROMs), 1 in neurodegenerative disease (n = 23, n = 1 PROM), and 1 with mixed diseases (n = 248, n = 1 PROM). Across 15 QOL PROMs and subscales, 47% (n = 7) were derived or validated in an ALLI population. The majority of these seven PROMs were exclusively derived or validated among people with cancer (57%, n = 4). QOL PROMs such as Quality of Life at End of Life, EuroQoL-5 Dimension 5-level, and 36-item Short Form Survey demonstrated validity in more than one terminal noncancer illness. Conclusions: Most QOL PROMs that measured the effect of palliative care on QOL in RCTs were neither derived nor validated in an ALLI population. These findings raise questions about the inferences that palliative care does not improve QOL among people with terminal noncancer illness.

Translation and validation of the multiple sclerosis walking scale 12 for the German population – the MSWS-12/D

BackgroundGait impairment is a relevant problem in persons with multiple sclerosis (pwMS). The Multiple Sclerosis Walking Scale 12 (MSWS-12) is a valid Patient Reported Outcome Measure (PROM) to evaluate walking ability in pwMS. The aim of this study was to provide a linguistically valid translation of MSWS-12 into German language (MSWS-12/D) and to evaluate its psychometric properties.MethodsThe MSWS-12 was translated in a process modified from guidelines for the cross-cultural adaption of PROMs, and a pre-test was applied in a small sample of 20 pwMS to evaluate comprehensibility and acceptance. Psychometric properties (floor and ceiling effects, internal consistency, construct validity) were then assessed in 124 pwMS seen at academic MS centers. Construct validity was evaluated against Expanded Disability Status Scale (EDSS) and maximum gait speed in the Timed 25-Foot Walk (T25FW).ResultsAlthough the sample covered a wide spectrum of symptom severity, the majority had rather low levels of disability (EDSS median 2.0) and 6.5% scored EDSS of 0. In this sample, MSWS-12/D showed floor effects (36% with score 0) and for internal consistency, a Cronbach’s alpha of 0.98 was calculated. MSWS-12/D score showed a relevant correlation to EDSS (ρ = 0.73) and T25FW speed (r=-0.72).ConclusionWe provide MSWS-12/D as a linguistically valid German version of MSWS-12. Psychometric properties (acceptance, floor and ceiling effects, internal consistency and construct validity) in pwMS were similar to those described for the original version. This indicates that MSWS-12/D can be applied as equivalent to the original version in German speaking pwMS. Results support the relevance of PROMs to capture patient perception of walking ability in addition to performance-based assessments such as maximum walking speed or maximum walking distance.

Development of an implementation strategy for routine collection of generic patient reported outcome measures: a qualitative study in multidisciplinary community rehabilitation

Purpose To explore staff perceptions of barriers and enablers towards implementing the EQ-5D-5L in community rehabilitation, and develop a theory-informed implementation approach for routine administration of generic patient-reported outcome measures (PROMs) using implementation science frameworks. Materials and methods A qualitative study was conducted at three sites. Multidisciplinary rehabilitation staff completed individual semi-structured interviews, which were transcribed and coded against the Theoretical Domains Framework (TDF). We identified and selected potentially effective behaviour change techniques using the Behavior Change Wheel. Hypothetical strategies were operationalised. Results Twenty-one interviews were conducted, and four themes emerged: (1) The Impact of PROMs on patient centered-care; (2) Considerations for validity of PROMs; (3) Service-level impact of embedding PROMs; (4) Practical issues of embedding PROMs within the service. Barriers and enablers were mapped to seven of the TDF domains; relating most to clinicians’ “belief about consequences”, “reinforcement”, and “environmental context and resources”. Five hypothetical strategies were developed to overcome identified barriers and strengthen enablers. Key behaviour change techniques underpinning the strategies include: restructuring the physical environment, incentivisation, persuasion and education, enablement, and, social support. Conclusions Our implementation approach highlights the importance of automating processes, engaging site champions, routinely reporting, and using PROM data to inform service provision.

Patient-Reported Outcomes of Omission of Breast Surgery Following Neoadjuvant Systemic Therapy

