RESUMO
BACKGROUND: Triptans are the first-line option for the acute treatment of migraine attacks; however, triptans are contraindicated in people with certain underlying cardiovascular risk factors and are associated with inadequate efficacy or poor tolerability in some individuals. Ubrogepant is an oral calcitonin gene-related peptide receptor antagonist approved for the acute treatment of migraine. METHODS: This post hoc analysis of the phase 3 ACHIEVE trials examined the impact of ubrogepant on the Functional Disability Scale (FDS), satisfaction with medication, and Patient Global Impression of Change (PGIC) in participants who were self-reported triptan insufficient responders (TIRs), defined as those who are unable to take triptans due to contraindications, tolerability issues, or insufficient efficacy. Responder definitions for the FDS, satisfaction measures, and PGIC were based on qualitative interpretation of the respective response options for the pooled ubrogepant 50 mg and placebo groups. RESULTS: In the pooled analysis population (n = 1799), 451 (25%) participants were TIRs, with most (80%) reporting insufficient efficacy with triptan use. A significantly higher proportion of TIRs treated with ubrogepant vs placebo reported being able to function normally from 2 to 8 h post dose (P < 0.05). Notably, significance was demonstrated at the time of the primary outcome assessments (2 h post dose), where rates of normal function were 38% for ubrogepant vs 29% for placebo (P = 0.048). A greater proportion of TIRs in the ubrogepant arm vs the placebo arm were satisfied with treatment at 2 (33% vs 21%, P = 0.006) and 24 h (58% vs 28%, P < 0.001) and indicated that their migraine improved at 2 h vs placebo (30% vs 18%, P = 0.006). Results were generally similar in the insufficient efficacy subpopulation of TIRs as in the overall TIRs group. Ubrogepant was safe and well tolerated in TIRs, with no new safety signals identified. CONCLUSIONS: In people with migraine who are TIRs, individuals treated with ubrogepant had favorable 2-h outcomes, as measured by the FDS, satisfaction with medication, and PGIC, compared with placebo. TRIAL REGISTRATION: ClinicalTrials.gov: NCT02828020 (ACHIEVE I), registered July 11, 2016; NCT02867709 (ACHIEVE II), registered August 16, 2016.
Assuntos
Transtornos de Enxaqueca , Triptaminas , Antagonistas do Receptor do Peptídeo Relacionado ao Gene de Calcitonina/farmacologia , Antagonistas do Receptor do Peptídeo Relacionado ao Gene de Calcitonina/uso terapêutico , Humanos , Transtornos de Enxaqueca/induzido quimicamente , Transtornos de Enxaqueca/tratamento farmacológico , Satisfação Pessoal , Piridinas , Pirróis , Ensaios Clínicos Controlados Aleatórios como Assunto , Agonistas do Receptor 5-HT1 de Serotonina/uso terapêutico , Triptaminas/uso terapêuticoRESUMO
BACKGROUND: Patient-reported outcome (PRO) data may be used at 2 levels: to evaluate impacts of disease and treatment aggregated across individuals (group-level) and to screen/monitor individual patients to inform their management (individual-level). For PRO data to be useful at either level, we need to understand their clinical relevance. PURPOSE: To provide clarity on whether and how methods historically developed to interpret group-based PRO research results might be applied in clinical settings to enable PRO data from individual patients to inform their clinical management and decision-making. METHODS: We first differentiate PRO-based decision-making required at group versus individual levels. We then summarize established group-based approaches to interpretation (anchor-based and distribution based), and more recent methods that draw on item calibrations and qualitative research methods. We then assess the applicability of these methods to individual patient data and individual-level decision-making. FINDINGS: Group-based methods provide a range of thresholds that are useful in clinical care: some provide screening thresholds for patients who need additional clinical assessment and/or intervention, some provide thresholds for classifying an individual's level of severity of symptoms or problems with function, and others provide thresholds for meaningful change when monitoring symptoms and functioning over time during or after interventions. Availability of established cut-points for screening and symptom severity, and normative/reference values, may play into choice of PRO measures for use in clinical care. Translatability of thresholds for meaningful change is more problematic because of the greater reliability needed at the individual-level versus group-level, but group-based methods may provide lower bound estimates. Caution is needed to set thresholds above bounds of measurement error to avoid "false-positive changes" triggering unwarranted alerts and action in clinic. CONCLUSIONS: While there are some challenges in applying available methods for interpreting group-based PRO results to individual patient data and clinical care-including myriad contextual factors that may influence an individual patient's management and decision-making-they provide a useful starting point, and should be used pragmatically.
