Quality of Care in Ulcerative Colitis: A Modified Delphi Panel Approach.

OBJECTIVES: To establish clinical consensus on important and relevant quality-of-care (QoC) attributes in ulcerative colitis (UC) treatment that may improve treatment outcomes and guide best practices. METHODS: Thirty-eight QoC attributes were identified in a literature review. Sixteen European-based experts were selected based on their contributions to UC guidelines, publications, and patient care. A 3-round, modified Delphi panel was conducted including an interview round, and 2 web-based rounds to reach consensus and finalize a QoC attribute list. RESULTS: The draft QoC attribute list derived from a literature review and Round 1, expert interviews, comprised 63 attributes. In Rounds 2 and 3, the QoC attributes frequently rated as critically important were diagnosis (n = 15, 93.8%), treatment adherence (n = 15, 93.8%), and access to care/treatment (Round 2: n = 14, 87.5%; Round 3: n = 15, 93.8%). The final QoC attribute list consisted of 61 attributes across 20 domains, with the most attributes reported in the "treatment goals" domain (n = 9). CONCLUSION: QoC is a complex and evolving concept that can improve outcomes while maximizing healthcare resources. Limited time and resources hamper clinicians' ability to openly and empathetically communicate with patients; novel technology may help to offer solutions.

Development of a new patient-reported outcome (PRO) measure on the Impact of Nighttime Urination (INTU) in patients with nocturia-Psychometric validation.

AIMS: To psychometrically evaluate the Impact of Nighttime Urination (INTU) questionnaire, a new patient-reported outcome measure developed to assess the impact of nocturia on health and functioning in a multicenter, behavioral modification (fluid restriction) study. METHODS: Participants aged 50-95 years with at least two voiding episodes/night for ≥6 months completed voiding diaries and the INTU on 3 consecutive days during weeks 1 and 2 (same day recall) and completed the Pittsburgh Sleep Quality Index (PSQI) and Nocturia Quality of Life Questionnaire (N-QOL) at baseline and days 8 and 15. Psychometric evaluations of the INTU were conducted. RESULTS: Rasch analysis showed the INTU to be a unidimensional construct, with most items located on the severe end of the symptom severity continuum. In addition to an Overall Impact Score (10 items), exploratory factor analysis affirmed by confirmatory factor analysis identified two domains: Daytime (six items) and Nighttime (four items) Impact Scores (comparative fit index = 0.968; root mean square error of approximation = 0.08). Concurrent validity met prespecified hypotheses, indicating similarity of concepts with the PSQI (correlation [r] = 0.627) and N-QOL (r = -0.784) total scores. The INTU differentiated among patients with different nocturic episode frequencies (P < 0.05 for all three summary scores). Statistically significant decreases were observed in mean Overall and Nighttime Impact Scores at week 2 versus week 1 in responders, indicating that the instrument can detect changes in response to symptom improvements. CONCLUSIONS: The INTU questionnaire demonstrated robust measurement properties and is a suitable tool for assessing the patient-reported impact of nocturia on health and functioning.

Psychometric assessment of the IBS-D Daily Symptom Diary and Symptom Event Log.

PURPOSE: Diarrhea-predominant irritable bowel syndrome (IBS-D) can considerably impact patients' lives. Patient-reported symptoms are crucial in understanding the diagnosis and progression of IBS-D. This study psychometrically evaluates the newly developed IBS-D Daily Symptom Diary and Symptom Event Log (hereafter, "Event Log") according to US regulatory recommendations. METHODS: A US-based observational field study was conducted to understand cross-sectional psychometric properties of the IBS-D Daily Symptom Diary and Event Log. Analyses included item descriptive statistics, item-to-item correlations, reliability, and construct validity. RESULTS: The IBS-D Daily Symptom Diary and Event Log had no items with excessive missing data. With the exception of two items ("frequency of gas" and "accidents"), moderate to high inter-item correlations were observed among all items of the IBS-D Daily Symptom Diary and Event Log (day 1 range 0.67-0.90). Item scores demonstrated reliability, with the exception of the "frequency of gas" and "accidents" items of the Diary and "incomplete evacuation" item of the Event Log. The pattern of correlations of the IBS-D Daily Symptom Diary and Event Log item scores with generic and disease-specific measures was as expected, moderate for similar constructs and low for dissimilar constructs, supporting construct validity. Known-groups methods showed statistically significant differences and monotonic trends in each of the IBS-D Daily Symptom Diary item scores among groups defined by patients' IBS-D severity ratings ("none"/"mild," "moderate," or "severe"/"very severe"), supporting construct validity. CONCLUSIONS: Initial psychometric results support the reliability and validity of the items of the IBS-D Daily Symptom Diary and Event Log.