Patients should have an active role in decisions about pursuing or forgoing specific therapies in treatment de-escalation trials. To evaluate longitudinal patient-reported outcomes (PROs) encompassing decisional comfort and health-related quality of life (HRQOL) among patients who elected to enroll in a clinical trial evaluating radiotherapy alone, without breast surgery, for invasive breast cancers with exceptional response to neoadjuvant systemic therapy (NST). Prospective, single-group, phase 2 clinical trial at 7 US medical centers. Women aged 40 years or older with invasive cT1-2 N0-1 M0 triple-negative or human epidermal growth factor receptor 2 (ERBB2)-positive breast cancer with no pathologic evidence of residual disease following standard NST enrolled from March 6, 2017, to November 9, 2021. Validated PRO measures were administered at baseline and 6, 12, and 36 months post-radiotherapy. Data were analyzed from January to February 2023. PRO measures included the Decision Regret Scale (DRS), Functional Assessment of Cancer Therapy-Lymphedema (FACT-B+4), and Breast Cancer Treatment Outcomes Scale (BCTOS). Changes in PRO measure scores and subscores over time. Among 31 patients, the median (IQR) age was 61 (56-66) years, 26 (84%) were White, and 26 (84%) were non-Hispanic. A total of 15 (48%) had triple-negative disease and 16 (52%) had ERBB2-positive disease. Decisional comfort was high at baseline (median [IQR] DRS score 10 [0-25] on a 0-100 scale, with higher scores indicating higher decisional regret) and significantly increased over time (median [IQR] DRS score at 36 months, 0 [0-20]; P < .001). HRQOL was relatively high at baseline (median [IQR] FACT-B composite score 121 [111-134] on a 0-148 scale, with higher scores indicating higher HRQOL) and significantly increased over time (median [IQR] FACT-B score at 36 months, 128 [116-137]; P = .04). Perceived differences between the affected breast and contralateral breast were minimal at baseline (median [IQR] BCTOS score 1.05 [1.00-1.23] on a 1-4 scale, with higher scores indicating greater differences) and increased significantly over time (median [IQR] BCTOS score at 36 months, 1.36 [1.18-1.64]; P < .001). At 36 months postradiotherapy, the cosmetic subscore was 0.45 points higher than baseline (95% CI, 0.16-0.74; P = .001), whereas function, pain, and edema subscores were not significantly different than baseline. In this nonrandomized phase 2 clinical trial, analysis of PROs demonstrated an overall positive experience for trial participants, with longitudinal improvements in decisional comfort and overall HRQOL over time and minimal lasting adverse effects of therapy. ClinicalTrials.gov Identifier: NCT02945579.

Patient-Reported Outcome Measures in Patients with Thrombotic Thrombocytopenic Purpura: A Systematic Review of the Literature.

Health-related quality of life (HRQoL) impacts of thrombotic thrombocytopenic purpura (TTP) have been captured in clinical studies using patient-reported outcome (PRO) measures (PROMs) that are validated for other diseases. However, the validity evidence to support the use of existing PROMs in patients with TTP is unknown. In a systematic review of the literature, including studies of adults and children with TTP, we assessed the validity evidence for use of PROMs in clinical research and clinical practice, characterized HRQoL, described the integration of PROMs in clinical practice and evaluated PRO scores for patients with TTP compared with reference populations. From an initial 4518 studies, we identified 14 studies using 16 PROMs to assess general HRQoL domains in patients in remission. No identified studies assessed the validity of PROMs for the context of use of TTP and no studies described PROM integration into TTP clinical practice or evaluated PROMs that were specific for patients with TTP. Moreover, PRO scores were worse in patients with TTP compared with reference populations and other chronic conditions. We conclude that, in patients with TTP, PROMs pick up on important patient experiences not captured by clinical outcomes at present. There is, therefore, a need for studies that assess the validity of existing PROMs in patients with TTP to determine if TTP-specific PROMs specific to patients with TTP should be developed.

Adaptation and validation of the Greek version of the Speech Pathology-Specific Questionnaire for Persons with Multiple Sclerosis (SMS)

Purpose The aim of the present study was to adapt and validate the Speech Pathology-Specific Questionnaire for Persons with Multiple Sclerosis (SMS) into the Greek language. Method The study sample consisted of 124 people with multiple sclerosis (PwMS) and 50 healthy controls (HCs). All PwMS underwent cognitive assessment using the Brief International Cognitive Assessment for Multiple Sclerosis (BICAMS). Both PwMS and HCs completed the SMS, the Eating Assessment tool (EAT-10), the Voice Handicap Index (VHI), and the Stroke and Aphasia Quality of Life Scale-39 (SAQOL-39). Result Significant difference was found between PwMS and HCs for the EAT-10, SAQOL-39, the total SMS, and the SMS subscales. Discriminant validity analyses revealed a statistically significant difference between PwMS and HCs for the total and subscales SMS. Convergent validity analyses between the total SMS and the SMS subscales, and scores on the BICAMS, EAT-10, SAQOL-39, and VHI in PwMS were significantly correlated, with exception of the SMS Speech/Voice with the Symbol Digit Modalities Test (SDMT) and the Greek Verbal Learning Test-II (GVLT-II). Scores on the EAT-10, SAQOL-39, and VHI in PwMS were also correlated with the total SMS and the SMS subscales in PwMS, HCs, and the total sample. Construct validity analyses revealed that the total SMS and the SMS subscales were significantly correlated with the Expanded Disability Status Scale (EDSS) and years of education, while no associations were found with regards to age, MS subtype (relapsing-remitting MS [RRMS] vs progressive MS [PMS]), disease duration, or sex. The internal consistency of all items was excellent in PwMS and the total sample (Cronbach’s alpha was >0.7 after deletion of one item), with the exception of two items, which still fell within the acceptable range (>0.6) for PwMS and the total sample. Conclusion The Greek version of the SMS is a reliable and valid patient-reported outcome measure to assess speech-language and swallowing pathology related symptoms in PwMS, and can be used for research and clinical purposes.

