The development and initial validation of the PROMIS®+HF-27 and PROMIS+HF-10 profiles.

AIMS: Heart failure (HF) is a common and morbid condition impacting multiple health domains. We previously reported the development of the PROMIS®-Plus-HF (PROMIS+HF) profile measure, including universal and HF-specific items. To facilitate use, we developed shorter, PROMIS+HF profiles intended for research and clinical use. METHODS AND RESULTS: Candidate items were selected based on psychometric properties and symptom range coverage. HF clinicians (n = 43) rated item importance and clinical actionability. Based on these results, we developed the PROMIS+HF-27 and PROMIS+HF-10 profiles with summary scores (0-100) for overall, physical, mental, and social health. In a cross-sectional sample (n = 600), we measured internal consistency reliability (Cronbach's alpha and Spearman-Brown), test-retest reliability (intraclass coefficient; n = 100), known-groups validity via New York Heart Association (NYHA) class, and convergent validity with Kansas City Cardiomyopathy Questionnaire (KCCQ) scores. In a longitudinal sample (n = 75), we evaluated responsiveness of baseline/follow-up scores by calculating mean differences and Cohen's d and comparing with paired t-tests. Internal consistency was good to excellent (α 0.82-0.94) for all PROMIS+HF-27 scores and acceptable to good (α/Spearman-Brown 0.60-0.85) for PROMIS+HF-10 scores. Test-retest intraclass coefficients were acceptable to excellent (0.75-0.97). Both profiles demonstrated known-groups validity for the overall and physical health summary scores based on NYHA class, and convergent validity for nearly all scores compared with KCCQ scores. In the longitudinal sample, we demonstrated responsiveness for PROMIS+HF-27 and PROMIS+HF-10 overall and physical summary scores. For the PROMIS+HF overall summary scores, a group-based increase of 7.6-8.3 points represented a small to medium change (Cohen's d = 0.40-0.42). For the PROMIS+HF physical summary scores, a group-based increase of 5.0-5.9 points represented a small to medium change (Cohen's d = 0.29-0.35). CONCLUSIONS: The PROMIS+HF-27 and PROMIS+HF-10 profiles demonstrated good psychometric characteristics with evidence of responsiveness for overall and physical health. These new measures can facilitate patient-centred research and clinical care, such as improving care quality through symptom monitoring, facilitating shared decision-making, evaluating quality of care, assessing new interventions, and monitoring during the initiation and titration of guideline-directed medical therapy.

Optimizing brief, focused assessment of priority symptoms and concerns in recurrent and/or metastatic squamous cell carcinoma of the head and neck: Content validation of the Functional Assessment of Cancer Therapy/National Comprehensive Cancer Network Head and Neck Symptom Index-10 (FHNSI-10).

BACKGROUND AND AIMS: Patients with recurrent and/or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN) experience vast disease and treatment burdens. Brief, focused instruments are needed to assess patient-reported priority symptoms and concerns as targeted outcome assessments for use in clinical research. Although the instrument was developed based on expert and patient input and is psychometrically valid, the Functional Assessment of Cancer Therapy (FACT)/National Comprehensive Cancer Network (NCCN) Head and Neck Symptom Index-10 (FHNSI-10) has yet to undergo content validation from the perspective of R/M SCCHN patients to evaluate its use as a brief symptom-focused targeted endpoint assessment for use in clinical research. METHODS: Interviews conducted with R/M SCCHN patients explored priority symptoms and concerns, followed by cognitive debriefing of the FHNSI-10 to evaluate face validity. Transcripts were analyzed, and results were mapped to the FHNSI-10. In accordance with published recommendations, expert input from the original development and published literature was considered for content validity assessment. RESULTS: A total of 18 patients participated in a concept elicitation interview; saturation was obtained at interview 17. Most (83%) were undergoing active treatment, male (94%), white (72%), and did not have a college degree (67%). The most commonly mentioned symptoms were lumps/swelling, pain, sore throat, difficulty swallowing, and voice changes. For all items, ≥75% reported each question was relevant to their R/M SCCHN experience and 94% reported the instrument captured their experiences with R/M SCCHN. CONCLUSION: Results provide support for the content validity of the FHNSI-10, inasmuch as all 10 items were spontaneously reported and considered relevant to R/M SCCHN. Content validity might be enhanced by adding cough and hearing impairment items; however, the existing FHNSI-10 covers the majority of symptoms uncovered in interviews with a small sample of R/M SCCHN patients.

