Abstract

The studies of hypothyroidism in children with transfusion-dependent hemoglobin E/β-thalassemia (TDT), especially in those who underwent hematopoietic stem cell transplantation (HSCT) are limited. We performed a longitudinal retrospective analysis of thyroid function test (TFT) results among TDT patients aged <25 years who received regular transfusion compared to those who underwent HSCT in Faculty of Medicine Siriraj hospital, Thailand during October 2003 to March 2019. Fifty patients (23 TDT, 27 HSCT) were included. The mean age at the last follow-up was 20.1 ± 2.8 vs. 14.5 ± 4.61 years, respectively. The median age at HSCT was 6 (range: 1.9–13.7) years. The prevalence of hypothyroidism among TDT and post-HSCT was 47.8% and 52.2%, respectively. No study patients showed symptoms or signs of hypothyroidism. Subclinical hypothyroidism was the most common type (63.6% of TDT, and 100% of post-HSCT). We found persistent hypothyroidism in 30.4% of TDT, and in 22.2% of post-HSCT. Thyroxine was given in 1 TDT patient with overt hypothyroidism, and in 3 of 6 post-HSCT patients with persistent subclinical hypothyroidism. The ex-thalassemia patients who underwent HSCT after the age of 10 years had a significantly higher risk of post-HSCT hypothyroidism compared to those who underwent HSCT at the age ≤10 years (hazard ratio: 12.01, 95% confidence interval: 1.65–87.41; p = 0.014). In conclusion, hypothyroidism was found to be common in both TDT and post-HSCT patients. Subclinical hypothyroidism without symptoms and signs was the most common type, and was diagnosed only by TFT screening. Long-term regular surveillance of TFT should be performed in both groups of patients.

摘要

对输血依赖性血红蛋白 E/β-地中海贫血 (TDT) 患儿甲状腺功能减退症的研究，尤其是接受造血干细胞移植 (HSCT) 的患儿甲状腺功能减退症的研究是有限的。我们对 2003 年 10 月至 2019 年 3 月期间在泰国诗里拉吉医学院医院接受定期输血的 25 岁以下 TDT 患者与接受 HSCT 的患者的甲状腺功能测试 (TFT) 结果进行了纵向回顾性分析。50 名患者（23 TDT，27 HSCT）被包括在内。最后一次随访的平均年龄分别为 20.1 ± 2.8 岁和 14.5 ± 4.61 岁。HSCT 的中位年龄为 6 岁（范围：1.9-13.7）。TDT 和 HSCT 后甲状腺功能减退症的患病率分别为 47.8% 和 52.2%。没有研究患者表现出甲状腺功能减退症的症状或体征。亚临床甲状腺功能减退症是最常见的类型（TDT 的 63.6%，HSCT 后的 100%）。我们在 30.4% 的 TDT 和 22.2% 的 HSCT 后发现持续性甲状腺功能减退症。1 名患有明显甲状腺功能减退症的 TDT 患者和 6 名患有持续性亚临床甲状腺功能减退症的 HSCT 后患者中的 3 名患者接受了甲状腺素治疗。与 ≤10 岁时接受 HSCT 的患者相比，10 岁后接受 HSCT 的前地中海贫血患者发生 HSCT 后甲状腺功能减退症的风险显着更高（风险比：12.01，95% 置信区间：1.65–87.41 ;p  = 0.014）。总之，发现甲状腺功能减退症在 TDT 和 HSCT 后患者中很常见。无症状和体征的亚临床甲状腺功能减退症是最常见的类型，仅通过 TFT 筛查即可确诊。两组患者均应长期定期监测 TFT。