Validation of a New Patient-Reported Outcome Measure for Facial Acne: The Acne Symptom and Impact Scale (ASIS).

OBJECTIVE: The objective of this study was to evaluate the psychometric performance of the Acne Symptom and Impact Scale (ASIS) for use in adolescents and adults with acne vulgaris. METHODS: Psychometric evaluation was performed using both traditional psychometrics in line with proposed US Food and Drug Administration (FDA) criteria and new psychometric methods, Rasch Measurement Theory (RMT). Assessment of equivalence was also evaluated between Caucasians and Non-Caucasians on individual items. RESULTS: One-hundred fifty subjects completed baseline and follow-up assessments (89 [59.33%] in the Caucasian group and 61 [40.67%] in the Non-Caucasian group). Psychometric analyses demonstrated that the ASIS Sign and Impact domains both performed well. Each domain fulfilled traditional psychometric criteria (Cronbach's alpha=0.79-0.92; test-retest reliability=0.75-0.78) and mostly satisfied Rasch psychometric criteria (person-reliability=0.72-0.93; person-separation=1.61-3.69). Select individual ASIS Items also performed well across all measures and were shown to be reliable and valid as stand-alone items. A similar pattern of results were found for both Caucasian and Non-Caucasian racial subgroups. CONCLUSIONS: These findings provide empirical evidence that the ASIS is a reliable and valid PRO measure that can accurately assess the severity of symptoms and impacts associated with acne vulgaris.

Key concepts of migraine postdrome: a qualitative study to develop a post-migraine questionnaire.

OBJECTIVE: To understand migraine postdrome by directly interviewing migraine patients with postdrome symptoms. To document these symptoms, as well as impacts, as a prelude to developing a postdrome migraine questionnaire. BACKGROUND: Migraine attacks are traditionally divided into 4 phases. Of these, the postdrome is the least studied, and no patient-reported outcomes to assess symptoms and impacts of this migraine phase have been published. METHODS: Qualitative concept elicitation focus groups were conducted with 34 patients in 3 geographically diverse US cities to elicit the symptoms and burden of migraine postdrome. Data elicited from focus groups were coded using Atlas.ti software to facilitate identification of concepts and terminologies of migraine postdrome. A draft questionnaire was developed based on the symptoms and impacts of migraine postdrome described by patients. Cognitive debriefing interviews were conducted with 15 patients in Connecticut and Chicago to confirm content validity, relevance, and comprehension. RESULTS: Patients defined the onset of postdrome as when they no longer experienced the migraine pain. Postdrome was often described as "[being] or [feeling] wiped out" and "headache hangover." The symptoms most frequently reported by the patients who participated in the focus groups and included in the draft post-migraine questionnaire were: tiredness, difficulty concentrating, weakness, dizziness, lightheadedness, and decreased energy. Patients also reported decreased activity level as a result of experiencing postdrome symptoms. Postdrome symptoms were reported to impact the ability to work, to affect family interactions and social life, and to cause cognitive impairment. A preliminary questionnaire measuring severity and duration of symptoms and severity of impacts of the post-migraine experience, with an 11-point (0 to 10) response scale, was developed. This preliminary questionnaire was tested for content validity, relevance, and comprehension using cognitive debriefing interviews. All patients reported that the questionnaire was relevant to their condition. Irrelevant and redundant items such as body tension and annoyance were eliminated. CONCLUSIONS: Migraine postdrome is debilitating for those who experience it. Concept elicitation and cognitive debriefing research support the relevance of the items in the post-migraine questionnaire. Future research will provide evidence of the post-migraine questionnaire's psychometric properties and interpretation guidelines.

