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Successful use of long-acting octreotide for intractable chronic gastrointestinal bleeding in children
Marie O'Meara1, Maria P Cicalese, Andrea Bordugo
1*Paediatric Liver, GI and Nutrition Centre †Department of Pharmacy, King's College Hospital NHS Foundation Trust, London, UK ‡Department of Paediatrics, S. Maria degli Angeli Hospital, Pordenone §Università Vita-Salute, San Raffaele Hospital ||San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy.
Insights
Monthly long-acting octreotide (OCT-LAR) effectively controlled severe gastrointestinal bleeding in children with portal hypertension. This treatment reduced bleeding episodes and hospitalizations, offering a viable option for pediatric patients.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Pharmacology
Background:
- Portal hypertension in children can cause severe gastrointestinal bleeding (GIB).
- Octreotide is effective in managing GIB, but daily injections can be challenging for long-term pediatric use.
- Monthly long-acting octreotide (OCT-LAR) offers a convenient alternative for sustained therapy.
Purpose of the Study:
- To evaluate the efficacy and safety of OCT-LAR in managing severe or recurrent GIB in pediatric patients.
- To assess OCT-LAR's impact on bleeding episodes, hospital admissions, and transfusion requirements.
- To determine if OCT-LAR can prevent the need for liver transplantation in children with intractable GIB.
Main Methods:
- Retrospective study of 9 pediatric patients (median age 21 months) with portal hypertension receiving OCT-LAR (2.5-20 mg monthly).
- Dosing was extrapolated from adult studies.
- Treatment outcomes were compared using data from the year before and the year after initiating OCT-LAR, focusing on bleeding events, hospitalizations, and blood transfusions.
Main Results:
- All patients experienced a reduction in bleeding episodes with OCT-LAR.
- Seven out of nine children achieved cessation of GIB.
- Two children were removed from the liver transplant list due to improved GIB control. No immediate serious adverse effects were noted.
Conclusions:
- OCT-LAR demonstrates significant efficacy in controlling severe, intractable, and recurrent GIB in children with portal hypertension.
- Further prospective randomized controlled trials and pharmacokinetic studies are warranted to optimize OCT-LAR dosage and treatment duration in pediatric populations.
- Long-term safety data for OCT-LAR in children require further investigation.
Background And Aims:
Octreotide reduces splanchnic blood flow and is effective in controlling gastrointestinal bleeding (GIB) caused by portal hypertension. Monthly long-acting octreotide (OCT-LAR) with an efficacy and safety profile similar to subcutaneous daily administration presents an attractive option for long-term therapy. We report our experience with OCT-LAR for severe/recurrent GIB in children with portal hypertension secondary to chronic liver disease or portal vein thrombosis who were unresponsive to standard interventions.
Methods:
A total of 9 patients, 7 boys, who received OCT-LAR between 2000 and 2009 were studied retrospectively (median age at first bleeding 21 months, range 1 month-14.5 years). The dose (2.5-20 mg intramuscularly monthly) was extrapolated from that used in adult acromegaly and neuroendocrine tumours (10-60 mg/mo). Response to treatment was assessed by comparing the number of bleeding events, hospital admissions for acute bleeding, and number of blood units required during the year before and year after starting OCT-LAR.
Results:
OCT-LAR led to a reduction in the number of bleeding episodes in all of the children and to cessation of bleeding in 7. Two children listed for transplantation because of severe GIB were removed from the list. No serious adverse effects immediately attributable to OCT-LAR were observed. One child developed growth hormone deficiency and hypothyroidism during a prolonged period of treatment with subcutaneous octreotide before commencing OCT-LAR.
Conclusions:
OCT-LAR can control severe intractable recurrent GIB in children with portal hypertension. Prospective randomised controlled trials and pharmacokinetic studies are indicated to establish the optimum dose and length of treatment of OCT-LAR and confirm its efficacy and long-term safety in children.
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