DESCRIPTION (provided by applicant): Douglas Mogul, MD, MPH, an Assistant Professor in the Division of Pediatric Gastroenterology at Johns Hopkins University, seeks a Mentored Clinical Investigator Award (K08) in patient-centered outcomes research (PCOR) to obtain essential skills and mentored research experience for an independent career as a pediatric health services researcher in the field of liver disease. Pediatric end-stage liver disease (ESLD) is associated with severe impairments in nutrition and cognitive development, and liver transplantation is the only life-saving option. Over the last 25 years, liver transplantation has saved the lives of more than 15,000 children in the US. Successful transplantation is hindered by a scarcity of suitable livers for children. Consequently, liver allocation is currently prioritied by the probability of waitlist mortality, but this strategy means that most individuals are extremely sick before they receive a new liver, and in some instances they will die on the waitlist. The specific aims of the research agenda are: Aim 1. To evaluate health care utilization among pediatric liver transplant candidates before, during, and after transplantation and to determine if healthcare utilization varies among people from different clinical characteristics, including patients who utilization different donation strategies such as living-related and split liver transplantation (LRLT and SLT, respectively); Aim 2. To determine if incentivization for SLT would be associated with better overall outcomes; and Aim 3. To solicit preferences among a range of key stakeholders - transplant surgeons, caretakers of children with ESLD, and adults with ESLD - for two strategies of organ allocation: the status quo, and a strategy where SLT is incentivized. This proposal has the potential to challenge the existing system of liver allocation and to determine if expansion of organ supply is associated with better outcomes. Furthermore, this study is novel in that it engages patient stakeholders throughout the proposal in order to address questions that are of primary concern to individuals with ESLD. Directed toward AHRQ-priority populations of children with chronic diseases and special needs, this proposal uses cutting edge methods in comparative effectiveness research as directed towards PCOR. This research will facilitate long-term goals including mastery of advanced techniques in PCOR, as well as independence as a health services researcher. These goals will be achieved through an academic curriculum integrated with the research plan, including doctoral-level coursework in comparative effectiveness and PCOR methodologies. Dr. Mogul will receive active mentorship by Drs. John F. P. Bridges, PhD, and Dorry Segev, MD, PhD. Completion of the proposed aims will yield important information on what children with ESLD, and their caregivers, can expect regarding healthcare utilization while they await liver transplantation, and whether individual characteristics influence this utilization. Through a better understanding of the healthcare utilization in these two different allocation strategies, and stakeholder preference for these strategies, there exists a tremendous potential to inform policy and improve outcomes for children with ESLD.
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