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Introduction: The standard of care for pediatric growth hormone deficiency (pGHD) is once-daily recombinant human growth hormone (rhGH). Somatrogon, a long-acting rhGH, requires less frequent, once-weekly, dosing. We describe physicians' preference for, experiences, and satisfaction with once-weekly somatrogon vs once-daily rhGH. Methods: English-speaking investigators from somatrogon's global phase III study (NCT02968004) with prior experience using once-daily rhGH were included. Participants answered an online survey containing 14 closed- and open-ended items. Results: Twenty-four pediatric endocrinologists (41.7% men; 79.2% practiced at public/private hospitals) from 12 countries with 25.8 ± 12.0 years' experience treating pGHD completed the survey. In terms of the time and effort required to explain device instructions, injection regimen, procedure for missed injection, and address patients'/caregivers' concerns, a similar proportion of physicians chose once-weekly somatrogon and once-daily rhGH; 62.5% physicians indicated that once-daily rhGH required greater effort to monitor adherence. Overall, 75% preferred once-weekly somatrogon over once-daily rhGH, 79.2% considered once-weekly somatrogon to be more convenient and less burdensome, and 83.3% were likely to prescribe somatrogon in the future. Overall, 50% felt that once-weekly somatrogon was more beneficial to patients, while 50% chose "No difference". Most physicians (62.5%) felt both regimens were equally likely to support positive long-term growth outcomes and reduce healthcare utilization. More physicians were "very satisfied" with once-weekly somatrogon (62.5%) than with once-daily rhGH (16.7%). Reduced injection frequency, patient and caregiver burden, increased convenience, and improved adherence were reasons for these choices. Conclusion: Physicians had a positive experience with, and perception of, treating pGHD with once-weekly somatrogon.

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BACKGROUND: Pediatric patients with eosinophilic esophagitis (EoE) experience heterogeneous symptoms and the patient's age may preclude reliable self-report of symptoms. OBJECTIVE: The goal of this study was to develop a patient-reported outcome and an observer-reported outcome questionnaire to evaluate the signs and symptoms of EoE in pediatric patients (≥1 to <12 y of age) in a clinical trial setting. METHODS: A concept-focused literature review, expert advice meetings, and concept elicitation interviews with pediatric EoE patients and their caregivers were conducted to identify disease-related signs and symptoms. Instructions, items, and response options were drafted. Cognitive debriefing interviews were conducted to evaluate children's and caregivers' ability to understand and respond to the questionnaires and to evaluate the comprehensiveness of the concepts measured. RESULTS: Results from the literature review, expert advice meetings (n = 6), and concept elicitation interviews (n = 24) informed the development of the Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire intended for use by patients (PESQ-P) with EoE 8 years or older to younger than 12 years and an observer-reported outcome questionnaire planned for use by caregivers of patients (PESQ-C) 1 year old or older to younger than 12 years. Both questionnaires measure the same concepts; the PESQ-P assesses the frequency, duration, and/or severity of symptoms and the PESQ-C assesses the presence/absence of the signs/symptoms. The cognitive debriefing interviews (n = 17) demonstrated that participants were able to comprehend and complete the questionnaires as intended. CONCLUSIONS: This study provides evidence of the content validity of 2 novel questionnaires, PESQ-P and PESQ-C, designed to evaluate the symptom experience of pediatric EoE patients in a clinical trial setting.

