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Abstract Background Patients with respiratory disease experience disturbed sleep, but there is no widely accepted measure of sleep impairment due to respiratory disease. We developed and evaluated the psychometric performance of a patient-reported measure to assess the impact on sleep due to respiratory disease, the COPD and Asthma Sleep Impact Scale (CASIS). Methods Identification of the items forming the CASIS was guided by patient interviews and focus groups. An observational study involving patients from the US and UK was then conducted to assess the psychometric characteristics of the measure. Results Qualitative data from 162 patients were used to develop the CASIS (n = 78 COPD; n = 84 asthma). The observational study included 311 patients with COPD and 324 patients with asthma. The final seven items used in the CASIS were identified based on factor and item response theory analyses. Internal consistency was 0.90 (COPD) and 0.92 (asthma), and test-retest reliability was 0.84 (both groups). In the COPD sample, CASIS scores were significantly correlated with the Saint George's Respiratory Questionnaire scores (all p < 0.0001) and differed significantly by patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.005). In the asthma sample, CASIS scores were significantly correlated with the Asthma Quality of Life Questionnaire scores (all p < 0.0001) and differed significantly by clinician and patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.0005). Conclusion The CASIS shows good internal consistency, test-retest reliability, and construct validity and may be useful in helping to understand the impact that COPD and asthma have on sleep outcomes.

Importance: Defining meaningful improvement using the Total Vitiligo Area Scoring Index (T-VASI) and the Facial VASI (F-VASI) aids interpretation of findings from clinical trials evaluating vitiligo treatments; however, clear and clinically meaningful thresholds have not yet been established., Objective: To assess concept validity and measurement performance of the T-VASI and F-VASI in patients with nonsegmental vitiligo and to identify meaningful change thresholds., Design, Settings, and Participants: This mixed-methods study consisted of a secondary analysis of a phase 2 multicenter double-blind dose-ranging randomized clinical trial and embedded qualitative interviews conducted at 35 sites in Canada, France, Japan, and the US. The secondary analysis included the trial's adult patients with nonsegmental vitiligo (T-VASI ≥5 and F-VASI ≥0.5 at baseline). Psychometric performance of the T-VASI and F-VASI and thresholds for meaningful change were evaluated using clinician- and patient-reported information. The trial's embedded interviews were used to qualitatively assess content validity and patient perceptions of meaningful repigmentation. Data analyses were performed from March to July 2023., Intervention: Participants were randomized to 6-, 11-, or 22-mg/day upadacitinib or placebo for 24 weeks., Main Outcomes and Measures: Psychometric performance of the T-VASI and F-VASI and thresholds for meaningful changed plus content validity and patient perceptions of meaningful repigmentation. Measurement instruments included the T-VASI, F-VASI, Vitiligo Noticeability Scale, Total-Patient Global Vitiligo Assessment, Face-Patient Global Vitiligo Assessment, Total-Physician Global Vitiligo Assessment (PhGVA-T), Face-Physician Global Vitiligo Assessment (PhGVA-F), Patient's Global Impression of Change-Vitiligo, Physician's Global Impression of Change-Vitiligo (PhGIC-V), Vitiligo Quality-of-Life Instrument, Dermatology Life Quality Index, the Hospital Anxiety and Depression Scale, and transcribed verbatim interviews with patients., Results: The psychometric analysis included 164 participants (mean [SD] age, 46 years; 103 [63%] females) and the qualitative analysis included 14 participants (mean [SD] age, 48.8 [12.2] years; 9 females [64%] and 5 males [36%]). Intraclass correlation coefficients were 0.98 for T-VASI and 0.99 for F-VASI in patients with clinically stable vitiligo between baseline and week 4, supporting test-retest reliability. At baseline and week 24, correlations were moderate to strong between T-VASI and PhGVA-T (r = 0.63-0.65) and between F-VASI and PhGVA-F (r = 0.65-0.71). Average baseline and week-24 VASI scores decreased with repigmentation (ie, increasing PhGVA scores). Least-square mean VASI scores increased with greater repigmentation as measured by the PhGIC-V. Least-square mean VASI scores also differed between patients with improved PhGIC-V and those with no change or worsened V-PhGIC scores. Using a multiple anchor approach, improvements of 30% in T-VASI and 50% in F-VASI scores reflected meaningful repigmentation between baseline and week 24., Conclusion and Relevance: This mixed-methods study found that the T-VASI and F-VASI are reliable, valid, able to differentiate between clinically distinct groups, and responsive in patients with nonsegmental vitiligo. The thresholds for meaningful change were lower than those historically used in clinical trials, suggesting that T-VASI 50 and F-VASI 75 are conservative estimates and reflect improvements that would be meaningful in patients with nonsegmental vitiligo., Trial Registration: ClinicalTrials.gov Identifier: NCT04927975.

