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OBJECTIVES: To explore the agreement between patient-reported flare status and clinically significant flare status in patients with rheumatoid arthritis (RA) in sustained remission. METHOD: Patients with RA in remission for ≥12 months on stable treatment were included in the ARCTIC REWIND tapering trials and pooled 12-month data used in current analyses. Patient-reported flare status was assessed according to the Outcome Measures in Rheumatology flare questionnaire; 'Are you having a flare of your RA at this time?' (yes/no). A clinically significant flare was defined as a combination of Disease Activity Score (DAS) >1.6, increase in DAS of ≥0.6 and 2 swollen joints, or the rheumatologist and patient agreed that a clinically significant flare had occurred. Agreement coefficient, sensitivity, specificity and predictive values of patient-reported flare status with regard to clinically significant flare status were determined. RESULTS: Of 248 patients, 64% were women, age 56.1 (11.8) years, disease duration 4.1 (2.8-7.4) years, DAS 0.8 (0.3). 35% of patients reported a flare at least once, clinically significant flares were recorded in 21%. 48/53 clinically significant flares (91%) led to an intensification of disease-modifying antirheumatic drugss. In 621/682 (91%) visits, patient-reported and clinically significant flare status were in agreement, agreement coefficient 0.89. Sensitivity and specificity were both 91%, positive predictive value of patient-reported flare status 46% and negative predictive value 99%. CONCLUSION: Among patients in sustained remission, patient-reported flare status was accurate in ruling out a clinically significant flare. About half of the patient-reported flares were assessed to be clinically significant. These findings support a potential for using patient-reported flare status in remote monitoring of patients with RA in sustained remission.
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Antirreumáticos , Artritis Reumatoide , Medición de Resultados Informados por el Paciente , Inducción de Remisión , Índice de Severidad de la Enfermedad , Humanos , Artritis Reumatoide/tratamiento farmacológico , Artritis Reumatoide/diagnóstico , Femenino , Masculino , Persona de Mediana Edad , Antirreumáticos/uso terapéutico , Anciano , Brote de los Síntomas , Adulto , Resultado del Tratamiento , Encuestas y CuestionariosRESUMEN
INTRODUCTION: The integration of Patient-Reported Experience Measures (PREM) alongside traditional clinical outcomes is crucial for improving quality of care. Although PREMs are frequently measured in inpatient treatment settings, they are rarely employed in digitally supported care processes or longitudinal assessment of care pathways. METHODS: To gain an overview of PREMs used to cover patients' experiences with digitally supported care processes in heart failure (HF), a scoping review was conducted in Medline. RESULTS: Out of 538 publications, 29 were identified that focus on PREMs in digitally supported care processes across 9 unspecific and 14 disease-specific groups, with 5 manuscripts focusing on HF. PREMs were mostly assessed using self-developed, study-specific questionnaires lacking standardization and validity. In total, 9 PREM dimensions and 25 sub-dimensions were identified. This included care delivery, privacy, physician-patient relationship, involvement, administration, information, knowledge, technology, and experiences in general. CONCLUSION: The findings suggest that the relevance of different dimensions assessed depends largely on the type of care rather than the underlying chronic disease.
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Insuficiencia Cardíaca , Medición de Resultados Informados por el Paciente , Insuficiencia Cardíaca/terapia , Humanos , Satisfacción del Paciente , TelemedicinaRESUMEN
BACKGROUND: N-methyl-D-aspartate-receptor (NMDAR) encephalitis is a rare neurological autoimmune disease with severe neuropsychiatric symptoms during the acute phase. Despite good functional neurological recovery, most patients continue to experience cognitive, psychiatric, psychological, and social impairments years after the acute phase. However, the precise nature and evolving patterns over time of these long-term consequences remain unclear, and their implications for the well-being and quality of life of predominantly young patients have yet to be thoroughly examined. METHODS: SAPIENCE is a European multi-center (n = 3) prospective observational cohort study studying the long-term cognitive, psychiatric, psychological, and social outcome in patients with NMDAR encephalitis. The study consists of three interconnected levels. Level 1 comprises a qualitative interview and focus groups with patients and their caregivers. Level 2 consists of a condensed form of the interview, standardized questionnaires, and a detailed neuropsychological examination of patients. Level 3 involves an online survey that will be open to patients world-wide and explores patient-reported outcomes (PROMs), and patient-reported experiences (PREMs) in association with clinical and cognitive outcomes. Levels 1 to 3 will progressively contribute developing of structured interviews, survey questions, and treatment guidelines by informing one another. DISCUSSION: SAPIENCE is an in-depth study of the long-term effects of NMDAR encephalitis and bridges the gap between standardized assessments and individual patient experiences, intending to improve patient care and to increase awareness of the psychosocial long-term consequences of the disease. Through collaboration of experts in clinical neurology and social and health psychology across Europe, SAPIENCE aims to create online assessment tools and formulate guidelines for patient-centered post-acute care that will help enhance the quality of life for patients and caregivers.
