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1.
J Cyst Fibros ; 21(5): 753-758, 2022 09.
Artigo em Inglês | MEDLINE | ID: mdl-35466039

RESUMO

BACKGROUND: Cystic fibrosis (CF) pulmonary exacerbation (PEx) treatment guidelines suggest that Pseudomonas aeruginosa (Pa) airway infection be treated with two antipseudomonal agents. METHODS: We retrospectively studied treatment responses for STOP2 PEx treatment trial (NCT02781610) participants with a history of Pa infection. Mean lung function and symptom changes from intravenous (IV) antimicrobial treatment start to Visit 2 (7 to 10 days later) were compared between those receiving one, two, and three+ antipseudomonal classes before Visit 2 by ANCOVA. Odds of PEx retreatment with IV antimicrobials within 30 days and future IV-treated PEx hazard were modeled by logistic and Cox proportional hazards regression, respectively. Sensitivity analyses limited to the most common one-, two-, and three-class regimens, to only IV/oral antipseudomonal treatments, and with more stringent Pa infection definitions were conducted. RESULTS: Among 751 participants, 50 (6.7%) were treated with one antipseudomonal class before Visit 2, while 552 (73.5%) and 149 (19.8%) were treated with two and with three+ classes, respectively. Females and participants with a negative Pa culture in the prior month were more likely to be treated with a single class. The most common single, double, and triple class regimens were beta-lactam (BL; n = 42), BL/aminoglycoside (AG; n = 459), and BL/AG/fluoroquinolone (FQ; n = 73). No lung function or symptom response, odds of retreatment, or future PEx hazard differences were observed by number of antipseudomonal classes administered in primary or sensitivity analyses. CONCLUSIONS: We were unable to identify additional benefit when multiple antipseudomonal classes are used to treat PEx in people with CF and Pa.


Assuntos
Fibrose Cística , Infecções por Pseudomonas , Aminoglicosídeos , Antibacterianos , Fibrose Cística/complicações , Fibrose Cística/diagnóstico , Fibrose Cística/tratamento farmacológico , Feminino , Fluoroquinolonas , Humanos , Infecções por Pseudomonas/diagnóstico , Infecções por Pseudomonas/tratamento farmacológico , Pseudomonas aeruginosa , Estudos Retrospectivos , beta-Lactamas
2.
J Cyst Fibros ; 21(4): 588-593, 2022 07.
Artigo em Inglês | MEDLINE | ID: mdl-34933824

RESUMO

BACKGROUND: C-reactive protein (CRP) has been proposed as a biomarker for pulmonary exacerbation (PEx) diagnosis and treatment response. CRP >75mg/L has been associated with increased risk of PEx treatment failure. We have analyzed CRP measures as biomarkers for clinical response during the STOP2 PEx study (NCT02781610). METHODS: CRP measures were collected at antimicrobial treatment start (V1), seven to 10 days later (V2), and two weeks after treatment end (V3). V1 log10CRP concentrations and log10CRP change from V1 to V3 correlations with clinical responses (changes in lung function and symptom score) were assessed by least squares regression. Odds of intravenous (IV) antimicrobial retreatment within 30 days and future PEx hazard associated with V1 and V3 CRP concentrations and V1 CRP >75 mg/L were studied by adjusted logistic regression and proportional hazards modeling, respectively. RESULTS: In all, 951 of 982 STOP2 subjects (92.7%) had CRP measures at V1. V1 log10CRP varied significantly by V1 lung function subgroup, symptom score quartile, and sex, but not by age subgroup. V1 log10CRP correlated moderately with log10CRP change at V3 (r2=0.255) but less so with lung function (r2=0.016) or symptom (r2=0.031) changes at V3. Higher V1 CRP was associated with greater response. CRP changes from V1 to V3 only weakly correlated with lung function (r2=0.061) and symptom (r2=0.066) changes. However, V3 log10CRP was associated with increased odds of retreatment (P = .0081) and future PEx hazard (P = .0114). DISCUSSION: Despite consistent trends, log10CRP change was highly variable with only limited utility as a biomarker of PEx treatment response.


