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OBJECTIVE: To evaluate the impact of LEAP, a volunteer-based, inpatient asthma education program for families of inner-city children with asthma. METHODS: 711 children ages 2-17 years admitted with status asthmaticus were randomized to receive usual care or usual care plus a supplemental education intervention. Both groups completed a baseline interview. Trained volunteer lay educators conducted individualized bedside education with the intervention group. Primary outcome was attendance at a post-hospitalization follow-up visit 7-10 days after discharge. Secondary outcomes included parent-reported asthma management behaviors, symptoms, and self-efficacy scores from a one month follow-up interview. RESULTS: Post-hospitalization asthma clinic attendance was poor (38%), with no difference between groups. Families randomized to the intervention group were more likely to report use of a controller (OR 2.4, 95% CI 1.3-4.2, p<0.01) and a valved-holding chamber (OR 2.9, 95% CI 1.1-7.4, p=0.03), and were more likely to have an asthma action plan at follow up (OR 2.0, 95% CI 1.3-3.0, p<0.01). Asthma self-efficacy scores were significantly improved among those who received the intervention (p=0.04). CONCLUSIONS: Inpatient asthma education by trained lay volunteers was associated with improved asthma management behaviors. PRACTICE IMPLICATIONS: This novel volunteer-based program could have widespread implications as a sustainable model for asthma education.
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RATIONALE: Asthma clinical guidelines suggest written asthma action plans are essential for improving self-management and outcomes. OBJECTIVES: To assess the efficacy of written instructions in the form of a written asthma action plan provided by subspecialist physicians as part of usual asthma care during office visits. METHODS: A total of 407 children and adults with persistent asthma receiving first-time care in pulmonary and allergy practices at 4 urban medical centers were randomized to receive either written instructions (n = 204) or no written instructions other than prescriptions (n = 203) from physicians. MEASUREMENTS AND MAIN RESULTS: Using written asthma action plan forms as a vehicle for providing self-management instructions did not have a significant effect on any of the primary outcomes: (1) asthma symptom frequency, (2) emergency visits, or (3) asthma quality of life from baseline to 12-month follow-up. Both groups showed similar and significant reductions in asthma symptom frequency (daytime symptoms [P < 0.0001], nocturnal symptoms [P < 0.0001], ß-agonist use [P < 0.0001]). There was also a significant reduction in emergency visits for the intervention (P < 0.0001) and control (P < 0.0006) groups. There was significant improvement in asthma quality-of-life scores for adults (P < 0.0001) and pediatric caregivers (P < 0.0001). CONCLUSIONS: Our results suggest that using a written asthma action plan form as a vehicle for providing asthma management instructions to patients with persistent asthma who are receiving subspecialty care for the first time confers no added benefit beyond subspecialty-based medical care and education for asthma. Clinical trial registered with www.clinicaltrials.gov (NCT 00149461).
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Asma/terapia , Planificación de Atención al Paciente/estadística & datos numéricos , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Niño , Preescolar , Femenino , Estudios de Seguimiento , Humanos , Masculino , Persona de Mediana Edad , New York , Cooperación del Paciente , Educación del Paciente como Asunto , Pautas de la Práctica en Medicina , Estudios Prospectivos , Calidad de Vida , Autocuidado , Especialización , Población Urbana , Adulto JovenRESUMEN
BACKGROUND: We hypothesized that adding 5 days of prednisone to standard therapy for acute pulmonary exacerbations in patients with cystic fibrosis (CF) would result in a more rapid and greater increase in lung function. METHODS: CF patients with an acute pulmonary exacerbation were randomized to receive oral placebo or prednisone, 2 mg/kg/d up to 60 mg, on days 1 to 5 in addition to standard therapy. Study evaluations on days 1 to 6, 14, and 42 included spirometry, glucose measurements, sputum analysis, and symptom scores. RESULTS: Twelve subjects were randomized to each arm. The slope of FEV(1) between day 1 and day 6 did not differ between evaluable subjects in the prednisone vs placebo groups (52 mL/d vs 51 mL/d, respectively). Mean increase in FEV(1) percentage of predicted did not differ significantly between prednisone vs placebo groups (day 6 [mean +/- SD], 12.2 +/- 5.2% vs 8.1 +/- 10.5%; day 14, 14.7 +/- 8.8% vs 10.2 +/- 11.2%, respectively). Sputum inflammatory markers and symptom scores decreased between day 1 and day 14, but mean values did not differ between groups. Glucosuria occurred in six prednisone subjects, two of whom had hyperglycemia develop. CONCLUSIONS: In this pilot study, addition of oral corticosteroids to standard CF pulmonary exacerbation therapy did not result in a statistically significant effect on lung function or sputum markers of inflammation. Based on a trend toward improvement in pulmonary function with prednisone therapy, we obtained information for power calculations for a definitive study: 250 randomized subjects are required to detect a four-percentage-point treatment effect in FEV(1) percentage of predicted at day 14 to discriminate between null and alternative hypotheses.