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Encefalitis Antirreceptor N-Metil-D-Aspartato , Humanos , Encefalitis Antirreceptor N-Metil-D-Aspartato/psicología , Encefalitis Antirreceptor N-Metil-D-Aspartato/complicaciones , Calidad de Vida/psicología , Estudios Prospectivos , Femenino , Estudios de Cohortes , Masculino , Europa (Continente)/epidemiología , Adulto , Pruebas Neuropsicológicas , Medición de Resultados Informados por el PacienteRESUMEN
BACKGROUND: Chronic cough, a cough lasting >8 weeks, includes refractory chronic cough (RCC) and unexplained chronic cough (UCC). Patient-reported outcome (PRO) measures are needed to better understand chronic cough impacts that matter most to patients. The 19-item Leicester Cough Questionnaire (LCQ), an existing PRO measure of chronic cough, assesses impacts of cough across physical, psychological, and social domains. However, the content validity of the LCQ evaluating these concepts in patients with RCC/UCC had not been established. OBJECTIVES: To evaluate the content validity of the LCQ in patients with RCC/UCC. DESIGN: A cross-sectional, qualitative interview study. METHODS: First, previously completed qualitative interview results in adults with RCC/UCC (N = 30) were evaluated and mapped to LCQ concepts. Next, a clinical cough expert reviewed each LCQ item and assessed the salience of its concepts for patients with RCC/UCC. Finally, semistructured interviews-including both concept elicitation and cognitive debriefing-were conducted in adults with RCC/UCC (N = 20) to elicit a comprehensive set of participant experiences and to assess the appropriateness of using the LCQ in this population. RESULTS: Concepts reported in the past and present qualitative interviews were included across all LCQ items, and most impacts reported to be the "most bothersome" were assessed in the LCQ. In the current study, all participants indicated that reduced cough frequency would be an important treatment target. During cognitive debriefing, each LCQ item was endorsed by ⩾70% of participants. Additionally, participants were generally able to understand, recall, and select a response for each LCQ item. All participants and the clinical expert indicated that the LCQ was appropriate and assessed the impacts most relevant to patients with RCC/UCC. CONCLUSION: Our findings support the content validity of the LCQ and demonstrate that this measure is fit-for-purpose and includes important cough impacts in adults with RCC/UCC.
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Tos , Entrevistas como Asunto , Medición de Resultados Informados por el Paciente , Humanos , Tos/diagnóstico , Tos/fisiopatología , Tos/psicología , Masculino , Femenino , Persona de Mediana Edad , Enfermedad Crónica , Estudios Transversales , Adulto , Anciano , Reproducibilidad de los Resultados , Investigación Cualitativa , Encuestas y Cuestionarios , Calidad de Vida , Valor Predictivo de las Pruebas , Tos CrónicaRESUMEN
BACKGROUND: Medication Treatment Satisfaction (M-TS) from the patients' perspective is important for comprehensively evaluating the effect of medicines. The extent to which current patient-reported outcome measures (PROMs) for M-TS are valid, reliable, responsive, and interpretable remains unclear. To assess the measurement properties of existing PROMs for M-TS and to highlight research gaps. METHODS: Using PubMed, Embase (Ovid), Cochrane library (Ovid), IPA (Ovid), PsycINFO, Patient-Reported Outcome and Quality of Life Questionnaires biomedical databases, and four Chinese databases, we performed a systematic search for studies addressing the development and validation of PROMs for M-TS. Based on the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) guideline, pairs of reviewers independently assessed the measurement properties of the PROMs and rated the quality of evidence on the measurement properties of each PROM. (The Open Science Framework registration: https://doi.org/10.17605/OSF.IO/8S5ZM ). RESULTS: This review identified 69 PROMs for M-TS in 114 studies (four generic, 32 disease-specific, and 33 drug-specific) of which 60 were intended for adults. All provided limited or no information regarding interpretability. Most demonstrated appropriate construct validity including convergent validity (39/69) and discriminative or known-groups validity (40/69) (high to moderate quality of evidence). Only a few provided evidence of sufficient content validity (8/69), structural validity (13/69), and internal consistency (11/69). Of 38 PROMs reporting test-retest reliability, results in 24 provided evidence of satisfactory test-retest reliability (18 with high to moderate, 6 with low to very low quality of evidence). Few PROMs reported responsiveness (16/69). Two generic PROMs (Treatment Satisfaction Questionnaire for Medication initial Version 1.4, TSQM-1.4; Treatment Satisfaction with Medicines Questionnaire, SATMED-Q) and one drug-specific PROM (Insulin Treatment Satisfaction Questionnaire, ITSQ) demonstrated both satisfactory validity and reliability. CONCLUSIONS: Most existing PROMs for M-TS require further exploration of measurement properties. Reporting guidelines are needed to enhance the reporting quality of the development and validation of PROMs for M-TS.
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Medición de Resultados Informados por el Paciente , Satisfacción del Paciente , Humanos , Calidad de Vida , Encuestas y Cuestionarios , Reproducibilidad de los ResultadosRESUMEN
PURPOSE: Positive patient experiences can lead to better adherence to cancer treatment and greater patient health outcomes. The primary aim of this descriptive study was to determine whether commonly used cancer PREMs have been developed according to health literacy best practices. The secondary and third aims were to examine the development of PREMs and to assess their comprehensiveness against principles of patient-centered care. METHODS: To assess adherence to best practice literacy principles regarding readability and understandability of commonly used cancer PREMs, three validated readability calculators and a validated instrument were utilized. To better understand how PREMs were developed, data about survey items, patient involvement, and expert consultation were collected. Finally, the Picker framework was used to evaluate the comprehensiveness of PREMs against principles of patient-centered care. RESULTS: Thirty-five PREMs studies met inclusion criteria for the study. The mean reading grade level of cancer PREMs was 9.7 (SD = 0.75, range = 8.2-11.2) with best practice recommendation being a grade 6 reading grade level. Twenty-eight PREMs were rated on understandability, with a mean score of 74% (SD = 10.6, range = 46-93%, with optimal score of greater than 80%). The mean number of items across PREMs was 49 (SD = 31, range = 13-136). Recommendations for the number of items to include in a questionnaire is 25-30 items. Most PREMs (n = 33, 94.3%) asked ≥ 1 double-barreled question. All PREMs addressed ≥ 2 patient-centered care principles. CONCLUSION: Cancer PREMs included in this study did not meet evidence-informed thresholds for readability and understandability. As such, it is possible that there may be gaps in how we understand the care experiences of low health literacy populations. Future development of PREMs should engage patients with low health literacy to ensure their perspectives are accurately captured and that PREMs are designed to meet the needs of all patients.