Assuntos
Gerenciamento Clínico , Medidas de Resultados Relatados pelo Paciente , Projetos de Pesquisa , Tomada de Decisões , Humanos , Valores de Referência , Reprodutibilidade dos Testes , Índice de Gravidade de DoençaRESUMO
BACKGROUND: Sickle cell disease (SCD) is a chronic condition associated with high mortality and morbidity. It is characterized by acute clinical symptoms such as painful vaso-occlusive crises, which can impair health-related quality of life (HRQL). This study was conducted to identify validated patient-reported outcome (PRO) instruments for use in future trials of potential treatments for SCD. METHODS: A systematic literature review (SLR) was performed using MEDLINE and EMBASE to identify United States (US)-based studies published in English between 1997 and 2017 that reported on validated PRO instruments used in randomized controlled trials and real-world settings. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist was used to assess the quality of PRO instruments. RESULTS: The SLR included 21 studies assessing the psychometric properties of 24 PRO instruments. Fifteen of those instruments were developed and validated for adults and 10 for children (one instrument was used in both children and young adults aged up to 21 years). Only five of the 15 adult instruments and three of the 10 pediatric instruments were developed specifically for SCD. For most instruments, there were few or no data on validation conducted in SCD development cohorts. Of the 24 PRO instruments identified, 16 had strong internal reliability (Cronbach's α ≥0.80). There was often insufficient information to assess the content validity, construct validity, responsiveness, or test-retest reliability of the instruments identified for both child and adult populations. No validated PRO instruments measuring caregiver burden in SCD were identified. CONCLUSIONS: The evidence on the psychometric properties of PRO instruments was limited. However, the results of this SLR provide key information on such tools to help inform the design of future clinical trials for patients with SCD in the US.
Assuntos
Anemia Falciforme , Medidas de Resultados Relatados pelo Paciente , Qualidade de Vida , Adulto , Anemia Falciforme/fisiopatologia , Anemia Falciforme/psicologia , Criança , Pré-Escolar , Humanos , Masculino , Psicometria , Ensaios Clínicos Controlados Aleatórios como Assunto , Reprodutibilidade dos Testes , Estudos de Validação como Assunto , Adulto JovemRESUMO
The focus of this article, the fourth in the series, Standards for Clinical Trials in Male and Female Sexual Dysfunction, is on aspects of clinical trial design and measurement that are specific to clinical trials for treatments of female sexual dysfunction. Challenges in this area include the limited extent of treatment development and clinical trial research across the spectrum of female sexual dysfunctions, changing regulatory considerations, changing diagnostic criteria for female sexual dysfunction, and the need to articulate assessment procedures to these changes. Discussion focuses on approaches to addressing these challenges in clinical trials in female sexual dysfunction.
Assuntos
Ensaios Clínicos como Assunto/normas , Disfunções Sexuais Fisiológicas/terapia , Disfunções Sexuais Psicogênicas/terapia , Feminino , Humanos , Disfunções Sexuais Psicogênicas/diagnósticoRESUMO
This series of articles, Standards for Clinical Trials in Male and Female Sexual Dysfunction, began with the discussion of a common expected standard for clinical trial design in male and female sexual dysfunction, a common rationale for the design of phase I to IV clinical trials, and common considerations for the selection of study population and study duration in male and female sexual dysfunction. The second article in this series discussed fundamental principles in development, validation, and selection of patient- (and partner-) reported outcome assessment. The third and present article in this series discusses selected aspects of sexual dysfunction that are that are unique to male sexual dysfunctions and relevant to the conduct of clinical trials of candidate treatments for men.