Objective: To better understand the symptoms and impacts of Raynaud phenomenon (RP) in patients with systemic sclerosis (SSc) and to evaluate the content validity and usability of a new electronic patient-reported outcome (PRO) measure for RP: the Raynaud Diary., Methods: The Raynaud Diary was developed as a daily eDiary for assessing the number and duration of symptomatic Raynaud attacks; worst pain, numbness, tingling, and discomfort in the fingers; and overall disease severity, captured using the Raynaud's Condition Score. The Raynaud Diary was debriefed in two waves of qualitative interviews with adults with self-reported RP secondary to SSc. All interviews included open-ended questions about participants' experiences of RP., Results: Participants (N = 39) had a mean age of 55.1 years, and 87% were female. Frequently reported RP symptoms were color change (reported by all participants), numbness (90%), tingling (82%), pain (77%), and discomfort (72%). Common attack triggers included temperature-related factors and stress. Participants reported being unable to be outside or do outdoor activities and had problems gripping objects. All participants demonstrated understanding of the Raynaud Diary instructions. Most participants indicated that they would be able to use the Raynaud Diary to record the worst severity of individual RP symptoms in the previous 24 hours., Conclusion: Patients with RP secondary to SSc bear a heavy symptom burden. The Raynaud Diary is a content valid PRO measure that captures the most frequent symptoms of RP in patients with SSc., (© 2023 Eicos Sciences, Inc. ACR Open Rheumatology published by Wiley Periodicals LLC on behalf of American College of Rheumatology.)

A survey was conducted in eight countries to examine conversations around, and experiences and treatments during, severe hypoglycemia among people with diabetes and caregivers of people with diabetes. This article reports a subgroup analysis from the United States involving 219 people with diabetes and 210 caregivers. Most respondents (79.7%) did not use professional health care services during their most recent severe hypoglycemic event, and 40.3% did not report the event to their health care providers at a subsequent follow-up visit. Hypoglycemic events left respondents feeling scared (70.9%), unprepared (42.7%), and helpless (46.9%). These clinically important psychosocial impacts on people with diabetes and caregivers underscore the need for conversations about hypoglycemia prevention and management., (© 2022 by the American Diabetes Association.)

Acid sphingomyelinase deficiency (ASMD), historically known as Niemann-Pick disease (NPD) types A, A/B, and B, is a rare, progressive, potentially fatal lysosomal storage disease with a spectrum of phenotypes. Little is known about how ASMD symptoms affect the lives of patients and their caregivers. In a cross-sectional qualitative study conducted in the US and UK, and in collaboration with the National Niemann-Pick Disease Foundation (US) and Niemann-Pick UK, we investigated the symptom experience of patients with ASMD types B and A/B and explored how the disease impacts their and their caregivers' lives. The study included 17 adult patients (mean age 38.7 years, 12 female), three caregivers of adults with ASMD, 12 pediatric/adolescent patients with ASMD (mean age 10.5 years, six female), and 12 caregivers of pediatric/adolescent patients with ASMD. The most commonly reported disease manifestations were respiratory (n = 26, 89.7%), abdominal (n = 25, 86.2%), and musculoskeletal symptoms (n = 23, 79.3%); excessive bleeding or bruising (n = 20, 69%); fatigue (n = 20, 69%); gastrointestinal symptoms (n = 18, 62.1%); and headache (n = 15, 51.7%). ASMD was reported to negatively impact patients' physical function (n = 23, 79.3%), self-esteem (n = 18, 62.1%), emotions (n = 16, 55.2%), social function and relationships (n = 16, 55.2%), and personal care (n = 9, 31%). Providing care for individuals with ASMD negatively affected caregivers' emotional well-being (n = 12, 80%), social function (n = 4, 26.7%), relationships (n = 6, 40%), and financial security (n = 7, 46.7%). The physical toll of providing care, the need for lifestyle changes, and the responsibility for making medical decisions added to the burden for caregivers. Alternatively, some caregivers noted that caring for a loved one enhanced their spirituality, providing them with a different outlook on life and a deeper personal resolve. This study showed that ASMD is a substantial burden for patients and caregivers, with long-term physical, emotional, social, and financial impacts. The study confirmed commonly known manifestations of ASMD, especially respiratory problems, but also identified less recognized ones, such as dermatological complications. The data collected and insight gained from this study should enhance clinical care, help evaluate new treatments, and inform health care decision making for patients with ASMD., (© 2021. The Author(s).)

