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The functional Scale for the Assessment and Rating of Ataxia (f-SARA) assesses Gait, Stance, Sitting, and Speech. It was developed as a potentially clinically meaningful measure of spinocerebellar ataxia (SCA) progression for clinical trial use. Here, we evaluated content validity of the f-SARA. Qualitative interviews were conducted among individuals with SCA1 (n = 1) and SCA3 (n = 6) and healthcare professionals (HCPs) with SCA expertise (USA, n = 5; Europe, n = 3). Interviews evaluated symptoms and signs of SCA and relevance of f-SARA concepts for SCA. HCP cognitive debriefing was conducted. Interviews were recorded, transcribed, coded, and analyzed by ATLAS.TI software. Individuals with SCA1 and 3 reported 85 symptoms, signs, and impacts of SCA. All indicated difficulties with walking, stance, balance, speech, fatigue, emotions, and work. All individuals with SCA1 and 3 considered Gait, Stance, and Speech relevant f-SARA concepts; 3 considered Sitting relevant (42.9%). All HCPs considered Gait and Speech relevant; 5 (62.5%) indicated Stance was relevant. Sitting was considered a late-stage disease indicator. Most HCPs suggested inclusion of appendicular items would enhance clinical relevance. Cognitive debriefing supported clarity and comprehension of f-SARA. Maintaining current abilities on f-SARA items for 1 year was considered meaningful for most individuals with SCA1 and 3. All HCPs considered meaningful changes as stability in f-SARA score over 1-2 years, 1-2-point change in total f-SARA score, and deviation from natural history. These results support content validity of f-SARA for assessing SCA disease progression in clinical trials.
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BACKGROUND: Measuring self-reported experience of health and functioning is important for understanding the changes in the health status of individuals switching from cigarettes to less harmful tobacco and/or nicotine products (TNP) or reduced-risk products (RRP) and for supporting tobacco harm reduction strategies. METHODS: This paper presents insights from three research activities from the preparatory phase of the development of a new self-report health and functioning measure. A scoping literature review was conducted to identify the positive and negative impact of TNP use on health and functioning. Focus groups (n = 29) on risk perception and individual interviews (n = 40) on perceived dependence in people who use TNPs were reanalyzed in the context of health and functioning, and expert opinion was gathered from five key opinion leaders and five technical consultants. RESULTS: Triangulating the findings of the review of 97 articles, qualitative input from people who use TNPs, and expert feedback helped generate a preliminary conceptual framework including health and functioning and conceptually-related domains impacted by TNP use. Domains related to the future health and functioning measurement model include physical health signs and symptoms, general physical appearance, functioning (physical, sexual, cognitive, emotional, and social), and general health perceptions. CONCLUSIONS: This preliminary conceptual framework can inform future research on development and validation of new measures for assessment of overall health and functioning impact of TNPs from the consumers' perspective.
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Sistemas Electrónicos de Liberación de Nicotina , Productos de Tabaco , Humanos , Nicotina , Autoinforme , NicotianaRESUMEN
BACKGROUND: Hereditary Angioedema (HAE), a rare genetic disease, manifests as intermittent, painful attacks of angioedema. Attacks vary in frequency and severity and include skin, abdominal and life-threatening laryngeal swellings. This study aimed to develop a patient reported outcome (PRO) tool for the assessment of HAE attacks, including their management and impact on patients' lives, for use in clinical studies, or by physicians in general practice. METHODS: The results of open-ended face to face concept elicitation interviews with HAE patients in Argentina (n = 10) and the US (n = 33) were used to develop the first draft questionnaire of the HAE patient reported outcomes questionnaire (HAE PRO). Subsequently, in-depth cognitive debriefing interviews were performed with HAE patients in the UK (n = 10), Brazil (n = 10), Germany (n = 11) and France (n = 12). Following input from eight multinational clinical experts further cognitive interviews were conducted in the US (n = 12) and Germany (n = 12). Patients who experienced abdominal, cutaneous or laryngeal attacks of varying severity levels were included in all rounds of interviews. Across the rounds of interviews patients discussed their HAE attack symptoms, impacts and treatments. Cognitive debriefing interviews explored patient understanding and relevance of questionnaire items. All interviews were conducted face to face following a pre-defined semi-structured interview guide in the patient's native language. RESULTS: Patients reported a variety of HAE symptoms, attack triggers, warning signs, attack impacts and treatment options which were used to develop the HAE PRO. The HAE PRO was revised and refined following input from patients and clinical experts. The final 18-item HAE PRO provides an assessment of the HAE attack experience including symptoms, impacts, treatment requirements, healthcare resource use and loss of productivity caused by HAE attacks. CONCLUSIONS: Patient and expert input has contributed to the development of a content valid questionnaire that assesses concepts important to HAE patients globally. HAE patients across cultures consider the HAE PRO a relevant and appropriate assessment of HAE attacks and treatment.
