Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

• ClinicalTrials.gov Identifier: NCT04629014
• Recruitment Status: Recruiting
• First Posted: November 16, 2020
• Last Update Posted: February 14, 2023
• Sponsor: Boston Children's Hospital
• Collaborators: Seattle Children's Hospital; University of Alabama at Birmingham; Ann & Robert H Lurie Children's Hospital of Chicago; Washington University School of Medicine; University of Michigan; Duke Health; University of Texas Southwestern Medical Center; Alberta Children's Hospital; British Columbia Children's Hospital; The Hospital for Sick Children
• Information provided by (Responsible Party): Biren Modi, Boston Children's Hospital

Study Description

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.

Condition or disease
Pediatric Intestinal Failure

Detailed Description:

Health related quality of life is becoming an increasingly important topic as medical advancements continue to increase life expectancies in various illnesses. Quality of life (QoL) encompasses the physical, psychological, and social well-being of a person. Studies focusing on health related QoL show that providers have a limited understanding of the QoL of their patients, demonstrating a need for further research. Mortality rates in pediatric intestinal failure (PIF) have significantly dropped over the past 3-4 decades and long-term morbidity rather than survival has come into focus as the outcome of interest. However, there remains a significant knowledge gap regarding the specific factors that contribute to QoL among PIF patients and their families. Insight into the challenges these patients face can promote enhanced support services and psycho-education for caregivers of these children as well as help to tailor medical and surgical treatment with a focus on improved QoL. Due to the rare nature of PIF, and despite a recent bloom in multidisciplinary PIF centers, experience is limited in individual centers and a multi-site study with a robust and diverse patient population is optimal to study QoL in pediatric intestinal failure.

Study Design

• Study Type: Observational
• Estimated Enrollment: 400 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure
• Actual Study Start Date: February 27, 2020
• Estimated Primary Completion Date: December 2025
• Estimated Study Completion Date: December 2025

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. Health-related quality of life (hrQOL) [ Time Frame: Baseline ]
   hrQOL as determined by PedsQL Generic Core at time of enrollment and corrected for time from onset of disease
2. Health-related quality of life (hrQOL) Change over time [ Time Frame: Trend over 5 years ]
   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
3. Disease-specific Health-related quality of life (hrQOL) [ Time Frame: Baseline ]
   Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale at time of enrollment and corrected for time from onset of disease
4. Disease-specific Health-related quality of life (hrQOL) [ Time Frame: Trend over 5 years ]
   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

Secondary Outcome Measures :

1. Family Impact [ Time Frame: Baseline ]
   Family impact as determined by the PedsQL Family Impact Module
2. Family Impact [ Time Frame: Trend over 5 years ]
   Family impact as assessed by the PedsQL Family Impact Module, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease

Eligibility Criteria

• Ages Eligible for Study: 6 Months to 25 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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.

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Contacts and Locations

Contacts

Contact: Biren P Modi, MD MPH; 857-218-4651; biren.modi@childrens.harvard.edu

Locations

United States, Alabama

Children's of Alabama; Not yet recruiting

Birmingham, Alabama, United States, 35233

Contact: David Galloway, MD dgalloway@peds.uab.edu

Principal Investigator: David Galloway, MD

United States, Illinois

Lurie Children's Hospital; Recruiting

Chicago, Illinois, United States, 60611

Contact: Valeria Cohran, MD vcohran@luriechildrens.org

Principal Investigator: Valeria Cohran, MD

United States, Massachusetts

Boston Children's Hospital; Recruiting

Boston, Massachusetts, United States, 02115

Contact: Biren P Modi, MD MPH 857-218-4651 biren.modi@childrens.harvard.edu

Principal Investigator: Biren P Modi, MD MPH

United States, Michigan

C.S. Mott Children's Hospital; Recruiting

Ann Arbor, Michigan, United States, 48109

Contact: Meghan Arnold, MD meghanar@med.umich.edu

Principal Investigator: Meghan Arnold, MD

United States, Missouri

St. Louis Children's Hospital; Recruiting

Saint Louis, Missouri, United States, 63100

Contact: Brad Warner, MD brad.warner@wustl.edu

Principal Investigator: Brad Warner, MD

United States, North Carolina

Duke Children's Hospital; Recruiting

Durham, North Carolina, United States, 27710

Contact: Debra Sudan, MD debra.sudan@duke.edu

Principal Investigator: Debra Sudan, MD

United States, Texas

Dallas Children's Hospital; Recruiting

Dallas, Texas, United States, 75390

Contact: Nandini Channabasappa, MD nandini.channabasappa@utsouthwestern.edu

Principal Investigator: Nandini Channabasappa, MD

United States, Washington

Seattle Children's Hospital; Recruiting

Seattle, Washington, United States, 98105

Contact: Patrick J Javid, MD patrick.javid@seattlechildrens.org

Principal Investigator: Patrick J Javid, MD

Canada, Alberta

Alberta Children's Hospital; Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contact: Dana Boctor, MD dana.boctor@albertahealthservices.ca

Principal Investigator: Dana Boctor, MD

Canada, British Columbia

BC Children's Hospital; Recruiting

Vancouver, British Columbia, Canada, V6H 3N1

Contact: Hannah Piper, MD hannah.piper@cw.bc.ca

Principal Investigator: Hannah Piper, MD

Canada, Ontario

Hospital for Sick Children; Recruiting

Toronto, Ontario, Canada, M5G 1X8

Contact: Paul Wales, MD paul.wales@sickkids.ca

Principal Investigator: Paul Wales, MD

Sponsors and Collaborators

Boston Children's Hospital

Seattle Children's Hospital

University of Alabama at Birmingham

Ann & Robert H Lurie Children's Hospital of Chicago

Washington University School of Medicine

University of Michigan

Duke Health

University of Texas Southwestern Medical Center

Alberta Children's Hospital

British Columbia Children's Hospital

The Hospital for Sick Children

Investigators

• Principal Investigator: Patrick J Javid, MD; Seattle Children's Hospital
• Principal Investigator: Biren P Modi, MD MPH; Boston Children's Hospital

More Information

Publications:

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.

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.

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.

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.

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.

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.

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.

• Responsible Party: Biren Modi, Associate Surgical Director, Center for Advanced Intestinal Rehabilitation, Boston Children's Hospital
• ClinicalTrials.gov Identifier: NCT04629014
• Other Study ID Numbers: P00032389
• First Posted: November 16, 2020
• Last Update Posted: February 14, 2023
• Last Verified: February 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Biren Modi, Boston Children's Hospital:

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

Additional relevant MeSH terms:

Intestinal Failure; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases