Percentage of Patients With Mean Urinary Free Cortisol (UFC) ≤ Upper Limit of Normal (ULN)
Time frame · Baseline, week 12, 24 and 48
Registry description
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
Percentage of Patients Achieving a Reduction of Mean UFC ≥ 50% From Baseline
Time frame · Baseline, week 12, 24 and 48
Registry description
The 24h-UFC concentration results from three samples during screening were averaged to obtain baseline. After baseline, mean 24h UFC was determined at week 24. At Week 4, 8, 16 and 20, mean 24h UFC was determined from two 24 hour urine collections collected on two consecutive days occurring before the visit. At Week 12, 24 and 48, the mean 24h-UFC from three 24 hour urine collections, collected over the week before the visit, was determined. After Week 24, the mean 24h UFC was determined at 12-week intervals until end of study visit, from two 24 hour collections during two consecutive days prior to each respective visit (except at Week 48). UFC was determined by liquid chromatography tandem mass spectroscopy (LC/MS/MS). The normal ranges were determined by the central laboratory's own reference range. All samples, including screening samples, were analyzed by a central laboratory.
Percent Change in Cushing Quality of Life and Work Productivity and Activity Impairment-General Health (WPAI-GH) Scores
Time frame · Baseline, week 12, 24 and 48
Registry description
A 12-item Cushing's syndrome HRQoL questionnaire (CushingQoL, cf. Webb et al 2008) was implemented and patients who completed 9 or more items at a visit were considered evaluable for that visit. The standardized scores were calculated as follows: 1) Obtain raw scores, denoted by X, as the sum of all the ratings on all the HRQoL questions for a single patient and the score can range from 12 (worst HRQoL) to 60 points (best HRQoL). Therefore, the lower the score, greater the negative impact on HRQoL and 2) obtain standardized score, Y, for a single patient
• Y = 100 (X-12) / (60-12) = 100 (X-12)/48. For example, if a patient answers all 12 items with 'Sometimes' or 'Somewhat', X = 36 and Y = 100 ∙ 24/48 = 50 The WPAI-GH questionnaire was used to assess work productivity and activity impairment. However, there was very limited baseline data and therefore the results and outcomes of the objective, 'change from baseline in WPAI-GH scores' are not included.
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Blood Pressure (BP)
Time frame · Baseline, week 12, 24 and 48
Registry description
Standing systolic and diastolic BP based on 1 assessment and sitting systolic and diastolic BP was mean of 3 assessments.
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Pulse
Time frame · Baseline, week 12, 24 and 48
Registry description
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Temperature
Time frame · Baseline week 12, 24 and 48
Registry description
degrees celius
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Body Mass Index (BMI)
Time frame · Baseline, week 12, 24 and 48
Registry description
Percent change in patients reducing by at least one class level. Class levels: \<25.0, 25.0 to \<30.0, ≥ 30.0. Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Weight
Time frame · Baseline, week 12, 24 and 48
Registry description
Clinically relevant threshold (at any time point) was reduction of ≥ 5%
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Muscle Strength
Time frame · Baseline, week 12, 24 and 48
Registry description
Direct observation of ability to stand unaided: 0=able to stand easily with arms extended, 1=able to stand after several efforts without using arms as assistance, 2=able to stand only by using arms as assistance 3=completely unable to stand
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Waist Circumference
Time frame · Baseline, week 12, 24 and 48
Registry description
Clinically relevant threshold (at any time point). Reduction of ≥ 5%, Reduction of ≥ 10%
Percent Change in Cushing's Disease Clinical Signs and Symptoms - Hirsutism
Time frame · Baseline, week 12, 24 and 48
Registry description
Change from baseline is shown as: Percent change from baseline (BL) =((Post BL value - BL value)/ BL value)\*100. Ferriman-Gallway scoring was used: 0=minimum and 36 was maximum in females only.
Percent Change From Baseline in Growth Hormone (GH) Values
Time frame · Baseline, week 12, 24 and 48
Registry description
Descriptive summary of the effect of pasireotide on GH.
Percent Change From Baseline in Insulin Growth Factor - 1 (IGF - 1) Values
Time frame · Baseline, week 12, 24 and 48
Registry description
Descriptive summary of the effect of pasireotide on IGF-1