Department of Pediatrics, Endocrine Unit, Vita-Salute San Raffaele University, IRCSS San Raffaele, Milan, Italy.
Sant'Orsola-Malpighi University Hospital, Pediatric Unit, Program of Pediatric Endocrinology, Bologna, Italy.
Eur J Endocrinol. 2021 Nov 30;186(1):45-52. doi: 10.1530/EJE-20-1444.
Oral solution and tablet formulations of levothyroxine (L-T4) are both used in the treatment of congenital hypothyroidism (CH). However, few studies and with a limited follow-up period have been published comparing these two formulations in children.
The aim of this multicenter study was to compare the effectiveness of L-T4 oral solution (with ethanol as excipient) and tablet formulation in children with CH up to 3 years of age.
Children diagnosed with CH between 2006 and 2015 were enrolled and divided into two groups according to the L-T4 formulation used: solution in drops (group D) or tablets (group T). Auxological parameters, thyroid-stimulating hormone (TSH) and free thyroxine (FT4) values and L-T4 dose were collected at diagnosis and at 15 days, 1, 3, 6, 12, 24 and 36 months of treatment. The developmental quotient (DQ) at 1 and 3 years of age was evaluated using Griffiths' Scale.
In this study, 254 children were enrolled among which 117 were treated with solution and 137 with tablets. Auxological parameters, dose and thyroid function values at diagnosis, 3, 6, 12, 24, 36 months were not significantly different. TSH at 15 days (P = 0.002) and 1 month (P = 0.009) was significantly reduced in group D. At 2-year follow-up, median TSH was significantly lower in group T (P = 0.03). No statistical difference was detected between the median DQ; however, group D showed lower values in the language subscale at 12 months and in eye-hand coordination at 36 months.
Both therapeutic strategies are effective in the treatment of CH. A higher risk of overtreatment in the first months of therapy seems to be associated with oral solution L-T4; therefore, a different strategy should be considered when starting and adjusting the dose. No negative effects on cognitive development were observed. The data obtained are encouraging but long-term follow-up is needed.
左甲状腺素（L-T4）的口服液和片剂制剂均用于治疗先天性甲状腺功能减退症（CH）。然而，很少有研究发表，且随访时间有限，比较了这两种制剂在儿童中的应用。
本多中心研究旨在比较左甲状腺素口服液（含乙醇作为赋形剂）和片剂制剂在 3 岁以下 CH 儿童中的疗效。
2006 年至 2015 年间诊断为 CH 的儿童被纳入研究，并根据所用 L-T4 制剂分为两组：滴剂（D 组）或片剂（T 组）。在诊断时以及治疗 15 天、1、3、6、12、24 和 36 个月时收集体格生长参数、促甲状腺激素（TSH）和游离甲状腺素（FT4）值和 L-T4 剂量。在 1 岁和 3 岁时使用 Griffiths 量表评估发育商（DQ）。
本研究共纳入 254 例儿童，其中 117 例接受口服液治疗，137 例接受片剂治疗。诊断时、3、6、12、24、36 个月时的体格生长参数、剂量和甲状腺功能值无显著差异。D 组治疗 15 天（P=0.002）和 1 个月（P=0.009）时 TSH 显著降低。2 年随访时，T 组 TSH 中位数显著较低（P=0.03）。两组 DQ 中位数无统计学差异；但 D 组在 12 个月时语言分量表和 36 个月时手眼协调分量表得分较低。
两种治疗策略在 CH 治疗中均有效。口服 L-T4 治疗初期似乎存在过度治疗的更高风险；因此，在开始和调整剂量时应考虑不同的策略。未观察到认知发育的负面影响。所获得的数据令人鼓舞，但需要长期随访。
