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
| Autor: | Osamu Arisaka, Hironori Nishibori, Tohru Yorifuji, Yuki Hosokawa, Tsutomu Ogata, Rie Kawakita, Keiichi Ozono, Toshimi Sairenchi, Yukihiro Hasegawa, Susumu Yokoya, Michiya Masue, Satoshi Kusuda |
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| Rok vydání: | 2017 |
| Předmět: |
Blood Glucose
Male medicine.medical_specialty Endocrinology Diabetes and Metabolism Octreotide 030209 endocrinology & metabolism Hypoglycemia 03 medical and health sciences 0302 clinical medicine Endocrinology Japan 030225 pediatrics Internal medicine medicine Humans Prospective Studies Registries Adverse effect Blood glucose monitoring medicine.diagnostic_test business.industry Remission Induction Infant Newborn Infant medicine.disease Clinical trial Treatment Outcome Anesthesia Congenital hyperinsulinism Observational study Congenital Hyperinsulinism Female Open label business Somatostatin medicine.drug |
| Zdroj: | Endocrine journal. 64(9) |
| ISSN: | 1348-4540 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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