Advancing our Understanding of Rare Pediatric Liver Diseases

NIH RePORTER · NIH · U01 · $372,499 · view on reporter.nih.gov ↗

Abstract

This proposal is in response to a request for applications for the Continuation of ChiLDReN, the Childhood Liver Disease Research Network. Over the past twenty years, through a coordinated effort, investigations of cholestatic pediatric disorders have been advanced and we have established a robust database and biorepository for further research. Little is known about the pathogenesis, natural history, and optimal treatment strategies for the rare pediatric liver diseases investigated by ChiLDReN. We at The Children’s Hospital of Philadelphia (CHOP) propose to continue to participate in this Consortium, and thereby advance the field through collaborative research. CHOP has been a highly productive member of ChiLDReN for the last 20 years. In this application, we propose to continue our participation in all aspects of the ChiLDReN consortium, including observational and interventional study protocols, genomics initiatives, ancillary studies and dissemination of research findings. Only through collaboration can we improve the quality and efficiency of care provided to all individuals diagnosed with one of the diseases studied by this network. We have assembled a team of physicians and scientists who contribute substantially to the intellectual and practical needs of the Consortium. The Core Staff includes the Principal Investigators, the Co-Investigators, the Program Manager and the Research Study Coordinators. Other interested scientists and clinicians will participate as determined by Consortium protocols. Our team will continue to collaborate fully with the SDCC and the other investigators to advance scientific discoveries in rare pediatric liver diseases. We will continue to follow patients in the active observational clinical protocols, including PROBE, BASIC, LOGIC, MITOHEP, PSC and Genetic Collection Study, and enroll new participants with biliary atresia and PSC. We will continue to collaborate with the SDCC to implement procedures for uniform data collection, handling and transmittal of data, as well as data audits and other data quality control procedures. We will continue leadership and committee membership in administrative roles, study protocols and disease working groups. The team at CHOP will continue to lead and actively participate in writing groups for Network studies, to promote the dissemination of new knowledge in these rare liver diseases. We will continue to develop new ancillary and translational studies leveraging the robust data and samples previously collected in the ongoing network-wide studies. With the continuation of the Childhood Liver Disease Research Network, robust collaboration among Centers will lead to advances in the field to improve outcomes for children with rare liver disease.

Key facts

NIH application ID
10983207
Project number
2U01DK062481-23
Recipient
CHILDREN'S HOSP OF PHILADELPHIA
Principal Investigator
Binita Maya Kamath
Activity code
U01
Funding institute
NIH
Fiscal year
2024
Award amount
$372,499
Award type
2
Project period
2002-09-15 → 2029-05-31