Psychometric validation of anti-clot treatment scale and treatment satisfaction questionnaire for medication version II in Japanese patients with atrial fibrillation.

Aims: The Anti-Clot Treatment Scale (ACTS) and Treatment Satisfaction Questionnaire for Medication version II (TSQM-II) are validated treatment satisfaction patient-reported outcome (PRO) instruments. The ACTS includes two domains: Burdens and Benefits; the TSQM-II includes four: Effectiveness, Side Effects, Convenience, and Global Satisfaction. Japanese-language versions of the ACTS and TSQM-II have been developed and linguistically validated. This study aimed to assess their psychometric properties in Japanese patients with atrial fibrillation (AF). Materials and methods: ACTS and TSQM-II data from 534 patients with AF were collected in a Japanese post-marketing surveillance study of a direct oral-anticoagulant, rivaroxaban. Four key psychometric properties, in line with best practice guidelines from the US Food and Drug Administration, were examined using traditional psychometric methods: acceptability, scaling assumptions, reliability (i.e. internal consistency reliability, test-retest reliability), and construct validity (i.e. convergent validity and known groups). Results: ACTS Burdens and Benefits and TSQM-II Effectiveness, Convenience, and Global Satisfaction scales were found to be acceptable (e.g. item-level missing data at baseline <4%), with all scales having good internal consistency (Cronbach's alpha > 0.80). test-retest reproducibility intraclass correlation coefficients for the ACTS Burdens and Benefits were 0.59 and 0.65, respectively, and between 0.54-0.61 for the TSQM-II scales. Known-groups validity for the ACTS and TSQM-II was supported by differences in scale scores by positive and negative impact (p < 0.05). Correlations between the ACTS and TSQM-II (convergent validity) were lower than expected (range r = 0.09-0.48), but in line with the original ACTS development study. Limitations: Evaluation of test-retest reproducibility was limited by assessment period, which was longer (3 months) than recommended guidelines (usually up to 2 weeks). Conclusions: Overall, Japanese versions of ACTS and TSQM-II scales satisfied internal consistency reliability and traditional validity criteria. Our study supports the ACTS and TSQM-II as appropriate PRO instruments to measure satisfaction with anticoagulant treatment in Japanese patients with AF. Trial registration: NCT01598051, clinicaltrials.gov; registered April 20, 2012.

Development and initial psychometric evaluation of patient-reported outcome questionnaires to evaluate the symptoms and impact of hidradenitis suppurativa.

BACKGROUND: Two patient-reported outcome (PRO) questionnaires, the Hidradenitis Suppurativa Symptom Assessment (HSSA) and Hidradenitis Suppurativa Impact Assessment (HSIA), were developed to measure signs, symptoms and impacts of HS in treatment efficacy studies. METHODS: In accordance with FDA guidelines and published best practices, four stages of research were conducted to create the questionnaires: concept elicitation, questionnaire construction, content evaluation and psychometric evaluation. RESULTS: Subjects (N = 20) who participated in the concept elicitation stage reported 15 unique HS-related signs and symptoms and 51 impacts. Following this, eight sign and symptom concepts and 21 impacts were selected for construction of the HSSA and HSIA, respectively. During content evaluation, cognitive debriefing interviews with HS subjects (N = 20) confirmed subjects could read, comprehend and meaningfully respond to both questionnaires. Modifications made after this stage of work resulted in a nine-item HSSA and a 17-item HSIA. The HSSA and HSIA were subsequently entered into a US-based observational study (N = 40), and the scores produced by each were found to be reliable, construct valid, and able to distinguish among clinically distinct groups. CONCLUSIONS: The HSSA and HSIA are content-valid, HS-specific, PRO questionnaires with demonstrated ability to generate reliable, valid scores when administered to patients with HS in a research setting.

Quality of Care in Rheumatoid Disease from the Clinician Perspective: A Modified Delphi Panel Approach.

