Efficacy and safety of long‐term, continuous subcutaneous octreotide infusion for patients with different subtypes of K<sub>ATP</sub>‐channel hyperinsulinism

Yorifuji, Tohru; Kawakita, Rie; Hosokawa, Yuki; Fujimaru, Rika; Matsubara, Kousaku; Aizu, Katsuya; Suzuki, Shigeru; Nagasaka, Hironori; Nishibori, Hironori; Masue, Michiya

doi:10.1111/cen.12075

Cited by 38 publications

(43 citation statements)

References 26 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…In this study, blood glucose was stabilized > 3.3 mmol/L in 48.0% of patients with diazoxide‐unresponsive CHI who received octreotide treatment; however, Yorifuji et al 9 reported that 88.0% of patients maintained blood glucose levels > 3.33 mmol/L after weaning off intravenous dextrose. Nevertheless, 84.0% of patients in our study maintained blood glucose > 2.8 mmol/L.…”

Section: Discussioncontrasting

confidence: 51%

“…On follow‐up, 50.0% of patients with monoallelic KATP‐channel mutations exhibited spontaneous remission at a median age of 15.5 months (range, 8.0–30.0 months); this indicates that octreotide treatment is a feasible alternative to surgery, especially for patients with monoallelic KATP‐channel mutations. Yorifuji et al 9 reported that the spontaneous remission rate in patients who received octreotide treatment was 25.0% (three of 12); these three patients had monoallelic KATP‐channel mutations. Salomon‐Estebanez et al 16 reported that seven patients receiving octreotide therapy exhibited remission during follow‐up; three patients had a biallelic KATP mutation, four patients had a monoallelic KATP mutation, and the mean age at spontaneous remission was 5.9 years (range, 1.6–9.0 years).…”

Section: Discussionmentioning

confidence: 99%

“…Another cohort study showed only transient gastrointestinal symptoms, including poor appetite, constipation or changes in stool color, (e.g. whitish stool) 9 . Some case reports described the occurrence of octreotide‐induced hepatitis with an increase of liver transaminases to 1000 IU/L, 18‐20 but this effect has not been observed in large sample cohort studies.…”

Section: Discussionmentioning

confidence: 99%

See 2 more Smart Citations

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Cao

et al. 2020

Pediatric Investigation

View full text Add to dashboard Cite

Importance Octreotide is an off‐label medicine for congenital hyperinsulinism (CHI), but is currently widely used for treatment of patients with CHI. Thus far, variable efficacy and adverse effects have been reported for octreotide. Objective The present study evaluated the efficacy and safety of a subcutaneous octreotide injection for treatment of diazoxide‐unresponsive CHI in China. Methods This study was a retrospective review of children with diazoxide‐unresponsive CHI who were treated with a subcutaneous octreotide injection. The efficacy and side effects of the treatment were assessed. Results Twenty‐five Chinese children (15 boys) were involved in the study. Their median age at diagnosis was 8 weeks (range, 1–24 weeks) and median age at the final follow‐up was 1.8 years (range, 0.3–3.3 years). Octreotide therapy effectively increased blood glucose levels in all patients. The intravenous glucose infusion rate was reduced in all patients. Twenty‐one patients gradually discontinued the intravenous glucose infusion while receiving octreotide combined with frequent carbohydrate/glucose‐rich feeding. Among patients with a monoallelic ATP‐sensitive potassium (KATP) channel mutation, 50.0% showed gradual remission during follow up, indicating that the octreotide treatment may be a feasible alternative to surgery, especially for patients with monoallelic KATP‐channel mutations. Transient elevation of liver enzymes occurred in 20.0% of patients, while asymptomatic gallbladder pathology occurred in one patient. The growth rates of these patients were normal (height standard deviation score was 0.3 ± 1.5 at the final follow‐up). Interpretation Octreotide was a well‐tolerated, effective therapy for most children with diazoxide‐unresponsive CHI.

show abstract

Section: Discussioncontrasting

confidence: 51%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Cao

et al. 2020

Pediatric Investigation

View full text Add to dashboard Cite

show abstract

“…Octreotide therapy enabled us to discontinue intravenous infusions of glucose in all 7 patients. The duration of octreotide therapy ranged from 9 to 45 days, which was much shorter than the treatment time for children with congenital hyperinsulinism [18]. …”

Section: Discussionmentioning

confidence: 99%

Experience of Octreotide Therapy for Hyperinsulinemic Hypoglycemia in Neonates Born Small for Gestational Age: A Case Series

