Psychometric validation of the Psoriasis Symptom Scale, Functional Assessment of Chronic Illness Therapy-Fatigue and pain-Visual Analogue Scale in patients with generalized pustular psoriasis.

BACKGROUND: Generalized pustular psoriasis (GPP) is a rare, chronic, inflammatory skin disease associated with considerable patient burden. The Psoriasis Symptom Scale (PSS), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and pain-Visual Analogue Scale (pain-VAS) are patient-reported outcomes (PROs) that have not yet been validated in patients with GPP. OBJECTIVES: To evaluate the psychometric properties of the PSS, FACIT-Fatigue and pain-VAS using data from Effisayil 1, a randomised trial of spesolimab in patients with moderate-to-severe GPP. METHODS: Inter-item correlations and confirmatory factor analysis (CFA) were performed using Week 1 data. Internal consistency was assessed with Cronbach's α coefficient using baseline and Week 1 data. Test-retest reliability was assessed using intraclass correlation coefficients (ICCs); change data for the GPP Physician Global Assessment total score and pustulation subscore were used to define a stable population. Convergent validity was assessed at baseline and Week 1 using Spearman's rank-order correlations. Known-groups validity was measured by analysis of variance using Week 1 data. Ability to detect change from baseline to Week 1 was evaluated by analysis of covariance. RESULTS: Inter-item and item-to-total correlations were moderate or strong for most PSS and FACIT-Fatigue items. CFA demonstrated the unidimensionality of the PSS and FACIT-Fatigue, with high factor loadings for most items (PSS range, 0.75-0.94; FACIT-Fatigue range, 0.11-0.93) and acceptable fit statistics. Both scores demonstrated internal consistency (Cronbach's α, 0.71 and 0.95, respectively). The PSS, FACIT-Fatigue and pain-VAS demonstrated test-retest reliability (ICCs ≥0.70) and good evidence of convergent validity. Furthermore, the PROs could differentiate between known groups of varying symptom severity (range, p < 0.0001-0.0225) and detect changes in symptom severity from baseline to Week 1 (range, p < 0.0001-0.0002). CONCLUSIONS: Overall, these results support the reliability, validity and ability to detect change of the PSS, FACIT-Fatigue and pain-VAS as PROs in patients with GPP.

Psychometric validation of the generalized pustular psoriasis physician global assessment (GPPGA) and generalized pustular psoriasis area and severity index (GPPASI).

BACKGROUND: Generalized pustular psoriasis (GPP) is a rare and life-threatening skin disease often accompanied by systemic inflammation. There are currently no standardized or validated GPP-specific measures for assessing severity. OBJECTIVE: To evaluate the reliability, validity and responder definitions of the Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) and Generalized Pustular Psoriasis Area and Severity Index (GPPASI). METHODS: The GPPGA and GPPASI were validated using outcome data from Week 1 of the Effisayil™ 1 study. The psychometric analyses performed included confirmatory factor analysis, item-to-item/item-to-total correlations, internal consistency reliability, test-retest reliability, convergent validity, known-groups validity, responsiveness analysis and responder definition analysis. RESULTS: Using data from this patient cohort (N = 53), confirmatory factor analysis demonstrated unidimensionality of the GPPGA total score (root mean square error of approximation <0.08), and GPPGA item-to-item and item-to-total correlations ranged from 0.58 to 0.90. The GPPGA total score, pustulation subscore and GPPASI total score all demonstrated good test-retest reliability (intraclass correlation coefficient: 0.70, 0.91 and 0.95 respectively), and good evidence of convergent validity. In anchor-based analyses, all three scores were able to detect changes in symptom and disease severity over time; reductions of -1.4, -2.2 and - 12.0 were suggested as clinically meaningful improvement thresholds for the GPPGA total score, GPPGA pustulation subscore and GPPASI total score respectively. Anchor-based analyses also supported the GPPASI 50 as a clinically meaningful threshold for improvement. CONCLUSIONS: Overall, our findings indicate that the GPPGA and GPPASI are valid, reliable and responsive measures for the assessment of GPP disease severity, and support their use in informing clinical endpoints in trials in GPP.

