Department of Internal Medicine, Ito Hospital , Tokyo, Japan .
Thyroid. 2014 Feb;24(2):200-7. doi: 10.1089/thy.2012.0612. Epub 2013 Nov 13.
The management of Graves' disease (GD) in children is associated with a dilemma. Although the established initial treatment for GD in children is antithyroid drug (ATD) treatment, the remission rate in children is said to be lower than in adults, and severe propylthiouracil-induced adverse events (AEs) are an issue. Definitive treatments are effective, but they often result in permanent hypothyroidism and the need for lifelong T4 supplementation.
The objective of this study was to investigate the outcome of ATD treatment, identify significant predictors of a remission, and evaluate the AEs of ATDs in a large pediatric population of GD patients.
We retrospectively assessed the reports of 1138 children up to 18 years of age who had been newly diagnosed with GD at our institution between 1982 and 2006. Their median age at diagnosis was 16 years (range: 3-18 years), and there were 995 females and 143 males. All patients were initially treated with an ATD. Remission was defined as maintenance of euthyroidism for more than 12 months after discontinuing ATD treatment and the absence of any relapses during the follow-up period.
Of the 1138 patients, 723 continued on ATD treatment, 271 underwent surgery or radioactive iodine therapy, and 144 dropped out. Of the 723 patients who continued on ATD treatment, ATD treatment was subsequently ongoing in 84 and was discontinued in 639 (median duration of treatment: 3.8 years; range: 0.3-24.8 years). Of the 639 patients who discontinued ATD treatment, 334 (46.2%) achieved a remission, 247 (34.2%) experienced a relapse, and 58 (8.0%) dropped out. The cumulative remission rate increased with the duration of ATD treatment up until five years. No significant predictors of a remission were identified. The overall incidences of AEs associated with methimazole and propylthiouracil were 21.4% and 18.8% respectively. There were no fatal AEs in our population. While most AEs (91.6%) occurred within the first three months of ATD treatment, 2.7% developed more than two years after the start of ATD treatment. Seven of the eight late-onset AEs were induced by propylthiouracil.
Long-term ATD treatment is a useful treatment option for GD in children.
儿童 Graves 病（GD）的治疗存在一个两难境地。虽然儿童 GD 的既定初始治疗是抗甲状腺药物（ATD）治疗，但据报道，儿童的缓解率低于成人，而且严重的丙硫氧嘧啶诱导的不良事件（AE）是一个问题。确定性治疗是有效的，但它们常常导致永久性甲状腺功能减退症和终身 T4 补充的需要。
本研究旨在探讨 ATD 治疗的结果，确定缓解的显著预测因素，并评估大型儿科 GD 患者人群中 ATD 的 AE。
我们回顾性评估了 1982 年至 2006 年间在我院新诊断为 GD 的 1138 例 18 岁以下儿童的报告。他们的中位诊断年龄为 16 岁（范围：3-18 岁），其中女性 995 例，男性 143 例。所有患者最初均接受 ATD 治疗。缓解定义为停止 ATD 治疗后 12 个月以上维持甲状腺功能正常，且在随访期间无任何复发。
1138 例患者中，723 例继续 ATD 治疗，271 例接受手术或放射性碘治疗，144 例退出。723 例继续 ATD 治疗的患者中，84 例继续 ATD 治疗，639 例（中位治疗时间：3.8 年；范围：0.3-24.8 年）停止 ATD 治疗。639 例停止 ATD 治疗的患者中，334 例（46.2%）达到缓解，247 例（34.2%）复发，58 例（8.0%）退出。随着 ATD 治疗时间的延长，累积缓解率增加，直至五年。未发现缓解的显著预测因素。与甲巯咪唑和丙硫氧嘧啶相关的 AE 总发生率分别为 21.4%和 18.8%。本研究人群无致命 AE。大多数 AE（91.6%）发生在 ATD 治疗的前三个月内，但 2.7%在 ATD 治疗开始两年后发生。8 例迟发性 AE 中有 7 例由丙硫氧嘧啶引起。
长期 ATD 治疗是儿童 GD 的一种有用的治疗选择。