The PROMISⓇ-Plus-Osteoarthritis of the Knee (OAK) profile measure integrates generic and condition-specific content to enhance relevance and efficiency.

OBJECTIVE: The Patient-Reported Outcomes Measurement Information System (PROMIS)-Plus-Osteoarthritis of the Knee (OAK) profile integrates universal PROMIS items with knee-specific items across 13 domains. We evaluated the psychometric properties of a subset of six domains associated with quality of life in people with OAK. STUDY DESIGN AND SETTING: In a cross-sectional study of OAK patients (n=600), we estimated reliability using Pearson and Spearman correlations with Knee Injury and Osteoarthritis Outcome Score (KOOS) subscores and known-groups validity with PROMIS Global Health. Measure responsiveness was tested via paired t-tests in a longitudinal study (n=238), pre/post total knee replacement. RESULTS: Across the six domains, internal consistency reliability (Cronbach's alpha) was 0.77-0.95 and test-retest reliability (intraclass correlation coefficients) was ≥0.90. Correlations with Knee Injury and Osteoarthritis Outcome Score (KOOS) subscores and PROMIS Global supported convergent and divergent validity. Known-groups validity testing revealed better scores in all domains for high vs. low global status groups, and knee-specific items added value in physical function and pain. All domains reflected (p<0.001) better health status scores at follow up. CONCLUSION: The six PROMIS-Plus-OAK profile domains demonstrated good psychometric characteristics. The measure integrates universal and knee-specific content to provide enhanced relevance, measurement precision and efficient administration for patient care and clinical research.

Development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index.

OBJECTIVE: To develop a symptom-focused index to evaluate representative symptoms, treatment side effects, and emotional and functional well-being of patients with carcinoid syndrome (CS). METHODS: The development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index (FACT-CSI) followed US Food and Drug Administration guidelines for the development of patient-reported outcome (PRO) measures and involved the following: (a) literature review; (b) interviews with 14 CS patients; (c) interviews with 9 clinicians; and (d) instrument development involving input from a range of PRO measure development and CS experts. The resulting draft instrument underwent cognitive interviews with 7 CS patients. RESULTS: Forty-six CS sources were reviewed. Analysis of patient interviews produced 23 patient-reported symptoms. The most frequently endorsed physical symptoms were flushing, diarrhea, abdominal pain, fatigue, and food sensitivity/triggers. Seven priority CS emotional and functional themes were also identified by patients. Expert interviews revealed 12 unique priority symptoms - the most common being diarrhea, flushing, wheezing, edema, abdominal pain/cramping, fatigue, and 8 emotional and functional concerns. Through an iterative process of team and clinical collaborator meetings, data review, item reduction and measure revision, 24 items were selected for the draft symptom index representing symptoms, emotional concerns, global assessment of treatment side effects, and functional well-being. Cognitive interview results demonstrated strong content validity, including positive endorsement of item clarity (>86% across items), symptom relevance (>70% for most items), and overall measure content (86%). CONCLUSIONS: The FACT-CSI is a content-relevant, symptom-focused index reflecting the highest priority and clinically relevant symptoms and concerns of people with CS.

Validation of the Functional Assessment of Cancer Therapy-Leukemia instrument in patients with acute myeloid leukemia who are not candidates for intensive therapy.

BACKGROUND: The objective of this study was to validate the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) in patients with acute myeloid leukemia (AML) who are not candidates for intensive therapy. METHODS: A sample of 317 patients with AML who were not eligible for intensive chemotherapy completed the FACT-Leu and EuroQol 5-Dimension (EQ-5D) measures (Utility Index and Visual Analogue Scale) every 28 days until the end of treatment. Internal consistency reliability was estimated with Cronbach's α. Concurrent validity was examined with correlations between FACT-Leu and EQ-5D scales, and known-groups validity was examined by determining whether FACT-Leu scales distinguished between Eastern Cooperative Oncology Group (ECOG) performance status ratings (PSRs) and between maximum adverse event toxicities at the baseline. This study examined responsiveness to change by anchoring change in the FACT-Leu scales to a 0.10 change in the EQ-5D Health Utility Index. RESULTS: Cronbach's α usually exceeded the threshold for good (≥0.80) or excellent reliability (≥0.90). Correlations between FACT-Leu and EQ-5D scales were moderate (r > 0.50) or high (r > 0.70). FACT-Leu scales distinguished between ECOG PSR groups with large effect sizes for an ECOG PSR of 0 versus an ECOG PSR of 2 (0.50 ≤ d < 0.80). In addition, Functional Assessment of Cancer Therapy-General, Additional Concerns, FACT-Leu Total, and Trial Outcomes Index scales distinguished between patients with grade 3 or lower maximum adverse event toxicities and those with maximum adverse event toxicities higher than grade 3, but effect sizes were small (d < 0.50). Finally, FACT-Leu scale coefficients for a 0.10 change in the 5-level version of the EQ-5D HUI ranged between -0.01 and 4.30. CONCLUSIONS: The FACT-Leu is a suitable outcome measure for AML clinical trials among patients not eligible for intensive therapy, and it may have value for clinical monitoring.

Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©).

PURPOSE: To develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH). PATIENTS AND METHODS: The development of the PNH-PPQ© was consistent with Food and Drug Administration guidelines for patient-reported outcome measure development, and included 1) a targeted literature review; 2) PNH expert clinician input on treatment preferences; 3) review of existing qualitative data on the PNH treatment and disease experience; 4) concept elicitation interviews with 8 PNH patients who received eculizumab and/or ravulizumab; 5) translatability review; and 6) cognitive debriefing with 5 patients. Interview participants were recruited through a United Kingdom PNH patient advocacy group and a Canadian clinical site involved in clinical trial ALXN1210-PNH-302. RESULTS: Six themes were identified as most relevant to the PNH treatment experience from the concept elicitation interviews: disease symptoms (n=8/8); treatment frequency (n=7/8); quality of life impact of treatment/disease (n=7/8); treatment burden (n=7/8); treatment efficacy (n=5/8); and treatment side effects (n=5/8). An initial list of 88 preference questions was reduced to 11 highly relevant and non-redundant questions reflecting the 6 themes. Cognitive interview participants unanimously agreed that the PNH-PPQ instructions were clear; response options were understandable, easy to use, and provided enough choices; and the questions captured the factors that inform treatment preferences. DISCUSSION: When new drugs have similar efficacy to existing medications, documenting patient preferences is important for confirming patient benefit from the new medication. Understanding what matters most to patients is essential for delivering patient-centered care and may play a particularly significant role in treatment decision making. The availability of such a tool may be especially important as new orphan drugs are developed and patients with rare diseases have more than one treatment option to consider. CONCLUSION: The PNH-PPQ provides a patient-centered approach for evaluating preferences for the treatment of PNH. The PNH-PPQ has subsequently assessed patient preference in the clinical trial sub-study ALXN1210-PNH-302s.

Financial burden and quality of life among thyroid cancer survivors.

BACKGROUND: Survivors of cancer in the United States are often financially encumbered by expenses and lost wages from cancer treatment. The majority of patients with thyroid cancer are diagnosed before age 65, when they are not eligible for Medicare. We hypothesized that financial distress would be common among thyroid cancer survivors and would be associated with poor health-related quality of life. METHODS: A financial distress questionnaire and Patient-Reported Outcomes Measurement Information System (29-item) were completed online by 1,743 adult thyroid cancer survivors living in the United States. Multivariable modeling was used to identify variables which independently predicted poor health-related quality of life. The magnitude of predicted change was estimated by ß coefficients and 95% confidence intervals. A ß ≥3 was considered clinically significant; α was set at 0.01. RESULTS: Financial difficulties were reported by 43% of thyroid cancer survivors and were associated with worse anxiety (ß = 5.07; P < .01) and depression (ß = 5.47; P < .01). Living in poverty was associated with worse anxiety (ß = 4.14; P < .01) and depression (ß = 4.35; P < .01). Lost productivity at work was associated with worse fatigue (ß = 5.99; P < .01) and social functioning (ß = -4.07; P < .01). Inability to change jobs was associated with worse fatigue (ß = 3.08; P < .01), pain interference (ß = 3.56; P < .01), and social functioning (ß = -3.09; P < .01). Receiving disability benefits was associated with worse pain interference (ß = 3.93; P < .01). Impaired ability to obtain a job was associated with worse social functioning (ß = -3.02; P < .01). Reported unemployment rate was 12.3%. CONCLUSION: Financial distress and negative financial events were common among thyroid cancer survivors and were associated with poorer health-related quality of life across 5 Patient-Reported Outcomes Measurement Information System health domains.

Responsiveness of PROMIS® to change in chronic obstructive pulmonary disease.