Assuntos
Anti-Infecciosos , Fibrose Cística , Antibacterianos , Anti-Infecciosos/uso terapêutico , Biomarcadores , Proteína C-Reativa , Humanos , Pulmão
3.
J Cyst Fibros ; 21(4): 574-580, 2022 07.
Artigo em Inglês | MEDLINE | ID: mdl-34857494

RESUMO

BACKGROUND: In the STOP2 (Standardized Treatment of Pulmonary Exacerbations-2) study, intravenous (IV) antimicrobial treatment duration for adults with cystic fibrosis (CF) experiencing pulmonary exacerbations (PEx) was determined based on initial treatment response. The impact of home vs hospital care remains an important clinical question in CF. Our hypothesis was that STOP2 participants treated at home would have less improvement in lung function compared to those treated in the hospital. METHODS: Treating clinicians determined PEx treatment location, which was a stratification factor for STOP2 randomization. Lung function, weight, and symptom recovery were evaluated by treatment location. Propensity scores and inverse probability treatment weighting were used to test for differences in clinical response by treatment location. RESULTS: In all, 33% of STOP2 participants received IV antimicrobials in the hospital only, 46% both in the hospital and at home, and 21% at home only. Mean (95% CI) ppFEV1 improvement was significantly (p < 0.05) lower for those treated at home only, 5.0 (3.5, 6.5), compared with at home and in the hospital, 7.0 (5.9, 8.1), and in the hospital only, 8.0 (6.7, 9.4). Mean weight (p < 0.001) and symptom (p < 0.05) changes were significantly smaller for those treated at home only compared to those treated in the hospital only. CONCLUSIONS: Compared to PEx treatment at home only, treatment in the hospital was associated with greater mean lung function, respiratory symptom, and weight improvements. The limitations of home IV therapy should be addressed in order to optimize outcomes for adults with CF treated at home.


Assuntos
Anti-Infecciosos , Fibrose Cística , Administração Intravenosa , Adulto , Antibacterianos , Anti-Infecciosos/uso terapêutico , Fibrose Cística/complicações , Fibrose Cística/diagnóstico , Fibrose Cística/tratamento farmacológico , Humanos , Pulmão
4.
J Cyst Fibros ; 20(1): 36-38, 2021 01.
Artigo em Inglês | MEDLINE | ID: mdl-32800708

RESUMO

INTRODUCTION: Symptom improvement was assessed as changes in the Chronic Respiratory Infection Symptom Score (CRISS) during intravenous antimicrobial exacerbation treatments among subjects from study NCT02109822. METHODS: Median daily CRISS reduction (i.e., improvement) and covariates associated with CRISS reduction by Day 14 were assessed by logistic regression. RESULTS: Among 173 subjects, median baseline CRISS was 49 [IQR 41, 56]; 93.6% had a CRISS reduction of ≥11 (minimal clinically important difference); median time to -11 reduction was 2 days [95% CI 2, 3]. The greatest median CRISS difference from baseline, on Day 17, was -26 [-29, -23]. Odds of -26 CRISS change by Day 14 were greater in subjects with higher baseline CRISS (P=.006) and younger ages (P=.041). CONCLUSIONS: CRISS response has good dynamic range and may be a useful efficacy endpoint for PEx interventional trials. The optimal use of CRISS change as an endpoint remains uncharacterized.


