Division of Endocrinology, Diabetes and Metabolism, First Department of Pediatrics Medical School, National and Kapodistrian University of Athens, "Aghia Sophia" Children's Hospital, Athens, Greece.
Institute of Child Health, "Aghia Sophia" Children's Hospital, Athens, Greece.
J Pediatr Endocrinol Metab. 2021 Oct 18;35(2):239-247. doi: 10.1515/jpem-2021-0458. Print 2022 Feb 23.
The aim of the current prospective randomized control study was to assess efficacy, safety, and non-inferiority of a new liquid L-thyroxine formulation dissolved in glycerol and water (T4 drops, produced by a Greek pharmaceutical Company, Uni-Pharma, Athens, Greece) in comparison to the standard Tablets form (T4 tablets, Uni-Pharma, Athens, Greece) in the substitutive treatment of children with congenital hypothyroidism (CH).
Thirty-nine children with CH, aged 3-12 years old, were enrolled in the study, after parental Informed Consent has been obtained, while three patients were lost from follow-up. At baseline, all participants had normal thyroid-stimulating hormone (TSH) and Free T4 values. Patients were randomly subdivided according to the assigned treatment in Group A (n=17)-Tablet Form and Group B (n=19)-Liquid Form. TSH and Free T4 levels were evaluated at 0, 2, 4, and 6 months.
TSH values showed a statistically significant difference (p=0.017) between groups only at six months (Group A having higher TSH levels than Group B, albeit within the normal range), while Free T4 levels had no statistical difference throughout the six month study period and were always within the normal range. Moreover, dose adjustments were more frequent in Group A (p=0.038) during the six months. Liquid L-thyroxine substitutive treatment exhibited no statistically significant adverse effects in comparison to the widely used tablets.
Levothyroxine (LT4) as liquid solution formulation is safe and noninferior to the widely used L-thyroxine Tablets, with less need for dose adjustment, and can therefore be safely used in the treatment of children with CH.
本前瞻性随机对照研究旨在评估一种新型液体左旋甲状腺素制剂（由希腊制药公司 Uni-Pharma 生产，以甘油和水溶解，商品名为 T4 滴剂）与标准片剂（同样由 Uni-Pharma 生产，商品名为 T4 片剂）在替代治疗先天性甲状腺功能减退症（CH）儿童方面的疗效、安全性和非劣效性。
本研究共纳入 39 例年龄在 3-12 岁的 CH 儿童，在获得家长知情同意后入组，其中 3 例患儿失访。基线时，所有参与者的促甲状腺激素（TSH）和游离 T4 值均正常。根据分配的治疗方案，将患者随机分为 A 组（n=17，片剂组）和 B 组（n=19，液体组）。分别于 0、2、4 和 6 个月时评估 TSH 和游离 T4 水平。
仅在 6 个月时，两组间 TSH 值存在统计学差异（p=0.017）（A 组 TSH 水平高于 B 组，但仍在正常范围内），而游离 T4 水平在整个 6 个月的研究期间无统计学差异，且始终在正常范围内。此外，A 组在 6 个月期间需要更频繁地调整剂量（p=0.038）。与广泛使用的片剂相比，液体左旋甲状腺素替代治疗未显示出统计学上的不良反应。
与广泛使用的 L-甲状腺素片剂相比，液体 L-甲状腺素制剂（LT4）安全且非劣效，需要调整剂量的情况较少，因此可安全用于治疗 CH 儿童。
