Expanding the Evidence Base for Understanding Quality of Life in Pediatric Patients with Intestinal Failure

A study led by Vikram Raghu, MD, MS, assistant professor of Pediatrics in the Division of Pediatric Gastroenterology, Hepatology and Nutrition at UPMC Children’s Hospital of Pittsburgh, aims to develop clinically meaningful, patient-informed data on quality of life in children with intestinal failure. Dr. Raghu’s project is supported by a 2025 UPMC Children's Hospital Foundation Children's Trust Grantmakers award.

Children's Trust is a group of rising philanthropists that collectively channels their philanthropic passions and energies in support of UPMC Children’s most promising pediatric researchers. Over the past ten years, Children's Trust has awarded 18 research grants and contributed $770,000 towards pediatric research.

The project expands upon previous research conducted by Dr. Raghu and colleagues using the Child Health Utility 9D, a standardized tool designed to assess health-related quality of life in children but is not specific to gastroenterology or intestinal failure. Dr. Raghu’s project involves a multicenter collaboration with five academic pediatric gastroenterology programs in the United States and Canada.

“Previous studies have used standardized quality of life instruments, but the results have been inconsistent, and none of them were developed or validated with direct input from patients or families living with intestinal failure,” Dr. Raghu says.

Clinical Context and Need for Better Assessment Tools

Intestinal failure (IF) is a rare condition in which the intestines cannot absorb sufficient nutrients and fluids to sustain normal growth and development. Children with IF often require long-term parenteral nutrition and, in some cases, consideration for intestinal transplant. While clinical outcomes in patients with IF, including survival, enteral autonomy, and complication rates are well established in the literature, less is known about how these interventions affect a patient’s overall quality of life.

In earlier phases of the project, Dr. Raghu’s team conducted qualitative interviews with caregivers and adolescents to understand which aspects of life they considered most relevant when thinking about well-being. These insights guided the selection of the Child Health Utility 9D because it evaluates nine dimensions of quality of life and yields a single utility score between 0 and 1.

“What makes this tool particularly valuable is that it produces a single score we can use in economic modeling, helping us evaluate not just survival or clinical outcomes, but whether a treatment meaningfully improves quality of life,” Dr. Raghu says.

The Child Health Utility 9D includes emotional state, ability to participate in daily activities, school performance, sleep, physical comfort, and other domains. For Dr. Raghu’s study, the instrument was selected because its structure aligned with concerns raised during stakeholder interviews conducted by Dr. Raghu and colleagues, but also because its utility-based scoring system allows integration into broader cost-effectiveness analyses.

Dr. Raghu’s team piloted use of the Child Health Utility 9D in a single-center study in partnership with the University of Toronto. The results of that study showed the instrument was acceptable to caregivers and captured meaningful dimensions of health status. Feedback from those interviews highlighted the importance of participation in activities and general happiness as key indicators of quality of life.

“In contrast to the consistency we saw among parents, our interviews with adolescents revealed a broader, more diverse set of values that are important to them and that diverged from what family members said on the matter,” Dr. Raghu says.

The findings from the caregiver interviews conducted by Dr. Raghu and colleagues were recently presented in oral abstract format at the Congress of the International Intestinal Rehabilitation and Transplant Association in Sweden.

Multicenter Expansion Supported by Children’s Trust Grantmakers Award

With support of the Children's Trust Grantmakers award, the current phase of Dr. Raghu’s project will expand recruitment to five additional institutions: Stanford University, Seattle Children’s Hospital, Children’s Hospital Colorado, Nationwide Children’s Hospital, and the University of Toronto.

The target enrollment for the study is 230 patients. With this larger sample size, the team will be able to explore variation in quality of life based on clinical and demographic characteristics, including patient age, intestinal anatomy, feeding modality, and medical therapy.

“Each center individually follows a small number of patients because of the relative rarity of IF, typically 60 to 70 on home parenteral nutrition. By combining our patient populations, we can build a large enough cohort and data set to begin exploring subgroup patterns and data that are statistically compelling,” Dr. Raghu says.

To facilitate participation by other children’s hospitals, Dr. Ragu’s research team has designed the study to minimize administrative burden for collaborating centers. Survey distribution, administration, data collection, and analysis will be conducted by the UPMC Children’s site.

“This structure makes it much easier for other centers to contribute without needing to allocate additional research staff or resources for the project,” Dr. Raghu says.

Working Toward Optimal Patient-Centered, Data-Informed IF Care

Dr. Raghu’s research is designed to support more personalized and evidence-based approaches to care for children living with IF. By measuring outcomes that reflect patient and family lived experiences, and accounting for those outcomes in clinical and economic analyses, Dr. Raghu’s project may help clarify when to pursue certain medical therapies, optimal support for long-term parenteral nutrition use, and when transplantation may provide patients with optimal long-term outcomes.

“We want to ensure that we are not only extending life but also improving the quality of life in ways that are meaningful to patients,” Dr. Raghu says.

Further Reading on Dr. Raghu’s and Colleagues IF Research at UPMC Children’s

Explore a selection of Dr. Raghu and colleagues published research on pediatric IF in the following papers:

• Raghu VK, Smith KJ. Review of cost-effectiveness analysis to study pediatric intestinal failure and transplant. Intest Fail. 2024 Oct–Dec; 4:100051.
• Raghu VK, Belaid S, Gutierrez S, Holzer P, Orris S, Rothenberger S, Presel T, Ackerman K, Alissa F, King D, Woo Baidal J, Rudolph JA, Bond G, Mazariegos GV, Horslen SP, Smith KJ. Social and financial costs of neonatal intestinal failure. JAMA Netw Open. 2025 Feb 3; 8(2): e2459548.
• Raghu VK, Rumbo C, Horslen SP. From intestinal failure to transplantation: Review on the current need for transplant indications under multidisciplinary transplant programs worldwide. Pediatr Transplant. 2024 May; 28(3) :e14756.
• Garcia J, Oltean M, Rumbo C, Sharkey L, Kaufman SS, Ramos Boluda E, Gupte G, Martinez M, Moon J, Raghu V, Segovia MC, Sudan D, Talbotec C, Varkey J, Gondolesi GE, Mazariegos G, Venick R. Endoscopic surveillance of the intestinal allograft: Recommendations from the Intestinal Rehabilitation and Transplant Association Working Group. Transplantation. 2024 Apr 1; 108(4): 827–835.
• Raghu VK, Abdelhadi R, Garcia MA, McDonnell E, Mezoff E, Namjoshi SS. Push and pull: The art of intestinal rehabilitation. JPEN J Parenter Enteral Nutr. 2023 Nov; 47(8): 960–962.
• Raghu VK, Leraas HJ, Samoylova M, Park C, Rothenberger SD, Sudan D, Avitzur Y. Predictors of 1-year enteral autonomy in children with intestinal failure: A descriptive retrospective cohort study. JPEN J Parenter Enteral Nutr. 2023 Nov; 47(8): 1047–1055.
• Raghu VK, Rothenberger SD, Rudolph JA, Mazariegos GV, Horslen SP, Smith KJ. Pediatric intestine transplant cost: Analysis of the Pediatric Health Information System database. JPEN J Parenter Enteral Nutr. 2023 May; 47(4): 511–518.