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BACKGROUND & AIMS: Tumor necrosis factor inhibitors, including infliximab and adalimumab, are a mainstay of pediatric Crohn's disease therapy; however, nonresponse and loss of response are common. As combination therapy with methotrexate may improve response, we performed a multicenter, randomized, double-blind, placebo-controlled pragmatic trial to compare tumor necrosis factor inhibitors with oral methotrexate to tumor necrosis factor inhibitor monotherapy. METHODS: Patients with pediatric Crohn's disease initiating infliximab or adalimumab were randomized in 1:1 allocation to methotrexate or placebo and followed for 12-36 months. The primary outcome was a composite indicator of treatment failure. Secondary outcomes included anti-drug antibodies and patient-reported outcomes of pain interference and fatigue. Adverse events (AEs) and serious AEs (SAEs) were collected. RESULTS: Of 297 participants (mean age, 13.9 years, 35% were female), 156 were assigned to methotrexate (110 infliximab initiators and 46 adalimumab initiators) and 141 to placebo (102 infliximab initiators and 39 adalimumab initiators). In the overall population, time to treatment failure did not differ by study arm (hazard ratio, 0.69; 95% CI, 0.45-1.05). Among infliximab initiators, there were no differences between combination and monotherapy (hazard ratio, 0.93; 95% CI, 0.55-1.56). Among adalimumab initiators, combination therapy was associated with longer time to treatment failure (hazard ratio, 0.40; 95% CI, 0.19-0.81). A trend toward lower anti-drug antibody development in the combination therapy arm was not significant (infliximab: odds ratio, 0.72; 95% CI, 0.49-1.07; adalimumab: odds ratio, 0.71; 95% CI, 0.24-2.07). No differences in patient-reported outcomes were observed. Combination therapy resulted in more AEs but fewer SAEs. CONCLUSIONS: Among adalimumab but not infliximab initiators, patients with pediatric Crohn's disease treated with methotrexate combination therapy experienced a 2-fold reduction in treatment failure with a tolerable safety profile. CLINICALTRIALS: gov, Number: NCT02772965.
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Metotrexato , Inhibidores del Factor de Necrosis Tumoral , Niño , Humanos , Femenino , Adolescente , Masculino , Metotrexato/efectos adversos , Adalimumab/efectos adversos , Anticuerpos Monoclonales/efectos adversos , Infliximab/efectos adversos , Factor de Necrosis Tumoral alfa , Resultado del TratamientoRESUMEN
INTRODUCTION: Evidence about specific carbohydrate diet (SCD) for inflammatory bowel disease (IBD) is limited. We conducted 54 single-subject, double-crossover N-of-1 trials comparing SCD with a modified SCD (MSCD) and comparing each with the participant's baseline, usual diet (UD). METHODS: Across 19 sites, we recruited patients aged 7-18 years with IBD and active inflammation. Following a 2-week baseline (UD), patients were randomized to 1 of 2 sequences of 4 alternating 8-week SCD and MSCD periods. Outcomes included fecal calprotectin and patient-reported symptoms. We report posterior probabilities from Bayesian models comparing diets. RESULTS: Twenty-one (39%) participants completed the trial, 9 (17%) completed a single crossover, and 24 (44%) withdrew. Withdrawal or early completion occurred commonly (lack of response [n = 11], adverse events [n = 11], and not desiring to continue [n = 6]). SCD and MSCD performed similarly for most individuals. On average, there was <1% probability of a clinically meaningful difference in IBD symptoms between SCD and MSCD. The average treatment difference was -0.3 (95% credible interval -1.2, 0.75). There was no significant difference in the ratio of fecal calprotectin geometric means comparing SCD and MSCD (0.77, 95% credible interval 0.51, 1.10). Some individuals had improvement in symptoms and fecal calprotectin compared with their UD, whereas others did not. DISCUSSION: SCD and MSCD did not consistently improve symptoms or inflammation, although some individuals may have benefited. However, there are inherent difficulties in examining dietary changes that complicate study design and ultimately conclusions regarding effectiveness.