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BACKGROUND: Patients treated with peanut oral immunotherapy (OIT) may experience adverse reactions, particularly during up-dosing. OBJECTIVE: To develop the Side Effects of Peanut Oral Immunotherapy Diary (SEPOD), an electronic questionnaire assessing the daily side effects of peanut OIT in clinical trials. METHODS: Content and design of the SEPOD were informed by empirical literature review and meetings with 3 allergy-immunology experts. Interviews to confirm content and inform revisions were conducted in 24 pediatric patients with peanut allergy (14 treated with peanut OIT) aged 6 to 17 years; children aged 6 to 11 years were interviewed with their caregiver. RESULTS: The SEPOD was drafted after literature review and expert interviews; the initial measurement approach comprised 2 SEPOD versions, a patient-reported outcome (PRO) version for children aged 12 to 17 years, and a caregiver-administered PRO version for children aged 6 to 11 years with instructions for caregiver questionnaire administration. Pediatric patients were expected to respond independently on both versions. Patient interviews indicated that some younger children (ie, aged 6-8 years) had difficulty understanding questions, even when reading aloud; therefore, a caregiver-administered outcome version, identical in content to the caregiver-administered PRO version, was developed for this age group. The final electronic SEPOD covered 23 peanut OIT side effects within the following 7 domains: gastrointestinal, dermatologic, itching, nasal, and respiratory, swelling (eyelid or periorbital, lip, tongue, and throat), pain (tongue, mouth, and throat), and dizziness. CONCLUSION: This study yielded the SEPOD, a new clinical outcome assessment instrument with various methods of administration that can be used to assess the side effects of peanut OIT experienced by pediatric patients in a clinical trial setting.

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BACKGROUND: Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development. METHODS: A concept-focused literature review and two advisory panel meetings involving researchers, clinicians, and measurement scientists were conducted to identify acceptability attributes that may be relevant to children taking targeted oral medicine formulations. The Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQs), including patient (P-OMAQ-P) and caregiver (P-OMAQ-C) versions, were drafted to assess these attributes. Qualitative concept elicitation (CE) and cognitive debriefing (CD) patient and caregiver interviews were conducted to confirm key acceptability attribute concepts for measurement and to evaluate patient and caregiver ability to understand and respond to the questions. RESULTS: A full-text review of 40 articles identified 24 acceptability attributes that were categorized into 10 overarching domains and organized into a preliminary conceptual model. Feedback from the advisory panel refined the preliminary model. In total, 14 attributes were reported during the CE phase of the interviews (n = 23 pediatric patients, n = 13 caregivers); six attributes were included in the final model. The draft P-OMAQ was refined over four waves of CD interviews (n = 31 pediatric patients, n = 48 caregivers). The final version of the P-OMAQ-P is a 12-item questionnaire designed for young people aged 8-17 years. The P-OMAQ-C is a 19-item questionnaire designed for adult caregivers of young people aged 6 months to 17 years. There are two versions of each questionnaire: one with a 24-h recall period and one with a 7-day recall period. All items are answered on a 5-point numerical rating scale. CONCLUSIONS: This research supports the content validity of the patient and caregiver versions of the P-OMAQ. Both questionnaires appropriately assess the acceptability of oral medicine formulations from the perspective of pediatric patients and their caregivers.

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INTRODUCTION: The daily injection burden of recombinant human growth hormone (r-hGH) replacement therapy to treat growth hormone deficiency (GHD) may reduce compliance and limit treatment benefit. Research is needed to evaluate patient preferences for GHD injection regimen and device features. OBJECTIVE: Quantitatively evaluate factors driving preferences for r-hGH injection regimen and device features among pediatric (3-17 years, and caregivers) and adult (≥25 years) patients with GHD using a discrete choice experiment (DCE) approach. METHODS: The DCE was part of a broader, cross-sectional observational field study to develop clinical outcome assessments (COAs) that assess the experience of patients taking r-hGH injections. Following ethics approval, discrete choice data were collected through an online questionnaire from consented participants recruited from eight sites in the United States. Participants were presented with 20 choice tasks, each comprising different combinations of two profiles. Participants were then shown the same set of three hypothetical device and injection profiles (ie, storage, preparation, injection type device, maintenance, dose setting, injection schedule) and asked whether they would choose each profile over their current device and schedule. Choice-based conjoint analyses were used to estimate the marginal utilities and values for treatment attributes. Subject preferences were estimated at individual and aggregate levels. RESULTS: Two hundred and twenty-four participants completed the DCE (n=75 adults, n=79 adolescent/caregiver dyads, n=70 child/caregiver dyads). Injection schedule was the strongest predictor of choice for the total sample and each patient group. Less frequent injection schedules were more likely to be chosen by participants. A "ready to use" injection was preferred, with no preference for auto-injector versus needle-free device. Most participants would choose the hypothetical injection devices and less frequent dosing over their current daily administered device schedule. CONCLUSION: Patients prefer a less frequent injection regimen for treating GHD. Addressing patient preferences may improve compliance, adherence, and ultimately, clinical outcomes.