BACKGROUND: Chronic obstructive pulmonary disease (COPD) is a progressive chronic disease characterized by airflow obstruction that leads to shortness of breath and substantial negative impacts on health-related quality of life (HRQL). The course of COPD includes periodic acute exacerbations that require changes in treatment and/or hospitalizations. This study was designed to examine the responsiveness of Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures to changes associated with COPD exacerbation recovery. METHODS: A longitudinal analysis using mixed-effects models was conducted of people who were enrolled while stable (n = 100) and those who experienced an acute exacerbation (n = 85). PROMIS (physical function, pain interference, pain behavior, fatigue, anxiety, depression, anger, social roles, discretionary social activities, Global Health, dyspnea severity and dyspnea functional limitations) and COPD-targeted HRQL measures were completed at baseline and at 12 weeks. RESULTS: We administered PROMIS measures using computer adaptive testing (CAT), followed by administration of any remaining short form (SF) items that had not yet been administered by CAT. Examination of the difference between group differences from baseline to 12 weeks in the stable and exacerbation groups revealed that the exacerbation group changed (improved) significantly more than the stable group in anxiety (p < .001 to p < .01; f2 effect size [ES] = 0.023/0.021), fatigue (p < .0001; ES = 0.036/0.047) and social roles (p < .001 to p < .05; ES = 0.035/0.024). All effect sizes were small in magnitude and smaller than hypothesized. Depression was also statistically significant (p < .05, SF only) but the ES was trivial. For all other PROMIS domains, the differences were not significant and ES were trivial. CONCLUSIONS: This longitudinal study provides some support for the validity of the PROMIS fatigue, anxiety, and social roles domains in COPD, but further evaluation of responsiveness is warranted.

Establishing clinically-relevant terms and severity thresholds for Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures of physical function, cognitive function, and sleep disturbance in people with cancer using standard setting.

PURPOSE: Patient-Reported Outcomes Measurement Information System® (PROMIS®) physical function, cognitive function, and sleep disturbance measures are increasingly used in cancer care. However, there is limited guidance for interpreting the clinical meaning of scores. This study aimed to apply bookmarking, a standard setting methodology, to identify PROMIS score thresholds in the context of cancer care. METHODS: Using item parameters, we constructed vignettes of five items covering the range of possible scores. Focus groups were held with cancer care providers and people with cancer. Terminology for categorizing levels of severity was explored. Participants rank ordered vignettes by severity and then placed bookmarks between vignettes representing different levels of severity. Group discussion was held until consensus on bookmark placement was reached. RESULTS: Clinicians selected "within normal limits," "mild," "moderate," and "severe" to describe levels of severity. Both patients and clinicians were able to apply these labels, but there was not unanimous support for any set of descriptors. Clinicians and patients agreed on all severity thresholds for sleep disturbance. For cognitive and physical function, clinicians and patients agreed on the threshold between "within normal limits" and "mild." However, patients required greater dysfunction than clinicians before applying "moderate" and "severe" labels. CONCLUSIONS: Bookmarking can be applied to develop provisional score interpretation for PROMIS measures. Patients and clinicians were frequently consistent in their bookmark placement. When there was variance, patients required more dysfunction before assigning more severity. Additional research with other cancer samples is needed to evaluate the replicability and generalizability of our findings.

Development and Initial Validation of the PROMIS®-Plus-HF Profile Measure.

Background Bringing together generic and heart failure (HF)-specific items in a publicly available, patient-reported outcome measure may facilitate routine health status assessment for improving clinical care and shared decision-making, assessing quality of care, evaluating new interventions, and comparing groups with different conditions. Methods and Results We performed a mixed-methods study to develop and validate the PROMIS®-Plus-HF (Patient-Reported Outcomes Measurement Information System®-Plus-Heart Failure) profile measure-a HF-specific instrument based on the generic PROMIS. We conducted 8 focus groups with 61 patients with HF and phone interviews with 10 HF clinicians. The measure was developed via an iterative process of reviewing existing PROMIS items and developing and testing new HF items. In a 600-patient sample, we estimated reliability (internal consistency; test-retest, with n=100 participants). We conducted validity analyses using Pearson r and Spearman ρ correlations with Kansas City Cardiomyopathy Questionnaire subscores. In a longitudinal sample, we performed responsiveness testing (paired t tests) with 75 patients with HF receiving interventions with expected health status improvement. The PROMIS-Plus-HF measure comprises 86 items (64 existing; 22 new) across 18 domains. Internal consistency reliability (Cronbach α) coefficients ranged from 0.52 to 0.96, with α≥0.70 in 12 of 17 domains. Test-retest intraclass correlation coefficients were ≥0.90. Correlations with Kansas City Cardiomyopathy Questionnaire subscores supported expected convergent ( r/ρ>0.60) and divergent validity ( r/ρ<0.30). In the longitudinal sample, 10 of 18 domains had improved ( P<0.05) scores from baseline to follow-up. Conclusions The PROMIS-Plus-HF profile measure-a complete assessment of physical, mental, and social health-exhibited good psychometric characteristics and may facilitate patient-centered care and research. Subsets of domains and items can be used depending on the clinical or research purpose.