Pan¹,

Zhang²,

Li³

2015

Horm Res Paediatr

View full text Add to dashboard Cite

Aims: Hyperinsulinemic hypoglycemia (HH) is common in small-for-gestational-age (SGA) neonates. Diazoxide is often used as the first-line medication for HH in SGA neonates. Unfortunately, diazoxide is not authorized in China. We examined the effectiveness of octreotide as an alternative therapy to treat HH in SGA neonates. There is limited data on the use of octreotide in HH of SGA neonates. Methods: Seven SGA neonates with HH who were admitted to the Department of Neonatology at the Third Affiliated Hospital of Sun Yat-sen University between January 2013 and December 2014 received octreotide at an initial dose of 5 μg/kg/day through subcutaneous injection at 8-hour intervals. Depending on the glycemic control, the dose of octreotide was increased in increments of 2-5 μg/kg/day every 3-5 days to the maximum dose of 30 μg/kg/day. Results: The age of neonates with HH diagnosis ranged from 1 to 4 days. The maximum dose of octreotide ranged from 8 to 18 μg/kg/day. The duration of octreotide therapy ranged from 9 to 45 days. All patients had a clear glycemic response to octreotide, and no major adverse events were observed during the treatment. Conclusions: Octreotide may be a useful alternative therapy in HH of SGA neonates when diazoxide is unavailable.

show abstract

“…It is administered as multiple daily subcutaneous injections (3-4 times/day) or by continuous subcutaneous infusions using an insulin pump. In our experience, many patients with K ATP channel CHI can be maintained on long-term treatment until spontaneous remission at 2-5 years of age69). Common adverse events include gastrointestinal symptoms, white stool, dilated gall bladder with or without gall stones, and growth deceleration after 2 years of age.…”

Section: Current Treatment Strategiesmentioning

confidence: 99%

Congenital hyperinsulinism: current status and future perspectives

Yorifuji

2014

Ann Pediatr Endocrinol Metab

Self Cite

147

View full text Add to dashboard Cite

The diagnosis and treatment of congenital hyperinsulinism (CHI) have made a remarkable progress over the past 20 years and, currently, it is relatively rare to see patients who are left with severe psychomotor delay. The improvement was made possible by the recent developments in the understanding of the molecular and pathological basis of CHI. Known etiologies include inactivating mutations of the KATP channel genes (ABCC8 and KCNJ11) and HNF4A, HNF1A, HADH, and UCP2 or activating mutations of GLUD1, GCK, and SLC16A1. The understanding of the focal form of KATP channel CHI and its detection by 18F-fluoro-L-DOPA positron emission tomography have revolutionized the management of CHI, and many patients can be cured without postoperative diabetes mellitus. The incidence of the focal form appears to be higher in Asian countries; therefore, the establishment of treatment systems is even more important in this population. In addition to diazoxide or long-term subcutaneous infusion of octreotide or glucagon, long-acting octreotide or lanreotide have also been used successfully until spontaneous remission. Because of these medications, near-total pancreatectomy is less often performed even for the diazoxide-unresponsive diffuse form of CHI. Other promising medications include pasireotide, small-molecule correctors such as sulfonylurea or carbamazepine, GLP1 receptor antagonists, or mammalian target of rapamycin inhibitors. Unsolved questions in this field include the identification of the remaining genes responsible for CHI, the mechanisms leading to transient CHI, and the mechanisms responsible for the spontaneous remission of CHI. This article reviews recent developments and hypothesis regarding these questions.

show abstract

Efficacy and safety of long‐term, continuous subcutaneous octreotide infusion for patients with different subtypes of K_ATP‐channel hyperinsulinism

Abstract: Long-term, continuous, subcutaneous octreotide infusion is a feasible alternative to surgery especially for patients with monoallelic KATP-channel mutations.

Cited by 38 publications

References 26 publications

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Experience of Octreotide Therapy for Hyperinsulinemic Hypoglycemia in Neonates Born Small for Gestational Age: A Case Series

Congenital hyperinsulinism: current status and future perspectives

Contact Info

Product

Resources

About

Efficacy and safety of long‐term, continuous subcutaneous octreotide infusion for patients with different subtypes of KATP‐channel hyperinsulinism

Abstract: Long-term, continuous, subcutaneous octreotide infusion is a feasible alternative to surgery especially for patients with monoallelic KATP-channel mutations.

Cited by 38 publications

References 26 publications

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Efficacy and safety of octreotide treatment for diazoxide‐unresponsive congenital hyperinsulinism in China

Experience of Octreotide Therapy for Hyperinsulinemic Hypoglycemia in Neonates Born Small for Gestational Age: A Case Series

Congenital hyperinsulinism: current status and future perspectives

Contact Info

Product

Resources

About

Efficacy and safety of long‐term, continuous subcutaneous octreotide infusion for patients with different subtypes of K_ATP‐channel hyperinsulinism