Assuntos
Antibacterianos/uso terapêutico , Fibrose Cística/complicações , Progressão da Doença , Infecções Respiratórias/complicações , Infecções Respiratórias/tratamento farmacológico , Avaliação de Sintomas/métodos , Adolescente , Adulto , Doença Crônica , Ensaios Clínicos como Assunto , Humanos , Infecções Respiratórias/diagnóstico , Adulto Jovem
5.
J Cyst Fibros ; 19(5): 677-687, 2020 09.
Artigo em Inglês | MEDLINE | ID: mdl-32522463

RESUMO

As CFTR modulator therapy transforms the landscape of cystic fibrosis (CF) care, its lack of uniform access across the globe combined with the shift towards a new standard of care creates unique challenges for the development of future CF therapies. The advancement of a full and promising CF therapeutics pipeline remains a necessary priority to ensure maximal clinical benefits for all people with CF. It is through collaboration across the global CF community that we can optimize the evaluation and approval process of new therapies. To this end, we must identify areas for which harmonization is lacking and for which efficiencies can be gained to promote ethical, feasible, and credible study designs amidst the changing CF care landscape. This article summarizes the counsel from core advisors across multiple international regions and clinical trial networks, developed during a one-day workshop in October 2019. The goal of the workshop was to identify, in consideration of the highly transitional era of CFTR modulator availability, the drug development areas for which global alignment is currently uncertain, and paths forward that will enable advancement of CF therapeutic development.


Assuntos
Regulador de Condutância Transmembrana em Fibrose Cística/efeitos dos fármacos , Fibrose Cística/tratamento farmacológico , Desenvolvimento de Medicamentos/organização & administração , Cooperação Internacional , Fibrose Cística/genética , Humanos
6.
Curr Pulmonol Rep ; 8(4): 160-165, 2019 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-31875166

RESUMO

PURPOSE OF REVIEW: a)We conducted a review of the current evidence relating to antibiotic duration in the short and long-term management of non-cystic fibrosis bronchiectasis. RECENT FINDINGS: b)In non-cystic fibrosis pulmonary exacerbations, evidence is primarily based on expert consensus and recent guidelines recommend antibiotic durations of approximately 14 days. Chronic antibiotics (oral or inhaled) are recommended in patients with frequent exacerbations or with chronic Pseudomonas aeruginosa airways infection. Macrolides are the best studied therapies for long-term use with evidence for effect limited to a 12 month duration. Encouragingly, there are increased efforts to develop registries and conduct larger population level studies to improve patient care. SUMMARY: c)There is a paucity of evidence for optimal antibiotic strategies in exacerbations and chronic maintenance in persons with non-cystic fibrosis bronchiectasis. Rationally designed studies which utilize a registry and population-based approach will be critical to build evidence-based strategies to optimize management of non-cystic fibrosis bronchiectasis.

7.
J Cyst Fibros ; 16(5): 607-615, 2017 09.
Artigo em Inglês | MEDLINE | ID: mdl-28438499

RESUMO

BACKGROUND: Given the variability in pulmonary exacerbation (PEx) management within and between Cystic Fibrosis (CF) Care Centers, it is possible that some approaches may be superior to others. A challenge with comparing different PEx management approaches is lack of a community consensus with respect to treatment-response metrics. In this analysis, we assess the feasibility of using different response metrics in prospective randomized studies comparing PEx treatment protocols. METHODS: Response parameters were compiled from the recent STOP (Standardized Treatment of PEx) feasibility study. Pulmonary function responses (recovery of best prior 6-month and 12-month FEV1% predicted and absolute and relative FEV1% predicted improvement from treatment initiation) and sign and symptom recovery from treatment initiation (measured by the Chronic Respiratory Infection Symptom Score [CRISS]) were studied as categorical and continuous variables. The proportion of patients retreated within 30days after the end of initial treatment was studied as a categorical variable. Sample sizes required to adequately power prospective 1:1 randomized superiority and non-inferiority studies employing candidate endpoints were explored. RESULTS: The most sensitive endpoint was mean change in CRISS from treatment initiation, followed by mean absolute FEV1% predicted change from initiation, with the two responses only modestly correlated (R2=.157; P<0.0001). Recovery of previous best FEV1 was a problematic endpoint due to missing data and a substantial proportion of patients beginning PEx treatment with FEV1 exceeding their previous best measures (12.1% >12-month best, 19.6% >6-month best). Although mean outcome measures deteriorated approximately 2-weeks post-treatment follow-up, the effect was non-uniform: 62.7% of patients experienced an FEV1 worsening versus 49.0% who experienced a CRISS worsening. CONCLUSIONS: Results from randomized prospective superiority and non-inferiority studies employing mean CRISS and FEV1 change from treatment initiation should prove compelling to the community. They will need to be large, but appear feasible.


