Pediatric Acute Liver Failure Immune Response Network (PALF IRN)

September 2019 Council

Lead Division/Office

DDN

Point(s) of Contact

Edward Doo, M.D.

Executive Summary

Pediatric acute liver failure (PALF) is a complex, rapidly progressive, and potentially devastating clinical syndrome that is the final common pathway for many acute liver diseases in children; some known and others yet to be identified. In up to one-third of cases of PALF, the cause cannot be ascertained. Children with indeterminant PALF have lower rates of spontaneous survival and higher rates of liver transplantation which are medically and resource intensive, leaving the child with life-time need for immunosuppression and careful clinical management. In October 2015, the NIDDK convened a one-day clinical research workshop on the NIH campus, bringing together national and international experts and focusing entirely on the etiology, clinical course, management and potential means of prevention and therapy of indeterminant PALF. A summary of the meeting proceedings and list of research challenges has been published (Hepatology 2017; 65 (3): 1026-37). Among the research opportunities identified, a major priority was given to the design and conduct of a rigorous, randomized controlled trial assessing whether intensive immune modulation would improve survival in this condition for which there is currently no effective therapy. This initiative would fund a prospective trial that has been designed during the preparatory phase of an approved U34 research grant.