A prospective longitudinal study of Pasireotide in Nelson's syndrome.

Daniel E, Debono M, Caunt S, Girio-Fragkoulakis C, Walters SJ, Akker SA, Grossman AB, Trainer PJ, Newell-Price J

Pituitary1 Jun 2018Cited by 21 (Europe PMC)

PMID29313180PMCIDPMC5942349DOI10.1007/s11102-017-0853-3PepBankPUB-29313180

Summary

PurposeNelson's syndrome is a challenging condition that can develop following bilateral adrenalectomy for Cushing's disease, with high circulating ACTH levels, pigmentation and an invasive pituitary tumor. There is no established medical therapy. The aim of the study was to assess the effects of pasireotide on plasma ACTH and tumor volume in Nelson's syndrome.MethodsOpen labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.ResultsSeven patients had s.c. treatment and 5 proceeded to LAR treatment. There was a significant reduction in morning plasma ACTH during treatment (mean ± SD; 1823 ± 1286 ng/l vs. 888.0 ± 812.8 ng/l during the s.c. phase vs. 829.0 ± 1171 ng/l during the LAR phase, p < 0.0001). Analysis of ACTH levels using a random intercept linear mixed-random effects longitudinal model showed that ACTH (before the morning dose of glucocorticoids) declined significantly by 26.1 ng/l per week during the 28-week of treatment (95% CI - 45.2 to - 7.1, p < 0.01). An acute response to a test dose predicted outcome in 4/5 patients. Overall, there was no significant change in tumor volumes (1.4 ± 0.9 vs. 1.3 ± 1.0, p = 0.86). Four patients withdrew during the study. Hyperglycemia occurred in 6 patients.ConclusionsPasireotide lowers plasma ACTH levels in patients with Nelson's syndrome. A longer period of treatment may be needed to assess the effects of pasireotide on tumor volume.Trial registrationClinical Trials.gov ID, NCT01617733.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

