WHY ISTURISA

Clinical Studies

ISTURISA efficacy was assessed in two Phase 3 clinical studies.1,2
Select a study to review efficacy data.

Linc 3
Linc 4

LINC 3 was a phase 3 trial that included an open-label, randomized, double-blind, placebo-controlled withdrawal period.1,3

Inclusion criteria1,3

  • Patients aged 18-75 years with a confirmed diagnosis of persistent, recurrent, or de novo (if not surgical candidates) Cushing's syndrome (CS)1,3
  • Confirmed CS as determined by mUFC >1.5 x ULN at screening3
  • Patients with a history of pituitary surgery had to be at least 30 days post surgery1
  • Evidence of a pituitary source of excess ACTH1

Baseline characteristics1,3

  • Mean age at enrollment was 41 years1,3
  • 77% of patients were female1,3
  • 88% of enrolled patients had previous surgery1,3
  • Mean mUFC at baseline was 365 μg/24 hr (~7 x ULN)3
  • Median mUFC was 173 μg/24 hr (~3.5 x ULN)3

ACTH, adrenocorticotropic hormone; mUFC, mean urinary free cortisol; ULN, upper limit of normal.

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Chart Showing the ISTURISA LINC 3 Study Design

Dose was up- or down-titrated every 2 weeks if mUFC was greater than ULN (defined as 138 nmol/24 h or 50 μg/24 h) or if mUFC was lower than LLN (defined as 11 nmol/24 h or 4 μg/24 h), respectively.1

Dose adjustments were permitted during study period 2.1

Patients who did not require further dose increase, tolerated the drug, and had mUFC levels ≤ULN at week 24 were considered responders and eligible to enter the randomized withdrawal period. Patients whose mUFC became elevated during the maintenance period could have their dose increased further, if tolerated, up to 30 mg twice daily. These patients were considered nonresponders and did not enter the randomized withdrawal period, but continued open-label treatment together with the patients who did not achieve normal mUFC levels at week 12.3

Patients remained on assigned dose throughout the randomized withdrawal period if their mUFC levels were WNL. Blinded dose reduction or temporary discontinuation for safety or tolerability issues were permitted; however, dose increases were not permitted. Patients with mUFC levels >1.5 x ULN or who required a dose increase were considered nonresponders and discontinued from the randomized withdrawal period, but allowed to receive open-label treatment during the open-label period.3

LLN, lower limit of normal; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal; WNL, within normal limits.

ISTURISA maintained normalization of mUFC in a majority (86%) of patients in the randomized withdrawal period3

Demonstrated superiority over placebo at maintaining mUFC ≤ULN at the end of the randomized withdrawal period (N=71, Difference 57% [95% CI: 38-76]; P<.001).3

PRIMARY ENDPOINT1,3

Complete responder rate after 8-week randomized withdrawal period (week 34)a

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mUFC, Randomized withdrawal period (week 26-34)

continued Isturisa chart
86%

(31/36) of patients who received ISTURISA maintained mUFC levels ≤ULN

Complete response rate

Value at randomized withdrawal baseline

Last value on randomly assigned treatment

Placebo chart
29%

(10/34) of patients who received placebo maintained mUFC levels ≤ULN

Complete response rate

Value at randomized withdrawal baseline

Last value on randomly assigned treatment

Complete responder rate was the percentage of patients who had mUFC levels ≤ULN (defined as 138 nmol/24 h or 50 μg/24 h) at the end of the randomized withdrawal period (week 34) and who neither discontinued randomized treatment or the study nor had any dose increase above their week 26 dose.1,3

CI, confidence interval; mUFC, mean urinary free cortisol; ULN, upper limit of normal.

ISTURISA may rapidly normalize mUFC levels1,a

More than half of patients maintained normalized mUFC with ISTURISA at the therapeutic dose established during the titration period.1,a

KEY SECONDARY ENDPOINT1,3

Complete responder rate at week 24 (end of maintenance period) without up-titration after week 12b

53%

(72/137; 95% CI, 43.9-61.1) of patients who received ISTURISA maintained mUFC ≤ULN at week 24 without dose up-titration after week 121,3

  • The lower bound of the 95% CI exceeded 30%, the prespecified threshold for statistical significance and minimum threshold for clinical benefit3

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Graph showing individual patient changes in mean urinary free cortisol (mUFC) levels from baseline to Week 24.