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BACKGROUND: Best practices for pediatric clinical outcomes assessment (COA) development rely on guidelines that have been developed for adult populations. While some useful resources are available to support pediatric COA development, this information has primarily come from within the measurement development field. To our knowledge, no research has explored the experiences of professionals from other disciplines who interact with children on a routine basis. AIMS AND OBJECTIVES: The goal of this research was to explore the experiences of professionals from outside of the measurement science field, who work closely with children every day, in settings relevant to the context of concept elicitation and cognitive debriefing interviews for pediatric COA development. The objectives were to 1) learn new ways to engage children in conversations regarding their health state; 2) understand how methods used in other disciplines can be used to improve the amount and quality of data emerging from pediatric qualitative interviews; and 3) generate a list of references to support pediatric COA development. METHODS: Individual, one-to-one expert advice meetings were conducted over the phone or in person and lasted approximately 60 minutes in duration. One child life specialist, one speech/language pathologist, and three reading specialists were consulted, given their role in evaluating children's comprehensive abilities as well as their daily interactions with children. Two experienced COA researchers conducted the expert advice meetings using a semi-structured interview guide to provide a framework for discussion. RESULTS: These experts reported that factors such as interview setting, time taken to build rapport with the child, the child's comfort level, presence or absence of caregiver during the interview, the child's communication style, disease-related factors, and the child's developmental age may influence the amount and type of information that is possible to elicit during qualitative interviews. Several of these factors are also important for cognitive debriefing interviews. In addition, experts provided input that may improve the debriefing procedure, such as having the child read the text aloud in small increments, re-read text, and highlight text that they do not understand. Best practice tips from the experts were consolidated into a set of references for use by those conducting pediatric COA development research. CONCLUSION: Incorporating interdisciplinary perspectives into pediatric COA development may improve both the methods used to elicit information from children and the quality of the resulting questionnaires.

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BACKGROUND: Current recombinant human growth hormone (r-hGH) replacement therapy involves long-term daily subcutaneous injections to treat growth hormone deficiency (GHD) in children and adults. Daily r-hGH injections can be burdensome, often resulting in poor treatment compliance. Clinical outcome assessments (COAs) can capture the burden of these injections from the patient (and caregiver) perspective and may demonstrate the benefit of a less-frequent r-hGH injection regimen, which may ultimately improve treatment compliance and long-term outcomes. OBJECTIVE: To address this knowledge gap, qualitative research was conducted to inform the development of a new Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD), designed to measure the experiences of patients taking r-hGH GHD injections. A second objective was to evaluate the hypothesized factor structure and preliminary performance of the LIQ-GHD in a cross-sectional observational study. METHODS: An empirical literature review and expert advice meetings were conducted to inform development of the draft LIQ-GHD (pediatric and adult versions). In-person concept elicitation and cognitive debriefing interviews were conducted with GHD patients (and patient dyads including caregivers) to explore and confirm concept coverage and evaluate respondents' ability to understand the questionnaire. The draft LIQ-GHD was then tested in a cross-sectional field study involving pediatric and adult patients receiving daily r-hGH injections for GHD. The factor structure, reliability, and validity were analyzed for the overall sample and for pediatric, adolescent, and adult subgroups. RESULTS: Results from the literature review and input from six experts were used to develop and refine the LIQ-GHD, with content covering pen ease of use; regimen convenience; life interference due to regimen; benefit/satisfaction/willingness to continue treatment; regimen choice/preference; intent to comply with regimen; injection-related signs/symptoms; and reasons for missed injections. Twenty-one patient interviews confirmed comprehensive concept coverage and patient/caregiver comprehension of the LIQ-GHD. A total of 224 patients (n = 70 children/caregiver dyads, n = 79 adolescents/caregiver dyads, n = 75 adults) participated in the field study. While most items showed floor effects, confirmatory factor analysis fit statistics were good for the overall sample (root mean square error of approximation = 0.07, comparative fit index = 0.98) and for the full pediatric sample after dropping co-dependent questions from the model. Cronbach's alpha (α) ranged from 0.746 to 0.905 and intra-class correlation coefficients ranged from 0.761 to 0.918 for the overall sample on LIQ-GHD domains. Scores correlated as predicted with an existing criterion measure in the overall sample and LIQ-GHD domain scores distinguished known groups as expected. CONCLUSIONS: The LIQ-GHD is a new COA for the measurement of r-hGH injection treatment burden. This research provides evidence supporting its content validity, hypothesized factor structure, score reliability, and construct validity in pediatric and adult populations.

