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儿童期格雷夫斯-巴塞多病:2000 - 2020年期间某单一三级中心的临床特征描述及疾病转归预测因素并文献综述

Graves-Basedow disease in pediatric age: description of the clinical features and predictive factors for disease outcome in a single third-level center in the period 2000-2020 and review of the literature.

作者信息

Tuli Gerdi, Munarin Jessica, Tessaris Daniele, Matarazzo Patrizia, DE Sanctis Luisa

机构信息

Department of Pediatric Endocrinology, Regina Margherita Children's Hospital, Turin, Italy -

Department of Public Health and Pediatrics, University of Turin, Turin, Italy -

出版信息

Minerva Pediatr (Torino). 2025 Aug;77(4):352-359. doi: 10.23736/S2724-5276.21.06653-2.

DOI:10.23736/S2724-5276.21.06653-2
PMID:40625067
Abstract

BACKGROUND

Graves-Basedow disease (GD) is a rare disease in pediatric age and its management is still controversial. Age, goiter size, fT4 and fT3 levels, TRAb titration and the duration of antithyroid drug (ATD) were considered predictors of remission.

METHODS

The aim of this retrospective study was to analyze the clinical features at onset of GD and the outcome of the disease in a single-center pediatric cohort.

RESULTS

Of the 55 subjects analyzed, 16 had remission of GD (group A: 29.1%), 7 underwent definitive treatment (group B: 12.7%), 27 are still on long-term ATD treatment (group C: 49.1%). The relapse rate was 37.5% reducing the overall remission rate to 18.2% with remission plateau observed after approximately 5 years of medical treatment. Serum fT3 levels at diagnosis were higher in group B (17.9±4.02 pg/mL) and group C (12.99±1.21 pg/mL) than in group A (10.79±1.09 pg/mL, P=0.03). The titer of TRAb at diagnosis was also higher in group B and C (24.03±11.45 and 22.23±11.95 UI/respectively) compared to subjects in group A (19.7±7.1 pg/mL, P=0.04) as well as thyroid volume (3.56±0.68 vs. 2.6±0.25 vs. 2.04±0.18, P=0.02). The ATD dose at diagnosis was similar in the 3 groups but significantly lower in the group A at 6, 12 and 24 months (P=0.005, P<0.005 and P<0.005 respectively).

CONCLUSIONS

Given the low remission rate in GD, in presence of negative prognostic factors for remission or when the thyrotoxic state persists or when remission does not occur in approximately 5 years, definitive approach might be considered.

摘要

背景

格雷夫斯-巴塞多病(GD)在儿童期是一种罕见疾病,其治疗仍存在争议。年龄、甲状腺肿大小、游离甲状腺素(fT4)和游离三碘甲状腺原氨酸(fT3)水平、促甲状腺素受体抗体(TRAb)滴定度以及抗甲状腺药物(ATD)治疗时长被视为缓解的预测因素。

方法

这项回顾性研究的目的是分析单中心儿科队列中GD发病时的临床特征以及疾病转归。

结果

在分析的55名受试者中,16人GD缓解(A组:29.1%),7人接受确定性治疗(B组:12.7%),27人仍在接受长期ATD治疗(C组:49.1%)。复发率为37.5%,使总体缓解率降至18.2%,药物治疗约5年后观察到缓解平台期。诊断时B组(17.9±4.02 pg/mL)和C组(12.99±1.21 pg/mL)的血清fT3水平高于A组(10.79±1.09 pg/mL,P = 0.03)。诊断时B组和C组的TRAb滴定度(分别为24.03±11.45和22.23±11.95 UI)也高于A组受试者(19.7±7.1 pg/mL,P = 0.04),以及甲状腺体积(3.56±0.68 vs. 2.6±0.25 vs. 2.04±0.18,P = 0.02)。诊断时3组的ATD剂量相似,但A组在6个月、12个月和24个月时显著更低(分别为P = 0.005、P < 0.005和P < 0.005)。

结论

鉴于GD缓解率低,存在缓解的负面预后因素时,或甲状腺毒症状态持续存在时,或约5年未出现缓解时,可考虑采用确定性治疗方法。

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