SECONDARY ENDPOINT1,c

Irrespective of dose increase

68%

(93/137) of patients achieved mUFC ≤ULN at week 24 (prespecified endpoint)

The ULN for UFC was defined as 138 nmol/24 h or 50 μg/24 h.1

Complete responder rate was the percentage of patients who had mUFC levels ≤ULN (defi ned as 138 nmol/24 h or 50 μg/24 h).1,3

The study was not powered to assess statistical significance in non-key secondary endpoints and no defi nitive conclusions can be drawn.

CI, confidence interval; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal.

Post hoc analysis: additional analyses indicate other signs of improvement with ISTURISA.1

96%

(132/137) of patients achieved cortisol normalization at least once during the study

41 days

The median time to first complete response (mUFC ≤ULN)d was

These data were not included in primary or secondary endpoint analyses and were assessed post hoc; therefore, no definitive conclusions should be drawn.1,3

ISTURISA sustained normal mUFC up to 48 weeks in a majority of patients.3
Mean mUFC decreased rapidly during the titration period, then remained below baseline through week 48.4,a

48-WEEK STUDY Secondary ENDPOINT1,3

(ITT analysis, end of open-label period)

66%

of patients who received ISTURISA maintained mUFC levels ≤ (91/137; 95% CI, 57.9-74.3)3,6

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Graph showing mean mUFC at time posts up to week 48 among all patients who received treatment in the randomized withdrawal period
  • Average daily ISTURISA dose for all patients through 48 weeks was 10 mg/d6,b
  • The study was not powered to assess statistical significance in non-key secondary endpoints and no definitive conclusions can be drawn

Patients who received ISTURISA had an open-label titration period (weeks 1-12), then a maintenance period (weeks 13-24), and a randomized withdrawal period (weeks 26-34). Patients could participate in an open-label period (weeks 34-48) if they had mUFC ≤ULN at week 24 without a dose increase after week 12.1

Among patients who were ineligible for the randomized withdrawal period, the average daily ISTURISA dose was 11 mg/d.6

mUFC, mean urinary free cortisol.

ISTURISA was associated with cardiometabolic, HRQOL, and psychosocial improvements1,3,a

In patients taking ISTURISA, mean improvements were observed from baseline in the following parameters.4,6

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mean improvements were observed from baseline table

The study was not powered to assess statistical significance in non-key secondary endpoints and no definitive conclusions can be drawn.

Because the study allowed initiation of anti-hypertensive and anti-diabetic medications and dose increases in patients already receiving such medications and there was absence of a control group, the individual contribution of ISTURISA or of anti-hypertensive and anti-diabetic medication adjustments cannot be clearly established.3

CushingQoL is a disease-generated questionnaire proven to be a reliable and valid instrument for measuring health-related quality of life in patients with Cushing’s syndrome. Scores correlate with relevant clinical parameters. The CushingQoL is comprised of 12 items that capture patient responses on 7 concepts: daily activities, healing and pain, mood and self-confidence, social concerns, physical appearance, memory and concern about the future.9

BDI-II is a proven valid and reliable 21-item self-report multiple-choice inventory tool used to evaluate depressive states that occur at high prevalence among the medically ill.10

BDI-II, Beck Depression Inventory-II; CS, Cushing’s syndrome; CushingQoL, Cushing’s Quality of Life questionnaire; HbA1c, hemoglobin A1c; HRQOL, health-related quality of life; mUFC, mean urinary free cortisol; ULN, upper limit of normal.