Assuntos
Ensaios Clínicos como Assunto/normas , Disfunções Sexuais Fisiológicas/terapia , Disfunções Sexuais Psicogênicas/terapia , Humanos , Masculino , Avaliação de Resultados em Cuidados de SaúdeRESUMO
BACKGROUND: The Hypogonadism Impact of Symptoms Questionnaire Short Form (HIS-Q-SF) is a patient-reported outcome measurement designed to evaluate the symptoms of hypogonadism. The HIS-Q-SF is an abbreviated version including17 items from the original 28-item HIS-Q. AIM: To conduct item analyses and reduction, evaluate the psychometric properties of the HIS-Q-SF, and provide guidance on score interpretation. METHODS: A 12-week observational longitudinal study of hypogonadal men was conducted as part of the original HIS-Q psychometric evaluation. Participants completed the original HIS-Q every 2 weeks. Blood samples were collected to evaluate testosterone levels. Participants completed the Aging Male's Symptoms Scale, the International Index of Erectile Function, the Short Form-12, and the PROMIS Sexual Activity, Satisfaction with Sex Life, Sleep Disturbance, and Applied Cognition Scales (baseline and weeks 6 and 12). Clinicians completed the Clinical Global Impression of Severity and Change scales and a clinical form. MAIN OUTCOME MEASURES: Item performance was evaluated using descriptive statistics and Rasch analyses. Reliability (internal consistency and test-retest), validity (concurrent and know groups), and responsiveness were assessed. RESULTS: One hundred seventy-seven men participated (mean age = 54.1 years, range = 23-83). Similar to the full HIS-Q, the final abbreviated HIS-Q-SF instrument includes five domains (sexual, energy, sleep, cognition, and mood) with two sexual subdomains (libido and sexual function). For key domains, test-retest reliability was very good, and construct validity was good for all domains. Known-groups validity was demonstrated for all domain scores, subdomain scores, and total score based on the Clinical Global Impression-Severity. All domains and subdomains were responsive to change based on patient-rated anchor questions. CLINICAL IMPLICATIONS: The HIS-Q-SF could be a useful tool in clinical practice, epidemiologic studies, and other academic research settings. STRENGTHS AND LIMITATIONS: Careful consideration was given to the selection of the final HIS-Q-SF items based on quantitative data and clinical expert feedback. Overall, the reduced set of items demonstrated strong psychometric properties. Testosterone levels for the participating men were not as low as anticipated, which could have limited the ability to examine the relations between the HIS-Q-SF and testosterone levels. Further, the analyses used data collected through administration of the full HIS-Q, and future studies should administer the standalone HIS-Q-SF to replicate the psychometric analyses reported in the present study. CONCLUSION: Similar to the original HIS-Q, the HIS-Q-SF has evidence supporting reliability, validity, and responsiveness. The short form includes a smaller set of items that might be more suitable for use in clinical practice or academic research settings. Gelhorn HL, Roberts LJ, Khandelwal N, et al. Psychometric Evaluation of the Hypogonadism Impact of Symptoms Questionnaire Short Form (HIS-Q-SF). J Sex Med 2017;14:1046-1058.
Assuntos
Hipogonadismo/psicologia , Psicometria/métodos , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Hipogonadismo/sangue , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Satisfação Pessoal , Estudos Prospectivos , Qualidade de Vida , Reprodutibilidade dos Testes , Inquéritos e Questionários , Testosterona/sangue , Adulto JovemRESUMO
OBJECTIVES: The Psoriasis Symptom Inventory (PSI) is a patient-reported outcome instrument that measures the severity of psoriasis signs and symptoms. This study evaluated measurement properties of the PSI in patients with moderate to severe plaque psoriasis. METHODS: This secondary analysis used pooled data from a phase 3 brodalumab clinical trial (AMAGINE-1). Outcome measures included the PSI, Psoriasis Area and Severity Index (PASI), static Physician's Global Assessment (sPGA), psoriasis-affected body surface area, 36-item Short-Form Health Survey version 2, and the Dermatology Life Quality Index (DLQI). The PSI was evaluated for dimensionality, item performance, reliability (internal consistency and test-retest), construct validity, ability to detect change, and agreement between PSI response and response measures based on the PASI, sPGA, and DLQI. RESULTS: Results supported unidimensionality, good item fit, ordered responses, and PSI scoring. The PSI demonstrated reliability: baseline Cronbach's alpha ≥ 0.92 and intraclass correlation coefficients ≥ 0.95. Correlations between PSI total score and DLQI item 1 (r = 0.86), DLQI symptoms and feelings (r = 0.87), and 36-item Short-Form Health Survey version 2 bodily pain (r = -0.61) supported convergent validity. PSI scores differed significantly (P < 0.001) among severity groups based on the PASI (< 12/≥ 12), sPGA (0-1/2-3/4-5), body surface area (< 5%/5%-10%/> 10%), and DLQI (≤ 5/> 5) at weeks 8 and 12. At week 12, the PSI detected significant changes in severity based on PASI responses (< 50/50- < 75/≥ 75) and sPGA (0-1/≥ 2), and showed good agreement (k ≥ 0.66) between PSI response and PASI, sPGA, and DLQI responses. CONCLUSION: The PSI demonstrated excellent validity, reliability, and ability to detect change in the severity of psoriasis signs and symptoms.
