RESUMO
OBJECTIVE: To assess if a heterogeneous pattern on research liver ultrasound examination can identify children at risk for advanced cystic fibrosis (CF) liver disease. STUDY DESIGN: Planned 4-year interim analysis of a 9-year multicenter, case-controlled cohort study (Prospective Study of Ultrasound to Predict Hepatic Cirrhosis in CF). Children with pancreatic insufficient CF aged 3-12 years without known cirrhosis, Burkholderia species infection, or short bowel syndrome underwent a screening research ultrasound examination. Participants with a heterogeneous liver ultrasound pattern were matched (by age, Pseudomonas infection status, and center) 1:2 with participants with a normal pattern. Clinical status and laboratory data were obtained annually and research ultrasound examinations biannually. The primary end point was the development of a nodular research ultrasound pattern, a surrogate for advanced CF liver disease. RESULTS: There were 722 participants who underwent screening research ultrasound examination, of which 65 were heterogeneous liver ultrasound pattern and 592 normal liver ultrasound pattern. The final cohort included 55 participants with a heterogeneous liver ultrasound pattern and 116 participants with a normal liver ultrasound pattern. All participants with at least 1 follow-up research ultrasound were included. There were no differences in age or sex between groups at entry. Alanine aminotransferase (42 ± 22 U/L vs 32 ± 19 U/L; P = .0033), gamma glutamyl transpeptidase (36 ± 34 U/L vs 15 ± 8 U/L; P < .001), and aspartate aminotransferase to platelet ratio index (0.7 ± 0.5 vs 0.4 ± 0.2; P < .0001) were higher in participants with a heterogeneous liver ultrasound pattern compared with participants with a normal liver ultrasound pattern. Participants with a heterogeneous liver ultrasound pattern had a 9.1-fold increased incidence (95% CI, 2.7-30.8; P = .0004) of nodular pattern vs a normal liver ultrasound pattern (23% in heterogeneous liver ultrasound pattern vs 2.6% in normal liver ultrasound pattern). CONCLUSIONS: Research liver ultrasound examinations can identify children with CF at increased risk for developing advanced CF liver disease.
Assuntos
Fibrose Cística/complicações , Hepatopatias/etiologia , Fígado/patologia , Estudos de Casos e Controles , Criança , Pré-Escolar , Feminino , Humanos , Fígado/diagnóstico por imagem , Hepatopatias/diagnóstico , Masculino , Estudos Prospectivos , Medição de Risco , UltrassonografiaAssuntos
Hepatite C Crônica , Hepatite C , Feminino , Hepacivirus , Humanos , Transmissão Vertical de Doenças Infecciosas , GravidezRESUMO
BACKGROUND: Congenital hypopituitarism is an uncommon cause of neonatal cholestasis. Little is known about the effect of anterior pituitary hormone on hepatic functions. METHODS: A retrospective review of the medical charts of eight infants with congenital hypopituitarism and neonatal cholestasis was performed. The results of endocrinological investigations, eye examinations, and magnetic resonance imaging were used to classify these infants. RESULTS: Eight infants (4 male and 4 female; mean age, 1.7 weeks) who presented with cholestatic jaundice subsequently (mean age, 7.6 weeks) developed isolated or multiple anterior pituitary hormone deficiencies. Persistent hypoglycemia, ocular abnormalities, and microphallus were often clinical signs prompting further endocrinological and radiological investigations. Septo-optic dysplasia was prevalent, occurring in five cases. Cholestasis and hepatosplenomegaly resolved within a mean of 9.7 and 10 weeks, respectively, in the majority of cases after replacement of glucocorticoid and thyroid hormones. However, transaminase levels remained high after hormone replacement. Cortisol deficiency and hypoglycemia were noted in all cases, often following stress. Hyperlipidemia persisted in one case after the resolution of cholestasis and after corticosteroid and thyroid hormone replacement therapy. Growth hormone deficiency was not corrected due to the absence of hypoglycemia after corticosteroid hormone, an infant's age, and/or a lack of financial resources. CONCLUSIONS: In our series, it appears that glucocorticoid and thyroid hormones play a significant role in the resolution of cholestasis and hepatosplenomegaly. A persistently elevated transaminase level and hyperlipidemia after corticosteroid and thyroid hormone replacement may indicate the need for long-term follow-up and/or growth hormone therapy.
Assuntos
Colestase/etiologia , Hepatomegalia/etiologia , Hipopituitarismo/complicações , Fígado/fisiopatologia , Hormônios Adeno-Hipofisários/deficiência , Feminino , Glucocorticoides/uso terapêutico , Hormônio do Crescimento/deficiência , Terapia de Reposição Hormonal , Humanos , Hidrocortisona/deficiência , Hipoglicemia/etiologia , Hipopituitarismo/congênito , Hipopituitarismo/tratamento farmacológico , Lactente , Recém-Nascido , Fígado/patologia , Masculino , Esplenomegalia/etiologia , Hormônios Tireóideos/uso terapêutico , Transaminases/sangueRESUMO
Vanishing bile duct syndrome (VBDS) is a rare disorder and requires a liver biopsy for a diagnosis. The condition has not been reported in children with toxic epidermal necrolysis (TEN). The etiology of VBDS in our patient with TEN is most likely from drug hypersensitivity. A high index of suspicion will prompt clinicians to start more specific investigations and treatments. The use of immunosuppressive agents, intravenous immunoglobulin and ursodeoxycholic acid has not been consistently successful in these patients. A new approach with biologic agents such as anti-tumor necrosis factor-alpha may be a promising therapy and reduce severe adverse outcomes.