Improvements were maintained for over a year in the majority of patients who entered the 72-week open-label extension (N=106).7,a

LINC 3 EXTENSION RESULTS

Complete responder rate at week 727,a

81%

of patients who entered the extension maintained mUFC ≤ULN at week 72(86/106; 95% CI, 72.4-88.1)7,a

  • Patients who had mUFC ≤ULN (defined as 138 nmol/24 h or 50 μg/24 h) were classified as complete responders7
  • Patients were classified as having a complete response if mUFC ≤ULN and a partial response if mUFC >ULN but with a ≥50% reduction from baseline7

PRESPECIFIED SECONDARY ENDPOINT

Improvements in cardiometabolic-related parameters were maintained through the open-label extension.1,7,a

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  • No new safety signals reported during long-term treatment3
  • 106 of 137 enrolled patients entered the optional extension7
  • Median duration of ISTURISA treatment from baseline to study end for all enrolled patients was 130 weeks with a median dose of 7.4 mg/day7
  • Most hypocortisolism-related AEs emerged during the first 26 weeks of treatment and AEs related to adrenal hormone precursor accumulation were less frequent in the extension7
  • Mean testosterone levels in women increased upon initiation of ISTURISA but tended to return to baseline levels and within the normal range at 72 weeks (0.8 x ULN)8

The study was not powered to assess statistical significance in non-key secondary endpoints and no definitive conclusions can be drawn.

Because the study allowed initiation of antihypertensive and antidiabetic medications and dose increases in patients already receiving such medications and there was absence of a control group, the individual contribution of ISTURISA or of antihypertensive and antidiabetic medication adjustments cannot be clearly established.3

BMI, body mass index; CI, confidence interval; HbA1c, hemoglobin A1c; mUFC, mean urinary free cortisol; ULN, upper limit of normal.



LEARN ABOUT THE SAFETY PROFILE OF ISTURISA FROM THE LINC 3 CLINICAL TRIAL

References: 1. Pivonello R, Fleseriu M, Newell-Price J, et al; LINC 3 investigators. Efficacy and safety of osilodrostat in patients with Cushing’s disease (LINC 3): a multicentre phase III study with a double-blind, randomised withdrawal phase. Lancet Diabetes Endocrinol. 2020;8(9):748-761. doi:10.1016/S2213-8587(20)30240-0 2. Gadelha M, Bex M, Feelders RA, et al. Randomized trial of osilodrostat for the treatment of Cushing disease. J Clin Endocrinol Metab. 2022;107(7):e2882-e2895. doi:10.1210/clinem/dgac178 3. Isturisa. Package insert. Recordati Rare Diseases Inc; 2025. 4. Pivonello R, Fleseriu M, Newell-Price J, et al. Efficacy and safety of osilodrostat in patients with Cushing’s disease (LINC 3): a multicentre phase III study with a double-blind, randomised withdrawal phase. Lancet Diabetes Endocrinol. 2020;8(9)(suppl). doi:10.1016/S2213-8587(20)30240-0 5. 5. Petersenn S, Newell-Price J, Findling JW, et al. High variability in baseline urinary free cortisol values in patients with Cushing’s disease. Clin Endocrinol (Oxf). 2014;80(2):261–269. doi:10.1111/cen.12259 6. Data on file 1. Recordati Rare Diseases Inc; 2019. 7. Fleseriu M, Newell-Price J, Pivonello R, et al. Long-term outcomes of osilodrostat in Cushing’s disease: LINC 3 study extension. Eur J Endocrinol. 2022;187(4):531-541. doi:10.1530/EJE-22-0317 8. Fleseriu M, Biller B, Pivonello R, et al. Osilodrostat is an effective and well tolerated treatment option for patients with Cushing’s disease (CD): final results from the LINC3 study. Abstract presented at: European Congress of Endocrinology 2021; May 22-26, 2021; Abstract OC8.2. 9. Webb SM, Badia X, Barahona MJ, et al. Evaluation of health-related quality of life in patients with Cushing’s syndrome with a new questionnaire. Eur J Endocrinol. 2008;158(5):623-630. doi:10.1530/EJE-07-0762 10. Wang YP, Gorenstein C. Assessment of depression in medical patients: a systematic review of the utility of the Beck Depression Inventory-II. Clinics (Sao Paulo). 2013;68(9):1274-1287. doi:10.6061/clinics/2013(09)15