Qualitative study of patients with venous malformations: symptom experiences and content validity of patient-reported outcome measures.

PURPOSE: To determine important symptoms and functional effects of venous malformations (VMs) to assess the content validity of commonly used patient-reported outcome (PRO) measures for use with VM patients. METHODS: This cross-sectional, qualitative study involved cognitive interviews with participants with VM aged ≥ 14 years. From February to June 2016, 11 participants (8 female) with a mean (± standard deviation) age of 31 ± 15 years were recruited from three clinical sites. The following subgroups were evaluated: 5 adults (aged ≥ 18) with trunk/extremity VMs; 3 adolescents (aged 14-17) with trunk/extremity VMs; and 3 adults with head/neck VMs. We evaluated the content validity of the Worst Pain Numeric Rating Scale (NRS), Patient-Reported Outcomes Measurement Information System (PROMIS®) Pain Interference 8-item short form, and PROMIS Physical Function 8-item short form. RESULTS: The most common participant-reported VM symptoms were swelling (n = 10), skin discoloration (n = 8), acute episodic pain (n = 8), chronic pain (n = 7), numbness (n = 7), and tingling/burning (n = 6). Participants reported that VMs affected their physical function (n = 10), appearance (n = 10), relationships/social activities (n = 7), and emotional health (n = 3). The Worst Pain NRS and PROMIS Pain Interference measures were relevant to all participants' VM experience. Only adults with head/neck VMs found the PROMIS Physical Function measure to be irrelevant. The assessed PRO measures did not address several symptoms commonly reported by VM patients (swelling, skin discoloration, numbness, and appearance). CONCLUSION: These results suggest that several VM symptoms are not assessed fully by commonly used PRO measures, and that the relevance of functional limitation questions may vary by VM location.

Content validity and electronic PRO (ePRO) usability of the Lung Cancer Symptom Scale-Mesothelioma (LCSS-Meso) in mesothelioma patients.

PURPOSE: Obtaining qualitative data directly from the patient perspective enhances the content validity of patient-reported outcome (PRO) instruments. The objective of this qualitative study was to evaluate the content validity of the Lung Cancer Symptom Scale for Mesothelioma (LCSS-Meso) and its usability on an electronic device. METHODS: A cross-sectional methodological study, using a qualitative approach, was conducted among patients recruited from four clinical sites. The primary target population included patients with pleural mesothelioma; data were also collected from patients with peritoneal mesothelioma on an exploratory basis. Semi-structured interviews were conducted consisting of concept elicitation, cognitive interviewing, and evaluation of electronic patient-reported outcome (ePRO) usability. RESULTS: Participants (n = 21) were interviewed in person (n = 9) or by telephone (n = 12); 71% were male with a mean age of 69 years (SD = 14). The most common signs and symptoms experienced by participants with pleural mesothelioma (n = 18) were shortness of breath, fluid build-up, pain, fatigue, coughing, and appetite loss. The most commonly described symptoms for those with peritoneal mesothelioma (n = 4) were bloating, changes in appetite, fatigue, fluid build-up, shortness of breath, and pain. Participants with pleural mesothelioma commonly described symptoms assessed by the LCSS-Meso in language consistent with the questionnaire and a majority understood and easily completed each of the items. The ePRO version was easy to use, and there was no evidence that the electronic formatting changed the way participants responded to the questions. CONCLUSIONS: Results support the content validity of the LCSS-Meso and the usability of the electronic format for use in assessing symptoms among patients with pleural mesothelioma.

Perceived health status and cardiometabolic risk among a sample of youth in Mexico.