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Angioedemas Hereditarios/diagnóstico , Angioedemas Hereditarios/psicología , Evaluación del Resultado de la Atención al Paciente , Calidad de Vida/psicología , Adulto , Anciano , Argentina , Brasil , Femenino , Alemania , Humanos , Masculino , Persona de Mediana Edad , Psicometría , Reproducibilidad de los Resultados , Índice de Severidad de la Enfermedad , Encuestas y Cuestionarios , Reino Unido , Estados Unidos , Adulto JovenRESUMEN
BACKGROUND: Herpes zoster (HZ) is a painful condition that can have a substantial negative impact on patients' lives. However, UK-specific data on the debilitating impact of HZ, in terms of patients' experience of pain and impairments in Health-Related Quality of Life (HRQoL) are limited. The Zoster Quality of Life (ZQOL) study, a large-scale UK cross-sectional study, was conducted to quantify the burden of HZ in UK patients. METHODS: A total of 229 HZ patients aged 50 years or over were recruited from primary and secondary/tertiary care centres throughout the UK. Patients completed a battery of validated questionnaires, including the Zoster Brief Pain Inventory (ZBPI), the Medical Outcomes Study Short-Form 36 (SF-36) and the EuroQol-5 Dimensions (EQ-5D) on initial presentation to the doctor and again 7-14 days later. At follow-up patients also completed the Treatment Satisfaction with Medication (TSQM) questionnaire. Where available, mean questionnaire scores in the HZ population were compared to scores for age-matched norms to investigate the burden associated with HZ. RESULTS: Pain was prominent among patients, with 57.9% at the initial study visit reporting pain in the preceding 24 hours at levels typically considered to have a significant impact on HRQoL (i.e. ZBPI worst pain ≥ 5). This was reflected in SF-36 and EQ-5D scores that were significantly lower for patients when compared to age-matched norms (p < 0.05) - except for the SF-36 domain of physical functioning. HRQoL was inversely associated with levels of reported pain, with those patients in the greatest amount of pain reporting the greatest HRQoL impact. However, there was no association between pain severity and participant age. The majority of patients (69.4%) received antivirals within 72 hours of rash appearing and 69.9% of patients were also taking analgesics for the management of HZ pain. TSQM scores indicated that patients were least satisfied with the effectiveness of their prescribed treatment. CONCLUSIONS: The acute presentation of HZ is a painful experience that can have a significant impact on the physical and mental wellbeing of sufferers. Findings highlight significant unmet need among patients, particularly in terms of the effectiveness of therapies for the management of HZ.
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Herpes Zóster/epidemiología , Herpes Zóster/patología , Calidad de Vida , Anciano , Analgésicos/uso terapéutico , Estudios Transversales , Recolección de Datos , Femenino , Herpes Zóster/complicaciones , Humanos , Masculino , Persona de Mediana Edad , Dolor/tratamiento farmacológico , Dolor/etiología , Encuestas y Cuestionarios , Reino Unido/epidemiologíaRESUMEN
Patient-reported outcome (PRO) measures must provide evidence that their development followed a rigorous process for ensuring their content validity. To this end, the collection of data is performed through qualitative interviews that allow for the elicitation of in-depth spontaneous reports of the patients' experiences with their condition and/or its treatment. This paper provides a review of qualitative research applied to PRO measure development. A clear definition of what is a qualitative research interview is given as well as information about the form and content of qualitative interviews required for developing PRO measures. Particular attention is paid to the description of interviewing approaches (e.g., semi-structured and in-depth interviews, individual vs. focus group interviews). Information about how to get prepared for a qualitative interview is provided with the description of how to develop discussion guides for exploratory or cognitive interviews. Interviewing patients to obtain knowledge regarding their illness experience requires interpersonal and communication skills to facilitate patients' expression. Those skills are described in details, as well as the skills needed to facilitate focus groups and to interview children, adolescents and the elderly. Special attention is also given to quality assurance and interview training. The paper ends on ethical considerations since interviewing for the development of PROs is performed in a context of illness and vulnerability. Therefore, it is all the more important that, in addition to soliciting informed consent, respectful interactions be ensured throughout the interview process.