LINC 4 is a randomized, double-blind, parallel, placebo-controlled clinical trial that evaluated the safety and efficacy of ISTURISA among 73 patients with Cushing's disease (CD) (mUFC >1.3 × ULN).2

Inclusion criteria2

  • Patients aged 18 to 75 years with a confirmed diagnosis of persistent, recurrent, or de novo (if not surgical candidates) CD
  • Confirmed CD as determined by mean of 3 UFC concentrations of >1.3 x ULN
  • Evidence of a pituitary source of excess ACTH

Baseline characteristics2

  • Median age at enrollment was 39 years
  • 84% (n=61/73) of patients were female
  • 88% (n=64/73) of enrolled patients had previous surgery
  • Mean mUFC at baseline was 157 μg/24 h (≈3.1 . ULN)
  • Median mUFC at baseline was 123 ug/24 h
  • Median mUFC at baseline was 123 μg/24 h(≈2.5 . ULN)

ACTH, adrenocorticotropic hormone; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal.

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Chart showing ISTURISA LINC 4 Study Design

Patients started on ISTURISA 2 mg twice daily (baseline median mUFC was 2.5 X ULN [0.7-12.5]) or matching placebo (baseline median mUFC was 2.2 X ULN [0.2-18.9]). Dose adjustments to normalize mUFC or to address safety issues were permitted at week 2, week 5, and week 8 (range 1-20 mg twice a day) based on considerations of all data for each patient, including mUFC level, rate of decrease of mUFC, and tolerability of ISTURISA. ULN was defined as 138 nmol/24 h or 50 μg/24 h, and LLN was defined as 11 nmol/24 h or 4 μg/24 h.2,3

Starting at week 12, all patients restarted open-label ISTURISA (2 mg twice a day unless they were receiving a lower dose), with dose adjustments permitted (range 1 mg every other day to 30 mg twice a day).2

LLN, lower limit of normal; mUFC, mean urinary free cortisol; ULN, upper limit of normal.

ISTURISA rapidly normalized cortisol levels in the majority of patients, and demonstrated superiority over placebo at normalizing cortisol (mUFC ≤ULN) at week 12 (N=73, 95% CI, 7.1-343.2; P<0.0001) 2,a

PRIMARY ENDPOINT2

Proportion of randomized patients with mUFC ≤ULN at week 12. Significantly more patients had mUFC ≤ULN with ISTURISA at week 12 (P<.0001).2

12-week, randomized, double-blind, placebo-controlled period (N=73)²

Among patients who received
ISTURISA

77%

(37/48) achieved
normal mUFC2,b

Among patients who received
placebo

8%

(2/25) achieved
normal mUFC2,b

P<0.0001

(OR 43.4;

95% CI, 7.1-343.2)

Dose adjustments were made at weeks 2, 5, and 8 followed by adjustments approximately every 3 weeks based on considerations of all data for each patient, including mUFC level, rate of decrease of mUFC, and tolerability of ISTURISA.2,b

Note: Adverse events related to hypocortisolism occurred in 15% of patients who received ISTURISA vs 0% of patients who received placebo during titration.2

Patients taking ISTURISA achieved mUFC ≤ULN rapidly, as early as 12 weeks. Among patients who received ISTURISA, 77% (37/48) achieved normal mUFC compared to 8% (2/25) of patients who received placebo (OR 43.4; 95% CI, 7.1-343.2; P<.0001).2

Patients started on ISTURISA 2 mg twice daily (baseline median mUFC was 2.5 X ULN [0.7-12.5]) or matching placebo (baseline median mUFC was 2.2 X ULN [0.2-18.9]). Dose adjustments to normalize mUFC or to address safety issues were permitted at week 2, week 5, and week 8 (range 1-20 mg twice a day) based on considerations of all data for each patient, including mUFC level, rate of decrease of mUFC, and tolerability of ISTURISA. ULN was defined as 138 nmol/24 h or 50 μg/24 h, and LLN was defined as 11 nmol/24 h or 4 μg/24 h.2,4

CI, confidence interval; LLN, lower limit of normal; mUFC, mean urinary free cortisol; OR, odds ratio; ULN, upper limit of normal.

