Octreotide-associated cholestasis and hepatitis in an infant with congenital hyperinsulinism

Levy‐Khademi, Floris; Shchors, Irina; Avnon-Ziv, Carmit; Levmore-Tamir, Michal; Leder, Oren

doi:10.1515/jpem-2014-0119

Cited by 9 publications

(8 citation statements)

References 3 publications

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“…We recruited only 7 cases receiving octreotide treatment, 4 of these cases were responsive. Although octreotide treatment could be used in diazoxide-unresponsive cases, it could cause cholestasis and hepatitis ( 31 , 32 ). Elevated levels of liver enzymes and gallbladder pathology were detected in the patients treated.…”

Section: Discussionmentioning

confidence: 99%

Congenital Hyperinsulinism in China: A Review of Chinese Literature Over the Past 15 Years

Wang¹,

Sun²,

Zhao³

et al. 2017

Jcrpe

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Objective:Congenital hyperinsulinism (CHI) is a rare but severe cause of hypoglycemia. The present study investigates the clinical presentation, therapeutic outcomes and genetic mutations of CHI in Chinese individuals over the past 15 years.Methods:The authors retrospectively reviewed one case in their department and 206 cases reported from January 2002 to October 2016 in China. PubMed, Ovid Medline, Springer and Wanfang Database, CBMD database, and CKNI database were the sources used to collect the data.Results:In total, 207 cases were recruited. Of these, the ages of 100 (48.3%) were within the 4th week after birth. Seventy-seven cases (37.2%) were born large for gestational age (LGA). Seizures occurred in 140 cases (67.6%). Among 140 cases (67.6%) who were administered diazoxide treatment, 90 (64.3%) were responsive. Seven cases (3.4%) received octreotide treatment and 19 cases (9.2%) underwent surgery. 63/129 cases (48.8%) were detected to have gene mutations, including ABCC8 (69.8%), KCNJ11 (12.7%), GLUD1, GCK, HADH, and HNF4A. Among the diazoxide-unresponsive cases, gene mutations were detected in 20/36 (55.6%) cases with ABCC8 and in 2 (5.6%) cases with KCNJ11. Among the diazoxide-responsive cases, gene mutations were detected in 8 patients with ABCC8, 4 with KCNJ11, 5 with GLUD1, and 1 with GCK.Conclusion:The present study indicates that most CHI cases occurred in neonates and that 1/3 of the cases were born LGA. ABCC8 and KCNJ11 are the most common gene mutations. More than half of the diazoxide-unresponsive CHI detected mutations are in ABCC8 and KCNJ11 genes. The GLUD1 gene mutations cause diazoxide-responsive CHI. Identifying the gene mutations can assist in the diagnosis and treatment of CHI.

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Section: Discussionmentioning

confidence: 99%

Congenital Hyperinsulinism in China: A Review of Chinese Literature Over the Past 15 Years

Wang¹,

Sun²,

Zhao³

et al. 2017

Jcrpe

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“…As previously reported [14,23,24], mild to moderate gastrointestinal events at the start of treatment and gall bladder pathology were the most common adverse events, and no severe adverse event related to the use of octreotide was observed. Of note, rare adverse events, such as necrotizing enterocolitis [14,28,29], long QT syndrome [30], convulsion after cessation of treatment [31], or prolonged hepatic dysfunction [32][33][34][35], have been associated with the use of octreotide for CHI. Necrotizing enterocolitis is of particular concern, due to the high fatality rate [28].…”