Assuntos
Anticorpos Monoclonais/uso terapêutico , Fármacos Dermatológicos/uso terapêutico , Psoríase/fisiopatologia , Qualidade de Vida , Adulto , Anticorpos Monoclonais Humanizados , Ensaios Clínicos Fase III como Assunto , Feminino , Inquéritos Epidemiológicos , Humanos , Masculino , Pessoa de Meia-Idade , Psoríase/tratamento farmacológico , Ensaios Clínicos Controlados Aleatórios como Assunto , Reprodutibilidade dos Testes , Índice de Gravidade de DoençaRESUMO
OBJECTIVE: The objective of this study was to evaluate the measurement properties of the Migraine Physical Function Impact Diary (MPFID), a novel patient-reported outcome (PRO) measure for assessing the impact of migraine on physical functioning. METHODS: In a prospective, observational study, adults with episodic migraine (EM) or chronic migraine (CM) used an eDiary to complete the MPFID (assessing daily impacts of migraine on physical function) and a headache diary (capturing migraine days, migraine pain intensity, and migraine interference) each day, and other PRO instruments related to migraine. Item-level evaluation, item response theory (IRT), and exploratory factor analysis (EFA) methods were applied to identify domains, select final MPFID items, and develop scoring procedures. Psychometric properties of the final 13-item MPFID were evaluated using confirmatory factor analysis and tests of reliability (Cronbach's α for internal consistency and intra-class correlation [ICC] for test-retest) and validity (convergent and known-groups). RESULTS: The study enrolled 569 adults with chronic or episodic migraine, mean (SD) age 39.9 (12.0) years and 87.2% female. Item-level analyses based on interim data informed selection of a set of 13 items for the MPFID, through evaluation of floor/ceiling effects, item-to-item correlations, factor loadings, and IRT-based fit/misfit statistics. Two domain scores (EA: Impact on Everyday Activities; PI: Physical Impairment) and a global item score for impact on everyday activities were identified. EA and PI domains exhibited high internal consistency (α = 0.97; α = 0.93) and good test-retest reliability among stable subjects (ICCs = 0.74 and 0.77). Convergent validity was demonstrated by moderate correlations (r = ±0.50-0.68; P < .0001) between MPFID domain scores and number of migraine days, headache days, bed days, and other migraine-related PRO instruments. EA and PI scores differentiated between groups who varied by number of migraine days, migraine interference levels, migraine pain intensity, and median split groups of scores based on other PROs instruments (P < .05). CONCLUSIONS: The MPFID has robust psychometric properties (ie, reliability and validity). Findings supported two distinct domains about the impact of migraine on physical functioning: Impact on Everyday Activities and Physical Impairment. Both domain scores showed evidence of excellent reliability and construct validity in assessing the impacts of migraine on physical functioning.
Assuntos
Atividades Cotidianas/psicologia , Exercício Físico/psicologia , Prontuários Médicos/normas , Transtornos de Enxaqueca/diagnóstico , Transtornos de Enxaqueca/psicologia , Medidas de Resultados Relatados pelo Paciente , Adolescente , Adulto , Idoso , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Psicometria , Adulto JovemRESUMO
This series of articles outlines standards for clinical trials of treatments for male and female sexual dysfunctions, with a focus on research design and patient-reported outcome assessment. These articles consist of revision, updating, and integration of articles on standards for clinical trials in male and female sexual dysfunction from the 2010 International Consultation on Sexual Medicine developed by the authors as part of the 2015 International Consultation on Sexual Medicine. We are guided in this effort by several principles. In contrast to previous versions of these guidelines, we merge discussion of standards for clinical trials in male and female sexual dysfunction in an integrated approach that emphasizes the common foundational practices that underlie clinical trials in the two settings. We present a common expected standard for clinical trial design in male and female sexual dysfunction, a common rationale for the design of phase I to IV clinical trials, and common considerations for selection of study population and study duration in male and female sexual dysfunction. We present a focused discussion of fundamental principles in patient- (and partner-) reported outcome assessment and complete this series of articles with specific discussions of selected aspects of clinical trials that are unique to male and to female sexual dysfunction. Our consideration of standards for clinical trials in male and female sexual dysfunction attempts to embody sensitivity to existing and new regulatory guidance and to address implications of the evolution of the diagnosis of sexual dysfunction that have been brought forward in the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition. The first article in this series focuses on phase I to phase IV clinical trial design considerations. Subsequent articles in this series focus on the measurement of patient-reported outcomes, unique aspects of clinical trial design for men, and unique aspects of clinical trial design for women.
Assuntos
Ensaios Clínicos como Assunto/normas , Disfunções Sexuais Fisiológicas/terapia , Disfunções Sexuais Psicogênicas/terapia , Manual Diagnóstico e Estatístico de Transtornos Mentais , Feminino , Humanos , Masculino , Avaliação de Resultados em Cuidados de Saúde , Comportamento SexualRESUMO
The second article in this series, Standards for Clinical Trials in Male and Female Sexual Dysfunction, focuses on measurement of patient-reported outcomes (PROs). Together with the design of appropriate phase I to phase IV clinical trials, the development, validation, choice, and implementation of valid PRO measurements-the focus of the present article-form the foundation of research on treatments for male and female sexual dysfunctions. PRO measurements are assessments of any aspect of a patient's health status that come directly from the patient (ie, without the interpretation of the patient's responses by a physician or anyone else). PROs are essential for assessing male and female sexual dysfunction and treatment response, including symptom frequency and severity, personal distress, satisfaction, and other measurements of sexual and general health-related quality of life. Although there are some relatively objective measurements of sexual dysfunction (ie, intravaginal ejaculatory latency time, frequency of sexual activity, etc), these measurements do not comprehensively assess the occurrence and extent of sexual dysfunction or treatment on the patient's symptoms, functioning, and well-being. Data generated by a PRO instrument can provide evidence of a treatment benefit from the patient's perspective.