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OBJECTIVE: Evidence-based recommendations for the a priori estimation of sample size are needed for qualitative concept elicitation (CE) interview studies in clinical outcome assessment (COA) instrument development. Saturation is described as the point at which no new data is expected to emerge from the conduct of additional qualitative interviews. STUDY DESIGN: A retrospective evaluation of 26 CE interview studies conducted with patients between 2006 and 2013 was completed to assess the point at which saturation of concept was achieved in each study. METHODS: For each of the 26 interview studies, saturation of symptom concepts was assessed by dividing the sample into quartiles and then comparing the number of responses elicited from the first 25% of participants to the next 25% of participants, from the first 50% of participants to the next 25% of participants, and then from the first 75% of participants to the last 25% of participants. The number of interviews required to achieve saturation was documented for each study and then summarized across studies. RESULTS: Findings indicate that 84% of symptom concepts emerged by the 10th interview, 92% emerged by the 15th interview, 97% emerged by the 20th interview, and 99% by the 25th interview. CONCLUSIONS: Results provide practical guidance for estimating the number of interviews that may be needed to achieve saturation in a qualitative CE interview study for COA instrument development; address an important gap in qualitative research for the development of COAs in the context of medical product development; and offer useful information for study design and implementation.

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BACKGROUND: Varicose veins are common and can impact patients' quality of life, but consensus regarding the evaluation of varicose vein symptoms is lacking and existing measures have limitations. OBJECTIVE: This research aimed to develop and establish the content validity of a new electronic patient-reported outcome (PRO) measure, the VVSymQ® instrument, to assess symptoms of superficial venous insufficiency (varicose veins) in clinical trials. METHODS: The development of the VVSymQ® instrument began with qualitative interviews with patients based on the symptom domain of the VEINES-QOL/Sym, an existing PRO instrument for chronic venous disorders of the leg. Three phases of qualitative research were conducted to examine the relevance and importance of the symptoms to patients with varicose veins, and the patients' ability to understand and use the VVSymQ® instrument. The development included evaluating questions that had 1-week and 24-h recall periods, and paper and electronic versions of the new instrument. RESULTS: Five symptoms (heaviness, achiness, swelling, throbbing, and itching [HASTI™]) were consistently reported by patients across all sources of qualitative data. The final version of the VVSymQ® instrument queries patients on the HASTI™ symptoms using a 24-h recall period and a 6-point duration-based response scale ranging from "None of the time" to "All of the time," and is administered daily via an electronic diary. Cognitive interviews demonstrated varicose vein patients' understanding of and their ability to use the final version of the VVSymQ® instrument. CONCLUSION: Content validity was established for the VVSymQ® instrument, which assesses the five HASTI™ symptoms of varicose veins daily via an electronic diary and has promise for use in research and practice.