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Fibrosis Quística/tratamiento farmacológico , Glucocorticoides/administración & dosificación , Prednisona/administración & dosificación , Adulto , Progresión de la Enfermedad , Método Doble Ciego , Quimioterapia Combinada , Femenino , Volumen Espiratorio Forzado , Humanos , Masculino , Proyectos Piloto , EspirometríaRESUMEN
RATIONALE: Chronic infection with Burkholderia cepacia complex bacteria in cystic fibrosis is associated with accelerated decline in pulmonary function and increased mortality. Clinical implications of the recently characterized genomovar VI, B. dolosa, are unknown. OBJECTIVES: Characterization of impact of B. dolosa on pulmonary function and mortality in cystic fibrosis. METHODS: We compared patients chronically infected with B. dolosa (n = 31) with unmatched patients with B. multivorans (n = 24) and with age- and sex-matched control subjects without Burkholderia species (n = 58). We analyzed rates of pulmonary function decline (% predicted FEV(1)) using a random effects model assuming segmented linear trends. All available FEV(1) measurements from 5 yr (median, 4.8) before until 2.5 yr (median, 1.5) after the first positive culture for Burkholderia (reference date) were analyzed. Survival was compared using the Kaplan-Meier method and proportional hazards model. MEASUREMENTS AND MAIN RESULTS: Baseline FEV(1) and rate of decline were similar in the cohorts. Decline in FEV(1) after the reference date accelerated in patients with B. dolosa (-2.3 percentage points/yr pre vs. -7.1 post, p = 0.002), but was unchanged in the B. multivorans and control patients (-2.3 vs. -0.8 post, p = 0.38, and -2.1 pre vs. -0.5 post, p = 0.20, respectively). The probability of dying within 18 mo of the reference date was 13, 7, and 3% for B. dolosa, B. multivorans, and control patients, respectively (B. dolosa vs. control hazard ratio, 10.8; 95% confidence interval, 1.3-92.8; p = 0.03). CONCLUSIONS: B. dolosa chronic infection in cystic fibrosis is associated with accelerated loss of lung function and decreased survival.
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Infecciones por Burkholderia/complicaciones , Infecciones por Burkholderia/fisiopatología , Fibrosis Quística/mortalidad , Fibrosis Quística/fisiopatología , Adulto , Estudios de Cohortes , Fibrosis Quística/complicaciones , Femenino , Humanos , Masculino , Pruebas de Función Respiratoria , Tasa de SupervivenciaRESUMEN
OBJECTIVE: Describe and define limitations of early pilocarpine iontophoresis (sweat testing) for cystic fibrosis (CF) newborn screening (NBS). STUDY DESIGN: Population-based results from follow-up of CF NBS-positive newborns. RESULTS: Insufficient quantity of sweat is more likely if the sweat test is done too early, but testing is generally successful after 2 weeks of age. Sweat chloride levels drop over the first weeks of life. CF carriers have higher sweat chloride concentrations than non-carriers. CONCLUSIONS: Sweat testing can be performed effectively after 2 weeks of age for CF NBS-positive newborns. Earlier testing has a higher risk of insufficient sweat for completing testing.
