Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

NCT04629014 | Recruiting

• 1 other identifier: P00032389
• Study Type: observational
• Target: 750
• Locations: 2 countries
• Sites: 14

Brief Summary

This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using data from multiple multidisciplinary intestinal failure centers across the United States and Canada specializing in the care of these participants.

Conditions

Pediatric Intestinal Failure

Keywords

intestinal failure; short bowel syndrome; short gut syndrome; parenteral nutrition; specialized nutrition

Outcome Measures

Primary Outcomes (4)

• Health-related quality of life (hrQOL)

  hrQOL as determined by PedsQL Generic Core at time of enrollment and corrected for time from onset of disease

  Baseline

• Health-related quality of life (hrQOL) Change over time

  hrQOL as determined by PedsQL Generic Core, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease

  Trend over 5 years

• Disease-specific Health-related quality of life (hrQOL)

  Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale at time of enrollment and corrected for time from onset of disease

  Baseline

• Disease-specific Health-related quality of life (hrQOL)

  Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease

  Trend over 5 years

Secondary Outcomes (2)

• Family Impact

  Baseline

• Family Impact

  Trend over 5 years

Eligibility Criteria

• Age: 6 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Participants will be children with a specific diagnosis of intestinal failure as determined by parenteral nutrition dependence at any point in their history for more than 60 out of 74 consecutive days who are managed at one of the intestinal rehabilitation programs located at the study participating sites.

You may qualify if:

• Participants will be followed in a participating institution's intestinal rehabilitation program
• Participants will have a diagnosis of intestinal failure due to functional or structural intestinal dysfunction with current or prior history of specialized nutritional support (parenteral nutrition requirement for 60 out of 74 consecutive days)
• Participants will be age 6 months to 25 years old.
• Parents/caregivers must be able to complete questionnaire without assistance.
• English or Spanish speaking

You may not qualify if:

• Participants aged less than 6 months or greater than 25 years will not be included in this study
• Participants will not be enrolled less than 3 months from index admission or initial outpatient evaluation
• Participants will not be enrolled less than 1 month from inpatient admission (any admission greater than 24 hours in duration)
• Participants will not be enrolled less than 1 month from any operative intervention requiring general anesthesia.
• Primary language other than English or Spanish.
• Participants who have a currently functional small bowel, liver/small bowel or multivisceral transplant
• While other major co-morbidities may be excluded at a later time during data analysis, or may be analyzed as a specific sub-group, they will not be excluded outright. For example, participants with global developmental delay may have parent surveys only, and therefore would need to be excluded from paired analysis of proxy vs. participant perception of HRQOL

Sponsors & Collaborators

• Seattle Children's Hospital: collaborator
• University of Alabama at Birmingham: collaborator
• Washington University School of Medicine: collaborator
• Alberta Children's Hospital: collaborator
• British Columbia Children's Hospital: collaborator
• The Hospital for Sick Children: collaborator
• The University of Texas Health Science Center, Houston: collaborator
• Connecticut Children's Medical Center: collaborator
• Boston Children's Hospital: lead
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator
• University of Michigan: collaborator
• Duke Health: collaborator
• University of Texas Southwestern Medical Center: collaborator
• Children's Hospital Colorado: collaborator

Study Sites (14)

Children's of Alabama

Birmingham, Alabama, 35233, United States

NOT YET RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

RECRUITING

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

RECRUITING

St. Louis Children's Hospital

St Louis, Missouri, 63100, United States

RECRUITING

Duke Children's Hospital

Durham, North Carolina, 27710, United States

RECRUITING

Dallas Children's Hospital

Dallas, Texas, 75390, United States

RECRUITING

UT Health Houston

Houston, Texas, 77030, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

RECRUITING

BC Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada

RECRUITING

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Related Publications (8)

• Diamond IR, de Silva N, Pencharz PB, Kim JH, Wales PW; Group for the Improvement of Intestinal Function and Treatment. Neonatal short bowel syndrome outcomes after the establishment of the first Canadian multidisciplinary intestinal rehabilitation program: preliminary experience. J Pediatr Surg. 2007 May;42(5):806-11. doi: 10.1016/j.jpedsurg.2006.12.033.

  PMID: 17502188 BACKGROUND

• Javid PJ, Malone FR, Reyes J, Healey PJ, Horslen SP. The experience of a regional pediatric intestinal failure program: Successful outcomes from intestinal rehabilitation. Am J Surg. 2010 May;199(5):676-9. doi: 10.1016/j.amjsurg.2010.01.013.

  PMID: 20466115 BACKGROUND

• Modi BP, Langer M, Ching YA, Valim C, Waterford SD, Iglesias J, Duro D, Lo C, Jaksic T, Duggan C. Improved survival in a multidisciplinary short bowel syndrome program. J Pediatr Surg. 2008 Jan;43(1):20-4. doi: 10.1016/j.jpedsurg.2007.09.014.

  PMID: 18206449 BACKGROUND

• Mutanen A, Kosola S, Merras-Salmio L, Kolho KL, Pakarinen MP. Long-term health-related quality of life of patients with pediatric onset intestinal failure. J Pediatr Surg. 2015 Nov;50(11):1854-8. doi: 10.1016/j.jpedsurg.2015.05.012. Epub 2015 Jun 3.

  PMID: 26078213 BACKGROUND

• Norsa L, Artru S, Lambe C, Talbotec C, Pigneur B, Ruemmele F, Colomb V, Capito C, Chardot C, Lacaille F, Goulet O. Long term outcomes of intestinal rehabilitation in children with neonatal very short bowel syndrome: Parenteral nutrition or intestinal transplantation. Clin Nutr. 2019 Apr;38(2):926-933. doi: 10.1016/j.clnu.2018.02.004. Epub 2018 Feb 15.

  PMID: 29478887 BACKGROUND

• Sanchez SE, McAteer JP, Goldin AB, Horslen S, Huebner CE, Javid PJ. Health-related quality of life in children with intestinal failure. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):330-4. doi: 10.1097/MPG.0b013e3182999961.

  PMID: 23648789 BACKGROUND

• Varni JW, Limbers CA, Burwinkle TM. Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 2007 Jul 16;5:43. doi: 10.1186/1477-7525-5-43.

  PMID: 17634123 BACKGROUND

• Modi BP, Piper HG, Belza C, Staffa S, Arnold MA, Boctor DL, Channabasappa N, Cohran VC, Galloway DP, Sudan D, Wales PW, Warner BW, Murtadi G, Javid PJ. Health-Related Quality of Life in Pediatric Intestinal Failure from Infancy to Adolescence: An International, Multi-Center Evaluation. J Pediatr. 2025 Jul;282:114566. doi: 10.1016/j.jpeds.2025.114566. Epub 2025 Mar 28.

  PMID: 40158842 DERIVED

MeSH Terms

Conditions

Intestinal Failure; Short Bowel Syndrome; Hyperphagia

Condition Hierarchy (Ancestors)

Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Malabsorption Syndromes; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Digestive; Signs and Symptoms

Study Officials

• Patrick J Javid, MD

  Seattle Children's Hospital

  PRINCIPAL INVESTIGATOR

• Biren P Modi, MD MPH

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Biren P Modi, MD MPH

CONTACT

857-218-4651; biren.modi@childrens.harvard.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Surgical Director, Center for Advanced Intestinal Rehabilitation

Study Record Dates

• First Submitted: October 29, 2020
• First Posted: November 16, 2020
• Study Start: February 27, 2020
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (11); CA (3)