SECONDARY ENDPOINT
The median duration of ISTURISA exposure until first controlled response (mUFC ≤ULN) was 35 days for patients randomized to ISTURISA.2,c

• 4 out of 5 patients on ISTURISA maintained mUFC ≤ULN at week 36 in the open-label period

• The study was not powered to assess statistical significance in secondary endpoints and no definitive conclusions can be drawn

ISTURISA effectively sustained cortisol normalization in a majority of patients2,a

Maintained mUFC ≤ULN for 4 out of 5 patients at week 36 in the open-label period (N=73).2

KEY SECONDARY ENDPOINT2,a

Proportion of patients with mUFC ≤ULN at week 36

81%

of patients who received ISTURISA maintained normalized mUFC (59/73; 95% CI, 69.9-89.1)2

The lower bound of the 95% CI exceeded 30%, the prespecified threshold for statistical significance and minimum threshold for clinical benefit.

mUFC, mean urinary free cortisol; ULN, upper limit of normal; CI, confidence interval.

Key learnings from LINC 4 study2:

Icon of chart with arrow pointing down

ISTURISA provides rapid and sustained normalization of cortisol

Shield Icon

ISTURISA has a demonstrated safety profile

Figure next to checkmarks Icon

Response and tolerability may be managed with closing monitoring and dose adjustments



LEARN ABOUT THE SAFETY PROFILE OF ISTURISA FROM THE LINC 4 CLINICAL TRIAL

References: 1. Pivonello R, Fleseriu M, Newell-Price J, et al; LINC 3 investigators. Efficacy and safety of osilodrostat in patients with Cushing’s disease (LINC 3): a multicentre phase III study with a double-blind, randomised withdrawal phase. Lancet Diabetes Endocrinol. 2020;8(9):748-761. doi:10.1016/S2213-8587(20)30240-0 2. Gadelha M, Bex M, Feelders RA, et al. Randomized trial of osilodrostat for the treatment of Cushing disease. J Clin Endocrinol Metab. 2022;107(7):e2882-e2895. doi:10.1210/clinem/dgac178 3. Fleseriu M, Auchus RJ, Snyder PJ, et al. Effect of dosing and titration of osilodrostat on efficacy and safety in patients with Cushing’s disease (CD): results from two phase III trials (LINC3 and LINC4). Endocr Pract. 2021;27(suppl 6):S112. Abstract #999926. doi:10.1016/j.eprac.2021.04.707 4. Gadelha M, Bex M, Feelders R, et al. Osilodrostat is an effective and well-tolerated treatment for Cushing’s disease (CD): results from a phase III study with an upfront, randomized, double-blind, placebo-controlled phase (LINC 4). J Endocr Soc. 2021;5(suppl 1):A516-A517. doi:10:1210/jendso/bvab048.1055

INDICATIONS AND USAGE

ISTURISA® (osilodrostat) is indicated for the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome for whom surgery is not an option or has not been curative.

IMPORTANT SAFETY INFORMATION

Hypocortisolism: ISTURISA lowers cortisol levels and can lead to hypocortisolism and sometimes life-threatening adrenal insufficiency. Lowering of cortisol can cause nausea, vomiting, fatigue, abdominal pain, loss of appetite, and dizziness. Significant lowering of serum cortisol may result in hypotension, abnormal electrolyte levels, and hypoglycemia.

Hypocortisolism can occur at any time during ISTURISA treatment. Evaluate patients for precipitating causes of hypocortisolism (infection, physical stress, etc.). Monitor 24-hr urine free cortisol, serum or plasma cortisol, and patient’s signs and symptoms periodically during ISTURISA treatment.

Decrease or temporarily discontinue ISTURISA if urine free cortisol levels fall below the target range, there is a rapid decrease in cortisol levels, and/or patients report symptoms of hypocortisolism. Stop ISTURISA and administer exogenous glucocorticoid replacement therapy if serum or plasma cortisol levels are below target range and patients have symptoms of adrenal insufficiency. After ISTURISA interruption or discontinuation, cortisol suppression may persist beyond the 4-hour half-life of ISTURISA.