Assuntos
Antibacterianos/uso terapêutico , Fibrose Cística , Determinação de Ponto Final , Avaliação de Resultados em Cuidados de Saúde , Ensaios Clínicos Controlados Aleatórios como Assunto/métodos , Infecções Respiratórias , Adulto , Protocolos Clínicos/normas , Fibrose Cística/complicações , Fibrose Cística/diagnóstico , Fibrose Cística/tratamento farmacológico , Determinação de Ponto Final/métodos , Determinação de Ponto Final/normas , Estudos de Viabilidade , Feminino , Volume Expiratório Forçado/efeitos dos fármacos , Humanos , Masculino , Avaliação de Resultados em Cuidados de Saúde/métodos , Avaliação de Resultados em Cuidados de Saúde/normas , Infecções Respiratórias/tratamento farmacológico , Infecções Respiratórias/etiologia , Tamanho da Amostra , Inquéritos e Questionários/normas , Exacerbação dos Sintomas
8.
J Cyst Fibros ; 14(2): 219-27, 2015 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-25228446

RESUMO

PURPOSE: To evaluate the effects of oral N-acetylcysteine (NAC), which replenishes systemic glutathione, on decreasing inflammation and improving lung function in CF airways. METHODS: A multicenter, randomized, double-blind proof of concept study in which 70 CF subjects received NAC or placebo orally thrice daily for 24 weeks. ENDPOINTS: primary, change in sputum human neutrophil elastase (HNE) activity; secondary, FEV(1) and other clinical lung function measures; and safety, the safety and tolerability of NAC and the potential of NAC to promote pulmonary hypertension in subjects with CF. RESULTS: Lung function (FEV(1) and FEF(25-75%)) remained stable or increased slightly in the NAC group but decreased in the placebo group (p=0.02 and 0.02). Log(10) HNE activity remained equal between cohorts (difference 0.21, 95% CI -0.07 to 0.48, p=0.14). CONCLUSIONS: NAC recipients maintained their lung function while placebo recipients declined (24 week FEV1 treatment effect=150 mL, p<0.02). However no effect on HNE activity and other selected biomarkers of neutrophilic inflammation were detected. Further studies on mechanism and clinical outcomes are warranted.


Assuntos
Acetilcisteína , Fibrose Cística , Inflamação , Pulmão , Estresse Oxidativo/efeitos dos fármacos , Acetilcisteína/administração & dosagem , Acetilcisteína/efeitos adversos , Administração Oral , Adolescente , Adulto , Antioxidantes/administração & dosagem , Antioxidantes/efeitos adversos , Criança , Fibrose Cística/complicações , Fibrose Cística/tratamento farmacológico , Fibrose Cística/metabolismo , Fibrose Cística/fisiopatologia , Método Duplo-Cego , Monitoramento de Medicamentos , Feminino , Humanos , Inflamação/tratamento farmacológico , Inflamação/metabolismo , Elastase de Leucócito/metabolismo , Pulmão/efeitos dos fármacos , Pulmão/metabolismo , Pulmão/fisiopatologia , Masculino , Testes de Função Respiratória/métodos , Escarro/efeitos dos fármacos , Escarro/metabolismo , Tempo , Resultado do Tratamento
9.
Contemp Clin Trials ; 36(2): 460-9, 2013 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-24055998

