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A Phase I/IIa Trial of Intravenous Immunoglobulin Following Portoenterostomy in Biliary Atresia.
Mack, Cara L; Spino, Cathie; Alonso, Estella M; Bezerra, Jorge A; Moore, Jeffrey; Goodhue, Catherine; Ng, Vicky L; Karpen, Saul J; Venkat, Veena; Loomes, Kathleen M; Wang, Kasper; Sherker, Averell H; Magee, John C; Sokol, Ronald J.
Afiliação
  • Mack CL; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
  • Spino C; University of Michigan, Ann Arbor, MI.
  • Alonso EM; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.
  • Bezerra JA; Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
  • Moore J; University of Michigan, Ann Arbor, MI.
  • Goodhue C; Children's Hospital Los Angeles, Los Angeles, CA.
  • Ng VL; The Hospital for Sick Children, University of Toronto, Toronto, Canada.
  • Karpen SJ; Emory University School of Medicine, Atlanta, GA.
  • Venkat V; Children's Hospital of Pittsburgh, Pittsburgh.
  • Loomes KM; Children's Hospital of Philadelphia, Philadelphia, PA.
  • Wang K; Children's Hospital Los Angeles, Los Angeles, CA.
  • Sherker AH; National Institute of Diabetes, Digestive and Kidney Diseases, NIH, Bethesda, MA.
  • Magee JC; University of Michigan, Ann Arbor, MI.
  • Sokol RJ; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
J Pediatr Gastroenterol Nutr ; 68(4): 495-501, 2019 04.
Article em En | MEDLINE | ID: mdl-30664564
OBJECTIVES: Biliary atresia (BA) is a progressive neonatal fibroinflammatory cholangiopathy. We hypothesized that intravenous immunoglobulin (IVIg) would be safe, feasible, acceptable, and efficacious for the treatment of BA. The primary objective of this study was to establish the feasibility, acceptability, and safety profile of IVIg administration after hepatoportoenterostomy (HPE) in BA. The secondary objective was to determine the treatment efficacy of IVIg based on good bile drainage and survival with the native liver. METHODS: A multicenter, prospective, open-labeled, phase I/IIA trial of IVIg was conducted, with 1 g/kg/dose of IVIg infused at 3-5, 30, and 60 days post-HPE, and subjects followed for 360 days post-HPE. Twenty-nine participants completed the study. RESULTS: Administration of IVIg infusions was feasible and acceptable in 79%. None of the serious adverse events (SAEs) were directly related to IVIg infusions; however, 90% of participants had an SAE. Compared with a historical placebo-arm group, there was no significant increase in the proportion of IVIg participants with a serum total bilirubin <1.5 mg/dL at 90, 180, or 360 days post-HPE. Survival with the native liver in the IVIg participants showed no significant benefit over the historical placebo arm, with a difference at 360 days of -11.9% (IVIg: 58.6%, placebo: 70.5%; 90% UCB: 2.1%; P > 0.05). CONCLUSIONS: Although IVIg infusions in infants with BA post-HPE were feasible, acceptable and safe, there was no trend to lower bilirubin levels or improved 360-day survival with the native liver. CLINICAL TRIAL: Safety Study of Intravenous Immunoglobulin Post-Portoenterostomy in Biliary Atresia; #NCT01854827.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atresia Biliar / Imunoglobulinas Intravenosas Tipo de estudo: Clinical_trials / Observational_studies Limite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2019 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atresia Biliar / Imunoglobulinas Intravenosas Tipo de estudo: Clinical_trials / Observational_studies Limite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2019 Tipo de documento: Article