PURPOSE: To examine differences in self-reported perceived mental and physical health status, as well as known cardiometabolic risk factors in a sample of normal weight, overweight, and obese Mexican youths. METHODS: Cross-sectional analysis of 164 youths aged 11-18 years recruited in Cuernavaca, Mexico. Participants completed a self-administered questionnaire that included measures of generic and weight-specific quality of life, perceived health, physical function, depressive symptoms, and body shape satisfaction. Height, weight, and waist circumference were measured, and body mass index (BMI) was determined. Fasting blood samples from participants yielded levels of glucose, triglycerides, and cholesterol (total, HDL, and LDL). RESULTS: Nearly 50 % of participants were female, 21 % had a normal BMI, 39 % were overweight, and 40 % were obese. Obese youths reported significantly lower measures of perceived health status (PHS) and showed an increase in cardiometabolic risk, compared with normal weight youths. Physical functioning, generic and weight-specific QoL were inversely associated with BMI, waist circumference, and glucose. Depressive symptoms were positively correlated with BMI, waist circumference, glucose levels, and HDL cholesterol. No correlation was found between PHS and cardiometabolic risk measures after controlling for BMI. CONCLUSIONS: In this sample of Mexican youths, obesity was associated with a significantly lower PHS and increased cardiometabolic risk.

Perceived body shape, standardized body-mass index, and weight-specific quality of life of African-American, Caucasian, and Mexican-American adolescents.

PURPOSE: To examine associations among perceived body shape, standardized body-mass index (zBMI), and weight-specific quality of life in African-American, Caucasian, and Mexican-American adolescents, aged 11-18 years. METHODS: Self-report questionnaires were administered to 454 adolescents between 11 and 18 years of age, of whom 53% were females, 33% were Caucasians, 30% were African-Americans, and 37% were Mexican-Americans. Thirty-four percent had a healthy zBMI, 20% were overweight, and 46% were obese. RESULTS: In examining the adjusted R-square and R-square changes among stepwise regression models, the model with depressive symptoms (adjusted R-square = 0.34), perceived body shape (adjusted R-square = 0.49), and female sex (adjusted R-square = 0.53) appears to be the most parsimonious and explanatory model for these data. Race/ethnicity and age did not enter the equations, due to their significance levels being greater than the probability of removal (0.1). CONCLUSIONS: This study demonstrates the importance of including the perceptual measures of weight-specific quality of life and perceived body shape in studies of and interventions with overweight and obese adolescents. Including these perceptual measures may provide increased insight into the motivations and values of overweight and obese youth and thus be useful for designing more effective weight interventions.

Validation of the EQ-5D in a general population sample in urban China.

PURPOSE: To evaluate the reliability and validity of the EQ-5D in a general population sample in urban China. METHODS: Thousand and eight hundred respondents in 18 communities of Hangzhou, China were recruited by multi-stage stratified random sampling. Respondents self-administered a questionnaire including the EQ-5D, the SF-36, and demographic questions. Test-retest reliability at 2-week intervals was evaluated using Kappa coefficient, the intraclass correlation coefficient. The standard error of measurement (SEM) was used to indicate the absolute measurement error. Construct validity was established using convergent, discriminant, and known groups analyses. RESULTS: Complete data for all EQ-5D dimensions were available for 1,747 respondents (97%). Kappa values were from 0.35 to 1.0. The ICCs of test-retest reliability were 0.53 for the EQ-5D index score and 0.87 for the EQ VAS score. The SEM values were 0.13 (9.22% range) and 4.20 (4.20% range) for the EQ-5D index and EQ VAS scores, respectively. The Pearson's correlation coefficients between the EQ-5D and the SF-36 were stronger between comparable dimensions than those between less comparable dimensions, demonstrating convergent and discriminant evidence of construct validity. The Chinese EQ-5D distinguished well between known groups: respondents who reported poor general health and chronic diseases had worse HRQoL than those without. Older people, females, people widowed or divorced, and those with a lower socioeconomic status reported poorer HRQoL. Respondents reporting no problems on any EQ-5D dimension had better scores on the SF-36 summary scores than those reporting problems. CONCLUSIONS: The Chinese version of the EQ-5D demonstrated acceptable construct validity and fair to moderate levels of test-retest reliability in an urban general population in China.

Symptom Experience and Content Validity of the Psoriasis Symptom Scale (PSS) in Patients with Generalized Pustular Psoriasis (GPP).