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Entrevistas como Asunto/métodos , Evaluación de Resultado en la Atención de Salud/métodos , Pacientes/psicología , Investigación Cualitativa , Autoinforme , Factores de Edad , Comunicación , Grupos Focales , Guías como Asunto , Investigación sobre Servicios de Salud , Humanos , Prioridad del Paciente , Relaciones Profesional-Paciente , Factores Sexuales , Encuestas y CuestionariosRESUMEN
BACKGROUND: Post-herpetic neuralgia (PHN) is the most common complication of herpes zoster (shingles). As a chronic condition, PHN can have a substantial adverse impact on patients' lives. However, UK-specific data concerning the burden of PHN on individual patients, healthcare systems and wider society, are lacking. As the first UK-wide cross-sectional study of its kind, The Zoster Quality of Life (ZQOL) study was designed to address these concerns. METHODS: Patients (n = 152) with a confirmed diagnosis of PHN (defined as pain persisting ≥ 3 months following rash onset) and aged ≥50 years were recruited from primary and secondary/tertiary care centres throughout the UK. All patients completed validated questionnaires, including the Zoster Brief Pain Inventory (ZBPI), the Medical Outcomes Study Short-Form 36 (SF-36), the EuroQol-5 Dimensions (EQ-5D) and the Treatment Satisfaction with Medication (TSQM) questionnaire. Where available, mean patient population scores on these questionnaires were compared to scores derived from age-matched normative samples to quantify the burden associated with PHN. RESULTS: Despite numerous consultations with healthcare professionals and receiving multiple medications for the management of their PHN, the majority of patients reported being in pain 'most of the time' or 'all of the time'. A total of 59.9% (n = 91) of all PHN patients reported pain in the preceding 24 hours to assessment at levels (ZBPI worst pain ≥ 5) typically considered to have a significant impact on Health Related Quality of Life (HRQoL). Accordingly, scores for SF-36 and EQ-5D indicated significant deficits in HRQoL among PHN patients compared to age-matched norms (p < 0.05) and patients reported being dissatisfied with the perceived efficacy of therapies received for the management of PHN. Increased pain severity was observed among older participants and higher levels of pain severity were associated with greater HRQoL deficits. CONCLUSIONS: The inadequate relief provided by PHN therapies available in the UK is associated with a significant burden among PHN patients in terms of pain severity and deficits in HRQoL which may persist for years. Therefore, alternative means such as prevention of shingles and PHN, are essential for reducing the impact on individual patients, healthcare systems and society as a whole.
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Neuralgia Posherpética/epidemiología , Calidad de Vida , Anciano , Anciano de 80 o más Años , Costo de Enfermedad , Estudios Transversales , Femenino , Humanos , Masculino , Persona de Mediana Edad , Neuralgia Posherpética/tratamiento farmacológico , Neuralgia Posherpética/psicología , Dimensión del Dolor , Satisfacción del Paciente , Calidad de Vida/psicología , Encuestas y Cuestionarios , Reino Unido/epidemiologíaRESUMEN
How nicotine is administered has evolved from cigarettes to various delivery systems. Assessing perceived dependence on nicotine-containing products now requires accounting for product specificity while allowing comparisons across products and users. This study aims to develop a new self-report measure to assess perceived dependence on tobacco and nicotine products (TNPs) among exclusive and poly-TNP users. A draft version of the new measure, the ABOUT-Dependence, was constructed based on literature review, qualitative research, and expert opinion. Data for scale formation and psychometric assessment was obtained through a US-based web survey (n = 2334) that included additional dependence measures for convergent validity assessment. Qualitative research confirmed a preliminary conceptual framework with seven sub-concepts. Following a cognitive debriefing, 19 items were considered to best represent the different sub-concepts. Psychometric findings supported a three-domain structure [i.e., behavioral impact (five items), signs and symptoms (five items), and extent/timing of use (two items)] and an overall total composite score. The data confirmed convergent and known-group validity, as well as test-retest reliability. The ABOUT-Dependence is a 12-item, psychometrically sound, self-report measure that may be used as a tool for research and further understanding of perceived dependence across the spectrum of TNP and TNP users.