Assuntos
Ensaios Clínicos como Assunto/normas , Medidas de Resultados Relatados pelo Paciente , Disfunções Sexuais Psicogênicas/terapia , Feminino , Humanos , Masculino , Qualidade de Vida , Comportamento SexualRESUMO
INTRODUCTION: Hypogonadism in men is often associated with poor libido, erectile dysfunction, irritability, fatigue, and psychological and relationship problems. Many of these symptoms can be best assessed through patient report. The 28-item Hypogonadism Impact of Symptoms Questionnaire (HIS-Q) was developed to evaluate hypogonadism symptoms in men with low testosterone in the context of clinical trials. AIM: To develop a briefer version of the HIS-Q that could be practical for use in treatment settings. METHODS: Participants with low testosterone levels and symptoms consistent with hypogonadism were recruited through clinical sites. Focus groups and interviews were conducted to elicit symptom concepts and identify those that were most relevant to patients, including changes as a consequence of treatment. MAIN OUTCOME MEASURES: Systematic analysis of the qualitative data and expert clinician input were used to develop the HIS-Q short form (HIS-Q-SF). One-on-one cognitive interviews were conducted to confirm the content validity of the HIS-Q-SF. RESULTS: Thirty-five men participated in this qualitative research. Concept elicitation was conducted through focus group discussions (n = 18) and telephone interviews (n = 2); then, the draft HIS-Q-SF was evaluated through cognitive interviews (n = 15). The mean age of total sample was 53.2 ± 6.8 years, and the mean serum total testosterone level was 184.9 ± 55.2 ng/dL. Results suggest that the HIS-Q-SF has demonstrated content validity, including the content coverage, comprehensibility, and the appropriateness of the response options and recall period. The final version of the HIS-Q-SF includes 17 items and is aligned with the original longer version of the instrument. CONCLUSION: The HIS-Q-SF is a comprehensive measurement of hypogonadism symptom severity in men. Content coverage and content validity were confirmed. The instrument will be evaluated further to establish the psychometric characteristics and to assess the utility of the measurement in clinical treatment settings.
Assuntos
Hipogonadismo/psicologia , Inquéritos e Questionários/normas , Adulto , Estudos Transversais , Disfunção Erétil/complicações , Disfunção Erétil/psicologia , Fadiga/etiologia , Grupos Focais , Humanos , Hipogonadismo/tratamento farmacológico , Libido/fisiologia , Masculino , Pessoa de Meia-Idade , Psicometria , Pesquisa Qualitativa , Testosterona/deficiênciaRESUMO
INTRODUCTION: The Hypogonadism Impact of Symptoms Questionnaire (HIS-Q) is a patient-reported outcome measurement designed to comprehensively evaluate the symptoms of hypogonadism and to detect changes in these symptoms in response to treatment. AIM: To conduct item analysis and reduction, evaluate the psychometric properties of the HIS-Q, and provide guidance on interpreting the instrument score. METHODS: A 12-week observational, longitudinal study of hypogonadal men was conducted. Participants completed the HIS-Q every 2 weeks. Blood samples were collected to evaluate testosterone levels. Participants also completed the Aging Male's Symptoms Scale, the International Index of Erectile Function, the Short Form-12 Health Survey, and the Patient-Reported Outcomes Measurement Information System Sexual Activity, Satisfaction with Sex Life, Sleep Disturbance, and Applied Cognition Scales (at baseline and weeks 6 and 12). Clinicians completed the Clinical Global Impression of Severity and Change measurements and a clinical form. MAIN OUTCOME MEASURES: Individual item performance was evaluated using descriptive statistics and Rasch analyses. Reliability (internal consistency and test-retest), validity (concurrent and know groups), and responsiveness were assessed. RESULTS: In total, 177 men participated in the study (mean age = 54.1 years, range = 23-83). The original 53-item draft HIS-Q was reduced to 28 items; the final instrument included five domains (sexual, energy, sleep, cognition, and mood) with two sexual subdomains (libido and sexual function). For all domains, test-retest reliability was acceptable (intraclass correlation coefficients > 0.70), construct validity was good (|r > 0.30| for all comparisons). Known-groups validity was demonstrated for all HIS-Q domain scores, subdomain scores, and the total score as measured by the Clinical Global Impression of Severity, and total testosterone level at baseline (P < .05 for all comparisons). All domains and subdomains were responsive to change based on patient-rated anchor questions (P < .05 for all comparisons). CONCLUSION: The final 28-item HIS-Q is reliable, valid, and responsive. The HIS-Q is suitable for inclusion in future clinical trials to help characterize the effects of testosterone replacement therapy.