INTRODUCTION: We sought to understand key symptoms of generalized pustular psoriasis (GPP) and to confirm the relevance to patients and content validity of the Psoriasis Symptom Scale (PSS) in GPP. METHODS: A targeted literature review and clinical expert interviews were conducted as background research. Patients were interviewed individually (involving concept elicitation and cognitive interviews), and a separate patient workshop was conducted to determine disease-specific symptoms of importance. RESULTS: Seven participants with moderate (n = 4), severe (n = 2), and mild (n = 1) GPP and clinician diagnosis were interviewed. During concept elicitation, all participants indicated that pustules may underlie other symptoms. Symptoms reported by all patients were pain, redness, itch, burning, and discomfort. The PSS symptoms of pain, itching, burning, and redness were reported by ≥ 86% of patients as most frequently experienced. Upon debriefing, the PSS was well understood. Relevance and importance of these symptoms was confirmed in the GPP patient workshop. CONCLUSION: Participant feedback found the PSS measure to be relevant and easy to understand. The symptoms included in the instrument, pain, redness, itch, and burning, were most frequently reported, important, and well understood by patients. Study results provided support for the content validity of the PSS for use as endpoints in GPP clinical trials.

Generalized pustular psoriasis (GPP) is a severe rare disease, including redness and boils that sometimes come with fever and other general symptoms. This study asked patients with GPP about their key symptoms, and whether the Psoriasis Symptom Scale (PSS) is relevant to them as patients. The PSS is a questionnaire with the symptoms pain, itching, burning, and redness. We searched the literature and interviewed clinical experts to guide the patient interviews. Patients were recruited through clinical sites and the National Psoriasis Foundation (NPF). The interviews discussed GPP symptoms and the PSS questionnaire. Patients with GPP were also asked about commonly experienced symptoms in a workshop. Most patients had moderate to severe GPP. Patients in both the interviews and workshop described experiencing pain, redness, itch, burning, and discomfort with their boils. During interviews, the patients said the PSS questionnaire was easy to understand. Patients in the workshop also found the PSS to be relevant and easy to understand. Patients agreed the symptoms in the PSS, pain, redness, itch, and burning, were common and important. Study results support the PSS for use with patients in clinical trials.

Validation and clinical value of the MANAGE-PD tool: A clinician-reported tool to identify Parkinson's disease patients inadequately controlled on oral medications.

INTRODUCTION: Making Informed Decisions to Aid Timely Management of Parkinson's Disease (MANAGE-PD) is a clinician-reported tool designed to facilitate timely identification and management of patients with advancing Parkinson's disease (PD) with suboptimal symptom control while on standard therapy. The objective of this study was to evaluate the validity and clinical value of the tool. METHODS: Driven by structured inputs from a steering committee and panel of PD experts, the tool was developed to classify patients into 3 categories. Validity and clinical value were elucidated using a two-pronged approach: (i) hypothetical patient vignettes (n = 10) developed based on the MANAGE-PD tool and rated by 17 PD specialists and 400 general neurologists (GN) and (ii) patients with PD (n = 2546) managed in real-world clinical settings. Vignette validity was based on concordance between PD experts' clinical judgement and MANAGE-PD vignette categorization. Patient-level data was used for known-group comparisons (validity) and discordant pair analysis (clinical value). RESULTS: The tool demonstrated strong validity and clinical value among PD specialists (intraclass coefficient [ICC] 0.843; Fleiss weighted kappa [ƙweighted] 0.79) and GN (ICC 0.690; ƙweighted 0.65) using patient vignettes. MANAGE-PD also demonstrated real-world validity and clinical value based on ability to identify patients with incrementally higher clinical, economic, and humanistic PD burden across categories of the tool (p < 0.01). CONCLUSIONS: MANAGE-PD demonstrated robust validity and clinical value in identifying patients with suboptimal PD symptom control. Clinical use of MANAGE-PD may complement treatment decision-making and facilitate timely and comprehensive management of patients with advancing PD.

Reliability, validity, and the ability to detect change of the Psoriasis Symptom Scale (PSS) in patients with plaque psoriasis.