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Alfabetización en Salud , Neoplasias , Atención Dirigida al Paciente , Humanos , Atención Dirigida al Paciente/normas , Medición de Resultados Informados por el Paciente , Comprensión , Encuestas y Cuestionarios , Guías de Práctica Clínica como AsuntoRESUMEN
BACKGROUND: Acute ankle sprains represent one of the most common traumatic injuries to the musculoskeletal system. Many individuals with these injuries experience unresolved symptoms such as instability and recurrent sprains, leading to chronic ankle instability (CAI), which affects their ability to maintain an active lifestyle. While rehabilitation programs focusing on sensorimotor, neuromuscular, strength and balance training are primary treatments, some patients require surgery when rehabilitation fails. A critical analysis of the patient-reported outcome tools (PROs) used to assess CAI surgical outcomes raises some concerns about their measurement properties in CAI patients, which may ultimately affect the quality of evidence supporting current surgical practice. The aim of this research is to develop and validate a new PRO for the assessment of ankle instability and CAI treatment outcomes, following recent methodological guidelines, with the implicit aim of contributing to the generation of scientifically meaningful evidence for clinical practice in patients with ankle instability. METHODS: Following the COnsensus-based Standards for the selection of Health Measurement Instruments (COSMIN), an Ankle Instability Treatment Index (AITI) will be developed and validated. The process begins with qualitative research based on faceâtoâface interviews with CAI individuals to explore the subjective experience of living with ankle instability. The data from the interviews will be coded following an inductive approach and used to develop the AITI content. The preliminary version of the scale will be refined through an additional round of faceâtoâface interviews with a new set of CAI subjects to define the AITI content coverage, relevance and clarity. Once content validity has been examined, the AITI will be subjected to quantitative analysis of different measurement properties: construct validity, reliability and responsiveness. DISCUSSION: The development of AITI aims to address the limitations of existing instruments for evaluating surgical outcomes in patients with CAI. By incorporating patient input and adhering to contemporary standards for validity and reliability, this tool seeks to provide a reliable and meaningful assessment of treatment effects. TRIAL REGISTRATION: Not applicable.
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Traumatismos del Tobillo , Inestabilidad de la Articulación , Medición de Resultados Informados por el Paciente , Humanos , Inestabilidad de la Articulación/cirugía , Inestabilidad de la Articulación/fisiopatología , Traumatismos del Tobillo/cirugía , Traumatismos del Tobillo/terapia , Articulación del Tobillo/fisiopatología , Articulación del Tobillo/cirugía , Reproducibilidad de los ResultadosRESUMEN
Background: The spreading adoption of value-based models of healthcare delivery has incentivized the use of patient-reported outcomes and experience measures (PROMs and PREMs) in clinical practice, with the potential to enrich the decision-making process with patient-reported data. Methods: This perspective article explores PROs and the shared decision-making (SDM) process as components of value-based healthcare. We describe the potential of PROMs and PREMs within the decision-making process and present a digital framework for informing the shared decision-making process using aggregated data from a healthcare system PROMs and PREMs program, including early results from implementation in hospital network in Madrid, Spain. Results: The proposed digital framework incorporates aggregated data from a hospital network PROMs and PREMs program as part of a digital patient decision aid (PDA) for patients with lymphoma. After the first hematologist appointment, participating patients access the PDA to review relevant information about clinical and patient-reported outcomes for each of the possible options, assign a personal order of priority to different outcomes, and then select their preferred course of action. Patients' answers are automatically uploaded to the EHR and discussed with hematologists at the next appointment. After beginning treatment, patients are invited to participate in the network PROMs program; participants' PROMs data are fed back into the PDA, thus "closing the circle" between the decision-making process and patient-reported data collection.During the first 14 months after launching the decision aid in October 2022, of 25 patients diagnosed with follicular lymphoma at the four participating hospitals, 13 patients decided to participate. No significant differences in age or sex were observed between groups. Average SDM Q-9 score for patients filling in the questionnaire (n = 6) was 36.15 of 45 points. Conclusion: Various obstacles toward widespread implementation of SDM exist such as time constraints, lack of motivation, and resistance to change. Support and active engagement from policy makers and healthcare managers is key to overcome hurdles for capturing patient-reported data and carrying out shared decision-making at healthcare system level. Early results of a digital framework for PRO-enriched SDM seem to be beneficial to the decision-making process.