RESUMO

BACKGROUND: Acute pulmonary exacerbations are central events in the lives of individuals with cystic fibrosis (CF). Pulmonary exacerbations lead to impaired lung function, worse quality of life, and shorter survival. We hypothesized that aggressive early treatment of acute pulmonary exacerbation may improve clinical outcomes. PURPOSE: Describe the rationale of an ongoing trial designed to determine the efficacy of home monitoring of both lung function measurements and symptoms for early detection and subsequent early treatment of acute CF pulmonary exacerbations. STUDY DESIGN: A randomized, non-blinded, multi-center trial in 320 individuals with CF aged 14 years and older. The study compares usual care to a twice a week assessment of home spirometry and CF respiratory symptoms using an electronic device with data transmission to the research personnel to identify and trigger early treatment of CF pulmonary exacerbation. Participants will be enrolled in the study for 12 months. The primary endpoint is change in FEV1 (L) from baseline to 12 months determined by a linear mixed effects model incorporating all quarterly FEV1 measurements. Secondary endpoints include time to first acute protocol-defined pulmonary exacerbation, number of acute pulmonary exacerbations, number of hospitalization days for acute pulmonary exacerbation, time from the end of acute pulmonary exacerbation to onset of subsequent pulmonary exacerbation, change in health related quality of life, change in treatment burden, change in CF respiratory symptoms, and adherence to the study protocol. CONCLUSIONS: This study is a first step in establishing alternative approaches to the care of CF pulmonary exacerbations. We hypothesize that early treatment of pulmonary exacerbations has the potential to slow lung function decline, reduce respiratory symptoms and improve the quality of life for individuals with CF.


Assuntos
Fibrose Cística/complicações , Volume Expiratório Forçado/fisiologia , Pneumopatias/etiologia , Testes de Função Respiratória/métodos , Protocolos Clínicos , Fibrose Cística/fisiopatologia , Serviços de Assistência Domiciliar , Humanos , Pulmão/fisiopatologia , Pneumopatias/fisiopatologia , Monitorização Fisiológica/métodos , Espirometria
10.
Thorax ; 68(9): 818-25, 2013 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-23749840

RESUMO

RATIONALE: Arikace is a liposomal amikacin preparation for aerosol delivery with potent Pseudomonas aeruginosa killing and prolonged lung deposition. OBJECTIVES: To examine the safety and efficacy of 28 days of once-daily Arikace in cystic fibrosis (CF) patients chronically infected with P aeruginosa. METHODS: 105 subjects were evaluated in double-blind, placebo-controlled studies. Subjects were randomised to once-daily Arikace (70, 140, 280 and 560 mg; n=7, 5, 21 and 36 subjects) or placebo (n=36) for 28 days. Primary outcomes included safety and tolerability. Secondary outcomes included lung function (forced expiratory volume at one second (FEV1)), P aeruginosa density in sputum, and the Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R). RESULTS: The adverse event profile was similar among Arikace and placebo subjects. The relative change in FEV1 was higher in the 560 mg dose group at day 28 (p=0.033) and at day 56 (28 days post-treatment, 0.093L±0.203 vs -0.032L±0.119; p=0.003) versus placebo. Sputum P aeruginosa density decreased >1 log in the 560 mg group versus placebo (days 14, 28 and 35; p=0.021). The Respiratory Domain of the CFQ-R increased by the Minimal Clinically Important Difference (MCID) in 67% of Arikace subjects (560 mg) versus 36% of placebo (p=0.006), and correlated with FEV1 improvements at days 14, 28 and 42 (p<0.05). An open-label extension (560 mg Arikace) for 28 days followed by 56 days off over six cycles confirmed durable improvements in lung function and sputum P aeruginosa density (n=49). CONCLUSIONS: Once-daily Arikace demonstrated acute tolerability, safety, biologic activity and efficacy in patients with CF with P aeruginosa infection.


