Evaluation of Long-term Benefit-risk Profile of Levothyroxine Treatment in Children With Congenital Hypothyroidism: Influence of Initial Levothyroxine Dose on Neurodevelopmental, Growth, Cardiovascular and Skeletal Outcomes
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 72
- 主要终点
- Linear Growth
研究概览
简要总结
The primary objective of this study is to evaluate the risk-benefit profile of long-term treatment of two different initials treatment schemes with L-T4 on the neurodevelopmental and auxological outcomes in children with congenital hypothyroidism, diagnosed by neonatal screening in order to find the best dose of initial thyroid hormone replacement to assure the best long-term developmental outcome without any adverse effects on auxological, cardiovascular and skeletal outcomes. The secondary objective of the study is to evaluate the role of other factors that, in addition to the initial L-T4 therapy,can influence long-term neurodevelopmental and auxological outcomes as well as the cardiovascular system and bone metabolism outcomes.
详细描述
Seventy-two neonates detected by the neonatal screening program for congenital hypothyroidism were randomly assigned to receive one of the two initial L-T4 replacement dose: Group A received an initial replacement dose of 10-12.5 mcg/kg/die and Group B received an initial replacement dose of 12.6-15 mcg/kg/die. The adequacy of treatment were monitored closely by clinical evaluation and regular measurement of FT4 and TSH. Cognitive development were evaluated through the Griffiths Mental Development Scales at the the age of 2 yeas. Cognitive and behavioural assessment at the age of 4 years were performed using the Wechsler Preschool and Primary scale of Intelligence. Growth were evaluate at the enrolment and at each visit. Skeletal maturation were evaluated at diagnosis and at the age of 1 and 4 years. At the age of 4 years a quantitative ultrasound measurements and cardiovascular evaluation were performed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Days 至 29 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Congenital Hypothyroidism diagnosed by neonatal screening program
- •Age less than 30 days at diagnosis
- •TSH value at confirmatory diagnosis above 30 mU/l
- •Caucasian ethnicity
排除标准
- •Prematurity
- •Major congenital malformations
- •Neonatal diseases
- •Chromosomopathies
- •Known maternal thyroid diseases
研究组 & 干预措施
10-12.5 μg/kg/day of L-T4
Children with congenital hypothyroidism who received an initial L-T4 dose of 10-12.5 μg/kg/day.
干预措施: Levothyroxin (Drug)
12.6-15 μg/kg/day of L-T4
Children with congenital hypothyroidism who received an initial L-T4 dose of 12.6-15 μg/kg/day.
干预措施: Levothyroxin (Drug)
结局指标
主要结局
Linear Growth
时间窗: 7-10 days after the start of treatment and at 1.5, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 months of life.
Linear growth was evaluated periodically during the study through the measurement of length up to 3 years and height subsequently. Length and height were measured in centimeters and are expressed as standard deviation score.
Neurodevelopmental outcomes
时间窗: At four years of age
Cognitive and behavioral assessment was performed using Wechsler Preschool and Primary scale of Intelligence (WIPPSI-III). The WIPPSI-III evaluates the intelligence of children between 2.6 and 7.3 years and provides a Total Intelligence Quotient, a Verbal Intelligence Quotient, a Performance Intelligence Quotient and a Processing Speed Quotient. Quotients \> 85 are considered normal.
次要结局
未报告次要终点
研究者
Prof.ssa Mariacarolina Salerno
Associate Professor of Pediatrics, Head of Pediatric Endocrinology Unit
Federico II University