Introduction: Information on experience/management of severe hypoglycaemic events (SHEs) among people with insulin-treated diabetes (PWD) and caregivers (CGs) providing care to PWD was sought., Materials and Methods: An online cross-sectional survey was conducted in eight countries., Inclusion Criteria: PWD (aged≥18 years; self-reported type 1 [T1D] or insulin-treated type 2 [T2D] diabetes; experienced ≥1 SHE [hypoglycaemia requiring external assistance] in past 3 years); CGs (layperson aged ≥18 years; caring for PWD meeting all criteria above except age [≥4 years]). This descriptive analysis provides data from Spain. SHE-associated data relate to the most recent SHE., Results: Across all groups (T1D PWD, n=106; T2D PWD, n=88, T1D CG, n=87; T2D CG, n=96), 76-89% reported that the SHE occurred at home; most common cause was eating less than planned (38-53%). Most usual action during the SHE was to intake carbohydrates (67-84%); glucagon use was low (9-36%). Discussion of the SHE with their healthcare provider (HCP) was reported by 70-75% of PWD. During the SHE, 35-69% of PWD/CGs reported feeling scared, unprepared and/or helpless., Conclusions: Most SHEs occurred outside the healthcare setting; treatment therefore depends greatly on CGs. SHEs have a negative emotional impact on PWD/CGs, underscoring the need for HCPs to discuss SHEs with PWD/CGs, and to provide tools and strategies to prevent and effectively manage SHEs., (Copyright © 2020 The Author(s). Published by Elsevier España, S.L.U. All rights reserved.)

Background: Hypoactive sexual desire disorder (HSDD) has a significant negative impact on women's overall health and relationships with their partners. Primary analyses from the RECONNECT clinical trials demonstrated statistically significant and clinically meaningful improvements in sexual desire and related distress with bremelanotide relative to placebo in premenopausal women with HSDD. Exit surveys and patient interviews were conducted to evaluate the impact of HSDD and bremelanotide treatment from the patient's perspective. Materials and Methods: Upon completion of the double-blind study but before participation in the open-label extension, up to 250 participants were recruited to complete the quantitative exit survey (17 questions). A subset of up to 90 patients was invited to participate in the telephone interview (17 questions). Patients who volunteered to participate remained blinded to study drug until the survey and interviews were completed. Results: Quantitative exit surveys were completed by 242 RECONNECT participants; 80 of these women also completed qualitative telephone exit interviews. Participants who received bremelanotide described increased feelings of sexual desire, physical arousal, and improvements in overall quality of their sexual activities in their partner relationship. In comparison, women taking placebo reported benefits that did not include the physiological responses described by women receiving bremelanotide, such as positive experiences of seeking HSDD treatment and improved communication with their partner. Conclusions: Exit surveys and patient interviews support the primary findings from RECONNECT and provide quantitative and qualitative assessments of the impact of HSDD on patients' quality of life and the patients' perspectives on the impact of bremelanotide. Clinical trial numbers NCT02333071, NCT02338960.