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Toma de Decisiones Conjunta , Participación del Paciente , Medición de Resultados Informados por el Paciente , Humanos , España , Femenino , Masculino , Persona de Mediana EdadRESUMEN
BACKGROUND AND PURPOSE: We aimed to systematically review studies of crosswalks for converting patient-reported outcome measure (PROM) scores used in joint replacement, and develop a database of published crosswalks. METHODS: 4 electronic databases were searched from January 2000 to May 2023 to identify studies reporting the development and/or validation of crosswalks to convert PROM scores in patients undergoing elective hip, knee, or shoulder replacement surgery. Data on study and sample characteristics, source and target PROMs, and crosswalk development and validation methods were extracted from eligible studies. Study reporting was evaluated using the Mapping onto Preference-based measures reporting Standards (MAPS) checklist. RESULTS: 17 studies describing 35 crosswalks were eligible for inclusion. Unidirectional crosswalks were available to convert hip-specific (Oxford Hip Score [OHS]) and knee-specific (Oxford Knee Score [OKS]) scores to the EQ-5D-3L/EQ-5D-5L. Similar crosswalks to convert disease-specific scores (WOMAC) to the EQ-5D-3L, EQ-5D-5L, and ICECAP-O Capability Index were identified. Bidirectional crosswalks for converting OHS and OKS to the HOOS-JR/HOOS-12 and KOOS-JR/KOOS-12, for converting WOMAC to the HOOS-JR/KOOS-JR, and for converting HOOS-Function/KOOS-Function to the PROMIS-Physical Function were also available. Additionally, crosswalks to convert generic PROM scores from the UCLA Activity Scale to the Lower Extremity Activity Scale in both directions were available. No crosswalks were identified for converting scores in shoulder replacement. Development methods varied with the type of target score; most studies used regression, item response theory, or equipercentile equating approaches. Reporting quality was variable, particularly for methods and results items, impacting crosswalk application. CONCLUSION: This is the first synthesis of published crosswalks for converting joint-specific (OHS, OKS, HOOS, KOOS), disease-specific (WOMAC), and generic PROMs scores (PROMIS-Physical Function, UCLA Activity Scale, Lower Extremity Activity Scale) used to assess joint replacement outcomes, providing a resource for data harmonization and pooled analysis. Crosswalks were developed using regression methods (9 studies), equipercentile equating methods (5 studies), a combination of equipercentile equating and item response theory methods (2 studies), and a combination of regression and equipercentile equating methods (1 study). A range of crosswalk validation approaches were adopted, including the use of external datasets, separate samples or subsets, follow-up data from additional time points, or bootstrapped samples. Efforts are needed to standardize crosswalk methodology and achieve consistent reporting.
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Artroplastia de Reemplazo de Cadera , Artroplastia de Reemplazo de Rodilla , Artroplastía de Reemplazo de Hombro , Medición de Resultados Informados por el Paciente , HumanosRESUMEN
Cleft palate presents multifaceted challenges impacting speech, hearing, appearance, and cognition, significantly affecting patients' quality of life (QoL). While surgical advancements aim to restore function and improve appearance, traditional clinical measures often fail to comprehensively capture patients' experiences. Patient-reported outcomes measure (PROMs) have emerged as crucial tools in evaluating QoL, offering insights into various aspects such as esthetic results, speech function, and social integration. This review explores PROMs relevant to cleft palate complications, including velopharyngeal insufficiency, oronasal fistulas, maxillary hypoplasia, sleep-disordered breathing, and caregiver QoL. Additionally, the review highlights the need for cleft palate-specific scales to better address the unique challenges faced by patients. By incorporating PROMs, healthcare providers can achieve more personalized, patient-centered care, improve communication, and enhance treatment outcomes. Future research should focus on developing and validating specialized PROMs to further refine patient assessments and care strategies.
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Fisura del Paladar , Medición de Resultados Informados por el Paciente , Calidad de Vida , Humanos , Fisura del Paladar/cirugía , Insuficiencia VelofaríngeaRESUMEN
BACKGROUND: The risk of developing avascular necrosis (AVN) in the setting of an unstable slipped capital femoral epiphysis (SCFE) that is undergoing treatment with the modified Dunn procedure is not well understood. In addition, since the Loder classification of unstable is reportedly different than actual intraoperatively observed instability (that is, discontinuity between the femoral head epiphysis and proximal femoral metaphysis), the overall risk of developing AVN, as well as the potential complications of treatment of these patients with the modified Dunn procedure, are unknown. QUESTIONS/PURPOSES: To evaluate the modified Dunn procedure for the treatment of patients with epiphyseal-metaphyseal discontinuity, we asked: (1) What was the survivorship free from AVN at 10 years? (2) What was the survivorship free from subsequent surgery and/or complications at 10 years? (3) What were the clinical and patient-reported outcome scores? METHODS: In a retrospective analysis, we identified 159 patients (159 hips) treated with a modified Dunn procedure for SCFE between 1998 and 2020, of whom 97% (155 of 159) had documentation about intraoperatively