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Colitis Ulcerosa , Enfermedad de Crohn , Complejo de Antígeno L1 de Leucocito , Adolescente , Teorema de Bayes , Niño , Colitis Ulcerosa/complicaciones , Colitis Ulcerosa/dietoterapia , Enfermedad de Crohn/complicaciones , Enfermedad de Crohn/dietoterapia , Dieta , Heces/química , Humanos , Inflamación/complicaciones , Inflamación/dietoterapia , Enfermedades Inflamatorias del Intestino/complicaciones , Enfermedades Inflamatorias del Intestino/dietoterapia , Complejo de Antígeno L1 de Leucocito/análisis , Medicina de PrecisiónRESUMEN
OBJECTIVE: Using a nested, cluster-randomized trial, we tested the hypothesis that a shared decision-making intervention, as part of consent, would improve study-related knowledge. METHODS: We developed a shared decision-makingintervention then randomized sites in a clinical trial to intervention or control (standard consent). We collected participants' knowledge (primary outcome) and decisional support data. Other data came from a clinical registry and research coordinator surveys. We compared outcomes between study arms using generalized estimating equation models, accounting for clustering. We used qualitative description to understand variation in intervention use. RESULTS: 265 individuals, from 34 sites, enrolled in the parent trial during our study period. Of those, 241 participants completed our survey. There was no knowledge difference between arms (mean difference = 0.56 (95 %CI: -3.8, 4.9)). Both groups had a considerable number of participants with misunderstandings. We also found no difference for decisional support (mean difference = 1.5 (95 %CI: -1.8, 4.8)) or enrollment rate between arms. Clinician use of the intervention varied between sites. CONCLUSIONS: We found no differences in outcomes but demonstrated the feasibility and acceptability of incorporating a shared decision-making intervention into consent. PRACTICE IMPLICATIONS: Future work should consider adapting our intervention to other trials and more robust measurement strategies.
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Toma de Decisiones Conjunta , Toma de Decisiones , Humanos , Consentimiento Informado , Conocimiento , Encuestas y CuestionariosRESUMEN
Measures of health care quality are produced from a variety of data sources, but often, physicians do not believe these measures reflect the quality of provided care. The aim was to assess the value to health system leaders (HSLs) and parents of benchmarking on health care quality measures using data mined from the electronic health record (EHR). Using in-context interviews with HSLs and parents, the authors investigated what new decisions and actions benchmarking using data mined from the EHR may enable and how benchmarking information should be presented to be most informative. Results demonstrate that although parents may have little experience using data on health care quality for decision making, they affirmed its potential value. HSLs expressed the need for high-confidence, validated metrics. They also perceived barriers to achieving meaningful metrics but recognized that mining data directly from the EHR could overcome those barriers. Parents and HSLs need high-confidence health care quality data to support decision making.
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Registros Electrónicos de Salud , Administradores de Instituciones de Salud , Padres , Pediatría , Indicadores de Calidad de la Atención de Salud , Femenino , Humanos , Entrevistas como Asunto , Masculino , Investigación Cualitativa , Calidad de la Atención de SaludRESUMEN
BACKGROUND: Shared decision making (SDM) between families and physicians may facilitate informed, timely decisions to proceed with biologic therapy in children with inflammatory bowel disease (IBD). Our team previously developed an SDM tool to aid communication between physicians and families when considering biologic therapy for children with IBD. OBJECTIVE: We are conducting a prospective, pre-post pilot trial of a new SDM tool. The primary aim of the study is to assess feasibility of both the intervention and trial procedures for a future large-scale trial. METHODS: We are enrolling physicians with experience prescribing biologic therapy in the past year and families of children with IBD. Families in the intervention arm receive a 3-step intervention including a letter sent before trial consent or clinic appointment, an in-clinic decision tool and a follow-up phone call. Our primary trial outcome is a measure of feasibility, with measures of clinical and decision outcomes secondary. We seek to enroll 27 families in each of 2 arms (usual-care and intervention) and plan data collection at the time of the initial visit or hospital stay, and at 1 week, 3 months, and 6 months after the initial visit. CONCLUSION: This study protocol is designed to demonstrate that integrating novel consent procedures, including timing and multiple versions of written consent, may increase trial feasibility while maintaining scientific rigor and full protection of study participants.