Objectives: This study examined Sheehan Disability Scale (SDS) performance in binge eating disorder (BED) and explored relationships between SDS and BED outcomes using data from three placebo-controlled lisdexamfetamine (LDX) studies (two short-term, dose-optimized studies and one double-blind, randomized-withdrawal study) in adults with Diagnostic and Statistical Manual of Mental Disorders, fourth edition, text revision (DSM-IV-TR)-defined BED., Methods: Analyses evaluated the psychometric properties of the SDS., Results: Confirmatory factor analysis supported a unidimensional total score in the short-term studies, with internal consistency (Cronbach's α) being 0.878. Total score exhibited good construct validity, with moderate and statistically significant correlations observed with Yale-Brown Obsessive Compulsive Scale modified for binge eating, Binge Eating Scale (BES), and EuroQol Group 5-Dimension 5-Level health status index scores. Known-groups validity analysis for the short-term studies demonstrated a significantly lower total score at end of study in participants considered "not ill" versus "ill" based on Clinical Global Impressions-Severity scores. SDS total score changes in the short-term studies were greater in responders than nonresponders based on binge eating abstinence or BES score. In the randomized-withdrawal study, SDS scores increased relative to baseline to a greater extent in participants randomized to placebo than LDX., Conclusions: These analyses support the reliability, validity, and responsiveness to change of the SDS in individuals with BED., (© 2020 The Authors. International Journal of Methods in Psychiatric Research published by John Wiley & Sons Ltd.)

Background: Endometriosis is a common problem in women of reproductive age and has impacts on health-related quality of life and productivity. Fatigue is an important part of the burden of endometriosis, it is not often included as an endpoint in clinical trials., Objectives: The study assessed the psychometric properties of the PROMIS Fatigue Short Form 6a in women with moderate-to-severe endometriosis-associated pain., Methods: In a phase III double-blind, placebo-controlled clinical trial (NCT01620528), women aged 18-49 years with moderate-to-severe endometriosis-related pain were randomized to elagolix 150 mg once daily, elagolix 200 mg twice daily, or placebo for 6 months. PROMIS Fatigue and dysmenorrhea and non-menstrual pelvic pain (NMPP) scores were assessed at baseline and months 1, 3, and 6, and Patient Global Impression of Change (PGIC) was assessed at months 1, 3, and 6. Reliability (internal consistency and test-retest reliability), construct validity (convergent and known groups validity), and responsiveness were evaluated., Results: The analysis included 871 women, mean age 31.5 years. Internal consistency supported a single concept (Cronbach's alpha 0.93). For the 238 patients with no change in PGIC at month 1, the intraclass correlation coefficient for the PROMIS Fatigue T-score was 0.7 and paired t-test statistically significant (2.84, p = 0.0049). Correlations with other measures were expected to be fairly low as concepts were not redundant. The PROMIS Fatigue discriminated among known groups with mean scores of 55.3, 62.3, and 65.8 at month 3 (PGIC improvement, no change, worsening, respectively). Statically significant discrimination, and change score responsiveness, were seen using clinically relevant anchors (dysmenorrhea and NMPP) at months 3 and 6 between responders and non-responders. Anchor-based (PGIC) responsiveness showed significant improvement from baseline to months 3 and 6 (p < 0.0001)., Conclusions: PROMIS Fatigue has good reliability, validity, and responsiveness in women with moderate-to-severe endometriosis-associated pain.

Objective: To evaluate the responsiveness of the Endometriosis Health Profile-30 (EHP-30) and ascertain score changes that are indicative of response to treatment. A post hoc analysis of two Phase III, double-blind, placebo-controlled, randomized clinical trials among women with moderate-to-severe endometriosis-associated pain (Elaris Endometriosis I and II [EM-I and EM-II]). Materials and Methods: EHP-30 core items and sexual relationship module were administered at day 1, month 3 (M3), and month 6 (M6) to monitor patient-reported impacts of endometriosis-related pain. A seven-response level Patient Global Impression of Change (PGIC) was administered at M3 and M6. Dysmenorrhea (DYS), nonmenstrual pelvic pain (NMPP), and dyspareunia (DYSP) were collected using a daily diary. Three psychometric approaches, "triangulation," were used to suggest responder thresholds for the EHP-30 domains. The three approaches were anchor- and distribution-based analyses and use of clinically relevant indicators (DYS, NMPP, DYSP). Results: EM-I and EM-II enrolled 871 and 815 women, respectively. All EHP-30 domains improved during the trials (M3, M6). Differences ( p  < 0.001) for all EHP-30 domains were found among the PGIC responses at M3 and M6, indicating greater change was associated with greater EHP-30 improvements. Large effect sizes were noted for all EHP-30 domains (EM-I range -0.59 to -1.80; EM-II range -0.52 to -1.59). EHP-30 thresholds of meaningful change ranged from -20 to -35, with greater changes indicating greater improvement in health status. Conclusion: Responder thresholds by EHP-30 domain are recommended to evaluate treatment efficacy. Clinicians can individualize goals of treatment by EHP-30 domain and track changes using the EHP-30.