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Cloruros/análisis , Fibrosis Quística/diagnóstico , Iontoforesis/métodos , Tamizaje Neonatal/métodos , Sudor/química , Factores de Edad , Algoritmos , Fibrosis Quística/genética , Análisis Mutacional de ADN , Árboles de Decisión , Diagnóstico Precoz , Reacciones Falso Negativas , Reacciones Falso Positivas , Estudios de Seguimiento , Humanos , Recién Nacido , Iontoforesis/normas , Modelos Lineales , Massachusetts , Agonistas Muscarínicos , Tamizaje Neonatal/normas , Selección de Paciente , Pilocarpina , Valores de Referencia , Derivación y Consulta , Factores de RiesgoRESUMEN
OBJECTIVE: To identify necessary components of a successful cystic fibrosis (CF) newborn screening (NBS) program. STUDY DESIGN: The approach to CF NBS used by the Massachusetts NBS program was examined. RESULTS: Several key components were identified that should be addressed when a state has made the decision to screen, and well in advance of actual implementation. These components include (1) inclusion of CF center directors in the development process; (2) logistics of choosing a screening algorithm relative to practices in place and community wishes; (3) projections of medical service needs from specific algorithms; (4) identification of critical reporting components; (5) identification of critical follow-up components; and (6) recognition of educational needs. CONCLUSIONS: Careful examination of a wide variety of issues is needed to ensure optimal implementation of NBS for CF.
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Fibrosis Quística/diagnóstico , Evaluación de Necesidades/organización & administración , Tamizaje Neonatal/organización & administración , Desarrollo de Programa/métodos , Cuidados Posteriores/organización & administración , Algoritmos , Actitud del Personal de Salud , Actitud Frente a la Salud , Toma de Decisiones en la Organización , Asesoramiento Genético/organización & administración , Conocimientos, Actitudes y Práctica en Salud , Humanos , Recién Nacido , Massachusetts , Modelos Organizacionales , Tamizaje Neonatal/psicología , Evaluación de Resultado en la Atención de Salud/organización & administración , Padres/educación , Padres/psicología , Ejecutivos Médicos/educación , Ejecutivos Médicos/organización & administración , Atención Primaria de Salud/organización & administración , Evaluación de Programas y Proyectos de Salud/métodos , Sensibilidad y Especificidad , Apoyo SocialRESUMEN
OBJECTIVE: To evaluate compliance with recommendations for sweat testing/specialty evaluation and genetic counseling after a positive cystic fibrosis newborn screening (CF NBS) result. STUDY DESIGN: All infants with positive CF NBS results require a diagnostic sweat test at a CF center. Results that were "screen positive and diagnosis negative" prompted family genetic counseling. Parent compliance with follow-up protocol recommendations was retrospectively analyzed relative to the communications model in place at a particular CF Center. RESULTS: At each of the 5 MA CF centers, 95% of the CF NBS-positive infants completed recommended sweat testing. In contrast, there was wide disparity in compliance (32%-90%) with completion of genetic counseling between CF centers. CONCLUSION: CF centers that escorted parents through the 2 recommended follow-up steps in 1 day had higher compliance with the second step (genetic counseling) than centers that required a return visit for genetic counseling.
