The purpose of this study is to evaluate if the treatment could maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve normal growth, to avoid parenteral nutrition complications and to achieve the best possible quality of life for the patient
The short bowel syndrome (SBS) may be defined as a severe malabsorption caused by reduction of intestinal absorptive surface following massive resection of the small intestine. Teduglutide (Revestive®) is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the functional and structural integrity of the cells lining the gastrointestinal tract. The aim of the treatment is to maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve the best possible quality of life for the patient. The rationale for the use of Revestive® is based on data obtained, especially in the trial in SBS patients. Treatment with 0.05 mg/kg/day was safe and well tolerated (no recorded side effects). Patients remained stable despite substantial reduction in parenteral nutrition (PN) supply as evidenced by stable body weight and height, serum electrolytes, pancreatic enzymes and renal function tests. Treatment was associated with: * Reduced PN volume and calories delivered by 25 and 45% respectively with 20% of patients weaned off PN during the study period * Increased Enteral Nutrition (EN) supply in volume and calories by 40 and 62% respectively * Increased in plasma citrulline during the treatment period, but decreased after Teduglutide discontinuation The recommended dose of Revestive® in children and adolescents (aged 1 to 17 years) is the same as for adults (0.05 mg/kg body weight once daily).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Daily sub cutaneous injection 0,05 mg/kg/day
Hôpital Necker - Enfants malades
Paris, France
Decrease in parenteral nutrition: Parenteral Nutrition/Resting Energy Expenditure (PN/REE)
Evaluate the efficacy of Revestive® treatment
Time frame: At week 24
Ostomy output defined as stool balance testing, urine output and plasma citrulline
Evaluate the impact of Revestive on ostomy flow
Time frame: up to week 48
Change in days per week of Parenteral Nutrition (PN)
Quantify the impact of Revestive on the number of perfusion in a week
Time frame: up to week 48
Change in number of stool per day
to evaluate the impact of Revestive on diarrhea
Time frame: up to week 48
Change in stools consistency (Bristol stool chart)
to evaluate the impact of Revestive on diarrhea
Time frame: up to week 48
Ingesta (calorimetric measure)
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
Stool weight/24h
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
Percentage of lipid in stool
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Percentage of nitrogen in stool
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
Percentage of carbohydrate in stool
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
Percentage of sodium in stool
to evaluate the impact of Revestive on Intestinal absorption
Time frame: Every 4 weeks up to week 48
Number of adverse events
to evaluate the long term safety of Revestive
Time frame: At week 48
Change in body weight
Time frame: At baseline, then at 6 and 12 months
Change in heart rate
Time frame: At baseline, then at 6 and 12 months
Change in blood pressure
Time frame: At baseline, then at 6 and 12 months
Endogenous GLP-2 rates (antibody ELISA)
to evaluate the response rate of Revestive
Time frame: up to week 48