RESUMO
The CFF Consensus Conference concluded with a summary of those outcome measures that would be most useful in studies of patients 6 years of age and older and those measures that would be most useful in studies of the younger population (< 6 years of age) (Table). These measures were further divided into biologic markers most appropriate for initial (phase I and phase II) clinical trials and those especially useful in large, multicenter (phase III) pivotal trials. There is an ongoing need to improve the accuracy and validity of currently available measures of biologic activity and clinical efficacy in CF, especially in the younger population. The conference participants recommended that the following eight issues be addressed as soon as possible: (1) definition of pulmonary exacerbation, (2) broadly applicable methods of testing pulmonary function in small children (ideally a single test for all ages), (3) a comprehensive severity-of-disease score for young children, (4) reliable methods of quantifying chest x-ray and CT scan changes in young patients, (5) simple, inexpensive measures of lung inflammation, (6) a centralized, uniform approach to the establishment of data monitoring committees, (7) a quality of well-being scale for small children, and (8) reliable, reproducible aerosol delivery systems with defined characteristics. In addition, participants recommended that better methods be developed for assessing patients' adherence to research protocols.
Assuntos
Ensaios Clínicos como Assunto , Fibrose Cística/terapia , Resultado do Tratamento , Adolescente , Adulto , Criança , Pré-Escolar , Protocolos Clínicos , Fibrose Cística/fisiopatologia , Feminino , Humanos , Masculino , Qualidade de Vida , Projetos de PesquisaAssuntos
Oxigênio/toxicidade , Traqueia/efeitos dos fármacos , Criança , Pré-Escolar , Técnicas de Cultura , Células Epiteliais , Epitélio/efeitos dos fármacos , Humanos , Lactente , Recém-Nascido , Doenças do Recém-Nascido/patologia , Doenças do Prematuro/patologia , Pulmão/patologia , Pneumopatias/patologia , Pesquisa , Traqueia/citologiaAssuntos
Obstrução das Vias Respiratórias/complicações , Ecocardiografia , Hipertensão Pulmonar/etiologia , Pressão Sanguínea , Dióxido de Carbono/sangue , Cateterismo Cardíaco , Pré-Escolar , Eletrocardiografia , Feminino , Humanos , Hipertensão Pulmonar/diagnóstico , Hipertensão Pulmonar/fisiopatologia , Lactente , Recém-Nascido , Masculino , Oxigênio/sangue , Pressão Parcial , Resistência VascularRESUMO
The course of 95 patients with cystic fibrosis is presented. Survivors have a mean follow-up period of over 14 years (minimum: 13 years). Of 45 patients diagnosed prior to extensive irreversible pulmonary involvement, only one has died and none is disabled. In contrast, of the other 50 patients diagnosed after substantial irreversible pulmonary disease was present, 26 have died. Mortality and morbidity has been greater in females. Possible factors contributing to the improving prognosis include early diagnosis, aggressive management with comprehensive care, easy access to specialized care, and improved antimicrobial therapy.
Assuntos
Fibrose Cística/diagnóstico , Adolescente , Criança , Pré-Escolar , Fibrose Cística/complicações , Feminino , Seguimentos , Humanos , Pneumopatias/complicações , MasculinoRESUMO
Seventeen black patients showed typical sweat gland, gastrointestinal, and pulmonary manifestations of cystic fibrosis. There was an unusually high incidence of meconium ileus equivalent (35%). Three infant siblings of patients in this study had died of cystic fibrosis prior to referral of a family member to this center. One patient died at age four years after emotional problems interfered with the treatment program; status asthmaticus played a major role in his terminal respiratory failure. The remainder of the patients have had substantially less morbidity from pulmonary disease than a comparable, much larger group of white patients. Only one patient, age 28, who is married and employed full-time has severe pulmonary involvement as assessed by pulmonary function studies and chest roentgenogram. The 16 surviving patients have a mean age of 13 1/2 years after a mean follow-up period of 10 years. If black patients with cystic fibrosis survive infancy, they then, as a group, may have a relatively good prognosis.
Assuntos
População Negra , Fibrose Cística/diagnóstico , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Lactente , Masculino , Testes de Função RespiratóriaRESUMO
Six black infants and young children with high titers of milk precipitins were identified by screening the sera of 160 children with idiopathic chronic lung disease. None of the six had immunoglobulin deficiency, elevation of sweat chlorides, SS hemoglobin, or recurrent aspiration. All six children had typical manifestations of milk-induced pulmonary hemosiderosis: recurrent pulmonary infiltrates (6/6), hemosiderin-laden pulmonary macrophages (5/6), intermittent wheezing (5/6), eosinophilia (4/6), anemia (4/6), iron deficiency (4/4), failure to thrive (4/6), and elevated levels of serum IgE (4/4). Three children also had chronic rhinitis and eventually developed large adenoids, hypercapnia and acidosis during sleep, and right heart failure. Elimination of cow milk from the diet, symptomatic therapy, and adenoidectomy when indicated resulted in improvement of all six patients. Pulmonary hemosiderosis and some cases of upper airway obstruction with pulmonary hypertension appear to be two stages, early and delayed, of the same immunophysiologic process. Early dietary intervention may prevent the cardiovascular complications of this process.