Assuntos
Hipogonadismo/psicologia , Inquéritos e Questionários/normas , Adulto , Afeto , Idoso , Idoso de 80 Anos ou mais , Terapia de Reposição Hormonal/métodos , Humanos , Libido/fisiologia , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Satisfação Pessoal , Psicometria/instrumentação , Qualidade de Vida , Reprodutibilidade dos Testes , Comportamento Sexual , Testosterona/metabolismo , Testosterona/uso terapêutico , Adulto JovemRESUMO
OBJECTIVES: Neuropathic pain (NP) is a consequence of many chronic conditions. This study aimed to develop an unidimensional NP scale with scores that represent levels of NP and distinguish between individuals with NP and non-NP conditions. METHODS: A candidate item pool of 42 pain quality descriptors was administered to participants with osteoarthritis, rheumatoid arthritis, diabetic neuropathy, and cancer chemotherapy-induced peripheral neuropathy. A subset of pain quality descriptors (items) that best distinguished between participants with and those without NP conditions were identified. Dimensionality of pain descriptors was evaluated in a development sample and cross-validated in a holdout sample. Item responses were calibrated using an item response theory model, and scores were generated on a T-score metric. NP scale scores were evaluated in terms of the reliability, validity, and ability to distinguish between participants with and without conditions typically associated with NP. RESULTS: Of the 42 initial items, 5 were identified for the Patient-Reported Outcome Measurement Information System (PROMIS) Neuropathic Pain Quality Scale. T scores exhibited good discriminatory ability on the basis of receiver-operator characteristic analysis. Score thresholds that optimize sensitivity and specificity were identified. Construct, criterion, and discriminant validity, and reliability of scale scores were supported. CONCLUSIONS: The five-item Patient-Reported Outcome Measurement Information System (PROMIS PQ-Neuro) Neuropathic Pain Quality Scale is a short and practical measure that can be used to identify patients more likely to have NP and to distinguish levels of NP. The data collected will support future research that targets other unidimensional pain quality domains (e.g., nociceptive pain).
Assuntos
Neuralgia , Medição da Dor/instrumentação , Autorrelato/normas , Adulto , Idoso , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Qualidade de Vida , Resultado do TratamentoRESUMO
BACKGROUND: Physical function (PF) is a common health concept measured in clinical trials and clinical care. It is measured with different instruments that are not directly comparable, making comparative effectiveness research (CER) challenging when PF is the outcome of interest. OBJECTIVE: Our goal was to establish a common reporting metric, so that scores on commonly used physical function measures can be converted into PROMIS scores. DESIGN: Following a single-sample linking design, all participants completed items from the NIH Patient Reported Outcomes Measurement Information System (PROMIS®) Physical Function (PROMIS PF) item bank and at least one other commonly used "legacy" measure: the Health Assessment Questionnaire (HAQ) or the Short Form-36 physical function ten-item PF scale (SF-36 PF). A common metric was created using analyses based on item response theory (IRT), producing score cross-walk tables. PARTICIPANTS: Participants (N = 733) were part of an internet panel, many of whom reported one or more chronic health conditions. MAIN MEASURES: PROMIS PF, SF-36 PF, and the HAQ-Disability Index (HAQ-DI). RESULTS: Our results supported the hypothesis that all three scales measure essentially the same concept. Cross-walk tables for use in CER are therefore justified. CONCLUSIONS: HAQ-DI and SF-36 PF results can be expressed on the PROMIS PF metric for the purposes of CER and other efforts to compare PF results across studies that utilize any one of these three measures. Clinicians seeking to incorporate PROs into their clinics can collect patient data on any one of these three instruments and estimate the equivalent on the other two.
Assuntos
Atividades Cotidianas , Inquéritos Epidemiológicos/normas , Aptidão Física/fisiologia , Autorrelato/normas , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Inquéritos Epidemiológicos/métodos , Humanos , Masculino , Pessoa de Meia-Idade , Inquéritos e Questionários/normas , Adulto JovemRESUMO
Measuring health and health-related quality of life (HRQoL) is important for tracking the health of individuals and populations over time. Generic HRQoL measures allow for comparison across health conditions. One form of generic HRQoL measures are profile measures, which provide a description of health across several different domains (such as physical functioning, depression, and pain). Recent advances in health profile measurement include the development of measures based on item response theory. The Patient-Reported Outcomes Measurement Information System (PROMIS®) has been constructed using this theory. Another form of generic HRQoL measures are utility measures, which assess the value of health states. Multi-attribute utility theory provides a framework for valuing disparate domains of health and aggregating them into a single preference-based score. Such a score provides an overall measure of health outcomes as well as a quality of life weight for use in decision analyses and cost-effectiveness analyses. Developing a utility score for PROMIS® would allow simultaneous estimation of both health profile and utility scores using a single measure. The purpose of this paper is to provide a roadmap of the methodological steps necessary to create such a scoring system.