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To understand how parents and physicians make decisions regarding antibiotics and whether a potential associated risk of obesity would alter decisions, we conducted a qualitative study of parents and physicians who care for children. Parent focus groups and physician interviews used a guide focused on experience with antibiotics and perceptions of risks and benefits, including obesity. Content analysis was used to understand how a risk of obesity would influence antibiotic decisions. Most parents (n = 59) and physicians (n = 22) reported limited discussion about any risks at the time of antibiotic prescriptions. With an acute illness, most parents prioritized symptomatic improvement and chose to start antibiotics. Physicians' treatment preferences were varied. An obesity risk did not change most parents' or physicians' preferences. Given that parent-physician discussion at the time of acute illness is unlikely to change preferences, public health messaging may be a more successful approach to counter obesity and antibiotics overuse.
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Antibacterianos , Actitud del Personal de Salud , Conocimientos, Actitudes y Práctica en Salud , Padres/psicología , Obesidad Infantil/psicología , Médicos/psicología , Adolescente , Adulto , Toma de Decisiones Clínicas , Femenino , Grupos Focales , Humanos , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Médicos/estadística & datos numéricos , Pautas de la Práctica en Medicina , Riesgo , Adulto JovenAsunto(s)
Corticoesteroides/uso terapéutico , Antiasmáticos/uso terapéutico , Asma/tratamiento farmacológico , Sistemas Recordatorios/instrumentación , Telemedicina/organización & administración , Envío de Mensajes de Texto , Adolescente , Asma/economía , Asma/etnología , Asma/psicología , Femenino , Humanos , Masculino , Programas Controlados de Atención en Salud/ética , Salud de las Minorías/economía , Monitoreo Fisiológico/instrumentación , Monitoreo Fisiológico/métodos , Proyectos Piloto , Pobreza/psicología , Sistemas Recordatorios/economía , RiesgoRESUMEN
We enrolled 64 patients age 12 to 22 years with a diagnosis of poorly controlled persistent asthma in a 6-month longitudinal crossover study. During the 3 intervention months, participants created personalized text messages to be sent to their phones. Adherence was objectively monitored in 22 of the participants. The adolescent participants gave high ratings on the acceptability of the text messaging system. Asthma control improved from baseline to month 1 regardless of whether teens were in the texting or control group. While participants were in the texting group, their quality of life improved and worry about their asthma decreased. Receiving the text intervention resulted in an increase in adherence of 2.75% each month relative to no intervention, but the improvements were not sustained. There was modest improvement in asthma control and quality of life outcomes, as well as improved adherence during the texting intervention.
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Antiasmáticos/uso terapéutico , Asma/tratamiento farmacológico , Cumplimiento de la Medicación/estadística & datos numéricos , Sistemas Recordatorios/estadística & datos numéricos , Envío de Mensajes de Texto/estadística & datos numéricos , Adolescente , Conducta del Adolescente/psicología , Adulto , Niño , Estudios Cruzados , Femenino , Humanos , Estudios Longitudinales , Masculino , Satisfacción del Paciente/estadística & datos numéricos , Proyectos Piloto , Calidad de Vida , Adulto JovenRESUMEN
This study assessed pediatric physicians' use of shared decision making (SDM) in 2 chronic conditions. Most physicians indicated that parent and adolescent trust and emotional readiness facilitated SDM, physicians' preferred approach to decision making. At the same time, they perceived few barriers, other than insurance limitations, to using SDM.
