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International Intestinal Failure Registry and The FDA Grant 101
0.0 - Information Session for Caregivers and Patients
Presenter: Yaron Avitzur, Simon Horslen, , , Authors: Yaron Avitzur, Simon Horslen, Christina Belza, Marie Neumann
Overview
Intestinal failure is a rare disease with challenging management strategies. Research is critically important to advancing the field and informing practice and engaging with patients and caregivers to facilitate current and future research initiatives is imperative. The session will provide a clear, accessible overview of the registry’s purpose and its role in advancing knowledge of long-term outcomes, treatment effectiveness, and patient-centred priorities in intestinal failure. It will also outline how registry data may inform clinical care, support research innovation, and contribute to improved therapies and health system planning. A goal of the webinar will be the importance of family engagement in research, emphasizing patients and caregivers as active partners whose lived experiences help shape research priorities, outcome selection, and study design. The session will promote opportunities for meaningful involvement through registry participation and advisory roles, while fostering transparency, trust, and open dialogue through an interactive question-and-answer component.
Learning Objectives
Will provide a clear, accessible overview of the registry’s purpose, including its role in advancing understanding of long-term outcomes, treatment effectiveness, and patient-centered priorities in intestinal failure
Will outline how registry data may inform clinical care, support research innovation, and contribute to improved therapies and health system planning
Will highlight the goals and scope of the FDA grant, including its focus on strengthening data infrastructure and accelerating meaningful research in rare pediatric conditions
Nurse Practitioner, Hospital for Sick Children
Toronto, Canada
Christina Belza graduated from McMaster University with an undergraduate degree in nursing and went on to complete her Master’s degree and Nurse Practitioner Certificate at the University of Toronto. She joined the GIFT (Group for Improvement of Intestinal Function and Treatment) program in 2008 as a Pediatric Nurse Practitioner, where she has worked across both inpatient and outpatient settings. Through her clinical work, she developed a strong interest in outcomes research, particularly related to the complications and long-term impacts of intestinal failure.
Christina has presented at numerous national and international conferences, showcasing work conducted at SickKids, and has contributed to several peer-reviewed publications. Her clinical and research interests led her to pursue a PhD in Clinical Epidemiology at the University of Toronto, with a focus on the role of caregivers in supporting children with intestinal failure requiring long-term parenteral nutrition.
University of Nebraska Medical Center
Omaha, Nebraska, USA
Marie Neumann, MA, is a social scientist who conducts research on how short bowel syndrome (SBS) or intestinal failure (IF) affects the well-being of patients and families. She holds a research position at the University of Nebraska Medical Center (UNMC) in Omaha, NE, and will earn a doctoral degree in May 2026. Together with a team of patient- and caregiver-researchers, Marie developed the PIF-QoL, the first quality-of-life assessment tool designed for pediatric IF. She also co-founded the gutsy perspective, an initiative intent on closing the gap between the IF community and the research that affects its members. Marie's personal journey inspired her work. Seven years ago, her youngest child was diagnosed with ultra-short bowel syndrome. Clinicians' communication at the time about what her child’s future with SBS would look like propelled her to systematically study the experiences of children with SBS and their families.
Pediatric Gastroenterologist, Hospital for Sick Children
Toronto, Canada
Dr. Avitzur is a Professor of Paediatrics at the University of Toronto, the Director of the Group for Improvement of Intestinal Function and Treatment (GIFT) program, and the Medical Director of Intestine Transplantation at SickKids hospital in Toronto, Canada. He is the president-elect of the International Intestinal Rehabilitation and Transplant Association, the leading world association for health professionals caring for children and adults with intestinal failure.
Dr. Avitzur research work is focused on strategies to improve the outcome of children with intestinal failure, liver and intestine transplantation and congenital diarrheas. In 2018 he established and currently leads the International Intestinal Failure Registry, a large high quality research platform for pediatric intestinal failure with close to 700 children enrolled to the registry from around the world. Dr. Avitzur published more than 170 papers in prominent journals and textbooks and is supported by grants from leading institutions such as the NIH and FDA.
Director of Hepatology, UPMC Children’s Hospital of Pittsburgh
Simon Horslen MB ChB FRCPCH FAASLD FAST is a Professor in the Department of Pediatrics and the Clinical & Translational Research Institute at the University of Pittsburgh. He is the Director of Pediatric Hepatology. Dr. Horslen earned his medical degree at the University of Bristol Medical School in England and is a Fellow and Founder Member of the Royal College of Paediatrics and Child Health.
He has worked in the US for over 25 years initially at the University of Nebraska Medical Center, then at Seattle Children’s for 16 years before moving to Pittsburgh Children’s Hospital in 2021. Clinical and research interests include metabolic liver disease, acute liver failure, and liver and intestine transplantation. He has over 200 publications in the areas of pediatric liver disease, intestinal failure and transplantation. Dr Horslen participates in several multicenter studies including SPLIT (Studies in Pediatric Liver Transplantation), PALF (Pediatric Acute Liver Failure) and ChiLDReN (Children’s Liver Disease Research Network). Dr Horslen is a past-Chair of the OPTN Pediatric Committee and has served as an At-Large member of the OPTN Board of Governors. He is a senior staff member for SRTR, focusing on Intestine, Liver, and Pediatrics. He has chaired the Pediatric Community of Practice of the American Society of Transplantation (AST), is a Past-President of SPLIT (Society of Pediatric Liver Transplantation) and of IIRTA (The International Intestinal Rehabilitation and Transplantation Association).
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