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Study · Non-randomized trial · n = 16 · 2017

12 results

Long-term treatment of Cushing's disease with pasireotide: 5-year results from an open-label extension study of a Phase III trial.

Petersenn S, Salgado LR, Schopohl J, Portocarrero-Ortiz L, Arnaldi G, Lacroix A, Scaroni C, Ravichandran S, Kandra A, Biller BMK

Endocrine9 Jun 2017Cited by 41 (Europe PMC)

PMID28597198 (opens in a new tab)PMCIDPMC5486525 (opens in a new tab)DOI10.1007/s12020-017-1316-3 (opens in a new tab)PepBankPUB-28597198
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Summary

BackgroundTreating hypercortisolism in patients with Cushing's disease after failed surgery often requires chronic medication, underlining the need for therapies with favourable long-term efficacy and safety profiles.MethodsIn a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide. Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.ResultsSixteen patients received 5 years of pasireotide treatment. Among these, median (95% confidence interval) percentage change from baseline in mean urinary free cortisol was -82.6% (-89.0, -41.9) and -81.8% (-89.8, -67.4) at months 12 and 60. Eleven patients had mean urinary free cortisol ≤ upper limit of normal at month 60. Improvements in clinical signs were sustained during long-term treatment. The safety profile of pasireotide at 5 years was similar to that reported after 12 months. Fifteen of 16 patients experienced a hyperglycaemia-related adverse event; glycated haemoglobin levels were stable between months 6 and 60. Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.ConclusionsThis represents the longest prospective trial of a medical therapy for Cushing's disease to date. A subset of patients treated with pasireotide maintained biochemical and clinical improvements for 5 years, with no new safety signals emerging. These data support the use of pasireotide as an effective long-term therapy for some patients with Cushing's disease.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study12

12 results from studies that gave the peptide. 12 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In five-year pasireotide completers with Cushing's disease, median urinary free cortisol changed by -82.6% at month 12 (95% CI -89.0 to -41.9).

    In plain wordsIn five-year pasireotide completers with Cushing's disease, median urinary free cortisol decreased by 82.6% at month 12.

    Peptide
    Pasireotide
    Outcome
    DecreaseUrinary free cortisol at month 12
    Study

    n = 16

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Median percent change from baseline in mean urinary free cortisol
    −82.6%
    month 12, patients who received 5 years of pasireotide
    95% CI of median percent change in mean urinary free cortisol
    −89 to −41.9%
    month 12, patients who received 5 years of pasireotide

    Source sentences

    Summary
    Among these, median (95% confidence interval) percentage change from baseline in mean urinary free cortisol was -82.6% (-89.0, -41.9) and -81.8% (-89.8, -67.4) at months 12 and 60.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  2. 02HumanNon-randomized trialEfficacy outcome

    In five-year pasireotide completers with Cushing's disease, median urinary free cortisol changed by -81.8% at month 60 (95% CI -89.8 to -67.4).

    In plain wordsIn five-year pasireotide completers with Cushing's disease, median urinary free cortisol decreased by 81.8% at month 60.

    Peptide
    Pasireotide
    Outcome
    DecreaseUrinary free cortisol at month 60
    Study

    n = 16

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Measurements

    Median percent change from baseline in mean urinary free cortisol
    −81.8%
    month 60, patients who received 5 years of pasireotide
    95% CI of median percent change in mean urinary free cortisol
    −89.8 to −67.4%
    month 60, patients who received 5 years of pasireotide

    Source sentences

    Summary
    Among these, median (95% confidence interval) percentage change from baseline in mean urinary free cortisol was -82.6% (-89.0, -41.9) and -81.8% (-89.8, -67.4) at months 12 and 60.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  3. 03HumanNon-randomized trialEfficacy outcome

    In five-year pasireotide completers with Cushing's disease, eleven had urinary free cortisol at or below the upper normal limit at month 60.

    In plain wordsIn five-year pasireotide completers with Cushing's disease, eleven had normal urinary free cortisol at month 60.

    Peptide
    Pasireotide
    Outcome
    Not applicableNormal urinary free cortisol at month 60
    Study

    n = 16

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Measurements

    Patients with mean urinary free cortisol at or below ULN
    11 patients
    month 60

    Source sentences

    Summary
    Eleven patients had mean urinary free cortisol ≤ upper limit of normal at month 60.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  4. 04HumanNon-randomized trialEfficacy outcome

    In patients with Cushing's disease receiving pasireotide, improvements in clinical signs persisted during long-term treatment.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, improvements in clinical signs persisted during long-term treatment.