PROMIS®: Standardizing the patient voice in health psychology research and practice.

The Patient-Reported Outcomes Measurement Information System® (PROMIS®) is a collection of person-centered measures of physical, mental, and social health in children and adults. Developed using state-of-the-art psychometric methods, these measures are offered as fixed-length questionnaires and computerized adaptive tests, with translations available in Spanish and other languages. This special issue presents articles that illustrate the use of PROMIS® measures to advance health behavior research across a wide range of health studies. (PsycINFO Database Record (c) 2019 APA, all rights reserved).

Post-thyroidectomy neck appearance and impact on quality of life in thyroid cancer survivors.

BACKGROUND: There is a paucity of patient-reported data on thyroidectomy scar perception. The effect of neck scarring on quality of life is not known. We hypothesized that worse perception of neck appearance would be related to worse health-related quality of life and that perception improves with time. METHODS: Survivors of thyroid cancer (n = 1,710) were surveyed online. Respondents were asked to score the appearance of their neck via a 5-point Likert scale. Quality of life was evaluated via the Patient-Reported Outcomes Measurement Information System 29. The relationships between neck appearance, patient characteristics, quality of life, and Patient-Reported Outcomes Measurement Information System domains were analyzed with multivariable models and Spearman partial correlations (rs). RESULTS: Older age was associated with better perception of neck appearance (odds ratio 0.975/year; 95% confidence interval 0.967-0.983; P < .001). Greater time since surgery was also associated with improved perception (odds ratio 0.962/year; 95% confidence interval 0.947-0.977; P < .0001). We observed no statistically significant difference between current and preoperative baseline perception >2 years after surgery. On multivariable analysis, age >45 years, >2 years since surgery, and higher self-reported quality of life were independent predictors of better self-reported neck appearance (P ≤ .0003). In patients ≤2 years after surgery (n = 568), the PROMIS domains of anxiety, depression, social function, and fatigue had weak but statistically significant correlations with worse perception of neck appearance (P < .0001). CONCLUSION: Age >45, >2 years since surgery, and higher quality of life were independently associated with better self-reported neck appearance. Perception of neck appearance returned to preoperative baseline 2 years after surgery. PROMIS domains had a weak but significant association with neck appearance perception in patients ≤2 years after surgery. The impact of post-thyroidectomy neck appearance on quality of life appears to be mild and transient and returns to preoperative levels after 2 years.

Clinical factors associated with worse quality-of-life scores in United States thyroid cancer survivors.

INTRODUCTION: Thyroid cancer survivors are a rapidly growing population in the United States. The factors that drive health-related quality of life (HRQOL) in this population have not been well characterized. We hypothesized that more aggressive treatments and greater treatment-related adverse effects would be associated with worse HRQOL scores in thyroid cancer survivors. METHODS: Thyroid cancer survivors (18-89 years of age) completed an online survey regarding their clinical history in addition to the Patient-Reported Outcomes Measurement Information System (PROMIS) 29 instrument. Univariable and multivariable modeling were performed to evaluate factors associated with worse HRQOL scores. We generated ß-values and 95% confidence intervals to quantify the effect of each independent variable in the model. RESULTS: Thyroid cancer survivors (n = 1,743) reported a high incidence of complications related to surgery and radioactive iodine ablation. Postoperative dysphonia (ß 1.83-3.07) and dysphagia (ß 2.05-3.65) predicted worse HRQOL scores across multiple PROMIS domains. Younger patient age (age <45 years) and short- or long-term complications of radioactive iodine, including gastrointestinal symptoms (51.9%), appetite changes (71.2%), sialadenitis (58.1%), xerostomia (73.3%), and xerophthalmia (45.1%) were associated with worse HRQOL scores (P < .01). CONCLUSION: The factors associated with significantly worse HRQOL scores across multiple PROMIS domains for thyroid cancer survivors included patient age <45 years, postoperative hypocalcemia, dysphonia, dysphagia, scar appearance, and complications from radioactive iodine. Methods of evaluation, management, and prevention of these factors might positively impact HRQOL.