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Psicometría , Autoinforme , Humanos , Adulto , Femenino , Masculino , Psicometría/métodos , Persona de Mediana Edad , Nicotina/efectos adversos , Tabaquismo/psicología , Reproducibilidad de los Resultados , Adulto Joven , Adolescente , Encuestas y Cuestionarios , Productos de Tabaco , AncianoRESUMEN
OBJECTIVES: Dryness, fatigue and joint/muscle pain are typically assessed in Sjögren's trials using European Alliance of Associations for Rheumatology Sjögren's Syndrome Patient Reported Index (ESSPRI). A Patient Acceptable Symptom State of <5 and a Minimal Clinically Important Improvement (MCII)/responder definition (RD) of ≥1 point or 15% on ESSPRI have previously been defined. This study explored alternative RDs to better discriminate between active treatment and placebo in trials. METHODS: Anchor-based and distribution-based methods were used to derive RD thresholds in blinded phase IIb trial data (N=190) and confirm these in blinded data pooled from three early phase II trials (N=126). The populations consisted of individuals with moderate-to-severe systemic primary Sjögren's. Anchors were prioritised by ESSPRI correlations and used in similar conditions. Triangulated estimates were discussed with experts (N=3). The revised RD was compared with the original using unblinded data to assess placebo and treatment responder rates. RESULTS: Patients were predominantly female (>90%), white (90%), with mean age of 50 years. Receiver operating characteristic estimates supported an MCII threshold of 1.5-1.6 in the phase II data, whereas correlation-weighted mean change estimates supported a low/minimal symptom severity threshold of ≥2. A low/minimal symptom severity of ≤3 showed the greatest sensitivity/specificity balance. Analyses in the pooled data supported these thresholds (MCII: 1.5-2.1; low/minimal symptom severity: 2.7-3.7). Unblinded analyses confirmed the revised RD reduced placebo rates. CONCLUSIONS: Completing a trial with an improvement of ≥1.5 points compared with baseline and an ESSPRI score of ≤3 points is a relevant RD for moderate-to-severe systemic Sjögren's and reduces placebo rates.
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Síndrome de Sjögren , Humanos , Femenino , Persona de Mediana Edad , Masculino , Síndrome de Sjögren/complicaciones , Síndrome de Sjögren/diagnóstico , Síndrome de Sjögren/tratamiento farmacológico , Fatiga , Curva ROC , Índice de Severidad de la Enfermedad , Medición de Resultados Informados por el PacienteRESUMEN
BACKGROUND: Respiratory Syncytial Virus (RSV) is a leading cause of hospitalization and serious respiratory illness in infants/young children. The objectives of this study were to (1) identify important RSV-related signs of illness in infants that were observed by the parent/caregiver of the child and (2) assess content validity and usability of the Pediatric RSV Electronic Severity and Outcomes Rating System (PRESORS) to monitor signs of RSV-related illness. METHODS: Review of medical literature identified signs of pediatric RSV-related illness in PRESORS. Semi-structured interviews with caregivers of infants (0-24 months of age) hospitalized with laboratory-confirmed RSV infection (in the two months prior to recruitment) were conducted to spontaneously elicit signs and impacts of the infant's illness from caregiver observations. Caregivers completed PRESORS using a "think-aloud" protocol to confirm comprehension, relevance, and usability of the smartphone application. Verbatim transcripts were analyzed using thematic analysis methods and Atlas.ti software. RESULTS: Interviews with 21 caregivers confirmed PRESORS captured 23/26 signs caregivers spontaneously reported. Cough, difficulty breathing, problems sleeping, and reduced feeding/drinking were the most worrying signs of severe RSV-related illness described. Cognitive debriefing indicated that caregivers: understood the wording of all PRESORS items and response options (except how to count heartbeats), recall periods were appropriate, and the PRESORS smartphone application was easy to use. Minor changes to enhance content validity were identified. CONCLUSIONS: In-depth interviews confirmed content validity and usability of the PRESORS by caregivers of infants with RSV. Next steps are to assess the revised PRESORS in clinical studies and evaluate its measurement properties.
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BACKGROUND: There is limited information available on the impact of moderate asthma exacerbations, often called "asthma attacks" (i.e., those not requiring hospitalisation or treatment with systemic corticosteroids) on patients' lives. This multi-country qualitative study explored the patient experience of these events. METHODS: Semi-structured concept elicitation interviews were conducted in the USA and Germany with adult patients with asthma who had experienced a moderate asthma exacerbation in the prior 30 days. Physicians with experience in managing patients with asthma were also interviewed. Interviews explored patients' experience of symptoms and impact of moderate exacerbations and associated exacerbation triggers and treatment patterns. Physicians were also asked about their interpretation of a clinical definition and treatment of a moderate exacerbation. RESULTS: Twenty-eight patient (n = 20 in the USA, n = 8 in Germany) and six physician (n = 3 in the USA, n = 3 in Germany) interviews were conducted. During their moderate exacerbation, all patients reported experiencing shortness of breath, which many considered to be severe and the most bothersome symptom. Wheezing was also reported by all patients and considered severe by two thirds of patients. Most patients also reported coughing and chest tightness. All or almost all patients reported that moderate exacerbation caused fatigue/tiredness and impacted their physical functioning, emotional functioning, activities of daily living and work/school life. Most patients reported using rescue or maintenance inhalers to alleviate symptoms of the exacerbation. Conceptual saturation (i.e., the point at which no new concepts are likely to emerge with continued data collection) was achieved. Findings were used to develop a patient-focused conceptual model of the experience of moderate asthma exacerbations, outlining concepts related to triggers, symptoms, impact, and treatment from the patient perspective. Physician data was consistent with patient reports and complemented the conceptual model. CONCLUSIONS: Findings from concept elicitation interviews highlight the increased frequency, duration and severity of asthma symptoms and increased rescue medication use during moderate asthma exacerbations compared with the typical daily asthma experience, which have a substantial impact on patients' lives.
