Program on Pediatric Health Services Research, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
BMC Pediatr. 2010 Feb 15;10:9. doi: 10.1186/1471-2431-10-9.
Thyroid hormone treatment in children with congenital hypothyroidism can prevent intellectual disability. Guidelines recommend that children diagnosed with congenital hypothyroidism through newborn screening remain on treatment to at least 3 years of age, after which a trial off therapy can determine which children have transient hypothyroidism. The purpose of this study was to describe the rate at which children with congenital hypothyroidism in the United States discontinue thyroid hormone treatment in early childhood.
Retrospective analysis of the 2002-2006 MarketScan(R) Commercial Claims and Encounters research databases and the 2001-2005 MarketScan Multi-State Medicaid databases. Children were classified as having congenital hypothyroidism based on billing codes and having filled a prescription for thyroid hormone treatment. Kaplan-Meier curve analysis was used to determine discontinuation rates.
There were a total of 412 Medicaid-enrolled children and 292 privately-insured children with presumed congenital hypothyroidism included in this study. The overall birth prevalence of congenital hypothyroidism across both datasets was about 1 per 2,300. By 36 months, the percentage who had discontinued thyroid replacement treatment was 38% (95% Confidence Interval: 32%-44%). Medicaid-enrolled children had a more rapid decline in the first 24 months of treatment compared to those with private insurance (P = 0.02).
More than one-third of children treated for congenital hypothyroidism discontinued treatment within 36 months, which is inconsistent with current guidelines. It is not known how many of these children required continued treatment or experience adverse effects from discontinuation. These findings emphasize the critical need for follow-up systems to monitor the outcome of newborn screening.
甲状腺激素治疗儿童先天性甲状腺功能减退症可预防智力残疾。指南建议通过新生儿筛查诊断为先天性甲状腺功能减退症的儿童至少继续治疗 3 年，然后停止治疗，以确定哪些儿童患有短暂性甲状腺功能减退症。本研究旨在描述美国先天性甲状腺功能减退症儿童在幼儿期停止甲状腺激素治疗的比率。
对 2002-2006 年 MarketScan(R)商业索赔和遭遇研究数据库和 2001-2005 年 MarketScan 多州医疗补助数据库进行回顾性分析。根据计费代码和甲状腺激素治疗处方，将儿童分类为患有先天性甲状腺功能减退症。使用 Kaplan-Meier 曲线分析来确定停药率。
本研究共纳入了 412 名参加医疗补助计划的儿童和 292 名私人保险的儿童，他们被认为患有先天性甲状腺功能减退症。两个数据集的先天性甲状腺功能减退症的总出生患病率约为每 2300 例中有 1 例。到 36 个月时，停止甲状腺替代治疗的百分比为 38%（95%置信区间：32%-44%）。与私人保险相比，参加医疗补助计划的儿童在治疗的前 24 个月内甲状腺替代治疗下降更快（P=0.02）。
超过三分之一接受先天性甲状腺功能减退症治疗的儿童在 36 个月内停止治疗，这与当前指南不一致。目前尚不清楚有多少儿童需要继续治疗或因停药而产生不良反应。这些发现强调了需要建立随访系统来监测新生儿筛查结果的重要性。