Development and Psychometric Validation of Patient-Reported Outcome Measures (PROMs BCC-20) for Assessing Comfort during Chemotherapy in Breast Cancer Patients.

The study aims to develop and psychometric validate Patient-Reported Outcomes Measures for Assessing Comfort during Chemotherapy in Breast Cancer Patients (PROMs BCC-20). This study was conducted in two phases: 1) items were developed from the literature review and in-depth interviews, and 2) Exploratory factor analysis (EFA), Confirmatory factor analysis (CFA), and concurrent validity were performed to evaluate construct validity. The participants were cancer stage I-IIIC, adult females, performance status was assessed by Eastern Cooperative Oncology Group (ECOG) score ≤ 2 after receiving the second cycle of adjuvant chemotherapy and selected by purposive sampling method. For each group of EFA and CFA was 250 participants. Five hundred breast cancer patients during adjuvant chemotherapy were recruited from three tertiary cancer centers. A succession of EFA using principal axis factoring with Promax rotation revealed four dimensions yielded a seven factors solution, explaining a 60.07 percent variance. CFA contains 20 items with five factors; 1) social function, four items; 2) digestive function, three items; 3) emotional function, six items; 4) environmental function, three items; and 5) sleep quality, four items via maximum likelihood with bootstrapping indicated a good fit model (SRMR = 0.045, RMSEA = 0.040, CFI = 0.947, and TLI = 0.935). The Cronbach's alpha of 0.86 demonstrated strong internal consistency reliability. Pearson's correlation coefficient showed acceptable criterion validity. The PROMS BCC-20 provides good psychometric properties and practical patients' direct reports of comfort in breast cancer patients during chemotherapy. The PROMs BCC-20 should be standardized for comfort measurement and tailor-made nursing care to provide patient satisfaction and good nursing outcomes.

Using Patient Blogs on Social Media to Assess the Content Validity of Patient-Reported Outcome Measures: Qualitative Analysis of Patient-Written Blogs.

Patient-reported outcome measures (PROMs) are questionnaires that measure patient outcomes related to quality of life, health, and functioning, and are increasingly used to assess important outcomes from the patient's perspective. For PROMs to contribute to better health and better care, it is vital that their content validity be adequate. This requires patient involvement in various steps of PROM development. PROM developers not only recognize the benefits of patient involvement but also report difficulties in recruiting patients and experience patient involvement as time-consuming, logistically challenging, and expensive. This study seeks to explore different strategies for disclosing the experiential knowledge of patients, namely through analyzing patient stories on the web and social media. The research questions are as follows: (1) how do bloggers living with a disease experience their health-related quality of life? (2) How are these experiences reflected in the domains and items of PROMs related to their disease? First, a qualitative analysis of blogs written by patients was performed. Second, subthemes and underlying codes resulting from this qualitative analysis were systematically compared with the domains and items in PROMs for the respective diseases that the bloggers write about. Blogs were identified via the Google search engine between December 2019 and May 2021. Bloggers describe a wide range of experiences regarding their physical functioning and health; mental well-being; social network and support; daily life, education, work, and leisure; coping; and self-management. Bloggers also write about their positive and negative experiences with health care delivery, the organization of health care, and health care professionals. In general, patients' experiences as described in blogs were reflected in the domains and items of the PROMs related to their disease. However, except for diabetes mellitus, in all the sets of PROMs, potentially missing topics could be identified. Similarly, with the exception of Parkinson disease, all PROMs address issues that patients did not write about in their blogs and that might therefore be redundant. Web-based patient stories in the form of blogs reveal how people living with a certain disease experience their health-related quality of life. These stories enable analyses of patients' experiences that can be used to assess the content validity of PROMs. This can be a useful step for researchers who are looking for sets of measuring instruments that match their purposes.

Hyaluronic Acid Fillers to Correct Cleft Lip Asymmetry in Adults: Description of Technique and Patient Reported Outcome Measure Using CLEFT-Q.

To determine the efficacy of hyaluronic acid (HLA) lip filler to correct subtle cleft lip asymmetries in adults using a validated patient reported outcome measure (PROM). Prospective cross sectional. UK National Health Service. Over 18 years old with repaired cleft lip and dissatisfaction of their lip appearance. HLA lip filler injection. A validated, cleft specific PROM, the lip module of CLEFT-Q™ prior to treatment and again after six weeks. 24 patients included. A mean total CLEFT-Q™ score pre-procedure was 14.9 (Stand deviation (SD) = 4.91) and 24 (SD = 6.08) post-procedure. Difference in mean total score pre- and post-procedure were statically significant (P = .0001) in all domains on the CLEFT-Q™. No adverse outcomes. HLA filler to correct subtle cleft lip asymmetries in adults is a simple low risk technique, which can significantly improve the patient's perception of lip appearance. HLA lip filler to correct subtle cleft lip asymmetries in adults is a simple technique, low risk procedure which can significantly improve the patient's perception of lip appearance.