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定义常规治疗未能充分控制的慢性甲状旁腺功能减退症的特征:三个欧洲德尔菲小组的共识结果。

Defining the Characteristics of Chronic Hypoparathyroidism Not Adequately Controlled on Conventional Therapy: Consensus Findings of Three European Delphi Panels.

机构信息

Shire Pharmaceuticals Ltd, a member of the Takeda group of companies, London, UK.

Shire Sweden AB, a member of the Takeda group of companies, Stockholm, Sweden.

出版信息

Adv Ther. 2019 Nov;36(11):3007-3016. doi: 10.1007/s12325-019-01102-5. Epub 2019 Oct 5.

Abstract

INTRODUCTION

European Society of Endocrinology (ESE) guidelines provide goals for hypoparathyroidism management but do not define characteristics of chronic hypoparathyroidism that is not adequately controlled. Three European country-specific Delphi panels were conducted to gain consensus on these characteristics.

METHODS

Delphi panels were conducted in the UK, Sweden, and Portugal using similar methodology. At each round, panellists considered patients with chronic hypoparathyroidism whose disease is not adequately controlled on conventional therapy according to a matrix of four presentations of patients with chronic hypoparathyroidism: normal biochemical levels/well (group 1), abnormal biochemical levels/well (group 2), normal biochemical levels/unwell (group 3), and abnormal biochemical levels/unwell (group 4), with wellness defined by the patient's persistent symptoms, comorbidities, and complications. For groups 2-4, panellists rated characteristics in five categories (patient characteristics, family history, comorbidities, biochemistry, and symptoms/impact on quality of life [QoL]) with respect to defining a patient as having chronic hypoparathyroidism that was not adequately controlled on conventional therapy. Consensus was achieved when more than 80% of respondents agreed.

RESULTS

Among the three countries, panellists agreed that characteristics within four of the five categories (patient characteristics, comorbidities, biochemistry, and symptoms/impact on QoL) were important for defining inadequate control. Characteristics deemed important in groups 2-4 included a history of compliance problems and chronic kidney disease stages 4 and 5. In groups 2 and 4, the biochemical parameters deemed important were serum calcium, urinary calcium, and serum creatinine. In groups 3 and 4, tingling or numbness in the hands/feet and face was the only symptom deemed important in all three countries.

CONCLUSION

Delphi panels conducted in three European countries provided national consensus on key parameters of patient characteristics, biochemistry, comorbidities, and symptoms/impact on QoL that define not adequately controlled chronic hypoparathyroidism. These characteristics should be tested more widely for their applicability in clinical practice.

FUNDING

Shire International GmbH, Zug, Switzerland, a member of the Takeda group of companies.

摘要

简介

欧洲内分泌学会(ESE)指南为甲状旁腺功能减退症的管理提供了目标,但并未定义未经充分控制的慢性甲状旁腺功能减退症的特征。为此,进行了三项欧洲国家特定的德尔菲小组研究,以就这些特征达成共识。

方法

在英国、瑞典和葡萄牙进行了德尔菲小组研究,使用了类似的方法。在每一轮中,小组成员都考虑了根据慢性甲状旁腺功能减退症患者的四种表现矩阵来评估慢性甲状旁腺功能减退症患者,这些患者的疾病未经常规治疗充分控制:生化水平正常/良好(第 1 组),生化水平异常/良好(第 2 组),生化水平正常/不适(第 3 组)和生化水平异常/不适(第 4 组),其中“不适”由患者的持续性症状、合并症和并发症定义。对于第 2-4 组,小组成员根据五项类别(患者特征、家族史、合并症、生化和症状/对生活质量的影响[QoL])对定义常规治疗未充分控制的慢性甲状旁腺功能减退症患者的特征进行了评分。当超过 80%的受访者表示同意时,就达成了共识。

结果

在这三个国家中,小组成员一致认为,在五个类别中的四个类别(患者特征、合并症、生化和症状/对 QoL 的影响)内的特征对于定义治疗不足很重要。在第 2-4 组中,被认为重要的特征包括有遵医嘱问题和慢性肾脏病 4 期和 5 期的病史。在第 2 组和第 4 组中,被认为重要的生化参数是血清钙、尿钙和血清肌酐。在第 3 组和第 4 组中,手/脚和面部刺痛或麻木是三个国家中唯一被认为重要的症状。

结论

在三个欧洲国家进行的德尔菲小组研究为定义治疗不足的慢性甲状旁腺功能减退症的患者特征、生化、合并症和症状/对 QoL 的关键参数提供了国家共识。这些特征应在更广泛的范围内进行测试,以检验其在临床实践中的适用性。

资金

Shire International GmbH,Zug,瑞士,武田集团的成员公司。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f30a/6830839/bb3b9e674e9e/12325_2019_1102_Fig1_HTML.jpg

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