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Artritis Juvenil/tratamiento farmacológico , Enfermedad Crónica/terapia , Toma de Decisiones , Enfermedades Inflamatorias del Intestino/tratamiento farmacológico , Médicos , Adolescente , Adulto , Artritis Juvenil/diagnóstico , Actitud del Personal de Salud , Niño , Enfermedad Crónica/economía , Femenino , Gastroenterología , Humanos , Enfermedades Inflamatorias del Intestino/diagnóstico , Seguro de Salud , Masculino , Persona de Mediana Edad , Padres , Participación del Paciente , Pediatría/métodos , Relaciones Médico-Paciente , Reumatología , Factor de Necrosis Tumoral alfa/antagonistas & inhibidores , Recursos HumanosRESUMEN
OBJECTIVE: To compare factors considered by parents to those considered by adolescents making decisions about chronic disease treatments. METHODS: We conducted individual interviews with 15 parent-adolescent dyads in which the adolescent had either juvenile idiopathic arthritis or Crohn's disease. Questions focused on treatment decisions, with an emphasis on the factors that influenced each individual's preferences related to biologic therapies. A multidisciplinary team developed a coding structure. All interviews were coded by two people with disagreements resolved through discussion. We used content analysis and coding matrices to examine decision factors within and between parent-adolescent dyads. RESULTS: Parents and adolescents both participated in decisions about treatment with biologic therapies but considered decision factors differently. In only half of cases did parents and adolescents agree on the factor that most influenced their decision. Although their decision factors often fell into similar categories (e.g. treatment risks, quality of life), in many cases the specifics varied between adolescents and their parents. Adolescents were more likely to focus on immediate treatment effects and quality of life while parents took a longer term view of the decision. Agreement within dyads was most consistent when a special circumstance influenced the treatment decision. CONCLUSIONS: Differences regarding influential decision factors exist within parent-adolescent dyads. Continued research is needed to determine the extent to which such differences are due to individual preferences or to variations in the information available to each person. Future decision support interventions will need to address parents' and adolescents' potentially disparate views and information needs.
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Conducta del Adolescente/psicología , Artritis Juvenil/psicología , Enfermedad de Crohn/psicología , Toma de Decisiones , Padres/psicología , Centros Médicos Académicos , Adolescente , Salud del Adolescente , Adulto , Artritis Juvenil/terapia , Niño , Enfermedad Crónica , Enfermedad de Crohn/terapia , Femenino , Hospitales Pediátricos , Humanos , Entrevistas como Asunto , Masculino , Persona de Mediana Edad , Ohio , Relaciones Padres-Hijo , Calidad de Vida , RiesgoRESUMEN
OBJECTIVE: To use observation to understand how decisions about higher-risk treatments, such as biologics, are made in pediatric chronic conditions. METHODS: Gastroenterology and rheumatology providers who prescribe biologics were recruited. Families were recruited when they had an outpatient appointment in which treatment with biologics was likely to be discussed. Consent/assent was obtained to video the visit. Audio of the visits in which a discussion of biologics took place were transcribed and analyzed. Our coding structure was based on prior research, shared decision making (SDM) concepts, and the initial recorded visits. Coded data were analyzed using content analysis and comparison with an existing model of SDM. RESULTS: We recorded 21 visits that included discussions of biologics. In most visits, providers initiated the decision-making discussion. Detailed information was typically given about the provider's preferred option with less information about other options. There was minimal elicitation of preferences, treatment goals, or prior knowledge. Few parents or patients spontaneously stated their preferences or concerns. An implicit or explicit treatment recommendation was given in nearly all visits, although rarely requested. In approximately one-third of the visits, the treatment decision was never made explicit, yet steps were taken to implement the provider's preferred treatment. CONCLUSIONS: We observed limited use of SDM, despite previous research indicating that parents wish to collaborate in decision making. To better achieve SDM in chronic conditions, providers and families need to strive for bidirectional sharing of information and an explicit family role in decision making.