Educate patients on the symptoms associated with hypocortisolism and advise them to contact a healthcare provider if they occur.

QTc Prolongation: ISTURISA is associated with a dose-dependent QT interval prolongation which may cause cardiac arrhythmias. Perform an ECG to obtain a baseline QTc interval measurement prior to initiating therapy with ISTURISA and monitor for an effect on the QTc interval thereafter.

Correct hypokalemia and/or hypomagnesemia prior to ISTURISA initiation and monitor periodically during treatment with ISTURISA. Use with caution in patients with risk factors for QT prolongation and consider more frequent ECG monitoring.

Elevations in Adrenal Hormone Precursors and Androgens: ISTURISA blocks cortisol synthesis and may increase circulating levels of cortisol and aldosterone precursors and androgens. This may activate mineralocorticoid receptors and cause hypokalemia, edema and hypertension. Hypokalemia should be corrected prior to initiating ISTURISA. Monitor patients treated with ISTURISA for hypokalemia, worsening of hypertension and edema. Inform patients of the symptoms associated with hyperandrogenism and advise them to contact a healthcare provider if they occur.

The most common adverse reactions (incidence >20%) are adrenal insufficiency, fatigue, nausea, headache, edema, decreased appetite, arthralgia, myalgia, and diarrhea.

To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Drug Interactions:

Use in Specific Populations:

Dosage Interruptions and Modifications: If treatment is interrupted, re-initiate ISTURISA at a lower dose when cortisol levels are within target ranges and patient symptoms have been resolved.

ISTURISA® (osilodrostat) tablets, for oral use, is available as 1 mg and 5 mg tablets.

Please see full Prescribing Information.

Patient portrayal.

Meghan, Actor Portrayal
BASELINE CHARACTERISTICS Age: 34 Race: White Height: 5'6" Weight: 80.1 kg (177 lb) BMI: 28.6 kg/m2
Diagnosis Cushing’s disease: recurrent
Time since diagnosis  6 years
Treatment
  • Transsphenoidal surgery was performed within 2 months of diagnosis 
  • Pituitary radiotherapy was performed 2 years ago
Status
  • In remission, but beginning to develop symptoms of recurrence
Symptoms
  • Weight gain in the abdomen
  • Fatigue and sleep disturbances
  • Anxiety and depression
  • Easy bruising
  • Increased thirst and urination
Comorbidities
  • Depression/anxiety
  • Hyperlipidemia
  • Prediabetes
Vital signs and labs Blood pressure: 124/86 mm Hg HR: 72 bpm Total cholesterol: 213 mg/dL (reference range: <200 mg/dL) LDL: 112 mg/dL (reference range: <100 mg/dL) HDL: 45 mg/dL (reference range: >60 mg/dL) Glucose: 123 mg/dL (reference range: <100 mg/dL) HbA1c: 6.2% (reference range: <5.7%) UFC: 217.5 nmol/24 h (78.8 μg/24 h) (reference range: <145 nmol/24 h [approximately 11-53 μg/24 h]) UFC fold ULN: 1.5 x ULN mUFC

Patient portrayal.

Questions to consider when developing a treatment plan

  1. What would be the consequences if this patient’s cortisol levels remain uncontrolled?
  2. How do you evaluate severity of disease in your patients?
  3. What are your treatment goals for this patient?
  4. What do you consider to be the appropriate next therapy option for this patient?
  5. Would you consider ISTURISA an appropriate option for this patient? Why or why not?

BMI, body mass index; bpm, beats per minute; HbA1c, hemoglobin A1c; HDL, high-density lipoprotein; HR, heart rate; LDL, low-density lipoprotein; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal.

Why ISTURISA May be Right for Your Patient

Patient portrayal.

Mike, Actor Portrayal
BASELINE CHARACTERISTICS Age: 34 Race: White Height: 5'10” Weight: 95.9 kg (211 lb) BMI: 30.3 kg/m2
Diagnosis Cushing’s syndrome: treatment naive
Time since diagnosis 7 months
Treatment
  • Exogenous sources of hypercortisolemia were ruled out
  • Standard imaging techniques were unsuccessful in locating the tumor
  • Inferior petrosal sinus sampling indicated an ectopic source
  • Patient is not a candidate for surgical intervention
Status
  • Further imaging investigation is required
  • Patient is experiencing symptoms of hypercortisolemia that may benefit from medication therapy
  • Cardiometabolic parameters are currently not controlled despite lifestyle changes and maximized treatment
Symptoms
  • Weight gain in the abdomen
  • Rounded face
  • Hypertension
  • Diabetes
  • Daytime fatigue and nighttime insomnia
  • Muscle weakness
Comorbidities
  • Hypertension
  • Diabetes
  • Hyperlipidemia
Vital signs and labs Blood pressure: 146/92 mm Hg HR: 68 bpm Total cholesterol: 238 mg/dL (reference range: <200 mg/dL) LDL: 121 mg/dL (reference range: <100 mg/dL) HDL: 42 mg/dL (reference range: >60 mg/dL) Glucose: 136 mg/dL (reference range: <100 mg/dL) HbA1c: 7.6% (reference range: <5.7%) UFC: 304.5 nmol/24 h (110.3 μg/24 h) (reference range: <145 nmol/24 h [approximately 11-53 μg/24 h]) UFC fold ULN: 2.1 x ULN mUFC

Patient portrayal.

Questions to consider when developing a treatment plan

  1. What would be the consequences if this patient’s cortisol levels remain uncontrolled?
  2. What are your treatment goals for this patient?
  3. What do you consider to be the appropriate next therapy option for this patient?
  4. Would you consider ISTURISA an appropriate option for this patient? Why or why not?

BMI, body mass index; bpm, beats per minute; HbA1c, hemoglobin A1c; HDL, high-density lipoprotein; HR, heart rate; LDL, low-density lipoprotein; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal.

Why ISTURISA May be Right for Your Patient

Patient portrayal.

Stephanie, Actor Portrayal
BASELINE CHARACTERISTICS Age: 31 Race: African American Height: 5'4” Weight: 75.0 kg (165 lb) BMI: 28.3 kg/m2
Diagnosis High clinical suspicion of hypercortisolemia in Cushing’s syndrome; further testing is required
Time since diagnosis Pending diagnosis
Notes
  • UFC labs were repeated twice over several weeks with consistent findings of elevated cortisol
  • Exogenous sources of hypercortisolemia were ruled out
  • Imaging investigation is required
Treatment
  • None
Status
  • Patient has seen an endocrinologist to discuss lack of symptom relief despite lifestyle modification and reported that symptoms are worsening
Symptoms
  • Weight gain in the abdomen
  • Weight gain in the face
  • Facial plethora
  • Muscle weakness
  • Menstrual irregularities
  • Fatigue and sleep disturbances
  • Anxiety and depression
Comorbidities
  • Diabetes
  • Polycystic ovarian syndrome
Vital signs and labs Blood pressure: 130/84 mm Hg HR: 86 bpm Total cholesterol: 208 mg/dL (reference range: <200 mg/dL) LDL: 106 mg/dL (reference range: <100 mg/dL) HDL: 58 mg/dL (reference range: >60 mg/dL) Glucose: 131 mg/dL (reference range: <100 mg/dL) HbA1c: 7.2% (reference range: <5.7%) UFC: 246.5 nmol/24 h (89.3μg/24 h) (reference range: <145 nmol/24 h [approximately 11-53 μg/24 h]) UFC fold ULN: 1.7 x ULN mUFC

Patient portrayal.

Questions to consider when developing a treatment plan

  1. What symptoms lead you to a high clinical suspicion of hypercortisolemia in Cushing’s syndrome?
  2. What would be the consequences if this patient’s cortisol levels remain uncontrolled?
  3. What are your treatment goals for this patient?

BMI, body mass index; bpm, beats per minute; HbA1c, hemoglobin A1c; HDL, high-density lipoprotein; HR, heart rate; LDL, low-density lipoprotein; mUFC, mean urinary free cortisol; UFC, urinary free cortisol; ULN, upper limit of normal.

Why ISTURISA May be Right for Your Patient