Introduction: The primary objective of the study was to evaluate the measurement properties of the patient-reported four-item Psoriasis Symptom Scale (PSS).Methods: Analysis of phase-III data on the efficacy of risankizumab to assess psychometric characteristics of the PSS in patients with moderate-to-severe psoriasis.Results: PSS items had a good range of symptom severity coverage. The PSS had good test-retest reliability (ICCs >0.90). Convergent and discriminant validity was indicated by moderate-to-strong correlations between the PSS and Dermatology Life Quality Index (DLQI), PSS pain item and EQ-5D pain/discomfort item at week 12 (0.63), and moderate negative correlation with EQ-Visual Analog Scale score at week 12 (-0.37). Known groups validity demonstrated as mean PSS total scores varied by Psoriasis Area and Severity Index (PASI) and Static Physician's Global Assessment (sPGA) defined groups (p < .0001). PSS total scores were responsive to changes in PASI score (p < .0001) and sPGA (p < .0001). PSS minimal, clinical, and meaningful change is estimated to be 1 to 2 points; a preliminary responder definition is a total change score of 3 to 4 points.Conclusions: The PSS is a short, valid unidimensional measure of psoriasis symptom severity, well suited for use in clinical trials.

Exploring the implementation of patient-reported outcome measures in cancer care: need for more real-world evidence results in the peer reviewed literature.

BACKGROUND: To explore the existing evidence of the real-world implementation of patient-reported outcomes (PROs) in oncology clinical practice and address two aims: (1) summarize available evidence of PRO use in clinical practice using a framework based on the International Society for Quality of Life Research (ISOQOL) PRO Implementation Guide; and (2) describe reports of real-world, standardized PRO administration in oncology conducted outside of scope of a research study. METHODS: A Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) protocol was developed to guide the systematic literature review (SLR) that was conducted in MEDLINE and Embase databases. A two step search strategy was implemented including two searches based on previously completed reviews. Studies published from 2006 to 2017 were synthesized using a framework based on the ISOQOL PRO Implementation Guide. RESULTS: After screening 4427 abstracts, 36 studies met the eligibility criteria. Most elements of the ISOQOL PRO Implementation Guide were followed. Two notable exceptions were found: 1) providing PRO score interpretation guidelines (39% of studies); and 2) providing patient-management guidance for addressing issues identified by PROs (25% of studies). Of the 22 studies with an intervention component, 19 (86%) reported intervention effects on study outcomes. The European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire-Core 30 (EORTC QLQ-C30) was the most commonly used PRO (n = 10, 28%); use of 38 other PRO measures was also reported. Only three studies (8%) reported real-world PRO implementation. CONCLUSION: Reports of real-world PRO implementation are limited. Reports from studies conducted in clinical settings suggest gaps in information on PRO score interpretation and the use of PRO results to inform patient management. Before the promise of practice-based PRO assessment in oncology can be truly realized, investigators need to advance the state-of-the-art of real-time PRO score interpretation as well as developing guidance on how to use PRO insights to drive clinically-meaningful patient-management strategies.

Burden of renal angiomyolipomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

BACKGROUND: Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by benign tumors in multiple organs, including non-cancerous kidney lesions known as renal angiomyolipomas. This study's objective is to describe the age-stratified morbidity, treatment patterns, and health-related quality of life of TSC patients with renal angiomyolipomas in the United States. A cross-sectional, anonymous web-based survey was conducted with a convenience sample of TSC patients and caregivers identified through a patient advocacy organization. RESULTS: Out of the total sample of 676, 182 respondents reported having kidney complications with 33% of the pediatric group and 25% of the adult group with TSC reporting them. Of those with kidney complications, 110 (60%) reported a diagnosis of renal angiomyolipomas, of which 79 (72%) were adult patients and 31 (28%) were pediatric age patients. Eighty-four percent of the pediatric group and 76% of the adult group reported lesions on both kidneys. Of the patients experiencing involvement of only one kidney, 60% of the pediatric group and 21% of the adult group reported having multiple tumors within the affected kidney. Almost all of the sample (99%) reported seeing a physician and having a procedure or test for TSC in the past year. Less than half the respondents (44%) reported being hospitalized in the past year. Thirty-nine percent reported an emergency room visit as well. Compared to scores for patients with kidney disease, the angiomyolipoma adult patients reported significantly lower Mental Component Summary scores on the SF-12. CONCLUSIONS: Renal angiomyolipomas burden leads to frequent healthcare resource use including hospitalization, invasive treatments, and surgical procedures, which result in an impaired mental health related quality of life.