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BACKGROUND: Bone metastases are a common painful and debilitating consequence of castration-resistant prostate cancer (CPRC). Bone pain may predict patients' prognosis and there is a need to further explore CRPC patients' experiences of bone pain in the overall context of disease pathology. Due to the subjective nature of pain, assessments of pain severity, onset and progression are reliant on patient assessment. Patient reported outcome (PRO) measures, therefore, are commonly used as key endpoints for evaluating the efficacy of CRPC treatments. Evidence of the content validity of leading PRO measures of pain severity used in CRPC clinical trials is, however, limited. METHODS: To document patients' experience of CRPC symptoms including pain, and their impact on health-related quality of life (HRQL), semi-structured in-depth qualitative interviews were conducted with 17 patients with CRPC and bone metastases. The content validity of the Present Pain Intensity (PPI) scale from the McGill Pain Questionnaire (MPQ), and the 'Average Pain' and 'Worst Pain' items of the Brief Pain Inventory Short-Form (BPI-SF) was also assessed. RESULTS: Patients with CRPC and bone metastases present with a constellation of symptoms that can have a profound effect on HRQL. For patients in this study, bone pain was the most prominent and debilitating symptom associated with their condition. Bone pain was chronic and, despite being generally well-managed by analgesic medication, instances of breakthrough cancer pain (BTcP) were common. Cognitive debriefing of the selected PRO measures of pain severity highlighted difficulties among patients in understanding the verbal response scale (VRS) of the MPQ PPI scale. There were also some inconsistencies in the way in which the BPI-SF 'Average Pain' item was interpreted by patients. In contrast, the BPI-SF 'Worst Pain' item was well understood and interpreted consistently among patients. CONCLUSIONS: Study findings support the importance of PRO measures of pain severity as key endpoints for evaluating the efficacy of treatments for CRPC, particularly for patients with bone metastases where episodes of BTcP are common. Qualitative evidence from CRPC patients supports the content validity of the BPI-SF ''Worst Pain' item and promotes use of this item for measuring pain severity in this population.
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Neoplasias Óseas/secundario , Dolor Intratable/diagnóstico , Neoplasias de la Próstata/patología , Anciano , Anciano de 80 o más Años , Progresión de la Enfermedad , Humanos , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Dimensión del Dolor , Investigación Cualitativa , Índice de Severidad de la EnfermedadRESUMEN
BACKGROUND: Ulcerative colitis (UC) often first presents during adolescence and early adulthood. Primary symptoms of UC are well known, yet similarities and differences of disease experience in adults and adolescents are not well characterized. METHODS: To understand the health-related quality of life (HRQoL) and symptomatic experience of UC, in-depth interviews were conducted in the US with 21 adults (20-70 years) and 14 adolescents (12-17 years). Eligibility and medical history were confirmed by clinician report. A previously conducted literature review and resultant conceptual model informed the discussion guide to explore symptoms and HRQoL. Age appropriate creative tasks ("animal" task and collage) were employed to facilitate discussion. Transcripts and collages were subjected to thematic analysis using ATLAS.ti software. RESULTS: Clinician-reported UC severity included 24% mild, 38% moderate, 38% severe among adults; and 64% mild, 29% moderate, 7% severe among adolescents. Among adults, 52% were female, 67% were white. Among adolescents, 50% were female, 71% were white. During analysis it was noted that all participants reported stomach/abdominal pain. Other key symptoms identified were frequent bowel movements, diarrhea, blood in stools, sudden need for bowel movement, stomach cramping, bloating, and feeling gassy/passing gas (≥75% of participants). Key impacts identified were embarrassment, dietary limitations, having to plan around UC, worry/fear, anger, low mood/depression, and relationship with others, (≥75% of participants). In creative tasks, animals were chosen to represent their UC and content included in the collages reflected the most commonly discussed themes from the interviews. Only adults discussed feeling dehydrated, while only adolescents discussed the impact of UC on school life. CONCLUSIONS: Open-ended interviews highlighted the HRQoL and symptomatic experiences of UC from the patient's perspective, which were similar between adult and adolescent UC patients.
