Understanding and Reducing Variation in Outcomes of Cleft Lip and Palate Surgery

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DESCRIPTION (provided by applicant): The scientific basis for management of cleft lip and palate is weak at best. Almost no elements of care have been evaluated with adequately powered, well-controlled clinical trials. Without high-quality evidence, providers have relied on individual judgments and preferences. The result is a broad variation in both the care children receive and the outcomes they achieve. There is a critical need to establish the efficacy of existing treatments and to speed dissemination and implementation of research findings into practice. To address this unmet need, Thomas Sitzman, a plastic surgeon with clinical expertise in the care of children with cleft lip and palate, is submitting this application for a K3 Mentored Patient-Oriented Research Career Development award. Dr. Sitzman is ideally suited at this stage in his career to benefit from a K23 award, which will build on his extensive clinical training as a cleft surgeon, his prior research studying variation in care delivery, and his ongoing coursework toward a Master of Public Health degree. Through the research and training activities proposed in the application, he will acquire the tools necessary to achieve his long-term goal of becoming an independent investigator evaluating existing treatments and designing healthcare delivery interventions to reduce provider-based variation and speed implementation of effective treatments. The candidate will achieve five specific learning objectives during this award, each targeted at specific needs for his long-term career goal. He will develop expertise in secondary data analysis, enabling him to overcome the low prevalence of craniofacial anomalies at individual centers by using large national datasets. He will acquire advanced skills in longitudinal data analysis, enabling him to address the time-dependent nature of outcomes in cleft surgery. He will learn to apply implementation science in the design of healthcare delivery interventions, enabling him to accelerate implementation and dissemination of research findings. He will gain expertise in the design and management of clinical trials so that he can evaluate efficacy of individual treatments. Finally, Dr. Sitzman will strengthen his academic and professional skills, including grant preparation, presentation of scientific findings, and establishing a network of collaborators across institutions. These learning objectives will be achieved through coursework, directed readings, and hands-on learning. Dr. Sitzman will receive mentorship throughout the award from established investigators deeply invested in his success. He and his primary mentor, Maria Britto, MD, MPH, have assembled a strong team of co-mentors to guide Dr. Sitzman through the proposed training and research activities. This team includes two internationally recognized experts in cleft lip and palate clinical research, along with experts in biostatistics, implementation science and clinical trials. His mentors will provide training, critically evaluate his research, monitor his progress toward independence, and identify specific opportunities for further development. The proposed training activities and research will be enhanced by the institutional environment at Cincinnati Children's Hospital Medical Center (CCHMC) and the University Of Cincinnati College Of Medicine. This environment provides direct mentorship from senior investigators conducting descriptive, interventional, and implementation research; structured seminars to support training and foster exchange of ideas; and access to resources within the University of Cincinnati CTSA program. CCHMC has made a sustained investment to the candidate to support his past and ongoing career development, including protected time and start-up funds over the past three years that enabled him to build the multi-disciplinary mentoring team and conduct preliminary studies to conceptualize the proposed research. The research proposed in this application will test the central hypothesis that modifiable factors from the surgeon and treatment center, including both individual components of the treatment protocol and the system of care delivery, contribute to the outcomes of cleft surgery. This hypothesis will be tested through three specific aims: (1) Determine surgeon and treatment center factors associated with outcomes of primary surgery; (2) Compare the effectiveness of standardized treatment and outcome measurement to a historical control where care was delivered based on individual judgments by the surgeon and treatment center; and (3) Develop and pilot an implementation strategy for standardized measurement of cleft outcomes. Specific Aims 1 and 2 will be achieved by applying longitudinal data analysis to existing national databases. Specific Aim 3 will be achieved using implementation science. The results of this research will improve the health of children with cleft lip and palate in two ways: by identifying modifiable factors associated with their treatment outcomes, which will be evaluated in future prospective experimental studies; and by developing an implementation strategy for standardized outcome measurement that enables surgeons and treatment centers to critically evaluate outcomes, compare their results with those from other centers, and focus their improvement activities. Dr. Sitzman's clinical expertise and previous research training, his team of experienced mentors invested in his success, and his extensive institutional support make him ideally positioned to benefit from a K23 Career Development Award at this stage in his career. Through the proposed research and training activities, he will develop into an independent investigator with the tools and experience to evaluate the efficacy of existing treatments in cleft surgery and to accelerate implementation of research findings into clinical practice.