Assuntos
Nível de Saúde , Inquéritos Epidemiológicos , Avaliação de Resultados em Cuidados de Saúde/métodos , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Humanos , Autorrelato , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Evidence is sparse about whether body weight categories in adolescents are associated with differences in pediatric HRQoL rated by adolescents and parents. Additionally, it is unknown whether HRQoL rated by individuals with different body mass index (BMI) weight categories is psychometrically comparable. This study aimed to assess whether difference in pediatric HRQoL rated by adolescents and their parents was explained by BMI weight status, and to test measurement properties of HRQoL items related to weight categories using differential item functioning (DIF) methodology. DIF refers to the situation when the individuals across subgroups rate an item differently (e.g., item score three by one subgroup and four by another) given the same underlying construct. METHODS: A cross-sectional study utilizing a sample of parents (n = 323) and their adolescents aged 15-18 years old (n = 323) who enrolled in Florida's Medicaid. Adolescent self-reports and parent proxy-reports of the Pediatric Quality of Life Inventory was adopted to measure pediatric HRQoL. We classified body weight categories as normal weight, overweight, and obesity. A Multiple Indicator Multiple Cause (MIMIC) method was used to assess DIF associated with BMI weight status, especially testing the disparity in the parameters of different weight categories (reference: lower weight category) associated with a response to a HRQoL item conditioning on the same underlying HRQoL. DIF analyses were conducted by adolescent self-reports and parent proxy-reports. RESULTS: Parents reported lower pediatric HRQoL across all domains than adolescents did. Excess body weight (combined overweight and obese) was significantly associated with a greater discrepancy in the rating of emotional and total functioning between adolescents and parents (p < 0.05). DIF associated with BMI weight categories was identified by two items in adolescent self-reports and five items in parent proxy-reports. CONCLUSIONS: Adolescents' BMI weight categories significantly contribute to a difference in the rating of pediatric HRQoL by adolescents and parents.
Assuntos
Viés , Índice de Massa Corporal , Obesidade/psicologia , Pais , Psicometria/métodos , Qualidade de Vida/psicologia , Autorrelato , Adolescente , Adulto , Peso Corporal , Estudos Transversais , Emoções , Feminino , Florida , Humanos , Masculino , Sobrepeso/psicologia , Pediatria , Procurador , Psicometria/normas , Projetos de Pesquisa , Magreza , Adulto JovemRESUMO
BACKGROUND: The United States has a culturally and demographically diverse populace, and the aim of this study was to examine differences in health preferences by sex, age, ethnicity, and race. METHODS: We assessed preferences for health outcomes defined by the PROMIS-29 survey in a sample of the US population. On the basis of the survey's 540 paired-comparisons trading off lifespan and 7 domains of health-related quality of life (HRQoL), we compared the choices between men and women, adults age 18-54 years and 55 years and older, Hispanics and non-Hispanics, and non-Hispanic blacks and whites. For each subgroup, we estimated the value of 122 HRQoL outcomes on a quality-adjusted life year scale and tested for subgroup differences. RESULTS: Compared with men, women preferred reduced lifespan over losses in HRQoL, particularly for depression. Compared with the younger adults, older adults preferred reduced lifespan over the symptoms of depression, anxiety, and fatigue. Compared with non-Hispanic whites, Hispanics preferred reduced lifespan over depression and sleep disturbance, but held similar values on losses in physical functioning. Among non-Hispanics, blacks preferred reduced lifespan over losses in ability to climb stairs and to fall asleep compared with whites, but held similar values on mental health outcomes. CONCLUSIONS: With the growing emphasis on patient-centeredness and culturally sensitive treatment, it is important to recognize the diversity in values placed on potential losses in HRQoL, particularly mental health outcomes. Demographic differences in preferences may influence comparative or cost effectiveness of treatments as perceived by one or another subgroup.