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Comunicación , Fibrosis Quística , Asesoramiento Genético/estadística & datos numéricos , Tamizaje Neonatal/psicología , Padres/psicología , Aceptación de la Atención de Salud , Cuidados Posteriores/organización & administración , Fibrosis Quística/diagnóstico , Fibrosis Quística/terapia , Toma de Decisiones , Conocimientos, Actitudes y Práctica en Salud , Humanos , Recién Nacido , Massachusetts , Modelos Organizacionales , Modelos Psicológicos , Consentimiento Paterno , Padres/educación , Aceptación de la Atención de Salud/psicología , Aceptación de la Atención de Salud/estadística & datos numéricos , Evaluación de Programas y Proyectos de Salud , Estudios Retrospectivos , Factores de TiempoRESUMEN
OBJECTIVES: Newborn screening for cystic fibrosis (CF) provides a model to investigate the implications of applying multiple-mutation DNA testing in screening for any disorder in a pediatric population-based setting, where detection of affected infants is desired and identification of unaffected carriers is not. Widely applied 2-tiered CF newborn screening strategies first test for elevated immunoreactive trypsinogen (IRT) with subsequent analysis for a single CFTR mutation (DeltaF508), systematically missing CF-affected infants with any of the >1000 less common or population-specific mutations. Comparison of CF newborn screening algorithms that incorporate single- and multiple-mutation testing may offer insights into strategies that maximize the public health value of screening for CF and other genetic disorders. The objective of this study was to evaluate technical feasibility and practical implications of 2-tiered CF newborn screening that uses testing for multiple mutations (multiple-CFTR-mutation testing). METHODS: We implemented statewide CF newborn screening using a 2-tiered algorithm: all specimens were assayed for IRT; those with elevated IRT then had multiple-CFTR-mutation testing. Infants who screened positive by detection of 1 or 2 mutations or extremely elevated IRT (>99.8%; failsafe protocol) were then referred for definitive diagnosis by sweat testing. We compared the number of sweat-test referrals using single- with multiple-CFTR-mutation testing. Initial physician assessments and diagnostic outcomes of these screened-positive infants and any affected infants missed by the screen were analyzed. We evaluated compliance with our screening and follow-up protocols. All Massachusetts delivery units, the Newborn Screening Program, pediatric health care providers who evaluate and refer screened-positive infants, and the 5 Massachusetts CF Centers and their affiliated genetic services participated. A 4-year cohort of 323 506 infants who were born in Massachusetts between February 1, 1999, and February 1, 2003, and screened for CF at approximately 2 days of age was studied. RESULTS: A total of 110 of 112 CF-affected infants screened (negative predictive value: 99.99%) were detected with IRT/multiple-CFTR-mutation screening; 2 false-negative screens did not show elevated IRT. A total of 107 (97%) of the 110 had 1 or 2 mutations detected by the multiple- CFTR-mutation screen, and 3 had positive screens on the basis of the failsafe protocol. In contrast, had we used single-mutation testing, only 96 (87%) of the 110 would have had 1 or 2 mutations detectable by single-mutation screen, 8 would have had positive screens on the basis of the failsafe protocol, and an additional 6 infants would have had false-negative screens. Among 110 CF-affected screened-positive infants, a likely "genetic diagnosis" was made by the multiple-CFTR-mutation screen in 82 (75%) versus 55 (50%) with DeltaF508 alone. Increased sensitivity from multiple-CFTR-mutation testing yielded 274 (26%) more referrals for sweat testing and carrier identifications than testing with DeltaF508 alone. CONCLUSIONS: Use of multiple-CFTR-mutation testing improved sensitivity and postscreening prediction of CF at the cost of increased referrals and carrier identification.
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Regulador de Conductancia de Transmembrana de Fibrosis Quística/genética , Fibrosis Quística/diagnóstico , Análisis Mutacional de ADN , Tamizaje Neonatal/métodos , Tripsinógeno/sangre , Algoritmos , Fibrosis Quística/genética , Estudios de Factibilidad , Femenino , Tamización de Portadores Genéticos , Pruebas Genéticas , Humanos , Recién Nacido , Masculino , Mutación , Sensibilidad y EspecificidadRESUMEN
We conducted a double-blind, placebo-controlled, multicenter, randomized trial to test the hypothesis that 300 mg of tobramycin solution for inhalation administered twice daily for 28 days would be safe and result in a profound decrease in Pseudomonas aeruginosa (Pa) density from the lower airway of young children with cystic fibrosis. Ninety-eight subjects were to be randomized; however, the trial was stopped early because of evidence of a significant microbiological treatment effect. Twenty-one children under age 6 years were randomized (8 active; 13 placebo) and underwent bronchoalveolar lavage at baseline and on Day 28. There was a significant difference between treatment groups in the reduction in Pa density; no Pa was detected on Day 28 in 8 of 8 active group patients compared with 1 of 13 placebo group patients. We observed no differences between treatment groups for clinical indices, markers of inflammation, or incidence of adverse events. No abnormalities in serum creatinine or audiometry and no episodes of significant bronchospasm were observed in association with active treatment. We conclude that 28 days of tobramycin solution for inhalation of 300 mg twice daily is safe and effective for significant reduction of lower airway Pa density in young children with cystic fibrosis.