Assessment of CAI in Adults With PWS.
- Conditions
- Central Adrenal InsufficiencyPrader-Willi Syndrome
- Registration Number
- NCT04700644
- Lead Sponsor
- Medical University of Warsaw
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 21
- PWS
- age >=18 years
- age <18 years
- GC treatment at last 6 months
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The prevalence of CAI in adult PWS patients based on HDSST. 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 minuteSymptoms of CAI before hydrocortisone substitution: fatigue Before HCT treatment Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: fatigue 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 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 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 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 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 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 Before HCT treatment Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: myalgia 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 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 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 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 Before HCT treatment Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia 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 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 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 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 Before HCT treatment Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: weight loss 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 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 After 12 month HCT treatment Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: vomiting Before HCT treatment Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: vomiting 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 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 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 After 6 month HCT treatment Measurement of the body fat percentage using bioelectrical impedance method.
Symptoms of CAI before hydrocortisone substitution: abdominal pain 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 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 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 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 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 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 After 12 month HCT treatment Measurement of the body fat percentage using bioelectrical impedance method.
- Secondary Outcome Measures
Name Time Method Concentration of morning cortisol At first visit (before HCT treatment). Results of morning cortisol in the entire study group.
Laboratory findings - short Synacthen test At first visit (before HCT treatment). Results of the short Synacthen test in the entire study group.
Concentration of ACTH At first visit (before HCT treatment). Results of ACTH in the entire study group.
Concentration of dihydroepiandrosterone sulfate At first visit (before HCT treatment). Results of dihydroepiandrosterone sulfate measurements in the entire study group.
Trial Locations
- Locations (1)
Central Clinical Hospital Medical University of Warsaw
🇵🇱Warsaw, Poland