Economic burden, work, and school productivity in individuals with tuberous sclerosis and their families.

AIMS: Tuberous sclerosis complex (TSC) is a multi-organ autosomal-dominant, genetic disorder with incomplete penetrance. The multiple manifestations of TSC and impacts to numerous organ systems represent significant disease, healthcare, and treatment burden. The economic and employment burden of the disease on individuals and their families is poorly understood. This study assessed the cost of illness and work and school productivity burden associated with TSC in a cross-sectional web-survey sample. MATERIALS AND METHODS: Eligible TSC individuals and caregivers were invited through the Tuberous Sclerosis Alliance advocacy group to complete a web-based survey about illness characteristics, treatment, disease burden, direct and indirect healthcare costs, work and school impairment. RESULTS: Data from 609 TSC adults or caregiver respondents with no cognitive impairments were analyzed. TSC adults (>18 years of age) had significantly higher direct out-of-pocket costs for ER visits, expenses for medical tests and procedures, alternative treatments, medications and lifetime cost of surgeries compared to TSC pediatric individuals. Both TSC adults and TSC caregivers reported work and school absenteeism and presenteeism; however, adults reported significantly higher absenteeism and presenteeism and overall activity impairment due to TSC, as might be expected, compared to TSC caregivers. TSC adults had significantly higher absenteeism and presenteeism rates compared to adults with moderate-to-severe plaque psoriasis and muscular sclerosis. CONCLUSIONS: TSC results in considerable direct out-of-pocket medical costs and impairment to work productivity, especially for adults. Future studies should include the comparator group and examine direct cost burden in the US using electronic medical records and insurance databases.

Developing consensus among movement disorder specialists on clinical indicators for identification and management of advanced Parkinson's disease: a multi-country Delphi-panel approach.

BACKGROUND: Lack of a global consensus on the definition of advanced Parkinson's disease (APD) and considerations for timing of device-aided therapies may result in heterogeneity in care. OBJECTIVES: To reach consensus among movement disorder specialists regarding key patient characteristics indicating transition to APD and guiding appropriate use of device-aided therapies in the management of PD symptoms. METHODS: A Delphi-panel approach was utilized to synthesize opinions of movement disorder specialists and build consensus. RESULTS: A panel was comprised of movement disorder specialists from 10 European countries with extensive experience of treating PD patients (mean =24.8 ± 7.2 years). Consensus on indicators of suspected APD and eligibility for device-aided therapies were based on motor symptoms, non-motor symptoms, and functional impairments. Key indicators of APD included: (i) motor-moderate troublesome motor fluctuations, ≥1 h of troublesome dyskinesia/day, ≥2 h "off" symptoms/day, and ≥5-times oral levodopa doses/day; (ii) non-motor-mild dementia, and non-transitory troublesome hallucinations; (iii) functional impairment-repeated falls despite optimal treatment, and difficulty with activities of daily living. Patients with good levodopa response, good cognition, and <70 years of age were deemed as good candidates for all three device-aided therapies. Patients with troublesome dyskinesia were considered good candidates for both levodopa-carbidopa intestinal gel and Deep Brain Stimulation (DBS). PD patients with levodopa-resistant tremor were considered good candidates for DBS. CONCLUSION: Identifying patients progressing to APD and suitable for device-aided therapies will enable general neurologists to assess the need for referral to movement disorder specialists and improve the quality of care and patient outcomes.

Results of a Qualitative Study to Develop a Patient Reported Outcome Measure for Patients with 4 Subtypes of Soft Tissue Sarcoma.