Assuntos
Preferência do Paciente/estatística & dados numéricos , Atividades Cotidianas/psicologia , Adolescente , Adulto , Fatores Etários , Ansiedade/psicologia , População Negra/psicologia , População Negra/estatística & dados numéricos , Depressão/psicologia , Fadiga/psicologia , Feminino , Hispânico ou Latino/psicologia , Hispânico ou Latino/estatística & dados numéricos , Humanos , Masculino , Pessoa de Meia-Idade , Preferência do Paciente/psicologia , Anos de Vida Ajustados por Qualidade de Vida , Estados Unidos/epidemiologia , População Branca/psicologia , População Branca/estatística & dados numéricos , Adulto JovemRESUMO
OBJECTIVES: Health valuation studies enhance economic evaluations of treatments by estimating the value of health-related quality of life (HRQOL). The Patient-Reported Outcomes Measurement Information System (PROMIS) includes a 29-item short-form HRQOL measure, the PROMIS-29. METHODS: To value PROMIS-29 responses on a quality-adjusted life-year scale, we conducted a national survey (N = 7557) using quota sampling based on the US 2010 Census. Based on 541 paired comparisons with over 350 responses each, pair-specific probabilities were incorporated into a weighted least-squared estimator. RESULTS: All losses in HRQOL influenced choice; however, respondents valued losses in physical function, anxiety, depression, sleep, and pain more than those in fatigue and social functioning. CONCLUSIONS: This article introduces a novel approach to valuing HRQOL for economic evaluations using paired comparisons and provides a tool to translate PROMIS-29 responses into quality-adjusted life-years.
Assuntos
Comportamento de Escolha , Nível de Saúde , Modelos Econômicos , Qualidade de Vida/psicologia , Inquéritos e Questionários , Adolescente , Adulto , Ansiedade/psicologia , Depressão/psicologia , Fadiga/psicologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Dor/psicologia , Psicometria , Anos de Vida Ajustados por Qualidade de Vida , Reprodutibilidade dos Testes , Sono , Fatores Socioeconômicos , Adulto JovemRESUMO
PURPOSE: Large samples are generally considered necessary for Rasch model to obtain robust item parameter estimates. Recently, small sample Rasch analysis was suggested as preliminary assessment of items' psychometric properties. This study is to evaluate the Rasch analysis results using small sample sizes. METHODS: Ten PROMIS pain behavior items were used. Random samples of 30, 50, 100, and 250, and a targeted sample of 30 were drawn 10 times each from a total of 800 subjects. Rasch analysis was conducted for each of these samples and the full sample. RESULTS: In the full sample, there were 104 cases of extreme scores, no null categories, two incorrectly ordered items, and four misfit items. For samples of 250, 100, 50, 30, and targeted 30, the average numbers of extreme scores were 42.2, 17.1, 9.6, 6.1, and 1.2; the average numbers of null categories were 1.0, 3.2, 8.7, 13.4, and 8.3; the average numbers of items with incorrectly ordered item parameters were 0.1, 0.8, 2.9, 4.7, and 3.7; and the average numbers of items with fit residuals exceeding ± 2.5 were 0.8, 0.3, 0.1, 0.2, and 0.3, respectively. CONCLUSIONS: Rasch analysis based on small samples (≤ 50) identified a greater number of items with incorrectly ordered parameters than larger samples (≥ 100). However, fewer items were identified as misfitting. Results from small samples led to opposite conclusions from those based on larger samples. Rasch analysis based on small samples should be used for exploratory purposes with extreme caution.
Assuntos
Inquéritos Epidemiológicos/métodos , Modelos Estatísticos , Medição da Dor/métodos , Psicometria/métodos , Qualidade de Vida , Inquéritos e Questionários , Inquéritos Epidemiológicos/normas , Humanos , Dor/fisiopatologia , Dor/psicologia , Projetos Piloto , Psicometria/normas , Valores de Referência , Tamanho da AmostraRESUMO
PURPOSE: The assessment of pain sensation and quality is a key component in understanding the experience of individuals with chronic pain. This study evaluated the factor structure of the patient-reported outcome measurement information system (PROMIS) pain quality item bank. METHODS: As part of the PROMIS project, we developed a pool of 37 pain quality items, based on a review of existing pain questionnaires and development of new items. A web-based survey was designed and completed by 845 members of the general population and 967 individuals with different types of chronic pain. Exploratory factor analysis (EFA) was conducted on a random split-half sample of the data to examine the factor structure of the 37 PROMIS pain quality items in the general population and in a chronic pain sample. A confirmatory factor analysis was conducted in the holdout sample. RESULTS: The EFA of the pain quality items resulted in comparable six-factor solutions for the general and chronic pain samples: (1) pulling/tugging pain; (2) tingling/numbness pain; (3) sharp/stabbing pain; (4) dull/aching pain; (5) pounding/pulsing pain; and (6) affective pain. The confirmatory factor analysis in the holdout sample supported this factor structure. CONCLUSIONS: Further research is needed to evaluate the psychometric characteristics of the derived scales based on their factor scores.