    Peptide
    Pasireotide
    Outcome
    Direction not reportedClinical signs
    Study

    n = 16

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Source sentences

    Summary
    Improvements in clinical signs were sustained during long-term treatment.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  5. 05HumanNon-randomized trialSafety outcome

    In patients with Cushing's disease, pasireotide's safety profile at five years was similar to its profile after twelve months.

    In plain wordsIn patients with Cushing's disease, pasireotide's safety profile at five years resembled its profile after twelve months.

    Peptide
    Pasireotide
    Outcome
    Direction not reportedSafety profile at five years
    Study

    n = 16

    Comparator
    Safety profile after 12 months
    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Source sentences

    Summary
    The safety profile of pasireotide at 5 years was similar to that reported after 12 months.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  6. 06HumanNon-randomized trialSafety outcome

    In five-year pasireotide completers with Cushing's disease, fifteen of sixteen experienced a hyperglycaemia-related adverse event.

    In plain wordsIn five-year pasireotide completers with Cushing's disease, fifteen of sixteen experienced an adverse event related to high blood glucose.

    Peptide
    Pasireotide
    Outcome
    Not applicableHyperglycaemia-related adverse events
    Study

    n = 16

    Outcome group
    Adverse events · Endocrine and hormones
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Measurements

    Patients with a hyperglycaemia-related adverse event
    15 patients
    of 16 patients

    Source sentences

    Summary
    Fifteen of 16 patients experienced a hyperglycaemia-related adverse event;
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  7. 07HumanNon-randomized trialEfficacy outcome

    In pasireotide-treated patients with Cushing's disease, glycated haemoglobin remained stable between months 6 and 60.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, glycated haemoglobin remained stable between months 6 and 60.

    Peptide
    Pasireotide
    Outcome
    No significant changeGlycated haemoglobin
    Study

    n = 16

    Comparator
    Month 6
    Outcome group
    Glycemic control · Endocrine and hormones
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Source sentences

    Summary
    glycated haemoglobin levels were stable between months 6 and 60.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  8. 08HumanNon-randomized trialSafety outcome

    In pasireotide-treated patients with Cushing's disease, hyperglycaemia-related adverse events were most likely to first occur by month 6.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, adverse events related to high blood glucose were most likely to first occur by month 6.

    Peptide
    Pasireotide
    Outcome
    Not applicableHyperglycaemia event onset
    Study

    n = 16

    Outcome group
    Adverse events · Endocrine and hormones
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  9. 09HumanNon-randomized trialSafety outcome

    In pasireotide-treated patients with Cushing's disease, bradycardia-related adverse events were most likely to first occur by month 6.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, adverse events related to slow heart rate were most likely to first occur by month 6.

    Peptide
    Pasireotide
    Outcome
    Not applicableBradycardia event onset
    Study

    n = 16

    Outcome group
    Adverse events · Cardiovascular
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  10. 10HumanNon-randomized trialSafety outcome

    In pasireotide-treated patients with Cushing's disease, gallbladder/biliary adverse events were most likely to first occur by month 6.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, gallbladder or bile duct adverse events were most likely to first occur by month 6.

    Peptide
    Pasireotide
    Outcome
    Not applicableGallbladder/biliary event onset
    Study

    n = 16

    Outcome group
    Adverse events · Liver
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  11. 11HumanNon-randomized trialSafety outcome

    In pasireotide-treated patients with Cushing's disease, liver safety adverse events were most likely to first occur by month 6.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, liver safety adverse events were most likely to first occur by month 6.

    Peptide
    Pasireotide
    Outcome
    Not applicableLiver safety event onset
    Study

    n = 16

    Outcome group
    Adverse events · Liver
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.
  12. 12HumanNon-randomized trialSafety outcome

    In pasireotide-treated patients with Cushing's disease, adverse event severity did not tend to worsen over time.

    In plain wordsIn patients with Cushing's disease receiving pasireotide, adverse event severity did not tend to worsen over time.

    Peptide
    Pasireotide
    Outcome
    Direction not reportedAdverse event severity over time
    Study

    n = 16

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · 5 yearsAs given in this study (human).

    Source sentences

    Summary
    Adverse events related to hyperglycaemia, bradycardia, gallbladder/biliary tract, and liver safety were most likely to first occur by month 6; adverse event severity did not tend to worsen over time.
    3 context sentences
    Summary · context
    In a randomised, double-blind study, 162 adult patients with persistent/recurrent or de novo Cushing's disease received pasireotide.
    Summary · context
    Patients with mean urinary free cortisol at/below the upper limit of normal or clinical benefit at month 12 could continue receiving pasireotide during an open-ended, open-label phase, the outcomes of which are described herein.
    Summary · context
    Sixteen patients received 5 years of pasireotide treatment.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:34 UTC

    First added · source update from Europe PMC

Subjects

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