OBJECTIVE: The objective of this research was to develop a disease-specific symptom inventory for soft tissue sarcoma. METHODS: Literature review and clinical expert and patient interviews were conducted to determine disease-specific symptoms important to patients with one of the four STS subtypes. Clinical experts identified the most relevant STS symptom items from the item pool developed from literature review. Concept elicitation interviews were conducted with patients to elicit their STS symptom experiences followed by a completion of the draft symptom list via web survey. A cognitive interview was conducted on the comprehension and importance of the symptom items. RESULTS: Eighty-three symptom items were compiled and discussed with three clinical experts who identified 26 symptoms specific to the four STS subtypes. A total sample of 27 STS participants with self-reported leiomyosarcoma (74%), undifferentiated sarcoma (15%), synovial sarcoma (7%), or liposarcoma (4%) diagnosis completed the web survey and 10 were interviewed. The draft 12-item STS-specific symptom inventory includes abdominal pain, pressure in abdomen, early satiety, bloating, gastrointestinal pain, muscle pain, bone pain, heavy menstrual flow, shortness of breath, chest pain, cough, and painful menstruation. CONCLUSION: A number of symptoms are common across STS subtypes and may form a single STS symptom inventory.

Content validity and ePRO usability of the BPI-sf and "worst pain" item with pleural and peritoneal mesothelioma.

BACKGROUND: The Brief Pain Inventory-short form (BPI-sf) is widely used in self-reported pain assessment, incorporates pain numeric rating scales (NRS) and is commonly utilized in electronic format in clinical trials, however, there is no published information about its usability as an electronic patient-reported outcome (ePRO) measure. The objective of this qualitative study was threefold: 1) to better understand pain experiences among patients with pleural or peritoneal mesothelioma; 2) to assess the interpretability of the instructions, item stem, recall period, and response option of the "worst pain" item of the BPI-sf; and 3) to examine the usability of the TrialMax Touch™ (CRF Health, Inc., Plymouth Meeting, PA) screen-based handheld device and the electronic format of the BPI-sf in a sub-sample of pleural mesothelioma patients. METHODS: A cross-sectional qualitative study was conducted among participants with pleural and peritoneal mesothelioma recruited from 4 clinical sites in the US. Semi-structured telephone or in-person interviews were conducted consisting of concept elicitation, cognitive interviewing of the 11-item BPI-sf, and in-person interview evaluation of ePRO assessment usability in pleural mesothelioma patients. RESULTS: Twenty-one participants recruited from 4 clinical sites in the US were interviewed in-person (n = 9) and by telephone (n = 12); 71% male; mean age 68.7 ± 13.6 years. Pleural and peritoneal patients described pain as ranging from discomfort to intense pain and reported being able to distinguish tumor pain from treatment pain. The BPI-sf "worst pain" item was relevant to, and easily understood by, study participants with pleural and peritoneal mesothelioma. The ePRO version was found to be easy to use, but readability of small font may be an issue. Participants reported minimal differences between their responses on the paper and ePRO version for all of the pain severity and pain interference items. CONCLUSIONS: Results support the relevance and ease of understanding of the "worst pain" item and provide support for its content validity in patients with pleural and peritoneal mesothelioma. Usability of the ePRO format of the BPI-sf was confirmed for use in clinical trials among patients with pleural mesothelioma.

Tuberous sclerosis complex: a survey of health care resource use and health burden.

PURPOSE: Tuberous sclerosis complex (TSC) is a multiorgan, autosomal-dominant genetic disorder with incomplete penetrance. METHODS: This analysis of a web-based survey focuses on the clinical presentation, management, and associated burden of patients with TSC in the United States. RESULTS: A total of 676 TSC patients or caregivers responded. Both pediatric and adult patient groups experienced skin lesions (77% and 44%), seizures (77% and 24%), and kidney complications (33% and 25%) as well as other manifestations. Patient groups averaged 22 visits to a physician, nine procedures/tests, two emergency room visits, and two hospital admissions in the past year. Standardized tests were administered for health-related quality of life and TSC patients reported significantly worse mental health scores and better physical health scores compared to a normative sample of cancer patients. CONCLUSION: Results demonstrate that TSC is associated with significant clinical burden, resource utilization, and decreased mental health well-being.

Caring for Children With Tuberous Sclerosis Complex: What Is the Physical and Mental Health Impact on Caregivers?

Disease burden associated with tuberous sclerosis complex, a genetic disorder characterized by benign tumor growth including lesions in multiple organs, puts tremendous demands on families. This analysis examines the physical and mental health burden of tuberous sclerosis complex caregivers in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex caregivers collected information; descriptive analyses were conducted on age-based subgroups. A total of 275 caregivers of tuberous sclerosis complex patients responded. Mean patient age ≤ 18 years was 6.9 (±4.4) and 42.3 (±18.2) for patients >18 years of age. Caregivers reported multiple tuberous sclerosis complex manifestations and high health care utilization for patients. Caregivers spending more time on doctor visits or researching tuberous sclerosis complex had lower physical and mental health-related quality of life scores and more depressive symptoms. Tuberous sclerosis complex caregivers had significantly lower physical and mental health-related quality of life scores and more depressive symptomatology compared to US healthy adult population norms.

The burden of subependymal giant cell astrocytomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

Tuberous sclerosis complex is a genetic disorder characterized by benign tumor growth including lesions in the ventricular system of the brain known as subependymal giant cell astrocytomas. This analysis focuses on the clinical presentation, management, and associated burden of subependymal giant cell astrocytomas in patients with tuberous sclerosis complex in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex patients and caregivers collected information, and descriptive analyses were conducted on age-based subgroups. A total of 116 tuberous sclerosis complex-subependymal giant cell astrocytoma patients or caregivers responded (17% of the total tuberous sclerosis complex sample). Mean and median patient ages were 25.5 and 23.5 years. Besides subependymal giant cell astrocytomas, patients also experienced skin lesions (72%), seizures (65%), and cognitive concerns (60%). Forty-five percent reported having brain surgery (22% for subependymal giant cell astrocytoma). In the past year, 42% of patients were admitted at least once to the hospital whereas 39% went to the emergency department. Results demonstrate that tuberous sclerosis complex-subependymal giant cell astrocytoma is associated with significant clinical burden, resource utilization, and decreased well-being.

A systematic literature review of methodologies used to assess medication adherence in patients with diabetes.

OBJECTIVE: Adhering to prescribed medication is often a problem for patients with diabetes yet there is no consensus on how best to measure adherence in this patient population. This systematic literature review critically reviewed and summarized the methods used to measure medication adherence in patients with diabetes (on oral hypoglycemic agents [OHAs] and/or insulin) in original research published between 2007-2013. STUDY DESIGN: Literature review. METHODS: A systematic search for methods to assess medication adherence in patients with type I or type II diabetes was conducted using PubMed, EMBASE, PsychInfo, and Cochrane databases. Two researchers independently screened abstracts for initial eligibility and then applied the inclusion/exclusion criteria to the relevant full-text articles. RESULTS: Fifty-nine articles met the criteria for inclusion. Subjective assessment (observer-reported and patient-reported), pill counts, Medication Event Monitoring System (MEMS), cell-phone real-time assessment, and logbooks were used in prospective studies. In pharmacy claims databases, medication possession ratios (MPRs), or some derivation thereof, were utilized. Each method has strengths and weaknesses, but few approaches specifically addressed issues unique to assessing insulin adherence. Three novel approaches (using cell-phone real-time assessment, computerized logbooks, and a questionnaire about different dosing irregularities) provided insight on timing and dosing issues that could be useful for highlighting interventions to improve insulin adherence. CONCLUSION: No gold standard exists for measuring medication adherence in patients with diabetes. The plethora of adherence methods precludes the comparison of adherence rates across studies. Greater consistency is therefore needed in adherence measurement, including question content, recall period, and response options for self-report measures. Novel methods for understanding adherence to variable-dosed insulin require further research. Researchers should select a methodology that best fits their research question, study design, patient population and resources.