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脑肿瘤幸存者生长激素缺乏管理的专家意见:来自意大利调查的结果。

Expert Opinion on the Management of Growth Hormone Deficiency in Brain Tumor Survivors: Results From an Italian Survey.

机构信息

Department of Pediatrics, IRCCS Istituto Giannina Gaslini, Endo-European Reference Networks (ERN) Center for Rare Endocrine Conditions, Genova, Italy.

Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.

出版信息

Front Endocrinol (Lausanne). 2022 Jul 14;13:920482. doi: 10.3389/fendo.2022.920482. eCollection 2022.

Abstract

BACKGROUND

Growth hormone deficiency (GHD) is the first and most common endocrine complication in pediatric brain tumor survivors (BTS). GHD can occur due to the presence of the tumor itself, surgery, or cranial radiotherapy (CRT).

AIMS

This study aimed to evaluate management and adherence to current guidelines of the Italian centers engaged in the diagnosis and follow-up of GHD patients with BTS.

METHODS

A multidisciplinary scientific board of pediatric endocrinologists, oncologists and radiologists with neuroimaging expertise discussed and reviewed the main issues relating to the management of GHD in pediatric BTS and developed a survey. The survey included questions relating to organizational aspects, risk factors, diagnosis, definition of stable disease, and treatment. The online survey was sent to an expanded panel of specialists dedicated to the care of pediatric BTS, distributed among the three specialty areas and throughout the country (23 Italian cities and 37 Centers).

RESULTS

The online questionnaire was completed by 86.5% (32 out of 37) of the Centers involved. Most had experience in treating these patients, reporting that they follow more than 50 BTS patients per year. Responses were analyzed descriptively and aggregated by physician specialty. Overall, the results of the survey showed some important controversies in real life adherence to the current guidelines, with discrepancies between endocrinologists and oncologists in the definition of risk factors, diagnostic work-up, decision-making processes and safety. Furthermore, there was no agreement on the neuroimaging definition of stable oncological disease and how to manage growth hormone therapy in patients with residual tumor and GHD.

CONCLUSIONS

The results of the first Italian national survey on the management of GHD in BTS highlighted the difference in management on some important issues. The time to start and stop rhGH treatment represent areas of major uncertainty. The definition of stable disease remains critical and represents a gap in knowledge that must be addressed within the international guidelines in order to increase height and to improve metabolic and quality of life outcomes in cancer survivors with GHD.

摘要

背景

生长激素缺乏症(GHD)是儿科脑肿瘤幸存者(BTS)中第一个也是最常见的内分泌并发症。GHD 可能由于肿瘤本身的存在、手术或颅放射治疗(CRT)引起。

目的

本研究旨在评估参与诊断和随访 BTS 伴 GHD 患者的意大利中心对当前指南的管理和遵循情况。

方法

由儿科内分泌学家、肿瘤学家和具有神经影像学专业知识的放射科医生组成的多学科科学委员会讨论并审查了与儿科 BTS 中 GHD 管理相关的主要问题,并制定了一份调查问卷。该调查问卷包括与组织方面、危险因素、诊断、稳定疾病的定义以及治疗相关的问题。在线调查发送给专门从事儿科 BTS 护理的扩大专家小组,分布在三个专业领域和全国各地(23 个意大利城市和 37 个中心)。

结果

参与的 37 个中心中有 86.5%(32 个)完成了在线问卷。大多数中心都有治疗这些患者的经验,报告称他们每年治疗的 BTS 患者超过 50 例。对回复进行了描述性分析,并按医生专业进行了汇总。总的来说,调查结果显示在现实生活中遵循当前指南方面存在一些重要的争议,内分泌学家和肿瘤学家在危险因素的定义、诊断工作、决策过程和安全性方面存在差异。此外,对于稳定的肿瘤疾病的神经影像学定义以及如何管理残留肿瘤和 GHD 患者的生长激素治疗,也没有达成共识。

结论

第一项关于 BTS 中 GHD 管理的意大利全国性调查结果突出了在一些重要问题上管理方法的差异。开始和停止 rhGH 治疗的时间是主要的不确定领域。稳定疾病的定义仍然是关键,这是知识上的一个空白,必须在国际指南中加以解决,以增加身高,并改善 GHD 癌症幸存者的代谢和生活质量结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1763/9331278/8eb34507e383/fendo-13-920482-g001.jpg

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