Background: Adenomyosis is a poorly understood, benign disease of the uterus., Objective: In this study, patient interviews were conducted to characterize the symptoms and impact of adenomyosis., Methods: This was a cross-sectional study in which women with adenomyosis were recruited from five US clinics and a health-related social network forum. Participants (aged 18-55 years) were pre-menopausal with a history of regular menstrual cycles. Participants were interviewed about their experiences with adenomyosis, symptoms and impacts on day-to-day activities (concept elicitation), and subsequently about the occurrence, relative severity, and impact of symptoms (card-sorting exercise)., Results: In total, 31 women were interviewed. Mean duration since onset of first adenomyosis symptom was 5.7 years; 41.9% reported severe/very severe adenomyosis. Over 50 symptoms and 30 impacts of adenomyosis were reported in the concept elicitation; 87% of symptoms were reported after 7 interviews and 78% of impacts after 5 interviews, indicating a condition with a significant symptom burden and a consistent presentation. The most common symptoms were heavy menstrual bleeding (87%), cramps (84%), and blood clots during menstrual bleeding (84%). The most common impacts were burdensome self-care hygiene (71%), and fatigue/low energy (71%). In the card-sorting exercise, the most commonly endorsed symptoms were pain during menstruation/menstrual cramps and heavy menstrual bleeding (both frequently rated as severe). The symptom with the highest impact was heavy menstrual bleeding., Conclusion: Initiatives to understand women's experiences with adenomyosis may improve management of the condition. This study provides a first step in understanding their experience and new information on the symptom profile of adenomyosis.

Aim: To gather qualitative data from patients on the burden, impacts and costs of surgical site infections (SSI) requiring second surgeries., Patients & Methods: 15 adults with SSIs from spinal (n = 4), knee replacement (n = 3) or hip replacement (n = 8) surgery participated in a focus group or individual interview. Patients completed the PROMIS Physical Functioning (PF) Short Form 10A (PROMIS-PF)., Results: Patients reported impacts within four primary domains: PF/activity-related; social/emotional; financial/employment; and energy/sleep. The mean PROMIS-PF score was 39.3 (standard deviation = 12.1), over one standard deviation below 50, the US norm., Conclusion: SSIs impart a broad and significant impact on patients and their families. These burdens will be important to capture when selecting patient-reported outcome measures for this patient population.

Background: Engaging adolescents in decisions about their health may enhance their compliance with treatment and result in better health outcomes. Treatment outcomes in attention-deficit/hyperactivity disorder (ADHD) are rarely evaluated from the adolescents' point of view. There is also concern that adolescents with ADHD may not have insight about the impacts of their disease. This article describes research conducted to understand the experiences of adolescents with ADHD and how the research was used to develop an adolescent self-report instrument., Methods: This research involved an iterative process to ensure content validity and was conducted in the following stages: concept identification from literature reviews and interviews with teachers and clinicians; concept elicitation interviews with adolescents with ADHD and their caregivers, review of existing instruments; development of a new instrument and cognitive interviews. Experts in instrument development and translation and clinical practitioners in ADHD also participated., Results: A conceptual framework to measure the impact of ADHD on adolescent functioning identified from concept identification research informed concept elicitation interviews with 60 adolescents with ADHD and their primary caregivers. In the interviews, adolescents discussed difficulties with performing activities in various contexts: school, home, leisure activities and social interactions. Caregivers provided additional insights. The instrument review revealed that none of the existing instruments were suitable to collect data on the elicited concepts; therefore, a new instrument was developed. Revisions were made to the format and content of the instrument (a daily diary) based on feedback received from cognitive testing with 15 adolescents., Conclusions: Our research helped to obtain a comprehensive understanding of the impacts of ADHD on adolescent functioning, to inform the development of a new instrument for measuring outcomes. Adolescents were able to discuss the impact of ADHD on their lives in concept elicitation interviews and report the impacts of ADHD on a self-report instrument. The new instrument developed to reflect the perspective of adolescents with ADHD can be used to supplement outcome assessments in clinic and research settings. Scientific advocacy for the use of such measures can be valuable to measure outcomes meaningful to adolescents with ADHD and the clinical community.