INTRODUCTION: To establish clinical consensus on important and relevant quality-of-care (QoC) attributes in rheumatic disease (RD) treatment that may improve treatment outcomes and guide best practices. METHODS: Twenty-three QoC attributes were identified in a literature review. Fifteen European-based clinicians were selected based on their contributions to RD guidelines, publications, and patient care. A three-round (an interview round and two web-based rounds) modified Delphi panel was conducted to reach consensus and finalize a QoC attribute list. RESULTS: In round 1 (clinician interviews), clinicians reported 52 unique QoC attributes across 14 themes, with the greatest number of attributes reported in the "treatment goals" (n = 7) and "remote monitoring" (n = 7) themes. During rounds 2 and 3, the critically important QoC attributes most frequently reported were access to care/treatment (n = 14, 93.3%), safety of treatment (round 2 n = 14, 93.3%, round 3 n = 13, 86.7%), and access to clinicians and specialists (round 2: n = 13, 86.7%, round 3: n = 14, 93.3%). The final list contained 53 QoC attributes. CONCLUSION: The study demonstrates consensus across several themes of QoC. Quality of care is a complex, multidimensional, and fluid concept that can be improved by ensuring patients have access to care, open communication between patients and clinicians, and the use of novel strategies, such as remote monitoring. Utilization of the attribute list can potentially improve the lives of patients, provide clinicians with tools to provide greater QoC, and improve the healthcare system as a whole. FUNDING: Merck & Co., Inc.

Use of antipsychotic blood levels in clinician decision making: A cross-over study using clinical vignettes of patients with schizophrenia.

The cause of treatment failure of antipsychotic medications is often difficult to determine in patients with schizophrenia. Evaluation of antipsychotic blood levels (ABLs) may aid clinicians in determining the cause of antipsychotic failure. The Clinical Assessment of the Schizophrenia Patient (CASP) was developed to evaluate clinical decision making during outpatient visits. The CASP assesses changes in medications, psychosocial treatments, and acute interventions along with factors influencing clinical decision making. Nine vignettes representative of clinical situations in patients with schizophrenia were created in two versions (one with ABLs, one without ABLs). The CASP was used to evaluate clinical decisions using the vignettes. Thirty-four clinicians participated in the study. In 8 out of 9 vignettes, most clinicians (at least 89.7%) made a different clinical decision with ABLs compared to without ABLs. In assessing the usefulness of ABLs, a majority (60.7%-85.7%, depending on the vignette) of clinicians responded that ABLs changed their clinical decision for 8 vignettes. Most clinicians (79%-93%) responded that they were more confident in their decisions with ABL information. This study demonstrated that ABLs have the potential to influence clinical decision making in the treatment of patients with schizophrenia.

Patient-reported treatment satisfaction with rivaroxaban in Japanese non-valvular atrial fibrillation patients: an observational study.

OBJECTIVES: Rivaroxaban has previously been shown to be as efficacious and safe as warfarin for the prevention of stroke in non-valvular atrial fibrillation (NVAF). Therefore, treatment satisfaction becomes an important consideration. Here we examine treatment satisfaction in Japanese NVAF patients who were switched from warfarin to rivaroxaban. METHODS: Patient-reported outcome (PRO) data were collected as part of a prospective, multi-center, post-marketing surveillance (PMS) of a direct oral-anticoagulant, rivaroxaban, in Japan. The Anti-Clot Treatment Scale (ACTS) and the Treatment Satisfaction Questionnaire for Medication version II (TSQM-II) were collected at baseline, month 3, and month 6. Change in scores from baseline to month 3 and month 6 were assessed. Exploratory analyses included change in scores by patient characteristics. Safety and effectiveness of rivaroxaban were also assessed. RESULTS: ACTS Burdens scores significantly improved at month 3 (54.6 ± 6.3) and month 6 (54.5 ± 6.5) compared to baseline (51.0 ± 7.6) (p < .001). ACTS Benefits score remained stable over time (baseline = 10.1 ± 2.8, month 3 = 10.2 ± 3.1, month 6 = 10.1 ± 3.1). Mean TSQM-II sub-scale scores significantly improved at month 3 and month 6 compared to baseline for all four domains (all p < .001). CONCLUSIONS: Findings suggest treatment satisfaction may improve in Japanese NVAF patients after a switch from warfarin to rivaroxaban. Higher treatment satisfaction may translate into improved treatment adherence, which is critical for the long-term prevention of stroke.

Development of the Rheumatoid Arthritis Symptom Questionnaire (RASQ): a patient reported outcome scale for measuring symptoms of rheumatoid arthritis.

