Aspiration pneumonia (AP) is the most common cause of death in children with severe neurological impairment (Nl). Children with severe Nl commonly experience AP from gastroesophageal reflux disease (GERD), placing them at high risk for compromised quality of life, repeated hospitalization, respiratory failure, and death. The two treatment approaches, surgical fundoplication (SF) and jejunal feeding tubes (JT), have not been prospectively studied with sufficient rigor (e.g., in randomized clinical trials) to demonstrate which approach is more effective in reducing the risk of AP or in improving the quality of life of children with Nl and GERD. This has led to wide variation in treatments offered to these children. The research plan of this K23 proposal consists of two studies that will compare the outcomes of these two treatments (SF or JT) for children with Nl and AP or GERD. Study 1 is retrospective, comparing outcomes of care for patients who underwent SF or JT;it is sufficiently powered to demonstrate morbidity and resource utilization differences and employs statistical methodology to account for possible selection bias. Study 2 is a prospective observational cohort study, comparing quality of life outcomes over time following SF or JT, adjusting for functional status. The results of these complementary studies will provide evidence for judging which treatment is more effective for children with Nl and AP/GERD, a highly vulnerable group of children. In addition, depending on the findings on these studies, it may then be appropriate to recommend a multi- center randomized control trial of these treatments. The career development plan of this K23 includes training in advanced research methods (e.g., clinical trials, survival analysis modeling), quality of life measurement, and the design and execution of multi-site studies. In addition, a highly qualified mentorship and collaborative team has been assembled. Their expertise, coupled with the training that the Principal Investigator (PI) will receive, will result in the successful accomplishment of the focused research plan. The mentorship team, training and proposed research studies will help the PI in achieving his goal of becoming an independent investigator in patient-oriented clinical research that improves outcomes for hospitalized children with Nl and other complex problems. This proposal will support the PI to gain the knowledge and skills necessary to design and conduct studies that have sufficient rigor to create an evidence base that will change practice and improve outcomes for such children. Generating needed evidence bases and learning to conduct multi-site studies are critical steps towards maximizing the quality of life and improving the outcomes for children with Nl.
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