observed epiphyseal-metaphyseal stability. Of those, 37% (58 of 155) of patients were documented to have intraoperatively observed epiphyseal-metaphyseal discontinuity and were considered eligible for inclusion, whereas 63% (97 of 155) had documented epiphyseal-metaphyseal stability and were excluded. No patients were lost to follow-up before the 2-year minimum. All patients were assessed for survival, but 7% (4 of 58) did not fill out our outcomes score questionnaire. This resulted in 93% (54 of 58) of patients who were available for outcome score assessment. Additionally, 50% (29 of 58) of patients had not been seen within the last 5 years; they are included, but we note that there is uncertainty about their status. The median (range) age at surgery was 13 years (10 to 16), and the sex ratio was 60% (35 of 58) male and 40% (23 of 58) female patients. Sixty-four percent (37 of 58) of patients were classified as acute-on-chronic, and 17% (10 of 58) of patients were classified as acute. Forty-seven percent (27 of 58) of patients presented with severe slips and 43% (25 of 58) of patients with moderate slips based on radiographic classification. All patients underwent surgical hip dislocation with the modified Dunn procedure to correct the slip deformity and provide stabilization. Complications and reoperations were assessed from a review of electronic medical records, and a Kaplan-Meier estimator was used to estimate survivorship free from complications and reoperations at 10 years. Clinical examination results and questionnaire responses were evaluated at minimum 2-year follow-up. RESULTS: Kaplan-Meier survivorship free from AVN was 93% (95% CI 87% to 100%) at 10 years. Survivorship free from any reoperation was 75% (95% CI 64% to 88%) at 10 years. In addition, survivorship free from complications, defined as development of AVN, reoperation, or a Sink Grade II complication or higher, was 57% (95% CI 45% to 73%) at 10 years. The median (range) Merle D'Aubigne Postel score was 18 (14 to 18) for the patients who did not develop AVN, and 12 (6 to 16) for the four patients who developed AVN (p < 0.001). The median modified Harris hip score was 100 (74 to 100) in the non-AVN cohort and 65 (37 to 82) in the AVN cohort (p = 0.001). Median HOOS total score was 95 (50 to 100) in the non-AVN cohort and 53 (40 to 82) in the AVN cohort (p = 0.002). CONCLUSION: Although the modified Dunn procedure is technically challenging, this study shows that in experienced hands, patients with who have demonstrated epiphyseal-metaphyseal discontinuity can be treated with a low risk of AVN and subsequent surgery. Referral of these patients to specialists who have substantial expertise in this procedure is recommended to improve patient outcomes. Prospective, long-term observational studies will help us identify these high-risk patients preoperatively and determine the long-term success of this procedure. LEVEL OF EVIDENCE: Level IV, therapeutic study.
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Necrosis de la Cabeza Femoral , Epífisis Desprendida de Cabeza Femoral , Humanos , Femenino , Epífisis Desprendida de Cabeza Femoral/cirugía , Epífisis Desprendida de Cabeza Femoral/diagnóstico por imagen , Epífisis Desprendida de Cabeza Femoral/fisiopatología , Masculino , Estudios Retrospectivos , Adolescente , Niño , Necrosis de la Cabeza Femoral/cirugía , Necrosis de la Cabeza Femoral/diagnóstico por imagen , Complicaciones Posoperatorias/etiología , Procedimientos Ortopédicos/efectos adversos , Procedimientos Ortopédicos/métodos , Factores de Riesgo , Resultado del Tratamiento , Medición de Resultados Informados por el Paciente , Factores de Tiempo , Articulación de la Cadera/cirugía , Articulación de la Cadera/diagnóstico por imagen , Articulación de la Cadera/fisiopatologíaRESUMEN
BACKGROUND: Computerized adaptive testing (CAT) is an effective way to reduce time, repetitious redundancy, and respond burden, and has been used to measure outcomes in many diseases. This study aimed to develop and validate a comprehensive disease-specific CAT for chronic obstructive pulmonary disease (COPD) patient-reported outcome measurement. METHODS: The discrimination and difficulty of the items from the modified patient-reported outcome scale for COPD (mCOPD-PRO) were analyzed using item response theory. Then the initial item, item selection method, ability estimation method, and stopping criteria were further set based on Concerto platform to form the CAT. Finally, the reliability and validity were validated. RESULTS: The item discrimination ranged from 1.05 to 2.71, and the item difficulty ranged from - 3.08 to 3.65. The measurement reliability of the CAT ranged from 0.910 to 0.922 using random method, while that ranged from 0.910 to 0.924 using maximum Fisher information (MFI) method. The content validity was good. The correlation coefficient between theta of the CAT and COPD assessment test and modified Medical Research Council dyspnea scale scores using random method was 0.628 and 0.540 (P < 0.001; P < 0.001) respectively, while that using MFI method was 0.347 and 0.328 (P = 0.007; P = 0.010) respectively. About 11 items (reducing by 59.3%) on average were tested using random method, while about seven items (reducing by 74.1%) on average using MFI method. The correlation coefficient between theta of the CAT and mCOPD-PRO total scores using random method was 0.919 (P < 0.001), while that using MFI method was 0.760 (P < 0.001). CONCLUSIONS: The comprehensive disease-specific CAT for COPD patient-reported outcome measurement is well developed with good psychometric properties, which can provide an efficient, accurate, and user-friendly measurement for patient-reported outcome of COPD.
