RESUMO
OBJECTIVE: To evaluate lung transplantation for treatment of surfactant protein B (SP-B) deficiency. STUDY DESIGN: We compared surfactant composition and function from pretransplantation and posttransplantation samples of bronchoalveolar lavage fluid, somatic and lung growth, neurodevelopmental progress, pulmonary function, and pulmonary immunohistology in 3 infants with SP-B deficiency who underwent bilateral lung transplantation at 2 months of age and 3 infants who underwent lung transplantation for other reasons. RESULTS: Two years after transplantation, the 2 surviving infants with SP-B deficiency exhibited comparable somatic growth and cognitive development to the comparison infants. All infants had delays in gross motor development that improved with time. Both groups have exhibited normal gas exchange, lung growth, and pulmonary function. The SP-B-deficient infants have also exhibited normal SP-B expression and pulmonary surfactant function after lung transplantation. In two SP-B-deficient infants antibody to SP-B developed. No pathologic consequences of this antibody were identified. CONCLUSIONS: Apart from the development of anti-SP-B antibody, the outcomes for SP-B-deficient infants after lung transplantation are similar to those of infants who undergo lung transplantation for other reasons. Lung transplantation offers a successful interim therapy until gene replacement for this disease is available.
Assuntos
Transplante de Pulmão , Proteolipídeos , Surfactantes Pulmonares/deficiência , Síndrome do Desconforto Respiratório do Recém-Nascido/cirurgia , Anticorpos/sangue , Líquido da Lavagem Broncoalveolar/química , Feminino , Humanos , Terapia de Imunossupressão/métodos , Lactente , Recém-Nascido , Pulmão/patologia , Pulmão/fisiopatologia , Transplante de Pulmão/imunologia , Transplante de Pulmão/patologia , Transplante de Pulmão/fisiologia , Complicações Pós-Operatórias/epidemiologia , Proteolipídeos/análise , Proteolipídeos/imunologia , Surfactantes Pulmonares/análise , Surfactantes Pulmonares/imunologia , Síndrome do Desconforto Respiratório do Recém-Nascido/imunologia , Síndrome do Desconforto Respiratório do Recém-Nascido/patologia , Sobreviventes , Resultado do TratamentoRESUMO
OBJECTIVE: To determine the safety and efficacy of nasal continuous positive airway pressure (CPAP) for obstructive sleep apnea (OSA) during childhood and the effects of growth and maturation on CPAP requirements. DESIGN: Retrospective study with use of a written questionnaire administered to pediatric practitioners treating sleep disorders. SETTING: Nine academic pediatric sleep disorders centers. RESULTS: Data were obtained for 94 patients. Three percent of patients receiving CPAP were less than 1 year, 29% were 1 to 5 years, 36% were 6 to 12 years, and 32% were 13 to 19 years of age; 64% were boys. The longest duration of CPAP use was 4 years. Indications for CPAP included OSA associated with obesity (27%), craniofacial anomalies (25%), idiopathic OSA persisting after adenoidectomy and tonsillectomy (17%), and trisomy 21 (13%). Continuous positive airway pressure was effective in 81 patients (86%), in one patient it was unsuccessful, and in 12 patients compliance was inadequate. The median pressure required was 8 cm H2O (range, 4 to 20 cm H2O); pressure requirements were independent of age or diagnosis. Twenty-two percent of patients eventually required a modification of CPAP levels. Complications of CPAP were minor. Sixty-four percent of centers reported difficulty in obtaining funding for CPAP. CONCLUSIONS: Continuous positive airway pressure is safe, effective, and well tolerated by children and adolescents with OSA. Experience in infants is limited. As pressure requirements change with patient growth, we recommend that CPAP requirements be regularly reevaluated over time. The marked center-to-center variability in CPAP use suggests that specific indications for this therapy require clarification.