Psychological Functioning in Patients With Chronic Obstructive Pulmonary Disease: A Preliminary Study of Relations With Smoking Status and Disease Impact.

INTRODUCTION: Chronic obstructive pulmonary disease (COPD) is a tobacco-related disease associated with several comorbid conditions, including elevated rates of depression and anxiety. Psychological factors that commonly underlie nicotine dependence, depression, and anxiety may represent novel treatment targets, but have not yet been examined among patientswith COPD. We assessed three psychological factors-anxiety sensitivity (AS; fear of anxiety-related sensations), distress intolerance (DI; inability to withstand distressing states), and anhedonia (Anh; diminished sense of pleasure or interest)-in relation to smoking status, COPD symptom impact, and negative response to COPD symptoms. METHODS: We conducted a single-session laboratory assessment with 37 patients with COPD (17 current daily smokers and 20 former smokers). All participants completed self-report measures of psychological factors, COPD symptom impact, response to COPD symptoms, and anxiety and depression symptoms. RESULTS: Current versus former smokers with COPD reported higher levels of AS, DI, and Anh. In univariate regression models, AS, DI, and Anh were each associated with greater COPD symptom impact and breathlessness catastrophizing. Only AS remained a significant predictor of COPD symptom impact and breathlessness catastrophizing after adjusting for general depression and anxiety symptoms. CONCLUSIONS: Our preliminary study is the first to assess AS, DI, and Anh among patients with COPD. These psychological factors were elevated among current smokers and associated with more negative disease impact, suggesting their potential utility as treatment targets within this clinical population. IMPLICATIONS: While elevated rates of anxiety and depression among patients with COPD have been wellcharacterized, few studies have specifically addressed the causal, modifiable psychological factors that may underlie these disorders. Our preliminary findings demonstrate associations of three psychological factors-AS, DI, and Anh-with smoking status, COPD symptom impact, and negative reaction to symptoms. Cognitive-behavioral interventions targeted to these psychological factors may improve smoking cessation outcomes and disease adjustment among patients with COPD.

Benchmarking health-related quality of life in thyroid cancer versus other cancers and United States normative data.

BACKGROUND: Thyroid cancer survivors may experience long-lasting physical, psychosocial, and financial challenges. No previous studies have compared health-related quality of life in thyroid cancer survivors to United States normative data and patient-reported outcomes from other types of cancers. We hypothesized that thyroid cancer survivors would report health-related quality of life poorer than the general United States population but similar to individuals with other cancers. METHODS: Thyroid cancer survivors were recruited online January2017-June 2017. Individuals completed a two-part questionnaire to assess clinical characteristics and health-related quality of life, using the Patient-Reported Outcomes Measurement Information System 29-item profile. Patient-Reported Outcomes Measurement Information System T-scores obtained from the literature were compared with United States normative data and T-scores from patients with breast, prostate, uterine, cervical, colorectal, non-Hodgkin lymphoma, and lung cancers. RESULTS: A total of 1,743 US respondents completed the survey. Thyroid cancer survivors reported statistically significantly worse health-related quality of life across all seven Patient-Reported Outcomes Measurement Information System domains compared with United States normative data (P < .05). Surveyed individuals reported statistically significantly worse scores for anxiety, depression, fatigue, and sleep disturbance than respondents from non-Hodgkin lymphoma, breast, colorectal, uterine, and prostate cancer cohorts (P < .01) but less pain and greater physical functioning than most other groups in this comparison (P < .01). CONCLUSION: The importance of health-related quality of life among thyroid cancer survivors should not be obscured by the relatively high survival rate of thyroid cancer compared with other cancers. Our results demonstrate that thyroid cancer survivors may be encumbered with greater psychologic and social burdens than survivors of several cancers that have a worse prognosis.

Quality of life in multiple endocrine neoplasia type 2A compared with normative and disease populations.