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BACKGROUND: Atopic dermatitis (AD) is a common skin disorder characterized by chronic inflammation, altered skin barrier function, and inflammatory cell skin infiltration that decreases health-related quality of life (HRQoL). The study objective was to understand the patient perspective of AD burden and determine suitable patient-reported outcome (PRO) measures. METHODS: This mixed methods study involved the collection of qualitative and quantitative information from adults (≥ 18 years old) and adolescents (12 - 17 years old) with clinician-confirmed AD regarding their experiences of AD symptoms and its impact on HRQoL. The first part of the study included three stages: in-person concept elicitation (CE) interviews, a 2-week daily electronic diary (eDiary) study, and in-person cognitive debriefing (CD) interviews. An Itch numeric rating scale (NRS) (v1.0) and a Skin Pain NRS (v1.0) evaluation during CD interviews required participants to think about their 'worst' itch and 'worst' skin pain in the past 24 h. Other PRO measures allowed for psychometric testing. The second part of the study involved telephone-depth interviews (TDIs) and qualitative feedback from participants who had not participated in the CD interviews. Qualitative data were thematically analyzed. Psychometric evaluation of NRS measures was performed using eDiary data. RESULTS: In the CE interviews, itch and/or itching and skin pain were the most prevalent symptoms consistently discussed by participants. Both NRS measures demonstrated strong psychometric reliability and were applicable across ages with suitable concurrent validity. During the CD interviews, some participants focused their answers on their 'average' itch/itching in the past 24 h, rather than their 'worst' itch. Some participants answered the Skin Pain NRS thinking about general pain or other types of pain, rather than skin pain specifically. Consequently, modifications to both measures addressed these issues and re-tested as paper-and-pen versions in subsequent TDIs. Itch NRS (v2.0) modifications helped participants focus on their worst itching. Most participants preferred Skin Pain NRS v2.0b, which included skin pain descriptors. CONCLUSIONS: Itching and skin pain are the most important and relevant AD symptoms. The Itch NRS (v2.0) and Skin Pain NRS (v2.0b) appear to be appropriate endpoints for the assessment of itching and skin pain severity for clinical trials with adults and adolescents with AD.
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Background. Determining the public health impact of tobacco harm reduction strategies requires the assessment of consumer perception and behavior associated with tobacco and nicotine products (TNPs) with different exposure and risk profiles. In this context, rigorous methods to develop and validate psychometrically sound self-report instruments to measure consumers' responses to TNPs are needed. Methods. Consistent with best practice guidelines, including the U.S. Food and Drug Administration's "Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims," scientifically designed, fit-for-purpose, reliable, and valid instruments are now being applied to tobacco regulatory research. Results. This brief report presents the ABOUT™ Toolbox ( Assessment of Behavioral OUtcomes related to Tobacco and nicotine products) initiative. This communication: (1) describes the methodological steps followed for the development and validation of the measurement instruments included in the ABOUT™ Toolbox, (2) presents a summary of the high-priority tobacco-related domains that are currently covered in the ABOUT™ Toolbox (i.e., risk perception, dependence, product experience, health and functioning, and use history), and (3) details how the measurement instruments are made accessible to the scientific community. Conclusions. By making the ABOUT™ Toolbox available to the tobacco research and public health community, we envision a rapidly expanding knowledge base, with the goals of (1) supporting consumer perception and behavior research to allow comparisons across a wide spectrum of TNPs, (2) enabling public health and regulatory communities to make better-informed decisions for future regulation of TNPs, and (3) enhancing surveillance activities associated with the impact of TNPs on population health.
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Conducta , Nicotina/análisis , Autoinforme , Productos de Tabaco/análisis , Humanos , Psicometría , Reproducibilidad de los ResultadosRESUMEN
OBJECTIVES: The aim was to develop a clinical outcome assessment (COA) for itching in children with cholestatic pruritus. METHODS: This prospective study aimed to enroll patients aged 4-30 years with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis type 1 and caregivers of patients aged 5 months to 14 years. Eligible patients experienced itching during ≥3 of the 7 days before enrollment and had not undergone liver transplant or surgical interruption of the enterohepatic circulation. Open-ended qualitative interviews confirmed that itching was a primary concern for patients and caregivers. Diaries were modified and then evaluated by participants during cognitive debriefing. Interview results were reviewed by clinical, COA and statistical experts. Diary questions were revised following an interim analysis before finalizing the Itch Reported Outcome (ItchRO). RESULTS: Thirty-six interviews were analyzed, representing 25 families of patients with ALGS. Itching was reported spontaneously (without prompting by the interviewer) by ten of 12 patients with ALGS and 19 of 20 caregivers. Consequences of itching included skin damage (78%), mood changes (59%), and difficulties staying asleep (59%) or falling asleep (53%). Two versions of the ItchRO were developed: ItchRO(Patient) for self-completion by patients and ItchRO(Observer) for caregivers. The ItchRO diaries comprise a single scorable item to assess itch and are to be completed twice daily (morning and evening). CONCLUSIONS: Itching was the most bothersome ALGS symptom reported by study participants. We have developed the ItchRO(Patient) and ItchRO(Observer) to assess itching in children with ALGS and other cholestatic liver diseases. These diaries are being validated for use in clinical trials.