Background: In a randomized controlled trial (RCT) of patients with recurrent, platinum-sensitive ovarian cancer, the combination weekly docetaxel and carboplatin was associated a with progression-free survival (PFS) of 13.7 months compared with 8.4 months for sequential, single-agent docetaxel followed by carboplatin. The objective of the current study was to construct a cost-utility model to compare these 2 regimens with the incorporation of prospectively collected quality-of-life (QoL) data., Methods: An RCT of concurrent docetaxel and carboplatin (cDC) versus docetaxel followed by carboplatin (sequential docetaxel and carboplatin [sDC]) was the basis for a Markov decision model, and the primary effectiveness outcome was PFS. Costs were estimated using US dollars based on Medicare reimbursements for chemotherapy regimens, bone marrow support, and management of adverse events. QoL data obtained using the Functional Assessment of Cancer Therapy-General questionnaire were converted to utilities. Costs and incremental cost-effectiveness ratios (ICERs) were reported in US dollars per quality-adjusted life year (QALY). Extensive 1-way sensitivity analyses and a Monte Carlo probabilistic sensitivity analysis were performed., Results: The least expensive strategy was sDC, which cost an average of $20,381, compared with cDC, which cost an average of $25,122. cDC had an ICER of $25,239 per QALY compared with sDC. cDC remained cost-effective, with an ICER <$50,000 per QALY, over a range of costs and estimates. In Monte Carlo sensitivity analysis using a $50,000 per QALY willingness-to-pay threshold, cDC was either dominant or cost-effective with an ICER <$50,000 per QALY in 83% of simulations., Conclusions: Combined weekly cDC appeared to be cost-effective compared with sDC as treatment strategy for patients with platinum-sensitive ovarian cancer, even when accounting for slightly lower QoL during treatment., (Copyright © 2011 American Cancer Society.)

Objectives: A phase II clinical trial compared docetaxel in combination with carboplatin to sequential single agent docetaxel followed by carboplatin for treatment of recurrent platinum-sensitive ovarian, peritoneal, or tubal cancer. This manuscript reports prospectively collected health-related quality of life (HRQL)., Methods: Participants were randomized to either weekly docetaxel 30 mg/m(2)/days 1 and 8 and carboplatin AUC 6/day 1 every 3 weeks (cDC) or docetaxel 30 mg/m(2)/days 1 and 8, repeated every 3 weeks for 6 cycles followed by carboplatin AUC 6/day 1 every 3 weeks for 6 cycles or until disease progression (sDC). The primary HRQL endpoint was the trial outcome index (TOI) score of the Functional Assessment of Cancer Therapy-Ovarian (FACT-O) instrument, and was assessed as an intent-to-treat analysis. The secondary HRQL endpoints included the FACT-O total score, the FACT-General, and several domain scores of the FACT-O instrument (physical well-being (PWB), social/family well-being (SWB), emotional well-being (EWB), functional well-being (FWB), and the ovarian cancer specific (OCS) module). The FACT-O was administered at randomization, prior to each of 6 cycles of treatment, and at study endpoint., Results: One hundred forty-eight participants were randomized to each group. Sequential docetaxel followed by carboplatin (sDC) was associated with significant improvements in the FACT-O TOI (p=0.013), FACT-O total score (p=0.033), and OCS (p=0.029) compared to the combination docetaxel and carboplatin group (cDC)., Conclusions: Sequential single agent docetaxel followed by carboplatin is associated with improved HRQL when compared to cDC. The improved progression-free survival observed with cDC should be weighed against lower quality of life during treatment., (Copyright © 2011 Elsevier Inc. All rights reserved.)