BACKGROUND: Patient-reported outcomes are measured in chronic disease states to inform intervention and management decisions while minimizing negative outcomes. We hypothesized that health-related quality of life in patients with multiple endocrine neoplasia type 2A would be worse than the general US population but similar to other chronic diseases. METHODS: Adults ≥18 years with multiple endocrine neoplasia type 2A were recruited to complete the Patient-Reported Outcomes Measurement Information System 29-item questionnaire (n = 45). Scores based on the Patient-Reported Outcomes Measurement Information System T-score metric were compared with the general US population and with cohorts with low back pain, cancer, congestive heart failure, chronic obstructive pulmonary disease, major depressive disorder, rheumatoid arthritis, neuroendocrine tumors, primary hyperparathyroidism, and MEN1. RESULTS: Compared with US normative data, multiple endocrine neoplasia type 2A patients reported worse anxiety (58.2 ± 12.0, P < .0001), depression (55.4 ± 12.0, P < .01), fatigue (61.4 ± 10.8, P < .0001), pain interference (54.0 ± 11.5, P < .05), and sleep disturbance (56.9 ± 2.7, P < .001), as well as significantly lower physical functioning (45.7 ± 9.3, P < .01) and ability to participate in social roles (46.4 ± 9.7, P < .05). Multiple endocrine neoplasia type 2A patients reported greater fatigue than patients with cancer (P < .0001), chronic obstructive pulmonary disease (P = .01), rheumatoid arthritis (P = .0002), neuroendocrine tumors (P = .0007), and primary hyperparathyroidism (P < .0001) but higher physical functioning compared with patients with rheumatoid arthritis (P = .02), low back pain, congestive heart failure, and chronic obstructive pulmonary disease (P < .0001). CONCLUSIONS: This study is the first to use the Patient-Reported Outcomes Measurement Information System to compare patient-reported outcomes between multiple endocrine neoplasia type 2A and other chronic conditions. Individuals with multiple endocrine neoplasia type 2A reported worse health-related quality of life in all 7 domains compared with US normative data. Multiple endocrine neoplasia type 2A patients reported greater fatigue but greater physical function compared with several other conditions. Prospective longitudinal evaluation of patient-reported outcomes in multiple endocrine neoplasia type 2A should be conducted to identify treatments associated with the highest health-related quality of life.

A method to create a standardized generic and condition-specific patient-reported outcome measure for patient care and healthcare improvement.

PURPOSE: Patient-reported outcome measures (PROMs), which are generic or condition-specific, are used for a number of reasons, including clinical care, clinical trials, and in national-level efforts to monitor the quality of health care delivery. Creating PROMs that meet different purposes without overburdening patients, healthcare systems, providers, and data systems is paramount. The objective of this study was to test a generalizable method to incorporate condition-specific issues into generic PROM measures as a first step to producing PROMs that efficiently provide a standardized score. This paper outlines the method and preliminary findings focused on a PROM for osteoarthritis of the knee (OA-K). METHODS: We used a mixed-methods approach and PROMIS® measures to test development of a combined generic and OA-K-specific PROM. Qualitative methods included patient focus groups and provider interviews to identify impacts of OA-K important to patients. We then conducted a thematic analysis and an item gap analysis: identified areas covered by existing generic PROMIS measures, identified "gap" areas not covered, compared gap areas to legacy instruments to verify relevance, and developed new items to address gaps. We then performed cognitive testing on new items and drafted an OA-K-specific instrument based on findings. RESULTS: We identified 52 existing PROMIS items and developed 24 new items across 14 domains. CONCLUSIONS: We developed a process for creating condition-specific instruments that bridge gaps in existing generic measures. If successful, the methodology will create instruments that efficiently gather the patient's perspective while allowing health systems, researchers, and other interested parties to monitor and compare outcomes over time, conditions, and populations.

Health-related quality of life in MEN1 patients compared with other chronic conditions and the United States general population.

