Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

Обзор

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.

Подробное описание

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.

Первичные конечные точки

• Health-related quality of life (hrQOL) [Срок оценки: Baseline]
• Health-related quality of life (hrQOL) Change over time [Срок оценки: Trend over 5 years]
• Disease-specific Health-related quality of life (hrQOL) [Срок оценки: Baseline]
• Disease-specific Health-related quality of life (hrQOL) [Срок оценки: Trend over 5 years]

Вторичные конечные точки (2)

• Family Impact [Срок оценки: Baseline]
• Family Impact [Срок оценки: Trend over 5 years]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 11 центров

• Children's of Alabama — Birmingham
• Children's Hospital Colorado — Aurora
• Connecticut Children's Medical Center — Hartford
• Lurie Children's Hospital — Chicago
• Boston Children's Hospital — Boston
• C.S. Mott Children's Hospital — Ann Arbor
• St. Louis Children's Hospital — St Louis
• Duke Children's Hospital — Durham
• … и ещё 3 центра

Канада · 3 центра

• Alberta Children's Hospital — Calgary
• BC Children's Hospital — Vancouver
• Hospital for Sick Children — Toronto

Публикации

• 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
• 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
• 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
• 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
• 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
• 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
• 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
• 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

Идентификаторы

NCT: NCT04629014 · P00032389

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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