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OBJECTIVE: As a means to measure quantifiable signs, symptoms, and impacts of a disease or its treatment, patient-reported outcome (PRO) instruments can be applied to numerous settings, including use in drug development to support labeling claims. This research summarizes the use of PROs in trials for 16 commonly used regulatory approved treatments for advanced or metastatic breast cancer. METHODS: For each treatment (n = 16), a literature search was conducted in MEDLINE, Embase, and PsycINFO. The primary criterion for selection was the report of studies that used PROs to evaluate treatment benefit and/or toxicity in advanced or metastatic breast cancer. From this, a sub-set of articles for each treatment were selected for full-text review where PRO-related information was extracted and summarized. RESULTS: The searches yielded 1727 publications. Following abstract review, 1702 were excluded because they failed to meet criteria, or were duplicates or less relevant for PRO information reported. Thus, 25 articles were reviewed in detail for this evaluation. Eleven PRO instruments were identified from these publications. The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Core (EORTC QLQ-C30) was utilized the most frequently (n = 13, 52.0%). Most publications reported PROs positioned as secondary endpoints (n = 20, 80.0%); described some of the statistical analyses applied to PRO data (n = 21, 84.0%); and specified PRO results (n = 23, 92.0%). CONCLUSIONS: While several of the publications provided some information on how PROs were utilized, many did not describe details for PRO administration, scoring, analyses, and results interpretation. While it is encouraging that PROs are often used in clinical trials for patients with metastatic breast cancer, they are not commonly used to support endpoints that establish the basis for label claims. Because they yield direct insight into the patient experience of a condition, PROs may be used to provide a more comprehensive perspective of the benefits and risks from treatment.

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OBJECTIVE: To evaluate the psychometric properties of the VVSymQ(®) instrument, a new 5-item patient-reported outcome (PRO) measure for symptoms of varicose veins. METHOD: The VVSymQ(®) electronic daily diary was administered to outpatients who received routine treatment for varicose veins (N = 40). Compliance with diary administration and item score variability, reliability, construct validity, sensitivity to change, and clinically meaningful change were evaluated. RESULTS: Patients completed >97 % of scheduled diary assessments (at screening, baseline, and week 8). The VVSymQ(®) instrument captured patients' pre-treatment symptoms (all VVSymQ(®) symptoms were endorsed by ≥75 % of patients at baseline), and the change post-treatment (mean change in score -6.1), with a large Cohen effect size (1.6). Test-retest reliability was high (intraclass correlation coefficient 0.96); internal consistency was good (Cronbach's alpha ≥0.76; baseline, week 8). VVSymQ(®) scores were more strongly associated with PRO scores that reflect symptoms and symptom impact (the Venous Insufficiency Epidemiological and Economic Study-Quality of Life/Symptoms [VEINES-QOL/Sym] instrument and the Chronic Venous Insufficiency Quality-of-Life Questionnaire [CIVIQ-20]) than with PRO scores that reflect appearance (the Patient Self-Assessment of Appearance of Visible Varicose Veins [PA-V(3)]) or clinician-reported outcome scores (the Clinical-Etiology-Anatomy-Pathophysiology [CEAP] Classification of Venous Disorders and Venous Clinical Severity Score [VCSS]), demonstrating construct validity. Patients reporting that symptoms were "moderately" or "much improved" on the Patient Global Impression of Change (PGIC) anchor (i.e., >97 % of patients) had mean improvements of -6.3 VVSymQ(®) points, while a cumulative distribution curve showed that 50 % of patients improved by ≥-5.8 points; thus, a score change of approximately -6 demonstrated a clinically meaningful change in this study. The clinically meaningful change in the VVSymQ(®) score was greater in patients with a greater baseline VVSymQ(®) symptom burden, and the VVSymQ(®) instrument captured clinically meaningful treatment benefit even in patients with a low baseline symptom burden. CONCLUSION: The 5-item VVSymQ(®) instrument is a brief, psychometrically sound, useful tool for evaluating patient-reported varicose veins symptoms.

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BACKGROUND: Patient diaries and pain scales can capture the course and complications of pain managed at home in children. The Faces Pain Scale-Revised (FPS-R) is a validated scale showing reliability in children, but it has not been validated in children with sickle cell disease (SCD). OBJECTIVE: The purpose of this study was to evaluate comprehension and usability of an electronic modified version of the FPS-R among pediatric patients with SCD. METHODS: This was a cross-sectional, qualitative study involving in-person interviews with children/adolescents from the USA and their parents/legal guardians. Interviews involved cognitive debriefing and usability testing of the FPS-R. RESULTS: In total, 22 children with SCD aged 4-17 years participated. Children aged 4-6 were generally unable to demonstrate clear understanding of the FPS-R and its response scale. Overall, children aged ≥7 years understood the instrument and could complete it on the electronic device, although children aged 7-8 often needed assistance from the parent. Children aged 9-17 years were able to read and complete the instrument independently. Most participants considered the electronic device easy to use. CONCLUSIONS: The FPS-R was shown to be a comprehensible and usable pain measure for children aged 7-17 with SCD and to be beneficial for future clinical studies.

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Clinical outcome assessments (COAs), including patient-reported outcome (PRO) measures, are routinely used in drug development and other clinical research initiatives to assess the impact of treatment on patient health and well-being. The FDA Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (2009), the European Medicines Agency's Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life Measures in the Evaluation of Medicinal Products (2005), and the International Society for Pharmacoeconomics and Outcomes Research PRO Good Research Practices for the Assessment of Children and Adolescence Task Force (2013) outline key considerations and good measurement principles that are relevant to the selection and use of COAs in a pediatric population. However, challenges remain in the appropriate selection and use of COAs to assess treatment benefit in pediatric clinical research. The purpose of this paper is to summarize proceedings from a panel presentation at the Critical Path Institute's 2015 Annual PRO Consortium Workshop. This paper underscores the importance of considering children's specific needs and the numerous challenges faced when developing and implementing well-defined and reliable COAs in pediatric clinical trials evaluating medical products, and describes some approaches to addressing these unique needs and challenges.

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INTRODUCTION: No existing patient-reported outcome instrument focuses solely on assessment of varicose veins symptoms that are bothersome to patients. METHODS: The VVSymQ® instrument is a five-item patient-reported outcome that assesses symptoms most important to patients with varicose veins (heaviness, achiness, swelling, throbbing and itching). This paper describes how the VVSymQ® instrument was incorporated into an electronic daily diary to monitor key outcomes over time and capture treatment benefit in two randomized, controlled, phase 3 clinical trials. RESULTS: Patients were highly compliant in completing the electronic daily diary, and the VVSymQ® instrument demonstrated ability to detect overall change and ability to detect change that is meaningful to patients. CONCLUSION: The VVSymQ® instrument is a reliable, valid instrument responsive to measuring change in the patient experience of varicose vein symptoms pre- and post-intervention, and is uniquely focused on patient-reported symptoms compared with other widely used questionnaires completed by clinicians.