BACKGROUND: Health-related quality of life (HRQOL) in multiple endocrine neoplasia type-1 (MEN-1) is poorly described. HRQOL in MEN-1 was compared with other chronic conditions and the US general population. METHODS: Adults aged ≥18 years recruited from an MEN-1 support group (n=153) completed the Patient-Reported Outcomes Measurement Information System (PROMIS) 29-item profile. MEN-1 scores were compared with PROMIS scores reported in peer-reviewed literature from back pain (n=218), cancer (n=310), congestive heart failure (CHF; n=60), chronic obstructive pulmonary disease (COPD; n=79), major depressive disorder (n=196), rheumatoid arthritis (RA; n=521), neuroendocrine tumors (NET; n=619), and primary hyperparathyroidism (PHPT; n=45) cohorts. RESULTS: Patients with MEN-1 reported worse anxiety (mean=61.7), depression (57.9), fatigue (62.2), pain interference (55.4), sleep disturbance (58.0), physical functioning (44.4), and social functioning (44.7) compared to normative data (50, P < .05) and greater anxiety, depression, and fatigue than patients with back pain, cancer, COPD, RA, NETs, and PHPT (P < .001). MEN-1 respondents had greater pain interference (55.4) than those with cancer (51.9), NETs (52.3), and PHPT (38.4, P < .05). Physical functioning was higher in individuals with MEN-1 (44.4) than in those with back pain (37.5), CHF (34.8), COPD (38.0), and RA (40.7, P < .01). CONCLUSION: This is the first study to describe HRQOL in a large sample of adults with MEN-1. MEN-1respondents reported worse HRQOL across PROMIS 29-item profile measure domains compared with the US general population and higher levels of anxiety, depression, and fatigue compared with many other chronic conditions.

Disease and treatment factors associated with lower quality of life scores in adults with multiple endocrine neoplasia type I.

BACKGROUND: Physical and psychosocial morbidity of multiple endocrine neoplasia type-1 is ill-defined. How disease and treatment-related factors relate to patient-reported outcomes including health-related quality of life is unknown. We hypothesized that disease and treatment burden negatively impacts health-related quality of life in adults with multiple endocrine neoplasia type-1. METHODS: Adults (≥18 years) with multiple endocrine neoplasia type-1 completed an online survey of demographics, disease features, treatments, and Patient-Reported Outcomes Measurement Information System 29-item profile measure, and scores were compared with normative US data. Multivariable modeling was performed to evaluate factors associated with decreased health-related quality of life. RESULTS: Multiple endocrine neoplasia type-1 patients (n = 207) reported worse health-related quality of life compared with US normative data in all health-related quality of life domains (P < .001). Persistent hypercalcemia after parathyroid surgery was associated with higher levels of anxiety, depression, fatigue, and decreased social functioning (P < .05). Patients <45 years of age at diagnosis reported worse physical and social functioning (P < .01). Traveling >50 miles for doctor appointments and ≥20 doctor appointments/year (P < .05) were associated with worse health-related quality of life. History of pancreatic neuroendocrine tumors was not associated with worse health-related quality of life. CONCLUSION: This is the largest study to assess clinical and treatment factors associated with health-related quality of life in multiple endocrine neoplasia type-1. Persistent hyperparathyroidism, increased travel distance and frequency of doctor appointments were all associated with worse health-related quality of life.

Financial burden is associated with worse health-related quality of life in adults with multiple endocrine neoplasia type 1.

BACKGROUND: Health-related quality of life and financial burden among patients with multiple endocrine neoplasia type 1 is poorly described. It is not known how financial burden influences health-related quality of life in this population. We hypothesized that the financial burden attributable to multiple endocrine neoplasia type 1 is associated with worse health-related quality of life. METHODS: United States adults (≥18 years) with multiple endocrine neoplasia type 1 were recruited from the AMENSupport MEN online support group. Patient demographics, clinical characteristics, and financial burden were assessed via an online survey. The instrument Patient-Reported Outcomes Measurement Information System 29-item profile measure was used to assess health-related quality of life. Multivariable linear regression was used to identify significant variables in each Patient-Reported Outcomes Measurement Information System domain. RESULTS: Out of 1,378 members in AMENSupport, our survey link was accessed 449 times (33%). Of 153 US respondents who completed our survey, 84% reported financial burden attributable to multiple endocrine neoplasia type 1. The degree of financial burden had a linear relationship with worse health-related quality of life across all Patient-Reported Outcomes Measurement Information System domains (r = 0.36-0.55, P < .001); 63% reported experiencing ≥1 negative financial event(s). Borrowing money from friends/family (30%), unemployment (13%), and spending >$100/month out-of-pocket on prescription medications (46%) were associated consistently with impaired health-related quality of life (ß = 3.75-6.77, P < .05). Respondents were 3- and 34-times more likely to be unemployed and declare bankruptcy than the US population, respectively. CONCLUSION: This study characterizes the financial burden in patients with multiple endocrine neoplasia type 1. Individuals with multiple endocrine neoplasia type 1 report a high degree of financial burden, negative financial events, and unemployment. Each of these factors was associated with worse health-related quality of life.