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Toma de Decisiones , Participación del Paciente , Relaciones Médico-Paciente , Adolescente , Adulto , Atención Ambulatoria , Niño , Preescolar , Enfermedad Crónica , Femenino , Gastroenterología , Humanos , Lactante , Masculino , Atención Dirigida al Paciente , Médicos , Reumatología , Adulto JovenRESUMEN
PURPOSE: To understand adolescents' roles and preferences in chronic disease treatment decisions, using biologic therapy decisions as an example. METHODS: We conducted semistructured interviews with adolescents who have Crohn's disease (n = 7) or juvenile idiopathic arthritis (n = 8). Questions focused on biologic therapy decisions, including who participated, preferred roles, and factors considered in the decision. We used pie charts to help teens describe the division of decisional responsibility. We open-coded the initial interviews to develop a coding structure. Interviews then were coded by two people, with disagreements resolved through discussion. Interviews were analyzed using content analysis. RESULTS: Nearly all adolescents participated in the decision about biologic therapy. Roles varied from telling parents about specific symptoms to having the final say in the decision. In addition to discussing their own roles, adolescents identified specific decision-making roles for both parents and physicians. Most factors that influenced adolescents' decision-making related to their quality of life, such as the desire to feel better, concerns about painful injections, and the time needed for treatment. Some adolescents did consider potential side effects and the expected treatment efficacy. Most adolescents were satisfied with how the decision was made, but given a choice would have preferred a different role in the decision. CONCLUSION: Adolescents with chronic disease wish to be involved in treatment decision making, but also seek the involvement of parents and physicians. Parents and providers can assist by providing adolescents with relevant information and by helping teens develop the skills necessary for future medical decision-making.
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Artritis Juvenil/terapia , Enfermedad de Crohn/terapia , Toma de Decisiones , Participación del Paciente , Adolescente , Niño , Femenino , Humanos , Entrevistas como Asunto , MasculinoRESUMEN
OBJECTIVE: The aim of the study was to describe parents' experiences and the information used when making decisions about tumor necrosis factor-α inhibitor (TNFαi) treatment. METHODS: We interviewed parents of children with Crohn disease (CD) or juvenile idiopathic arthritis who had experience deciding about TNFαi treatment. Interview questions focused on information used to make decisions and factors that influenced decision making. We used thematic analysis for all coding and analysis. Coding structure was developed by a multidisciplinary team review of the initial interviews. Two coders then coded the remaining interviews, compared coding, and resolved disagreements through discussion. Data were analyzed by thematic grouping and then compared between diseases. RESULTS: We interviewed 35 parents. For nearly all parents the decision about TNFαi treatment was the most challenging medical decision they had made; however, parents of children with CD experienced more stress and anxiety than did other parents. Both groups of parents sought information from multiple sources including health care providers, the Internet, and social contacts. They looked for information related to treatment effectiveness, adverse effects, and other individuals' treatment experiences. In CD, information was used to help make the decision, whereas in juvenile idiopathic arthritis it was used to confirm the decision. CONCLUSIONS: The decision-making experience, and associated information seeking, leaves some parents with long-lasting concerns and worry about TNFαi treatment. Providing parents with structured decision-making support may lead to more effective and efficient decision making, decreased psychosocial distress, and, ultimately, improved outcomes for their children.
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Artritis Juvenil/tratamiento farmacológico , Enfermedad de Crohn/tratamiento farmacológico , Toma de Decisiones , Padres/educación , Educación del Paciente como Asunto , Participación del Paciente , Factor de Necrosis Tumoral alfa/antagonistas & inhibidores , Adolescente , Antiinflamatorios no Esteroideos/efectos adversos , Antiinflamatorios no Esteroideos/uso terapéutico , Antirreumáticos/efectos adversos , Antirreumáticos/uso terapéutico , Niño , Preescolar , Femenino , Hospitales Pediátricos , Humanos , Inmunosupresores/efectos adversos , Inmunosupresores/uso terapéutico , Conducta en la Búsqueda de Información , Masculino , Evaluación de Necesidades , OhioRESUMEN
This study examined the relation between clinically significant (CS) change in symptoms of attention-deficit/hyperactivity disorder (ADHD) and of oppositional defiant disorder (ODD), and reliable change in multiple domains of functioning in children who participated in the Multimodal Treatment Study of Children with ADHD. Children with CS change in symptoms were significantly more likely than children without CS change to have reliable change across five domains of functioning. Interestingly, however, depending on the measure of functioning, 14 to 52% of children who did not achieve CS change in symptoms showed reliable improvement in functional domains. The results have implications for the definition and measurement of CS change in child treatment-outcome studies.