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Síndrome de Alagille/complicaciones , Colestasis Intrahepática/complicaciones , Medición de Resultados Informados por el Paciente , Prurito/etiología , Prurito/psicología , Encuestas y Cuestionarios/normas , Adolescente , Adulto , Afecto , Niño , Preescolar , Femenino , Humanos , Lactante , Recién Nacido , Masculino , Estudios Prospectivos , Investigación Cualitativa , Reproducibilidad de los Resultados , Sueño , Factores Socioeconómicos , Adulto JovenRESUMEN
PURPOSE: Major depressive disorder (MDD) has detrimental effects on health-related quality of life (HRQoL). We describe the effect of vortioxetine on HRQoL in MDD patients by using patient-reported outcome instruments. METHODS: HRQoL was evaluated in 5 short-term (6-8 weeks), randomized studies of vortioxetine (5-20 mg/d; n = 2155) versus placebo (n = 1316) in adults with MDD by using the 36-item Short-Form Health Survey (SF-36), the Quality of Life Enjoyment and Satisfaction Questionnaire-Short Form, the EuroQol 5-Dimension Questionnaire (EQ-5D), and the 12-item Health Status Questionnaire in 1 study in elderly patients. Only patients receiving the approved doses of vortioxetine 5, 10, 15, or 20 mg/d were included in the analysis. A random effects meta-analysis was performed on the 4 adult MDD studies that used the SF-36. A within-studies mixed model for repeated measures analysis based on the full analysis set (FAS) was used unless otherwise specified. Standardized effect size (SES) was calculated to reflect clinical relevance, based on a Cohen's d of 0.2. FINDINGS: Vortioxetine produced significantly better results compared with placebo in the SF-36 mental component summary score (5 mg: 2.6, P = 0.001, SES of 0.22, n = 604; 10 mg: 4.8, P < 0.001, SES of 0.42, n = 328) and 4 domain scores (vitality, social functioning, role emotional, and mental health). Vortioxetine was also significantly better in the EuroQoL-5 Dimension Questionnaire Health State score (10 mg: 7.5, P < 0.05, SES of 0.33, n = 86) and Quality of Life Enjoyment and Satisfaction Questionnaire-Short Form total score (15 mg: 3.3, P < 0.01, SES of 0.38, n = 127; 20 mg: 4.5, P < 0.0001, SES of 0.52, n = 134) (FAS, last-observation-carried-forward). In the study of elderly patients, vortioxetine 5 mg (n = 136) improved 12-item Health Status Questionnaire scores significantly more than placebo (n = 148) for the domains of health perception (10.4, P < 0.0001, SES of 0.54), mental health (7.9, P < 0.001, SES of 0.44), and energy (6.4, P < 0.05, SES of 0.28) (FAS, mixed model for repeated measures). IMPLICATIONS: Vortioxetine yielded significant, meaningful HRQoL improvements in 6 MDD studies of 6 to 8 weeks' duration.
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Trastorno Depresivo Mayor/tratamiento farmacológico , Piperazinas/uso terapéutico , Calidad de Vida , Inhibidores Selectivos de la Recaptación de Serotonina/uso terapéutico , Sulfuros/uso terapéutico , Encuestas y Cuestionarios , Adulto , Anciano , Femenino , Estado de Salud , Humanos , Masculino , VortioxetinaRESUMEN
BACKGROUND: The responsibilities of caring for a person with schizophrenia may significantly impact informal caregivers' lives. The Zarit Burden Interview (ZBI) was originally developed to assess burden among caregivers of people with Alzheimer's disease. OBJECTIVE: This research was conducted to inform the development of a revised version of the ZBI, relevant to caregivers of people with schizophrenia. METHODS: Based on published qualitative research, the questionnaire was reviewed and modified in accordance with industry-standard guidelines. The resulting questionnaire [the Schizophrenia Caregiver Questionnaire (SCQ)] was then completed by 19 caregivers during cognitive debriefing interviews to assess understanding, relevance and comprehensiveness. RESULTS: Review of the ZBI resulted in a number of operational changes to improve face validity and potential sensitivity. Further questions were added based on key concepts identified in existing literature and minor phrasing alterations were made to improve content validity. Findings from caregiver interviews supported the content validity of the SCQ. CONCLUSION: The SCQ provides a comprehensive view of caregivers' subjective experiences of caregiving and demonstrated strong face and content validity. The questionnaire will be important in both clinical assessment and evaluating the efficacy of interventions designed to reduce or alleviate caregiver burden. Future research will seek to establish the psychometric validity of the questionnaire.
