Abstract | 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:
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Authors | Cara L Mack, Cathie Spino, Estella M Alonso, Jorge A Bezerra, Jeffrey Moore, Catherine Goodhue, Vicky L Ng, Saul J Karpen, Veena Venkat, Kathleen M Loomes, Kasper Wang, Averell H Sherker, John C Magee, Ronald J Sokol, The ChiLDReN Network |
Journal | Journal of pediatric gastroenterology and nutrition
(J Pediatr Gastroenterol Nutr)
Vol. 68
Issue 4
Pg. 495-501
(04 2019)
ISSN: 1536-4801 [Electronic] United States |
PMID | 30664564
(Publication Type: Clinical Trial, Phase I, Clinical Trial, Phase II, Journal Article, Research Support, N.I.H., Extramural)
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Chemical References |
- Immunoglobulins, Intravenous
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Topics |
- Biliary Atresia
(mortality, surgery)
- Child, Preschool
- Drainage
- Female
- Humans
- Immunoglobulins, Intravenous
(administration & dosage, therapeutic use)
- Infant
- Infant, Newborn
- Liver Transplantation
- Male
- Portoenterostomy, Hepatic
- Postoperative Complications
(drug therapy, surgery)
- Prospective Studies
- Treatment Outcome
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