UCSF is currently one of the ten original sites of the Childhood Liver Disease Research and Education Network (ChiLDREN) now referred to as the Childhood Liver Disease Research Network (ChiLDReN). The specific objectives of the ChiLDReN include but are not limited to: 1) Continuation of the ChiLDReN prospective longitudinal studies of children to continue to provide data and biospecimens for ancillary studies aimed at discovering new diagnostics, etiologic and treatment options for children both pre and post liver transplantation.2) Identification and validation of non-invasive markers of liver disease in our ChiLDReN patients including but not limited to new imaging modalities with the FORCE fibroscan machine. 3) Phase I/II study of new agents to treat cholestasis in children. 4) Study of modifier genes in Alagille Syndrome and Alpha- 1-antitrypsin deficiency 5) Expansion to include other diseases of cholestasis in children with industry and patient advocacy group support. 6) Continue to provide support for small pilot and demonstration projects within the Network.7) Continue to provide education about pediatric liver diseases to the scientific and lay communities through publications and the website.
At UCSF, we specifically plan to perform a pilot crossover study comparing ursodeoxycholic acid with rifampin and or naloxone for children with cholestatic pruritus. Further, in collaboration with Dr. Richard Thompson at King's College Hospital in London, UK, Dr. Laura Bull from UCSF, propose a translational study to further investigate genetic causes of cholestasis. We propose to shift to more cost-effective and comprehensive experimental approaches making use of current state-of-the art technologies.
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