Assessment of Central Adrenal Insufficiency in Adults With Prader-Willi Syndrome.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- The prevalence of CAI in adult PWS patients based on HDSST.
研究概览
简要总结
Introduction: The prevalence and clinical significance of central adrenal insufficiency (CAI) in adult patients with Prader Willi Syndrome (PWS) remains unclear.
Aim: To assess the prevalence of CAI in adults with PWS and to analyse the effects of replacement therapy with hydrocortisone (HCT) in patients with suspected CAI.
Material and Methods: Twenty one adult patients with PWS were evaluated. Based on peak cortisol at the 30 minute of the high dose short Synacthen test (HDSST), patients were divided into three groups: CAI (central adrenal insufficiency) - peak cortisol <500nmol/L, intermediate (partial AI) - peak cortisol ≥500 nmol/L and <600 nmol/L and AS (adrenal sufficiency) - peak cortisol ≥ 600 nmol/L. In patients with diagnosed CAI HCT replacement treatment was initiated. Body weight, body fat percentage, signs, and symptoms of CAI were evaluated after 6 and 12 months of treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 26 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age >=18 years
排除标准
- •age <18 years
- •GC treatment at last 6 months
结局指标
主要结局
The prevalence of CAI in adult PWS patients based on HDSST.
时间窗: At first visit (before HCT treatment).
The diagnosis of CAI was based on the high-dose short Synacthen test (HDSST). The following cut-off points were used: 1. CAI group (suspected CAI): • cortisol \< 500 nmol/L at the 30th minute 2. AS group (adrenal sufficiency - CAI excluded): • cortisol ≥ 600 nmol/L at the 30th minute 3. Intermediate group • cortisol ≥500 nmol/L and \<600 nmol/L at the 30th minute
Symptoms of CAI before hydrocortisone substitution: fatigue
时间窗: Before HCT treatment
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: fatigue
时间窗: After 6 month HCT treatment
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: fatigue
时间窗: After 12 month HCT treatment
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: loss of appetite
时间窗: After 6 month HCT treatment
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: muscle weakness
时间窗: Before HCT treatment
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: loss of appetite
时间窗: Before HCT treatment
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocortisone treatment: muscle weakness
时间窗: After 6 month HCT treatment
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: myalgia
时间窗: Before HCT treatment
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: myalgia
时间窗: After 6 month HCT treatment
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: myalgia
时间窗: After 12 month HCT treatment
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: loss of appetite
时间窗: After 12 month HCT treatment
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocortisone treatment: muscle weakness
时间窗: After 12 month HCT treatment
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: arthralgia
时间窗: Before HCT treatment
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia
时间窗: After 6 month HCT treatment
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: arthralgia
时间窗: After 12 month HCT treatment
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: weight loss
时间窗: After 6 month HCT treatment
Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: weight loss
时间窗: After 12 month HCT treatment
Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: nausea
时间窗: Before HCT treatment
Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: weight loss
时间窗: Before HCT treatment
Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: nausea
时间窗: After 6 month HCT treatment
Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: nausea
时间窗: After 12 month HCT treatment
Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: vomiting
时间窗: Before HCT treatment
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: vomiting
时间窗: After 6 month HCT treatment
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: vomiting
时间窗: After 12 month HCT treatment
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Body fat percentage before hydrocortisone substitution in PWS patients with CAI
时间窗: Before HCT treatment
Measurement of the body fat percentage using bioelectrical impedance method.
Body fat percentage after 6 month hydrocortisone substitution in PWS patients with CAI
时间窗: After 6 month HCT treatment
Measurement of the body fat percentage using bioelectrical impedance method.
Symptoms of CAI before hydrocortisone substitution: abdominal pain
时间窗: Before HCT treatment
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: abdominal pain
时间窗: After 6 month HCT treatment
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: abdominal pain
时间窗: After 12 month HCT treatment
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
BMI before hydrocortisone substitution in PWS patients with CAI
时间窗: Before HCT treatment
Measurement of weight and height will be combined to report BMI in kg/m\^2.
BMI after 6 month hydrocortisone substitution in PWS patients with CAI
时间窗: After 6 month HCT treatment
Measurement of weight and height will be combined to report BMI in kg/m\^2.
BMI after 12 month hydrocortisone substitution in PWS patients with CAI
时间窗: After 12 month HCT treatment
Measurement of weight and height will be combined to report BMI in kg/m\^2.
Body fat percentage after 12 month hydrocortisone substitution in PWS patients with CAI
时间窗: After 12 month HCT treatment
Measurement of the body fat percentage using bioelectrical impedance method.
次要结局
- Concentration of ACTH(At first visit (before HCT treatment).)
- Concentration of morning cortisol(At first visit (before HCT treatment).)
- Laboratory findings - short Synacthen test(At first visit (before HCT treatment).)
- Concentration of dihydroepiandrosterone sulfate(At first visit (before HCT treatment).)
研究者
Magda Goralska
Principal Investigator
Medical University of Warsaw