Quality of Life and Long-term Outcome of Transition-age Patients with Congenital Hypothyroidism Diagnosed by Newborn Screening and Adequately Treated
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 150
- 试验地点
- 2
- 主要终点
- Quality of life of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
研究概览
简要总结
The primary objective of this observational study is the evaluation of the quality of life and long-term outccome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening receiving replacement treatment with levothyroxine.
The secondary objective is to relate the results to the form of hypothyroidism, the initial dose of L-T4, treatment adherence and genetics
详细描述
The study protocol will be performed in 3 different phases:
Step1:
A) Enrollment of patients meeting the following criteria:
- diagnosis of congenital hypothyroidism through neonatal screening adequately treated with L-T4
- confirmation of the diagnosis of hypothyroidism upon re-evaluation of the diagnosis
- age 16-21 years at the time of enrollment
B) Information interview on the study and signing of informed consent by the patient and/or parent/guardian
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of congenital hypothyroidism by neonatal screening
- •Start of L-T4 therapy within the first month of life
- •Confirmation of hypothyroidism condition upon reassessment of diagnosis
排除标准
- •other chronic diseases and/or genetic syndromes
- •family history of neuropsychiatric pathology
- •familial dyslipidemia
结局指标
主要结局
Quality of life of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
时间窗: evaluation of the long term outcomes at the time of enrolment
Administration of the SF36 questionnaire for the evaluation of quality of life in transition-age children with adequately treated congenital hypothyroidism
Auxological outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
时间窗: evaluation of the long term outcomes at the time of enrolment
weigh SDS, height SDS and BMI SDS assessment in transition-age children with adequately treated congenital hypothyroidism
Intellectual outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
时间窗: evaluation of the long term outcomes at the time of enrolment
Assessment of cognitive abilities through the administration of the WAIS IV
Psychopathological condition of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
时间窗: evaluation of the long term outcomes at the time of enrolment
Assessment of psychopathological conditions through the SCL-90 questionnaire
Behovioral outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
时间窗: evaluation of the long term outcomes at the time of enrolment
Administration of the CBCL questionnaire to parents of patients up to 18 years of age to describe the patient's behavior
次要结局
- Correlation between quality of life and type of congenital hypothyroidism(at time of enrollment)
- Correlation between intellectual outcome and type of congenital hypothyroidism(at time of enrollment)
- Correlation between psychopathological evaluation and type of congenital hypothyroidism(at time of enrollment)
- Correlation between behovioral evaluation and type of congenital hypothyroidism(at time of enrollment)
研究者
Prof.ssa Mariacarolina Salerno
Full Professor
Federico II University