Assuntos
Amicacina/administração & dosagem , Amicacina/efeitos adversos , Antibacterianos/administração & dosagem , Antibacterianos/efeitos adversos , Fibrose Cística/fisiopatologia , Infecções por Pseudomonas/tratamento farmacológico , Pseudomonas aeruginosa , Adolescente , Adulto , Análise de Variância , Criança , Fibrose Cística/complicações , Método Duplo-Cego , Feminino , Volume Expiratório Forçado , Humanos , Lipossomos , Masculino , Testes de Sensibilidade Microbiana , Nebulizadores e Vaporizadores , Qualidade de Vida , Escarro/microbiologia , Adulto Jovem
11.
J Cyst Fibros ; 8(4): 245-52, 2009 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-19481983

RESUMO

BACKGROUND: Cystic fibrosis (CF) specific patient-derived and reported symptom tools are critical steps toward evaluating the outcomes of new therapies for CF. METHODS: We conducted 25 in-depth qualitative interviews using the Day Reconstruction Method and 9 cognitive interviews at two CF programs, the University of Washington and Seattle Children's Hospital and Regional Medical Center. The interviews were audio-recorded and transcribed, and then coded and analyzed for themes relating to pulmonary symptoms and related psychosocial impacts. RESULTS: Six pulmonary symptoms were identified as central to CF: cough, sputum production, wheeze, chest tightness, difficulty breathing/shortness of breath, and fever. Emotional impacts included frustration, sadness/depression, irritability, worry, difficulty sleeping; while activity impacts included time spent sitting or lying down, reduction of usual activities, and missing school or work. In all, 8 symptom items, 4 emotional impacts items, and 4 activity impacts were selected for inclusion on a new daily diary. We also assessed triggers for seeking care. CONCLUSIONS: Using a qualitative inductive methodology, we have obtained patient centered data regarding pulmonary symptoms and burdens and have created a novel patient reported outcome measure for CF. Future studies will assess the validity of the instruments.


Assuntos
Fibrose Cística/complicações , Fibrose Cística/fisiopatologia , Transtornos Respiratórios/etiologia , Transtornos Respiratórios/fisiopatologia , Índice de Gravidade de Doença , Adolescente , Criança , Pré-Escolar , Tosse/etiologia , Tosse/fisiopatologia , Dispneia/etiologia , Dispneia/fisiopatologia , Feminino , Humanos , Entrevistas como Assunto , Masculino , Prontuários Médicos , Pesquisa Qualitativa , Sons Respiratórios/etiologia , Sons Respiratórios/fisiopatologia
12.
J Cyst Fibros ; 7(2): 147-53, 2008 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-17728193

RESUMO

BACKGROUND: Centralized spirometry may significantly improve quality of spirometry and reduce variability of this outcome measure in clinical trials in cystic fibrosis (CF). METHODS: Spirometry was performed during the phase 2 randomized, placebo-controlled, double-blind clinical trial of denufosol in patients with mild to moderate CF using American Thoracic Society guidelines. Uniform spirometers were used with electronic data transmission of all the data to a reading center. Spirometry was evaluated for quality by a central reader based on start of test, cough during the test, and evidence of a plateau. RESULTS: A total of 1418 spirometry values were assessed in 89 subjects during the trial. In only 5 instances did the central reading center need to give feedback to sites regarding the quality of spirometry. The study site data matched the central reading center's data for all but 78 (6%) spirometry values in 33 patients. Many of these differences were small with only 35 (3%) values differing by more than 50 mL in 26 patients. CONCLUSION: Spirometry in this clinical trial was of high quality with low rate of significant centralized over-read.


Assuntos
Fibrose Cística/fisiopatologia , Nucleotídeos de Desoxicitosina/administração & dosagem , Espirometria/métodos , Uridina/análogos & derivados , Administração por Inalação , Adolescente , Fibrose Cística/diagnóstico , Fibrose Cística/tratamento farmacológico , Relação Dose-Resposta a Droga , Método Duplo-Cego , Feminino , Seguimentos , Humanos , Masculino , Fluxo Expiratório Máximo/efeitos dos fármacos , Fluxo Expiratório Máximo/fisiologia , Índice de Gravidade de Doença , Resultado do Tratamento , Uridina/administração & dosagem , Capacidade Vital/efeitos dos fármacos , Capacidade Vital/fisiologia
13.
Thorax ; 59(11): 955-9, 2004 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-15516471

