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:
Using RELiZORB in children with short bowel syndrome
This study is investigating absorption of enteral nutrition when administered through the RELiZORB enzyme cartridge, with a primary goal to reduce dependency on intravenous nutrition (parenteral nutrition, or PN). The secondary goal is to examine any changes in body weight from use of RELiZORB. The duration of this study is 90 days, and includes seven clinic visits. Participants are required to complete daily food diaries, as well as provide stool samples at the beginning and end of the study. Compensation is provided at each visit and includes travel, meal, and parking expenses. At the end of the study, participants have the option to enroll in the RELiZORB Extension Study, which extends the current study for an additional 90 days. This study is sponsored through Alcresta Therapeutics.
Who is eligible?
- Children aged 2-18 years old who are diagnosed with short bowel syndrome (SBS) and are PN dependent
- Those who have congenital or acquired gastrointestinal disease and received surgical intervention
- Those who have been on PN for at least six months and meet the caloric threshold on PN
- Must have a feeding tube and receive stable enteral nutrition, as determined by the study dietitian
Who is not eligible?
- Those with chronic liver disease for a cause other than SBS
- Those with active clinically significant pancreatic or biliary disease
- Patients who have received a bowel-lengthening procedure, including, but not limited to, a STEP procedure
- Those with untreated intestinal obstruction or active stenosis
- Those with severe known dysmotility disease
- Those using growth hormones or those using supplemental glutamine within three months of screening
- Patients receiving enteral formulas that are not compatible with the RELiZORB cartridge (example, insoluble fiber-containing formulas)
To determine if your child is eligible, please contact the principal investigator Mark Puder, MD, PhD, or email relizorbstudy@childrens.harvard.edu.
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.
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.