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Medición de Resultados Informados por el Paciente , Psicometría , Enfermedad Pulmonar Obstructiva Crónica , Humanos , Enfermedad Pulmonar Obstructiva Crónica/psicología , Masculino , Femenino , Reproducibilidad de los Resultados , Persona de Mediana Edad , Anciano , Encuestas y Cuestionarios/normas , Calidad de VidaRESUMEN
Living with chronic pain is associated with substantial suffering and high societal costs. Patient reported outcomes (PROM's) and cellular ageing should be considered in pain management. The aim of this study was to explore correlations of PROM's and cellular ageing (telomere length [TL] and telomerase activity [TA]) amongst patients with chronic non-malignant pain. This was an explorative pilot study with cross-sectional design and recruitment was done at two pain rehabilitation facilities in Sweden, with inpatient setting/integrative care and outpatient setting/multimodal care, respectively. Eighty-four patients were enrolled by referral to pain rehabilitation in Sweden. The main outcome measures collected after admission in addition to TL and TA were the following PROMs: Numerical Rating Scale (NRS), Chronic Pain Acceptance Questionnaire (CPAQ), Hospital Anxiety and Depression Scale (HADS), Five Facets Mindfulness Questionnaire (FFMQ), WHO Quality of Life-Spiritual, Religious and Personal Beliefs (WHOQoL-SRPB) and EuroQol 5 Dimensions (EQ-5D). All the PROM's showed evidence of poor overall health status among the participants. TL correlated negatively with HADS score (r = -.219, p = .047) and positively with WHOQoL-SRPB (r = .224, p = .052). TL did not correlate with any of the pain measures. TA correlated positively with pain spread (r = .222, p = .049). A mediation of the direct effect of spiritual well-being on TL by anxiety and depression could be shown (b = 0.008; p = .045). The correlations between TL and SRPB and anxiety and depression suggest some importance of emotional and SRPB dimensions in pain management, with implications for cellular aging, which may warrant further study. Trial registration: ClinicalTrials.gov Identifier: NCT02459639.
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Dolor Crónico , Espiritualidad , Telómero , Adulto , Anciano , Femenino , Humanos , Masculino , Persona de Mediana Edad , Dolor Crónico/psicología , Estudios Transversales , Depresión/psicología , Emociones , Medición de Resultados Informados por el Paciente , Proyectos Piloto , Calidad de Vida , Religión , Encuestas y Cuestionarios , Suecia , Telomerasa/metabolismo , Telomerasa/genética , Telómero/genéticaRESUMEN
Importance: Robotic exoskeletons leverage technology that assists people with spinal cord injury (SCI) to walk. The efficacy of home and community exoskeletal use has not been studied in a randomized clinical trial (RCT). Objective: To examine whether use of a wheelchair plus an exoskeleton compared with use of only a wheelchair led to clinically meaningful net improvements in patient-reported outcomes for mental and physical health. Design, Setting, and Participants: This RCT of veterans with SCI was conducted at 15 Veterans Affairs medical centers in the US from September 6, 2016, to September 27, 2021. Data analysis was performed from March 10, 2022, to June 20, 2024. Interventions: Participants were randomized (1:1) to standard of care (SOC) wheelchair use or SOC plus at-will use of a US Food and Drug Administration (FDA)-cleared exoskeletal-assisted walking (EAW) device for 4 months in the home and community. Main Outcomes and Measures: Two primary outcomes were studied: 4.0-point or greater improvement in the mental component summary score on the Veterans RAND 36-Item Health Survey (MCS/VR-36) and 10% improvement in the total T score of the Spinal Cord Injury-Quality of Life (SCI-QOL) physical and medical health domain and reported as the proportion who achieved clinically meaningful changes. The primary outcomes were measured at baseline, post randomization after advanced EAW training sessions, and at 2 months and 4 months (primary end point) in the intervention period. Device usage, reasons for not using, and adverse events were collected. Results: A total of 161 veterans with SCI were randomized to the EAW (n = 78) or SOC (n = 83) group; 151 (94%) were male, the median age was 47 (IQR, 35-56) years, and median time since SCI was 7.3 (IQR, 0.5 to 46.5) years. The difference in proportion of successes between the EAW and SOC groups on the MCS/VR-36 (12 of 78 [15.4%] vs 14 of 83 [16.9%]; relative risk, 0.91; 95% CI, 0.45-1.85) and SCI-QOL physical and medical health domain (10 of 78 [12.8%] vs 11 of 83 [13.3%]; relative risk, 0.97; 95% CI, 0.44-2.15) was not statistically different. Device use was lower than expected (mean [SD] distance, 1.53 [0.02] miles per month), primarily due to the FDA-mandated companion being unavailable 43.9% of the time (177 of 403 instances). Two EAW-related foot fractures and 9 unrelated fractures (mostly during wheelchair transfers) were reported. Conclusions and Relevance: In this RCT of veterans with SCI, the lack of improved outcomes with EAW device use may have been related to the relatively low device usage. Solutions for companion requirements and user-friendly technological adaptations should be considered for improved personal use of these devices. Trial Registration: ClinicalTrials.gov Identifier: NCT02658656.