RESUMO

BACKGROUND: Stenotrophomonas maltophilia (SM) is a Gram-negative non-fermenting bacteria cultured from the sputum of patients with cystic fibrosis (CF). To date, no information is available regarding the effect of this organism on lung function in CF. METHODS: A cohort study was conducted to assess the effect of SM on lung function among CF patients aged > or =6 years in the CF Foundation National Patient Registry from 1994 to 1999. Repeated measures regression was used to assess the association between SM and lung function. RESULTS: The cohort consisted of 20 755 patients with median age at entry of 13.8 years and median follow up time of 3.8 years; 2739 patients (13%) were positive at least once for SM and 18 016 (87%) were never positive. After adjusting for sex, height and age, patients with SM had a mean forced expiratory volume in 1 second which was 0.09 l less (95% CI 0.05 to 0.14) than those without SM. The mean rate of decline associated with SM positivity was 0.025 l/year (95% CI 0.012 to 0.037) but, after adjusting for confounders (sex, height, weight, intravenous antibiotic courses, hospital admissions, pancreatic insufficiency, and Pseudomonas aeruginosa and Burkholderia cepacia status), the mean rate of decline decreased to 0.008 l/year (-0.008, 95% CI -0.019 to 0.003). CONCLUSIONS: Although CF patients with SM have worse lung function at the time of positivity, no association was found between SM and increased rate of decline after controlling for confounders.


Assuntos
Fibrose Cística/microbiologia , Infecções por Bactérias Gram-Negativas/complicações , Stenotrophomonas maltophilia , Adolescente , Adulto , Criança , Pré-Escolar , Estudos de Coortes , Fibrose Cística/fisiopatologia , Feminino , Volume Expiratório Forçado/fisiologia , Infecções por Bactérias Gram-Negativas/diagnóstico , Infecções por Bactérias Gram-Negativas/fisiopatologia , Humanos , Lactente , Masculino
14.
Adv Drug Deliv Rev ; 54(11): 1505-28, 2002 Dec 05.
Artigo em Inglês | MEDLINE | ID: mdl-12458158

RESUMO

Clinical trials have become critical to the advancement of medical science and to the evolution of patient care in medicine. The science of clinical research has advanced from early studies in which treatment was assessed without controls to sophisticated multinational collaborative randomized, double-blind, placebo controlled trials of therapeutic interventions. To facilitate the advancement of clinical research, clinical trials networks have been developed to conduct multicenter studies. This review describes the history of clinical trials, clinical trials networks, and the goals of such networks in the United States. The Cystic Fibrosis Therapeutics Development Network, a network that represents the paradigm for genetic and orphan diseases, is described in detail. This network has been extremely successful in its first 3.5 years of existence conducting 18 different clinical trials in patients with Cystic Fibrosis. Unique aspects of the network include the use of internet applications for study conduct and communication, the development of statistical methodology to enhance the efficiency of clinical trial design, the development of outcome measures specific to Cystic Fibrosis, and the development of infrastructure necessary for expediting protocol development. In the current environment, clinical research faces significant challenges related to ensuring the safe and ethical conduct of clinical research while promoting fast and efficient clinical trials. To succeed and move forward to provide treatments and find cures for diseases, clinical trials networks must continue to evolve. The Cystic Fibrosis Therapeutics Development Network represents a network that has met this challenge and will continue to provide a venue for the safe and efficient conduct of clinical trials in Cystic Fibrosis.


Assuntos
Ensaios Clínicos como Assunto/tendências , Redes de Comunicação de Computadores/organização & administração , Fibrose Cística/terapia , Informática em Saúde Pública/organização & administração , Doenças Raras/terapia , Ensaios Clínicos como Assunto/história , Fibrose Cística/história , História do Século XX , História do Século XXI , Humanos , Internet , Estudos Multicêntricos como Assunto/história , Estudos Multicêntricos como Assunto/métodos , Doenças Raras/história , Projetos de Pesquisa , Estados Unidos
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