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OBJECTIVES: This research evaluated the psychometric properties of a new Psoriasis Symptom Diary, identified diary responder definitions for use in determining whether a patient has experienced clinically meaningful change, and refined diary item content for use in future clinical trials. METHODS: The Psoriasis Symptom Diary was administered in a phase 2 clinical trial of AIN457 to US adult outpatients (N = 172) with physician-diagnosed moderate to severe chronic plaque-type psoriasis. Participant compliance with daily diary administration and item score variability, reliability, construct and discriminant validity, sensitivity to change, and interpretation were all evaluated. RESULTS: Participants completed 94% of scheduled diary assessments across 12 study weeks. Diary items were generally normally distributed, and no floor or ceiling effects were observed. Item reliability (reproducibility) was acceptable (intraclass correlation coefficients > 0.80), with an exception for one item (skin color). At week 12, items significantly related to criterion measures as predicted (Psoriasis Area and Severity Index r = 0.27-0.57; Investigator's Global Assessment r = 0.25-0.59), with the exception of items that measured skin color and difficulty using hands. Most items generated change scores that were synchronous to changes as measured by the Psoriasis Area and Severity Index, Investigator's Global Assessment, Dermatology Life Quality Index (r > 0.37), as well as the Patient Global Impression of Change. Responders experienced a 2- to 3-point and 3- to 5-point change in item scores for minimal and large improvements, respectively. Four items that did not perform well were dropped from the diary. CONCLUSIONS: The 16-item Psoriasis Symptom Diary demonstrated favorable psychometric properties and is a brief, useful tool for measuring patient-based symptoms and the impact of chronic plaque psoriasis.

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BACKGROUND: Asthma medication adherence is related to better asthma outcomes, but identification of suboptimal patient adherence behavior is not standardized in clinical settings. OBJECTIVE: [corrected] The purpose of this study was to develop a practical questionnaire that reflects nonadherence risk and identifies potential adherence barriers. METHODS: A questionnaire that included 20 potential adherence questions was completed by 420 adult patients with asthma who filled a prescription for an inhaled corticosteroid (ICS) and a short-acting beta agonist (SABA) in the previous 6 months. Questions without substantial floor or ceiling effects that were significantly related to self-reported low adherence or previous ICS canister dispensings were identified. Internal consistency reliability was tested by Cronbach α. Relationships of these questions to Asthma Control Test scores, future percent of days covered for ICS dispensings, and future asthma exacerbations and SABA dispensings were determined. RESULTS: Five final questions were identified: following "my medication plan," forgetting, not "needing" the medications, side effects, and cost. Low internal consistency reliability (<0.50) suggested items should not be summarized by a single score. All five questions were related to Asthma Control Test scores. Following the medication plan, forgetting, and not needing medication were significantly related to prospective percent of days covered. Side effects were related to subsequent SABA and oral corticosteroid dispensings, and cost was significantly related to oral corticosteroid dispensings. CONCLUSIONS: We identified five questions related to other measures of adherence and to asthma control that can be used clinically to identify patients at risk of nonadherence and the specific adherence barriers involved.

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PURPOSE: To evaluate the psychometric properties and factor structure of a computerized electronic version of the SF-36v2 Health Survey (SF-36v2) with items administered one-per-page versus the traditional grid format used in the paper-and-pencil version in a sample of physician-diagnosed headache patients. METHODS: Patients (N = 180) completed the SF-36v2 administered as part of a broader study of health outcomes. Scaling assumptions, reliability, factor structure, and the tool's ability to discriminate between headache pain severity groups were examined. RESULTS: Frequency distributions showed notable ceiling effects for the role emotional, social functioning, physical functioning, and role physical scales, but negligible (<1.2%) floor effects for any of the scales. Internal consistency reliability coefficients ranged from 0.81 to 0.95 for the eight health domains. Items passed tests of internal consistency and discriminant validity. Principal components' analyses confirmed the 2-factor structure; the pattern of correlations across scales was consistent with expectations for the physical and mental health components. As expected, patients with severe headache pain had lower mean SF-36v2 scores than those with mild or moderate pain. No significant score differences were observed between mild and moderate pain severity groups. CONCLUSIONS: Single-item electronic administration of the SF-36v2 is reliable and valid for use with headache patients.