01HumanNon-randomized trialEfficacy outcome
In Nelson's syndrome, mean morning ACTH decreased from 1823±1286 ng/l to 888.0±812.8 ng/l during subcutaneous pasireotide treatment.
In plain wordsIn Nelson's syndrome patients, morning ACTH decreased from baseline during subcutaneous pasireotide treatment.
Peptide
Pasireotide
Outcome
DecreaseMorning ACTH during subcutaneous treatment
Study
n = 7
Outcome group
Endocrine Control · Endocrine and hormones
Studied dose
300-600 μg twice daily · subcutaneous · 1 monthAs given in this study (human).
Measurements
Morning plasma ACTH
1823 ng/l
first value in the three-period comparison; ±1286
Morning plasma ACTH
888 ng/l
subcutaneous phase; ±812.8
P value
< 0.0001
across treatment periods
Source sentences
Summary
There was a significant reduction in morning plasma ACTH during treatment (mean ± SD; 1823 ± 1286 ng/l vs. 888.0 ± 812.8 ng/l during the s.c. phase vs. 829.0 ± 1171 ng/l during the LAR phase, p < 0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318001
02HumanNon-randomized trialEfficacy outcome
In Nelson's syndrome, mean morning ACTH decreased from 1823±1286 ng/l to 829.0±1171 ng/l during pasireotide LAR treatment.
In plain wordsIn Nelson's syndrome patients, morning ACTH decreased from baseline during long-acting pasireotide treatment.
Peptide
Pasireotide
Outcome
DecreaseMorning ACTH during LAR treatment
Study
n = 5
Outcome group
Endocrine Control · Endocrine and hormones
Studied dose
40-60 mg monthly · 6 monthsAs given in this study (human).
Measurements
Morning plasma ACTH
1823 ng/l
first value in the three-period comparison; ±1286
Morning plasma ACTH
829 ng/l
LAR phase; ±1171
P value
< 0.0001
across treatment periods
Source sentences
Summary
There was a significant reduction in morning plasma ACTH during treatment (mean ± SD; 1823 ± 1286 ng/l vs. 888.0 ± 812.8 ng/l during the s.c. phase vs. 829.0 ± 1171 ng/l during the LAR phase, p < 0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318002
03HumanNon-randomized trialEfficacy outcome
In Nelson's syndrome patients receiving pasireotide, pre-glucocorticoid ACTH declined by 26.1 ng/l weekly over 28 weeks (95% CI -45.2 to -7.1, p<0.01).
In plain wordsIn Nelson's syndrome patients, ACTH decreased over time during pasireotide treatment.
Peptide
Pasireotide
Outcome
DecreaseLongitudinal ACTH decline
Study
n: not reported in source
Outcome group
Endocrine Control · Endocrine and hormones
Studied dose
Dose not stated · 28 weeksAs given in this study (human).
Measurements
Weekly change in morning plasma ACTH
−26.1 ng/l per week
pre-glucocorticoid ACTH over 28 weeks of treatment
95% CI of weekly change in ACTH
−45.2 to −7.1 ng/l per week
95% CI
P value
< 0.01
longitudinal mixed-effects model
Source sentences
Summary
Analysis of ACTH levels using a random intercept linear mixed-random effects longitudinal model showed that ACTH (before the morning dose of glucocorticoids) declined significantly by 26.1 ng/l per week during the 28-week of treatment (95% CI - 45.2 to - 7.1, p < 0.01).
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318003
04HumanNon-randomized trialEfficacy outcome
In Nelson's syndrome patients receiving pasireotide, tumor volumes did not change significantly (1.4±0.9 versus 1.3±1.0, p=0.86).
In plain wordsIn Nelson's syndrome patients, tumour volume did not change significantly during pasireotide treatment.
Peptide
Pasireotide
Outcome
No significant changeTumor volume
Study
n: not reported in source
Outcome group
Tumor growth · Cancer
Studied dose
s.c. phase 300-600 μg twice daily; LAR phase 40-60 mg monthly · subcutaneous (s.c. phase); LAR phase route not stated · 1 month s.c. phase, then 6 months LAR phaseAs given in this study (human).
Measurements
Tumor volume, first value
1.4
first of the two compared values
Tumor volume SD, first value
0.9
first of the two compared values
Tumor volume, second value
1.3
second of the two compared values
Tumor volume SD, second value
1
second of the two compared values
P value
0.86
tumor volume comparison
Source sentences
Summary
Overall, there was no significant change in tumor volumes (1.4 ± 0.9 vs. 1.3 ± 1.0, p = 0.86).
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318005
05HumanNon-randomized trialOther
In Nelson's syndrome patients treated with pasireotide, four withdrew during the study.
In plain wordsIn the Nelson's syndrome pasireotide study, four patients withdrew.
Peptide
Pasireotide
Outcome
Not applicableWithdrawal
Study
n: not reported in source
Outcome group
Treatment adherence and retention · Whole body
Studied dose
s.c. phase 300-600 μg twice daily; LAR phase 40-60 mg monthly · subcutaneous (s.c. phase); LAR phase route not stated · 1 month s.c. phase, then 6 months LAR phaseAs given in this study (human).
Measurements
Patients who withdrew
4 patients
during the study
Source sentences
Summary
Four patients withdrew during the study.
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318006
06HumanNon-randomized trialSafety outcome
In Nelson's syndrome patients treated with pasireotide, hyperglycemia occurred in six patients.
In plain wordsIn the Nelson's syndrome pasireotide study, six patients developed high blood sugar.
Peptide
Pasireotide
Outcome
Not applicableHyperglycemia
Study
n: not reported in source
Outcome group
Adverse events · Metabolic and weight
Studied dose
s.c. phase 300-600 μg twice daily; LAR phase 40-60 mg monthly · subcutaneous (s.c. phase); LAR phase route not stated · 1 month s.c. phase, then 6 months LAR phaseAs given in this study (human).
Measurements
Patients with hyperglycemia
6 patients
during the study
Source sentences
Summary
Hyperglycemia occurred in 6 patients.
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318007
07HumanObservational studyEfficacy outcome
In Nelson's syndrome, acute response to a pasireotide test dose predicted outcome in 4/5 patients.
In plain wordsIn Nelson's syndrome patients, response to a pasireotide test dose predicted later outcome in four of five patients.
Peptide
Pasireotide
Outcome
Not applicableAcute response predicts outcome
Study
n = 5
Outcome group
Endocrine Control · Endocrine and hormones
Studied dose
Dose not stated
Measurements
Patients in whom the acute response to a test dose predicted outcome
4 patients
of 5
Source sentences
Summary
An acute response to a test dose predicted outcome in 4/5 patients.
2 context sentences(design, population, supporting numbers)
Summary · context
Open labeled multicenter longitudinal trial in three steps: (1) a placebo-controlled acute response test; (2) 1 month pasireotide 300-600 μg s.c. twice-daily; (3) 6 months pasireotide long-acting-release (LAR) 40-60 mg monthly.
Summary · context
Seven patients had s.c. treatment and 5 proceeded to LAR treatment.
Show in the study text Report a problemFND-2931318004

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

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 stopped early · source ClinicalTrials.gov (opens in a new tab)

TerminatedPhase 2NCT01617733

Pasireotide Therapy in Patients With Nelson's Syndrome

Sheffield Teaching Hospitals NHS Foundation Trustn = 8Mar 2011 to Dec 2015

Nelson Syndrome

Trial page ClinicalTrials.govUpdated 2 Nov 2016

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.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:34 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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