Asunto(s)
Cuidadores/psicología , Esquizofrenia , Encuestas y Cuestionarios/normas , Adaptación Psicológica , Adulto , Anciano , Anciano de 80 o más Años , Costo de Enfermedad , Femenino , Humanos , Masculino , Persona de Mediana Edad , Psicometría , Reproducibilidad de los Resultados , Índice de Severidad de la EnfermedadRESUMEN
Spina bifida (SB) accounts for approximately 90% of total neural tube defects. According to the degree of SB severity, the range of associated symptoms and complications varies greatly. Given the complexity and diversity of these complications, individuals with SB might suffer from livelong impairment. This review presents an overview of the impact of SB on patients' health-related quality of life (HRQoL) and explores results from published quantitative and qualitative studies regarding the HRQoL impact of SB on patients, as well as comparing results of existing studies to national norms. A literature search using three electronic databases PUBMED, PsycINFO, and Embase was performed to identify relevant studies dating from January 1976 to November 2010. To satisfy the initial inclusion criteria, articles had to contain studies that were specific to HRQoL in patients with SB. Findings highlight that HRQoL is significantly impaired in patients with SB. Making sure that the public is aware of the disease and how to minimise the risk of NTD, such as SB (e.g., through adequate folate levels at time of conception, etc.) is essential to ensure that fewer individuals face the burden of NTDs in the future.
Asunto(s)
Estado de Salud , Calidad de Vida , Disrafia Espinal/complicaciones , Disrafia Espinal/psicología , Emociones , Humanos , Salud Mental , Dolor/etiología , Investigación Cualitativa , Autoimagen , Índice de Severidad de la Enfermedad , Participación Social , Disrafia Espinal/prevención & controlRESUMEN
Objectives. As a disease typified by early onset and chronic disease course, caring for a person with schizophrenia may have a significant impact on caregivers' lives. This study aimed to investigate the subjective experiences of caregivers of people with schizophrenia as a means of understanding "caregiver burden" in this population. Methods. Face-to-face qualitative interviews were conducted with a diverse sample of 19 US-English speaking caregivers of people with schizophrenia (who were at least moderately ill). Interview transcripts were analyzed using grounded theory methods and findings used to inform the development of a preliminary conceptual model outlining caregivers' experiences. Results. Findings support assertions that people with schizophrenia were largely dependent upon caregivers for the provision of care and caregivers subsequently reported lacking time for themselves and their other responsibilities (e.g., family and work). Caregiver burden frequently manifested as detriments in physical (e.g., fatigue, sickness) and emotional well-being (e.g., depression and anxiety). Conclusions. Caring for a person with schizophrenia has a significant impact on the lives of informal (unpaid) caregivers and alleviating caregiver burden is critical for managing individual and societal costs. Future research should concentrate on establishing reliable and valid means of assessing burden among caregivers of persons with schizophrenia to inform the development and evaluation of interventions for reducing this burden.
RESUMEN
BACKGROUND: Symptom measurement in pediatric chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) trials requires appropriately developed clinical outcome assessments (COAs). METHODS: Literature was reviewed to identify symptom COAs meeting regulatory standards. Searches were conducted in Pubmed/Medline, EMBASE, and PsychINFO. Title/abstracts were reviewed to identify qualitative studies and those using COAs to measure pediatric CIC/IBS symptoms. Pediatric functional gastrointestinal experts provided input on relevant symptom-concepts to measure. RESULTS: Review of 1,105 abstracts identified 1 relevant qualitative article and 113 articles including COAs. Symptoms most frequently measured in CIC studies were frequency of bowel movements, fecal incontinence/encopresis, abdominal pain, stool consistency, and painful defecation. Symptoms most frequently measured in IBS were abdominal pain, abdominal distention/bloating, stool consistency, frequency of bowel movements, and gas. Evidence of development/validity of COAs was limited. Expert feedback was broadly consistent with the literature. CONCLUSION: Findings demonstrate consistency in the literature on key CIC/IBS symptoms to measure in pediatric trials, but existing COAs do not meet regulatory standards.