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Ongoing Research Projects | Overview

At CAIR, we’re working continually to improve treatments for short bowel syndrome and related concerns. Some of our current and recent research projects include:

Markers for short bowel syndrome

Our researchers are measuring blood and stool markers of intestinal adaptation in children with short bowel syndrome over the course of a year. They will investigate how these markers relate to growth and weaning off of parenteral nutrition.

Development of a treatment algorithm for vitamin D deficiency

We have modified our treatment plan for vitamin D deficiency after looking at lab trends and made changes based on both age and levels of this vitamin. We will be collecting data to determine the effectiveness of our new recommendations in treating vitamin D deficiency.

Copper status in infants with prolonged dependence on parenteral nutrition

Prospective data was collected on a group of children with intestinal failure and dependence on parenteral nutrition. Our researchers are now analyzing their blood levels of copper and various potential risk factors for copper deficiency to help optimize prevention and treatment of this problem in pediatric short bowel syndrome.

Assessment of body composition

The Bodpod (also called air displacement plethysmography) is a safe, quick, non-invasive technique that measures both the muscle mass and fat mass in children. Since routine measures such as weight gain may not be sensitive enough to assess the actual gain of muscle mass, the Bodpod may allow for a better assessment of a child’s body composition.

Non-invasive assessment of liver fibrosis

Vibration-controlled transient elastography, also called FibroScan®, is a new diagnostic tool to assess liver fibrosis in pediatric intestinal failure patients with associated liver disease. FibroScan results for our CAIR patients have shown that the diagnostic tool is successful and a valid method for non-invasive monitoring of liver injury in this population.

Methionine breath test

Monitoring hepatic function has been limited to conventional blood testing or liver biopsy in patients with intestinal failure-associated liver disease. Metabolism of the stable isotope, L[1-13C] methionine has been shown to exclusively occur in the liver and to be a safe, non-invasive, and valid measure of liver mitochondrial function. The CAIR team is currently assessing the feasibility and relevance of serial breath testing to measure changes in liver function over time.

Distraction enterogenesis

The remaining length of small intestine is the most significant factor in predicting whether a patient with short bowel syndrome will be able to successfully wean off parenteral nutrition. Techniques for lengthening intestine through mechanical stretch have been described, and the CAIR team has ongoing studies applying extraluminal stretch to elongate small bowel and stimulate tissue growth.

Long-term outcomes of ultra-short bowel syndrome

Our CAIR team recently reviewed long-term outcomes in patients with a history of midgut volvulus leading to ultra-short bowel syndrome. We have found that midgut volvulus with extensive bowel loss is associated with favorable long-term survival, and that weaning off parenteral nutrition is feasible with intestinal rehabilitation.

Long-term outcomes

Tens of thousands of children have short bowel syndrome; however, with the advent of parenteral nutrition and advances in intestinal rehabilitation, many are now surviving to adulthood. The primary aim of this study is to observe long-term medical and surgical outcomes of adolescents nearing adulthood as we work towards transition of care for these patients.

Quality of life

The CAIR team is actively enrolling patients in a study to learn more about the quality of life experienced by children with intestinal failure. This is a collaborative study being conducted by intestinal rehabilitation centers throughout the country. It is being co-led by Dr. Biren Modi at Boston Children’s and Dr. Patrick Javid at Seattle Children’s Hospital. Your family may be approached to inquire about participation in this study at a future appointment. If you have questions or want to discuss participation please feel free to contact our office for more information.

Effects of RELiZORB

Led by Mark Puder, MD, the primary goal of this study is to determine whether RELiZORB is associated with a reduction in calories from parenteral nutrition. We will also be measuring changes in children’s body weight by the end of the study.

To determine if your child is eligible for this study, please contact relizorbstudy@childrens.harvard.edu.

PROMPT BOLUS (Pragmatic Pediatric Trial of Balanced vs Normal Saline Fluid in Sepsis)

This clinical study will compare two commonly used intravenous treatments for pediatric sepsis to see if one is more effective and safer than the other. All children who present to the Boston Children’s Hospital Emergency Department with sepsis will be eligible to participate in this study. For more information or to contact the study team, visit the trial website.

STARS

The CAIR team is about to begin an exciting new study called STARS in adults ages 18 and older with short bowel syndrome/intestinal failure (SBS-IF). STARS is a clinical trial looking at the efficacy and safety of weekly subcutaneous injections of apraglutide (a GLP-2 analogue) in reducing parenteral nutrition (PN) dependency. It is an international, multicenter, double-blind, randomized, placebo-controlled trial. The trial consists of a screening phase, a treatment phase of 48 weeks, and a safety follow-up phase. After successful screening, trial participants will receive once weekly apraglutide or placebo. This study is sponsored by VectivBio AG.

Who is eligible?
  • Men and women with SBS-IF who are receiving PN secondary to surgical resection of the small intestine with either stoma or colon-in-continuity
  • You must require PN at least three days per week and be considered stable.
  • You cannot undergo restorative surgery intended to change PN requirements in the trial period.
  • Age 18 or older
Who is not eligible?
  • Women who are pregnant or lactating
  • People who have undergone major abdominal surgery in the last six months prior to screening
  • Those with a history of cancer within five years
  • Those with evidence of active inflammatory GI conditions in the previous six months
  • Those with evidence of decompensated heart failure
  • Those with evidence of severe renal or hepatic impairment
  • Any previous use of growth factors such as growth hormone, native GLP-2, GLP-1, or GLP-2 or GLP-1 analogues should be discussed with the Investigator.