BACKGROUND: Rheumatoid arthritis (RA), a chronic, progressive inflammatory, autoimmune disease, can substantially reduce health-related quality of life (HRQoL) and lead to severe disability and early mortality. Patient-reported outcome (PRO) instruments are used to assess the patient experience of RA symptoms and impacts, and can capture RA treatment effects. To address limitations in existing PRO instruments, this research aimed to establish the content validity of a new instrument, the Rheumatoid Arthritis Symptom Questionnaire (RASQ), to assess the signs and symptoms of RA. METHOD: The most important and relevant sign and symptom concepts for RA patients were identified through a targeted review of the published literature, expert opinion, and concept elicitation patient interviews. Cognitive interviews were conducted with patients to test the comprehensibility and comprehensiveness of the RASQ. RESULTS: Seven symptoms emerged consistently across the conceptual research: joint pain, joint swelling, joint stiffness, joint tenderness, joint warmth, muscle pain, and tiredness. Draft item content was developed to assess these symptoms, in addition to a single impact item, resulting in three RASQ versions: two utilizing a 7 day recall period (one assessing symptoms at their worst, the other on average) and a third using a 24 hour recall period assessing symptoms at their worst. Cognitive interview results demonstrated patient understanding and ability to use the instrument. CONCLUSIONS: Content validity of the RASQ was established in accordance with instrument development guidelines. The RASQ fills a measurement gap by assessing the RA signs and symptoms most important to patients. Research evaluating the RASQ's psychometric properties is underway.

Diarrhea-predominant irritable bowel syndrome: creation of an electronic version of a patient-reported outcome instrument by conversion from a pen-and-paper version and evaluation of their equivalence.

BACKGROUND: Subjects with diarrhea-predominant irritable bowel syndrome (IBS-D) experience abdominal cramping, bloating, pressure, and pain. Due to an absence of clinical biomarkers for IBS-D severity, evaluation of clinical therapy benefits depends on valid and reliable symptom assessments. A patient-reported outcome (PRO) instrument has been developed, comprising of two questionnaires - the IBS-D Daily Symptom Diary and IBS-D Symptom Event Log - suitable for clinical trials and real-world settings. This program aimed to support instrument conversion from pen-and-paper to electronic format. MATERIALS AND METHODS: Digital technology (Android/iOS) and a traditional mode of administration study in the target population were used to migrate or convert the validated PRO IBS-D pen-and-paper measure to an electronic format. Equivalence interviews, conducted in three waves, each had three parts: 1) conceptual equivalence testing between formats, 2) electronic-version report-history cognitive debriefing, and 3) electronic version usability evaluation. After each inter-view wave, preliminary analyses were conducted and modifications made to the electronic version, before the next wave. Final revisions were based on a full analysis of equivalence interviews. The final analysis evaluated subjects' ability to read, understand, and provide meaningful responses to the instruments across both formats. Responses were classified according to conceptual equivalence between formats and mobile-format usability assessed with a questionnaire and open-ended probes. RESULTS: Equivalence interviews (n=25) demonstrated conceptual equivalence between formats. Mobile-application cognitive debriefing showed some subjects experienced difficulty with font/screen visibility and understanding or reading some report-history charts and summary screens. To address difficulties, minor revisions/modifications were made and landscape orientation and zoom-in/zoom-out features incorporated. CONCLUSION: This study indicates that the two administration modes are conceptually equivalent. Since both formats are conceptually equivalent, both are psychometrically reliable, as established in the pen-and-paper version. Subjects found both mobile applications (Android/iOS) offered many advantages over the paper version, such as real-time assessment of their experience.

Development and psychometric evaluation of the self-assessment of psoriasis symptoms (SAPS) - clinical trial and the SAPS - real world patient-reported outcomes.

OBJECTIVE: The Self-Assessment of Psoriasis Symptoms - Clinical Trials (SAPS-CT) and SAPS - Real World (SAPS-RW) were simultaneously created to assess the experience of plaque psoriasis in two unique contexts. METHODS: Qualitative and quantitative research was conducted in four phases namely concept elicitation, questionnaire construction, content evaluation and psychometric evaluation. RESULTS: Following concept elicitation, 18 concepts were selected to inform questionnaire construction of the SAPS-CT and SAPS-RW. To accommodate each context of use, the SAPS-CT asks respondents to rate the target symptom 'at its worst' in the 24 h prior to assessment, while the SAPS-RW asks respondents to rate the target symptom "on average" in the 7 days prior to assessment. Cognitive debriefing confirmed that patients could comprehend and provide meaningful responses to both versions and, after minor modifications, resulted in 11-item questionnaires administered in an observational study (N = 200). Results from the observational study informed further item reduction (SAPS-RW to six items and SAPS-CT to nine items) and demonstrated that scores from each were reliable (Cronbach's α > 0.90, test-retest intraclass correlation coefficient >0.70), construct valid and able to differentiate among clinically distinct groups. CONCLUSION: The SAPS-CT and SAPS-RW are content-valid PRO questionnaires capable of producing psychometrically sound scores when administered chronic to plaque psoriasis patients.

Psychometric evaluation of the Rheumatoid Arthritis Symptom Questionnaire (RASQ) in an observational study.

OBJECTIVE: To describe the psychometric performance of the scores produced by the Rheumatoid Arthritis Symptom Questionnaire (RASQ), a new patient-reported outcome (PRO) questionnaire developed to assess the signs and symptoms of rheumatoid arthritis (RA). METHODS: Adult subjects with clinically confirmed RA completed a set of questionnaires (including the RASQ) at an initial study visit (Day 1), and then completed the RASQ and the Patient Global Impression of Change (PGI-C) on their own on Day 8. Demographic and health data were summarized using descriptive statistics, and psychometric analyses were conducted, including: acceptability, item and scale distribution, reliability (internal consistency and test-re-test reliability), and construct-related validity (convergent validity and known-groups methods). RESULTS: In total, 200 subjects (females = 61.5%; white = 72.0%; and age [mean] = 60.7 years) with RA were recruited across the US and included in the analysis. There were no missing data recorded for the RASQ, and scores were well distributed for both timepoints. The RASQ Total Symptom Score surpassed the threshold (α ≥ 0.70) for internal consistency at Day 1 (α = 0.967) and test-re-test score reliability (intra-class correlation coefficient [ICC] > 0.70) (ICC = 0.960). Convergent validity analyses demonstrated that the RASQ items and Total Symptom Score had high correlations (convergent validity) with other PRO questionnaires. Known-groups methods demonstrated that the RASQ (Total Symptom Score and all single items) can differentiate between clinically distinct groups. CONCLUSIONS: The RASQ is capable of producing psychometrically sound scores when administered to adults with RA.

Evaluation and performance of a newly developed patient-reported outcome instrument for diarrhea-predominant irritable bowel syndrome in a clinical study population.

BACKGROUND: To evaluate the psychometric properties of the newly developed seven-item Irritable Bowel Syndrome - Diarrhea predominant (IBS-D) Daily Symptom Diary and four-item Event Log using phase II clinical trial safety and efficacy data in patients with IBS-D. This instrument measures diarrhea (stool frequency and stool consistency), abdominal pain related to IBS-D (stomach pain, abdominal pain, abdominal cramps), immediate need to have a bowel movement (immediate need and accident occurrence), bloating, pressure, gas, and incomplete evacuation. METHODS: Psychometric properties and responsiveness of the instrument were evaluated in a clinical trial population [ClinicalTrials.gov identifier: NCT01494233]. RESULTS: A total of 434 patients were included in the analyses. Significant differences were found among severity groups (p < 0.01) defined by IBS Patient Global Impression of Severity (PGI-S) and IBS Patient Global Impression of Change (PGI-C). Severity scores for each Diary and Event Log item score and five-item, four-item, and three-item summary scores were calculated. Between-group differences in changes over time were significant for all summary scores in groups stratified by changes in PGI-S (p < 0.05), two of six Diary items, and three of four Event Log items; a one-grade change in PGI-S was considered a meaningful difference with mean change scores on all Diary items -0.13 to -0.86 [standard deviation (SD) 0.79-1.39]. Similarly, for patients who reported being 'slightly improved' (considered a clinically meaningful difference) on the PGI-C, mean change scores on Diary items ranged from -0.45 to -1.55 (SD 0.69-1.39). All estimates of clinically important change for each item and all summary scores were small and should be considered preliminary. These results are aligned with the previous standalone psychometric study regarding reliability and validity tests. CONCLUSIONS: These analyses provide evidence of the psychometric properties of the IBS-D Daily Symptom Diary and Event Log in a clinical trial population.