Section: Safetymentioning

confidence: 99%

Efficacy and safety of octreotide for the treatment of congenital hyperinsulinism: a prospective, open-label clinical trial and an observational study in Japan using a nationwide registry

et al. 2017

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Octreotide, a long-acting somatostatin analog, has been used for treating hypoglycemia caused by congenital hyperinsulinism (CHI). However, octreotide has not been evaluated in clinical trials and has not been approved in any developed country. We aimed to test the efficacy and safety of octreotide for diazoxide-unresponsive CHI through a combination of a single-arm, open-label clinical trial (SCORCH study) and an observational study to collect data on the clinical course of patients treated off-label in Japan (SCORCH registry). In the SCORCH study, 5 patients were stabilized (blood glucose > 45 mg/dL) by hypertonic glucose infusion, and treated by continuous subcutaneous octreotide infusion at a dose of 5-25 μg/kg/day. Continuous blood glucose monitoring was performed between -24 and +48 hours. In 3 patients, a clinically meaningful rise in blood glucose was achieved and therapy was continued. The glucose infusion was gradually decreased and stopped after 5, 11, and 174 days, respectively. In one case, remission of CHI was reached after 606 days and octreotide was discontinued. The SCORCH registry included 19 diazoxide-unresponsive patients treated by subcutaneous octreotide, by continuous infusion or multiple daily injections. Of the 17 patients treated with hypertonic glucose infusion, the infusion rate was reduced after 4 weeks to less than 50% in 11 patients (64.7%) and stopped in 9 (52.9%). During the combined observation period of 695.4 patient-months in both studies, no severe adverse events related to octreotide were observed. In conclusion, subcutaneous octreotide injection was effective and well tolerated in the majority of patients with diazoxide-unresponsive CHI.

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“…Thus dose adjustment may be required ( 32 , 113 , 114 ). Although various side effects have been reported in case reports, in a study evalutaing the long-term safety and efficacy of octreotide in a large series of CHI patients, it was found to be a safe and effective treatment for diazoxide unresponsive CHI patients ( 102 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 ) ( Table 3 ). The effect of octreotide on linear growth have been found clinically insignificant ( 102 , 117 , 123 ).…”

Section: Introductionmentioning

confidence: 99%

Congenital Hyperinsulinism: Diagnosis and Treatment Update

Demirbilek¹,

Hussain²

2018

Jcrpe

110

112

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Pancreatic β-cells are finely tuned to secrete insulin so that plasma glucose levels are maintained within a narrow physiological range (3.5-5.5 mmol/L). Hyperinsulinaemic hypoglycaemia (HH) is the inappropriate secretion of insulin in the presence of low plasma glucose levels and leads to severe and persistent hypoglycaemia in neonates and children. Mutations in 12 different key genes (ABCC8, KCNJ11, GLUD1, GCK, HADH, SLC16A1, UCP2, HNF4A, HNF1A, HK1, PGM1 and PMM2) that are involved in the regulation of insulin secretion from pancreatic β-cells have been described to be responsible for the underlying molecular mechanisms leading to congenital HH. In HH due to the inhibitory effect of insulin on lipolysis and ketogenesis there is suppressed ketone body formation in the presence of hypoglycaemia thus leading to increased risk of hypoglycaemic brain injury. Therefore, a prompt diagnosis and immediate management of HH is essential to avoid hypoglycaemic brain injury and long-term neurological complications in children. Advances in molecular genetics, imaging techniques (18F-DOPA positron emission tomography/computed tomography scanning), medical therapy and surgical advances (laparoscopic and open pancreatectomy) have changed the management and improved the outcome of patients with HH. This review article provides an overview to the background, clinical presentation, diagnosis, molecular genetics and therapy in children with different forms of HH.

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Octreotide-associated cholestasis and hepatitis in an infant with congenital hyperinsulinism

Cited by 9 publications

References 3 publications

Congenital Hyperinsulinism in China: A Review of Chinese Literature Over the Past 15 Years

Congenital Hyperinsulinism in China: A Review of Chinese Literature Over the Past 15 Years

Efficacy and safety of octreotide for the treatment of congenital hyperinsulinism: a prospective, open-label clinical trial and an observational study in Japan using a nationwide registry

Congenital Hyperinsulinism: Diagnosis and Treatment Update

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