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BACKGROUND: Patient-reported outcomes (PRO) measures should be valid and accessible to a wide audience. OBJECTIVE: Cognitive item testing and readability studies were conducted to evaluate how adult headache sufferers (N = 9) understood and responded to the Headache Impact Test (HIT™) item bank, a PRO measure for headache that serves as the source of item content for the HIT-6™ (a widely used six-item short-form measure of headache impact with more than 30 language translations), and the Dynamic Health Assessment Headache Impact Test (DYNHA® HIT™) [a computerized adaptive test (CAT) of headache impact]. METHODS: During cognitive interviews, participants were asked to 'think aloud' as they read survey instructions, completed items, and formulated responses. Data analyses evaluated item comprehension, memory recall of relevant information, and decision and response processes; compared various item attributes; and tested shortened item versions. RESULTS: Survey readability was at the seventh-grade level. Respondents understood most revised items as intended, and found shorter items comparable to longer items with some exceptions. When recall period was included in instructions but not within the items themselves, respondents often expanded the recall period to answer the item. Some response scales (e.g. "Never" to "Always") were more readily understood than others (e.g. "Definitely true" to "Definitely false"). CONCLUSION: Qualitative research can improve the validity and accessibility of PRO measures that are used to monitor health conditions and aid patient-provider communication.

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DYNHA SF-36 is a computerized adaptive test version of the SF-36 Health Survey. The feasibility of administering a modified DYNHA SF-36 to adults with HIV was evaluated with Johns Hopkins University Moore (HIV) Clinic patients (N=100) and Internet consumer health panel members (N=101). Participants completed the DYNHA SF-36, modified to capture seven health domains [(physical function (PF), role function (RF, without physical or emotional attribution), bodily pain (BP), general health, vitality (VT), social function (SF), mental health (MH)], and scored to produce two summary components [Physical Component Summary (PCS), Mental Component Summary (MCS)]. Item-response theory-based response consistency, precision, mean scores, and discriminant validity were examined. A higher percentage of Internet participants responded consistently to the DYNHA SF-36. For each domain, three standard deviations were covered with five items (90% reliability); however, RF and SF scores were less precise at the upper end of measurement (better functioning). Mean scores were slightly higher for the Internet sample, with the exception of VT and MCS. Clinic and Internet participants reporting an AIDS diagnosis had significantly lower mean PCS and PF scores than those without a diagnosis. Additionally, significantly lower RF and BP scores were found for Internet participants reporting an AIDS diagnosis. The measure was well accepted by the majority of participants, although Internet respondents provided lower ratings for the tool's usefulness. The DYNHA SF-36 has promise for measuring the impact of HIV and its treatment in both the clinic setting and through telemonitoring.

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OBJECTIVE: The purpose of this research was to calibrate an item bank for a computerized adaptive test (CAT) of asthma impact on health-related quality of life (HRQOL), test CAT versions of varying lengths, conduct preliminary validity testing, and evaluate item bank readability. METHODS: Asthma Impact Survey (AIS) bank items that passed focus group, cognitive testing, and clinical and psychometric reviews were administered to adults with varied levels of asthma control. Adults self-reporting asthma (N = 1106) completed an Internet survey including 88 AIS items, the Asthma Control Test, and other HRQOL outcome measures. Data were analyzed using classical and modern psychometric methods, real-data CAT simulations, and known groups validity testing. RESULTS: A bi-factor model with a general factor (asthma impact) and several group factors (cognitive function, fatigue, mental health, physical function, role function, sexual function, self-consciousness/stigma, sleep, and social function) was tested. Loadings on the general factor were above 0.5 and were substantially larger than group factor loadings, and fit statistics were acceptable. Item functioning for most items and fit to the model was acceptable. CAT simulations demonstrated several options for administration and stopping rules. AIS distinguished between respondents with differing levels of asthma control. CONCLUSIONS: The new 50-item AIS item bank demonstrated favorable psychometric characteristics, preliminary evidence of validity, and accessibility at moderate reading levels. Developing item banks for CAT can improve the precise, efficient, and comprehensive monitoring of asthma outcomes and may facilitate patient-centered care.

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