Cultural adaptation: translatability assessment and linguistic validation of the patient-reported outcome instrument for irritable bowel syndrome with diarrhea.

BACKGROUND AND OBJECTIVE: Following a 2009 US Food and Drug Administration guidance, a new patient-reported outcome (PRO) instrument was developed to support end points in multinational clinical trials assessing irritable bowel syndrome with diarrhea (IBS-D) symptom severity. Our objective was to assess the translatability of the IBS-D PRO instrument into ten languages, and subsequently perform a cultural adaptation/linguistic validation of the questionnaire into Japanese and US Spanish. MATERIALS AND METHODS: Translatability assessments of the US English version of the IBS-D PRO were performed by experienced PRO translators who were native speakers of each target language and currently residing in target-language countries. Languages were Chinese (People's Republic of China), Dutch (the Netherlands), French (Belgium), German (Germany), Japanese (Japan), Polish (Poland), Portuguese (Brazil), Russian (Russia), Spanish (Mexico), and Spanish (US). The project team assessed the instrument to identify potential linguistic and/or cultural adaptation issues. After the issues identified were resolved, the instrument was translated into Spanish (US) and Japanese through a process of two forward translations, one reconciled translation, and one backward translation. The project team reviewed the translated versions before the instruments were evaluated by cognitive debriefing interviews with samples of five Spanish (US) and five Japanese IBS-D patients. RESULTS: Linguistic and cultural adaptation concerns identified during the translatability assessment required minor revisions, mainly the presentation of dates/times and word structure. During the cognitive debriefing interviews, two of five Spanish respondents misunderstood the term "bowel movement" to mean only diarrhea in the Spanish version. Consequently, the term was changed from "movimiento intestinal" to "evacuaciones". None of the Japanese respondents identified issues with the Japanese version. CONCLUSION: The translatability of the IBS-D PRO instrument into ten target languages was confirmed, with only minor changes made to the translations of the instrument. The translation and linguistic validation into Spanish (US) and Japanese provide evidence that this instrument can be used in multinational trials and clinical settings.

Development and validation of a novel patient-reported treatment satisfaction measure for hyperfunctional facial lines: facial line satisfaction questionnaire.

BACKGROUND: Facial lines or wrinkles are among the most visible signs of aging, and minimally invasive cosmetic procedures are becoming increasingly popular. AIMS: The aim of this study was to develop and validate the Facial Line Satisfaction Questionnaire (FLSQ) for use in adults with upper facial lines (UFL). METHODS: A literature review, concept elicitation interviews (n = 33), and cognitive debriefing interviews (n = 23) of adults with UFL were conducted to develop the FLSQ. The FLSQ comprises Baseline and Follow-up versions and was field-tested with 150 subjects in a US observational study designed to assess its psychometric performance. Analyses included acceptability (item and scale distribution [i.e. missingness, floor, and ceiling effects]), reliability, and validity (including concurrent validity). RESULTS: In total, 69 concepts were elicited during patient interviews. Following cognitive debriefing interviews, the FLSQ-Baseline version included 11 items and the Follow-up version included 13 items. Response rates for the FLSQ were 100% and 73% at baseline and follow-up, respectively; no items had excessive missing data. Questionnaire scale scores were normally distributed. Most domain scores demonstrated good internal consistency reliability (Cronbach's α ≥ 0.70). Most items within their respective domains exhibited good convergent (item-scale correlations > 0.40) and discriminant (items had higher correlation with their hypothesized scales than other scales) validity. Concurrent validity correlation coefficients of the FLSQ domain scores with the associated concurrent measures were acceptable (range: r = 0.40-0.70). Six FLSQ items demonstrated reliability and validity as stand-alone items outside their domains. CONCLUSIONS: The FLSQ is a valid questionnaire for assessing treatment expectations, satisfaction, impact, and preference in adults with UFL.