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长效奥曲肽治疗高胰岛素性低血糖症的临床特征与疗效

Clinical Profile and Efficacy of Long-Acting Octreotide in Hyperinsulinemic Hypoglycaemia.

作者信息

Kubsad Payal S, Vani H N, Sheshadri Tejasvi, Palany Raghupathy

机构信息

Department of Paedistrics(Paediatric Endocrinology), Yeneopya Medical College, Mangalore, Karnataka, India.

Department of Paediatrics and Paediatric Endocrinology, Indira Gandhi Institute of Child Health, Bangalore, Karnataka, India.

出版信息

Indian J Endocrinol Metab. 2024 May-Jun;28(3):289-294. doi: 10.4103/ijem.ijem_483_23. Epub 2024 Jun 26.

Abstract

INTRODUCTION

Hyperinsulinemic hypoglycaemia (HH) is characterised by unregulated insulin secretion, leading to persistent non-ketotic hypoglycaemia with a lack of alternate fuel that induces a severe risk for brain damage and neurodevelopmental abnormalities. Octreotide, a somatostatin analogue, has been effectively administered as subcutaneous injections or depot preparations in diazoxide-unresponsive HH.

METHODS

Children and infants with HH receiving short-acting octreotide injections were included. Anthropometric values, hypoglycaemic episodes, HbA1C, and side effects were noted from the records and were followed up for 12 months. Informed written consent was obtained from the parents before administration of a single dose of LAR (long-acting octreotide). Based on home-based glucose monitoring (HBGM), the dosage of LAR was modified, and short-acting octreotide was eventually withdrawn. The patients shared the injection's cost for cost-effectiveness. HH affects the quality of life (QoL) if not diagnosed and controlled adequately. A QoL questionnaire was given before starting LAR and after 6 months of receiving LAR, and the changes were noted accordingly.

RESULTS

Twenty-two patients were diagnosed with HH, of which 11 infants and children were included in the study. Mutations were identified in 7 (63.63%) children. Daily octreotide could be tapered and stopped with the addition of sirolimus in one patient with an increasing dose of LAR to maintain euglycaemia. The hypoglycaemic episodes decreased with increasing dose of LAR with a decrease in the severity. Eight (72.7%) patients showed an improved lifestyle on LAR quantified through a QoL questionnaire.

CONCLUSION

LAR was found effective in reducing hypoglycaemic episodes with no adverse effects. The patient's parent's satisfaction was higher. Given its high cost, this trial achieved cost-effectiveness by sharing a single sitting of LAR injection.

摘要

引言

高胰岛素血症性低血糖症(HH)的特征是胰岛素分泌不受调节,导致持续性非酮症性低血糖,且缺乏替代燃料,这会引发脑损伤和神经发育异常的严重风险。奥曲肽是一种生长抑素类似物,已在对二氮嗪无反应的HH患者中有效地作为皮下注射剂或长效制剂使用。

方法

纳入接受短效奥曲肽注射的HH儿童和婴儿。从记录中获取人体测量值、低血糖发作情况、糖化血红蛋白(HbA1C)和副作用,并进行12个月的随访。在给予单剂量长效奥曲肽(LAR)之前,已获得家长的书面知情同意。根据家庭血糖监测(HBGM)调整LAR的剂量,最终停用短效奥曲肽。患者分担注射费用以评估成本效益。如果HH未得到充分诊断和控制,会影响生活质量(QoL)。在开始使用LAR之前和接受LAR 6个月后发放QoL问卷,并记录相应变化。

结果

22例患者被诊断为HH,其中11例婴儿和儿童纳入研究。7例(63.63%)儿童检测到突变。在1例患者中,随着LAR剂量增加以维持血糖正常,添加西罗莫司后可逐渐减少并停用每日奥曲肽。随着LAR剂量增加,低血糖发作次数减少,严重程度降低。通过QoL问卷量化,8例(72.7%)患者使用LAR后生活方式得到改善。

结论

发现LAR可有效减少低血糖发作,且无不良反应。患者家长满意度较高。鉴于其成本高昂,本试验通过让患者分担单次LAR注射费用实现了成本效益。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2082/11288510/d26271fb22d6/IJEM-28-289-g001.jpg

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