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Dispositivo Exoesqueleto , Traumatismos de la Médula Espinal , Veteranos , Caminata , Humanos , Masculino , Persona de Mediana Edad , Femenino , Veteranos/psicología , Traumatismos de la Médula Espinal/psicología , Traumatismos de la Médula Espinal/rehabilitación , Adulto , Medición de Resultados Informados por el Paciente , Parálisis/rehabilitación , Parálisis/psicología , Estados Unidos , Calidad de Vida/psicologíaRESUMEN
INTRODUCTION: Posterior native hip dislocations (NHD) are high-energy injuries. Thompson-Epstein Type I dislocations describe those without significant associated femoral or acetabular fracture. The aim of this study was to compare the clinical and radiological outcomes of patients with Type I NHDs. We also evaluate the association between radiological indicators of femoroacetabular impingement (FAI) and NHD. PATIENTS AND METHODS: A retrospective study from January 2012 to May 2021 compared skeletally mature patients (⩾16 years) with Type I posterior NHD to age and gender-matched controls with Type II-V posterior NHD. Patient demographics, mechanism of injury, complications and patient-reported outcome measures (PROMs) are presented. Post reduction radiographs and computed tomography were used to assess for FAI. Univariate analyses were performed to evaluate radiological outcomes. RESULTS: 13 patients (77% male) with Type I posterior NHD were compared to a control group of 40 patients (80% male) with Type II-V posterior NHD. 11 patients in the study group and 14 in the control group experienced isolated injuries (p = 0.01). Post-reduction complications were similar. The study group had significantly lower post-injury osteoarthritis incidence (n = 0) compared to controls (n = 18, p = 0.0083). Patients reported a mean Oxford Hip Score of 43.5 ± 2.2 and EQ-5D-VAS score of 87.1 ± 7.4, with 6 patients indicating minimal symptoms across all EQ-5D-5L domains. Radiological femoroacetabular impingement (FAI) was prevalent in both groups, especially among males. CONCLUSIONS: Patients who underwent emergent closed reduction of Type I NHD demonstrated good short to medium term outcomes. Our radiological findings suggest a high prevalence of FAI. Future work should aim to quantify longer term outcomes following this injury. We call for further comparative studies of patients who suffer NHD with and without fractures to aid our understanding of risk factors. Given the rarity of this injury, multicentre efforts will be required to capture large numbers of patients.
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Pinzamiento Femoroacetabular , Luxación de la Cadera , Centros Traumatológicos , Humanos , Masculino , Femenino , Estudios Retrospectivos , Adulto , Reino Unido/epidemiología , Luxación de la Cadera/diagnóstico por imagen , Luxación de la Cadera/epidemiología , Persona de Mediana Edad , Pinzamiento Femoroacetabular/diagnóstico por imagen , Pinzamiento Femoroacetabular/epidemiología , Adulto Joven , Medición de Resultados Informados por el Paciente , Acetábulo/lesiones , Acetábulo/diagnóstico por imagen , Tomografía Computarizada por Rayos X/métodos , Fracturas del Fémur/diagnóstico por imagen , Fracturas del Fémur/epidemiología , Anciano , AdolescenteRESUMEN
Lebrikizumab has previously demonstrated efficacy in Phase 3 trials: ADvocate1 and ADvocate2 (as monotherapy), ADhere, and ADhere-J (in combination with topical corticosteroids). Here, the impact of lebrikizumab combined with low- to mid-potency topical corticosteroids on patient-reported outcomes at 16 weeks in Japanese patients with moderate-to-severe atopic dermatitis is evaluated. Eligible patients (n = 286) were randomized 2:2:3 to receive placebo+ topical corticosteroids, 250 mg lebrikizumab every 4 weeks (LEBQ4W+topical corticosteroids, 500 mg loading dose at baseline), or 250 mg lebrikizumab every 2 weeks (LEBQ2W+ topical corticosteroids, 500 mg loading dose at baseline and Week 2) by subcutaneous injection. All PRO endpoints for the study were met; patients in the lebrikizumab in combination with topical corticosteroids groups demonstrated statistically significant and clinically meaningful improvements compared with placebo in combination with topical corticosteroids in Skin Pain NRS, DLQI, POEM, WPAI-AD, and SCORAD scales. Lebrikizumab combined with topical corticosteroids compared with placebo+topical corticosteroids improved patient-reported outcomes in Japanese patients with moderate-to-severe atopic dermatitis.
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Dermatitis Atópica , Quimioterapia Combinada , Medición de Resultados Informados por el Paciente , Índice de Severidad de la Enfermedad , Humanos , Dermatitis Atópica/tratamiento farmacológico , Dermatitis Atópica/diagnóstico , Masculino , Adulto , Femenino , Japón , Resultado del Tratamiento , Método Doble Ciego , Persona de Mediana Edad , Administración Cutánea , Factores de Tiempo , Corticoesteroides/administración & dosificación , Inyecciones Subcutáneas , Adulto Joven , Anticuerpos Monoclonales/administración & dosificación , Anticuerpos Monoclonales/uso terapéutico , Fármacos Dermatológicos/administración & dosificación , Fármacos Dermatológicos/efectos adversos , Pueblos del Este de AsiaRESUMEN
BACKGROUND: Cost is a key outcome in quality and value, but it is often difficult to estimate reliably and efficiently for use in real-time improvement efforts. We describe a method using patient-reported outcomes (PROs), Markov modeling, and statistical process control (SPC) analytics in a real-time cost-estimation prototype designed to assess cost differences between usual care and improvement conditions in a national multicenter improvement collaborative-the IBD Qorus Learning Health System (LHS). METHODS: The IBD Qorus Learning Health System (LHS) collects PRO data, including emergency department utilization and hospitalizations from patients prior to their clinical visits. This data is aggregated monthly at center and collaborative levels, visualized using Statistical Process Control (SPC) analytics, and used to inform improvement efforts. A Markov model was developed by Almario et al to estimate annualized per patient cost differences between usual care (baseline) and improvement (intervention) time periods and then replicated at monthly intervals. We then applied moving average SPC analyses to visualize monthly iterative cost estimations and assess the variation and statistical reliability of these estimates over time. RESULTS: We have developed a real-time Markov-informed SPC visualization prototype which uses PRO data to analyze and monitor monthly annualized per patient cost savings estimations over time for the IBD Qorus LHS. Validation of this prototype using claims data is currently underway. CONCLUSION: This new approach using PRO data and hybrid Markov-SPC analysis can analyze and visualize near real-time estimates of cost differences over time. Pending successful validation against a claims data standard, this approach could more comprehensively inform improvement, advocacy, and strategic planning efforts.
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Enfermedades Inflamatorias del Intestino , Cadenas de Markov , Medición de Resultados Informados por el Paciente , Humanos , Enfermedades Inflamatorias del Intestino/terapia , Enfermedades Inflamatorias del Intestino/economía , Atención Ambulatoria/economía , Atención Ambulatoria/estadística & datos numéricos , Servicio de Urgencia en Hospital/estadística & datos numéricos , Servicio de Urgencia en Hospital/economía , Hospitalización/economía , Hospitalización/estadística & datos numéricos , Costos de la Atención en Salud/estadística & datos numéricosRESUMEN
This study examines the origins and utilization trends of top quality-of-life (QoL) measures in acne research. A literature search on PubMed identified the Dermatology Life Quality Index (DLQI), Cardiff Acne Disability Index (CADI), and Acne Quality-of-Life Questionnaire (Acne-QoL) as the most frequently used QoL measures in studies on Acne Vulgaris. The DLQI was implemented in 142 studies it since its inception, compared to 43 utilizing CADI and 21 utilizing Acne-QoL. Despite it not being acne-specific, DLQI's usage surpassed other measures by over 50% annually since 2006. While DLQI displayed the steepest rise in utilization, usage of all measures increased significantly from 2010 to 2020. This trend underscores the growing emphasis on patient-centered outcomes in acne research, highlighting the need to incorporate both patient-reported and objective outcomes to better capture disease severity and its impact on patients' lives. For dermatologists, QoL indices can expand disease severity beyond purely objective clinical measurements.
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Acné Vulgar , Atención Dirigida al Paciente , Calidad de Vida , Índice de Severidad de la Enfermedad , Acné Vulgar/psicología , Acné Vulgar/terapia , Acné Vulgar/diagnóstico , Humanos , Encuestas y Cuestionarios/estadística & datos numéricos , Medición de Resultados Informados por el PacienteRESUMEN
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Oncología Médica , Neoplasias , Medición de Resultados Informados por el Paciente , Humanos , Neoplasias/terapia , Calidad de VidaRESUMEN
Importance: Multicomponent electronic patient-reported outcome cancer symptom management systems reduce symptom burden. Whether all components contribute to symptom reduction is unknown. Objective: To deconstruct intervention components of the Symptom Care at Home (SCH) system, a digital symptom monitoring and management intervention that has demonstrated efficacy, to determine which component or combination of components results in the lowest symptom burden. Design, Setting, and Participants: This randomized clinical trial included participants who were older than 18 years, had been diagnosed with cancer, had a life expectancy of 3 months or greater, were beginning a chemotherapy course planned for at least 3 cycles, spoke English, and had daily access and ability to use a telephone. Eligible participants were identified from the Huntsman Cancer Institute, University of Utah (Salt Lake City), and from Emory University Winship Cancer Institute, including Grady Memorial Hospital (Atlanta, Georgia), from August 7, 2017, to January 17, 2020. Patients receiving concurrent radiation therapy were excluded. Dates of analysis were from February 1, 2020, to December 22, 2023. Interventions: Participants reported symptoms daily during a course of chemotherapy and received automated self-management coaching with an activity tracker without (group 1) and with (group 2) visualization, nurse practitioner (NP) follow-up for moderate-to-severe symptoms without (group 3) and with (group 4) decision support, or the complete SCH intervention (group 5). Main Outcomes and Measures: The primary outcome, symptom burden, was assessed as the summed severity of 11 chemotherapy-related symptoms rated on a scale of 1 to 10 (with higher scores indicating greater severity), if present. Results: The 757 participants (mean [SD] age, 59.2 [12.9] years) from 2 cancer centers were primarily female (61.2%). The most common cancer diagnoses were breast (132 [17.4%]), lung (107 [14.1%]), and colorectal (99 [13.1%]) cancers; 369 patients (48.7%) had metastatic disease. The complete SCH intervention including automated self-management coaching and NP follow-up with decision support (group 5) was superior in reducing symptom burden to either of the self-management coaching groups, as shown by the mean group differences in area under the curve (group 1, 1.86 [95% CI, 1.30-2.41] and group 2, 2.38 [95% CI, 1.84-2.92]; both P < .001), and to either of the NP follow-up groups (group 3, 0.57 [95% CI, 0.03-1.11]; P =.04; and group 4, 0.66 [95% CI, 0.14-1.19]; P = .014). Additionally, NP follow-up was superior to self-management coaching (group 1 vs group 3, 1.29 [95% CI, 0.72-1.86]; group 1 vs group 4, 1.20 [95% 12 CI, 0.64-1.76]; group 2 vs group 3, 1.81 [95% CI, 1.25-2.37]; and group 2 vs group 4, 1.72 [95% CI, 1.17-2.26]; all P < .001), but there was no difference between the 2 self-management coaching groups (-0.52 [95% CI, -1.09 to 0.05]; P = .07) or between the 2 NP groups (-0.10 [95% CI, -0.65 to 0.46]; P = .74). Conclusions and Relevance: In this randomized clinical trial of adult participants undergoing chemotherapy treatment for cancer, the complete intervention, rather than any individual component of the SCH system, achieved the greatest symptom burden reduction. These findings suggest that a multicomponent digital approach to cancer symptom management may offer optimal symptom burden reduction. Trial Registration: ClinicalTrials.gov Identifier: NCT02779725.