Assuntos
Respiração com Pressão Positiva , Síndromes da Apneia do Sono/terapia , Adolescente , Adulto , Proteção da Criança , Feminino , Humanos , Masculino , Cooperação do Paciente , Polissonografia , Respiração com Pressão Positiva/efeitos adversos , Estudos Retrospectivos , Síndromes da Apneia do Sono/diagnóstico , Resultado do TratamentoRESUMO
To examine whether hepatic drug metabolism is altered in patients with cystic fibrosis (CF), we evaluated the pharmacokinetics of three model pharmacologic substrates (antipyrine, a marker of hepatic oxidative metabolism; lorazepam, a marker of hepatic glucuronosyltransferase activity; and indocyanine green (ICG), a marker of hepatic blood flow and biliary secretion) in 14 patients with CF (14.6 to 29.2 years of age) and in 12 children and adolescents with cancer (7.2 to 19.4 years of age), which was treated with only surgery and radiation. Each study subject received a single intravenous dose of the combined model substrates (0.03 mg/kg lorazepam, 10 mg/kg antipyrine, and 0.5 mg/kg ICG) for 5 minutes, followed by repeated blood sampling (n = 10) during a 24-hour postinfusion period. Patients with CF had a significantly greater plasma clearance of lorazepam (56.5 +/- 5.2 vs 25.9 +/- 1.9 ml/min/m2) and ICG (892.5 +/- 176.4 vs 256.5 +/- 41.7 ml/min/m2) but not of antipyrine (27.2 +/- 3.8 vs 20.7 +/- 2.0 ml/min/m2) in comparison with control subjects. The apparent steady-state volume of distribution for lorazepam, ICG, and antipyrine was significantly higher in the patients with CF (2.0-, 3.1-, and 1.4-fold, respectively) than in control subjects. Clearance of the model substrates did not correlate with standard biochemical markers of hepatic function. Similarly, no significant relationships were observed between the clearance or steady-state volume of distribution of the compounds and the National Institutes of Health prognostic scores for the patients with CF. These data demonstrate that the plasma clearance of lorazepam and ICG is increased in patients with CF and suggest that hepatic glucuronosyltransferase activity and biliary secretory capacity are enhanced in this disease.
Assuntos
Antipirina/farmacocinética , Fibrose Cística/metabolismo , Verde de Indocianina/farmacocinética , Fígado/metabolismo , Lorazepam/farmacocinética , Adolescente , Adulto , Antipirina/administração & dosagem , Antipirina/química , Biotransformação , Fibrose Cística/sangue , Fibrose Cística/tratamento farmacológico , Feminino , Humanos , Verde de Indocianina/administração & dosagem , Verde de Indocianina/química , Infusões Intravenosas , Lorazepam/administração & dosagem , Lorazepam/química , Masculino , Neoplasias/metabolismo , Distribuição TecidualRESUMO
Forty-one children and adolescents with a history of breathing difficulty during sleep and morbid obesity, defined as greater than 150% ideal body weight, underwent polysomnography to determine the incidence of sleep-associated breathing disorders. Seventeen patients also performed pulmonary function testing. The mean patient age was 10.3 +/- 4.4 (SD) years, and mean percent of ideal body weight was 208 +/- 42.2. A sleep history questionnaire showed that all patients snored, but frank apnea was reported in only 32%. The pulmonary function tests showed 18% (3/17) with a restrictive defect and 47% (8/17) with obstructive changes. The polysomnograms in 37% (15/41) of the patients were abnormal because of apnea, hypopnea, excessive arousals, or abnormalities in gas exchange. Multiple regression analysis demonstrated no significant association between weight, age, or gender and any physiologic measure on the polysomnogram. Most of the abnormal polysomnograms (13/15) were mildly abnormal, but two showed sufficiently severe abnormalities to require clinical intervention. We conclude that children and adolescents with morbid obesity are at risk for sleep-associated breathing disorders; their polysomnographic abnormalities are usually mild but rarely may be severe enough to require clinical intervention.
Assuntos
Obesidade/complicações , Síndromes da Apneia do Sono/etiologia , Adolescente , Adulto , Criança , Feminino , Humanos , Hipoventilação/etiologia , Lactente , Masculino , Testes de Função Respiratória , Ronco/etiologia , Inquéritos e QuestionáriosRESUMO
To evaluate the incidence and significance of radiographic sinus opacification in infants, we performed computed tomography (CT) of the maxillary and ethmoid sinuses in conjunction with routine cranial CT in 100 infants from birth to 12 months of age. CT was performed for indications other than sinusitis. Prospective concurrent clinical history was obtained and physical examination of the upper respiratory tract was performed. Of 100 infants, 16 had hypoplasia of the maxillary sinuses; 81% (13/16) of these were less than 2 months of age. The antra showed progressive increase in size during the first year of life. Of the 100 infants, 70 had CT sinus opacification, including 67% of those without historical or physical evidence of upper respiratory tract infection. There was a positive correlation of CT findings between the maxillary and ethmoid sinuses in 80% of the infants older than 2 months of age but in only 49% of the younger infants. Radiographic sinus opacification in infants is of uncertain significance and is not diagnostic of upper respiratory tract infection, much less of sinusitis.