Purpose: Available patient-reported outcome (PRO) measures for chronic obstructive pulmonary disease (COPD) focus primarily on impairment (symptoms) and activities (functioning). The purpose of the study was to develop a patient-based PRO measure for COPD that captures the overall everyday impact of living with COPD from the patient's perspective., Methods: LCOPD items (Living with COPD Questionnaire) were generated from qualitative interviews in the U.K. and focus groups in the U.S.A. The draft measure was tested for face and content validity in both countries. Item reduction and testing for reproducibility and construct validity was conducted via Rasch and traditional psychometric analyses., Results: The draft LCOPD was found to be relevant and acceptable to patients in the U.K. (N = 19) and U.S. (N = 16). Application of Rasch analysis to data collected in validation studies (n = 162 in the U.K. and 145 in U.S.) identified a 22-item scale that measured a single construct in both countries. Psychometric analyses indicated that this version was internally consistent and reproducible. Scores on the measure were related as expected to clinician ratings of disease severity and patient ratings of COPD severity and general health., Conclusions: The LCOPD is a new measure examining the everyday impact of living with COPD. It demonstrates good scaling properties and may prove valuable in understanding treatment benefits.

Background: Current asthma patient-reported outcome (PRO) measures focus on symptoms and functioning and may not capture the holistic impact of asthma on the quality of life of the patient., Objective: To develop a PRO measure capturing the overall impact of asthma on patient's quality of life., Methods: Items for the Asthma Life Impact Scale (ALIS) were generated from patients with asthma during interviews in the UK and focus groups in the US. The ALIS was tested with UK and US asthma patients during cognitive debriefing interviews and included in large, two-administration, validation studies in the UK and US., Results: Issues raised by asthma patients during interviews (n = 39 patients) and focus groups (n = 16 patients) were included in the draft ALIS. Cognitive debriefing interviews with 29 UK and US asthma patients showed that the scale was relevant and comprehensive. 140 UK and 185 US asthma patients participated in the validation study. The analysis showed that the ALIS measures a single construct, namely the overall impact of asthma on patients' quality of life. Internal consistency (Cronbach's Alpha) was high (UK = 0.94; US = 0.92) as was test-retest reliability (UK = 0.93; US = 0.83). Patients reporting worse general health or more severe asthma had significantly higher ALIS scores (p < 0.001) (indicating greater negative impact of asthma). Correlations with the Asthma Quality of Life Questionnaire were moderate to high., Conclusions: The final 22-item ALIS is unidimensional, reliable and valid, and a valuable tool for comprehensively assessing the holistic impact of asthma from the patient's perspective.

Cancer inflicts great pain, burden and cost upon American society, and preventing cancer is important but not costless. The aim of this review was to explore the upper limits that American society is paying and appears willing to pay to prevent cancer, by enforced environmental regulations and implemented clinical practice guidelines. Cost-effectiveness studies of clinical and environmental cancer-prevention policies and programmes were identified through a comprehensive literature review and confirmed to be officially sanctioned and implemented, enforced or funded. Data were collected in 2005-6 and analysed in 2007. The incremental cost-effectiveness ratios (ICERs) for clinical prevention policies ranged from under $US2000 to over $US6 000 000 per life-year saved (LYS), exceeding $US100 000 per LYS for only 11 of 101 guidelines. Median ICERs for tobacco-related ($US3978/LYS), colorectal ($US22 694/LYS) and breast ($US25 687/LYS) cancer prevention were within generally accepted ranges and tended not to vary greatly, whereas those for prostate ($US73 603/LYS) and cervical ($US125 157/LYS) cancer-prevention policies were considerably higher and varied substantially more. In contrast, both the median and range of the environmental policies were enormous, with 90% exceeding $US100 000 per LYS, and ICERs ranging from $US61 004 to over $US24 billion per LYS. Notwithstanding a relatively large and accessible literature evaluating the cost effectiveness of clinical and environmental cancer-prevention policies as well as the availability of ICERs for the policies identified in this study, the apparent willingness to pay to prevent cancer in the US still varies greatly and can be extremely high, particularly for many of the environmental cancer-prevention policies.