Evidence map›Paper›PMID 28597198›Full record

Trial reportEndocrine2017

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

S Petersenn, L R Salgado, J Schopohl, L Portocarrero-Ortiz, G Arnaldi, A Lacroix, C Scaroni, S Ravichandran, A Kandra, B M K Biller

Open access · hybridAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Endocrine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed, 2 pooled it
4.4field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

28 citing papers in PubMed, 2 syntheses or guidelines pooled it, 59 citations in OpenAlex.

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  14. New Trends in Treating Cushing's Disease.TouchREVIEWS in endocrinology · 2024
    Article
  15. Medication-Induced Hyperglycemia and Diabetes Mellitus: A Review of Current Literature and Practical Management Strategies.Diabetes therapy : research, treatment and education of diabetes and related disorders · 2024
    Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 9 institutions in 7 countries.

S PetersennENDOC Center for Endocrine Tumors, Hamburg, Germany. stephan.petersenn@endoc-med.de.
L R SalgadoDivision of General Internal Medicine, Hospital das Clínicas, University of São Paulo Medical School, São Paulo, Brazil.
J SchopohlMedizinische Klinik IV, University of Munich, Munich, Germany.
L Portocarrero-OrtizDepartment of Neuroendocrinology, National Institute of Neurology and Neurosurgery, Mexico City, Mexico.
G ArnaldiDivision of Endocrinology, Polytechnic University of Marche Region, Ancona, Italy.
A LacroixDivision of Endocrinology, Centre hospitalier de l'Université de Montréal, Montreal, Canada.
C ScaroniEndocrinology Unit, Department of Medicine, Padova University Hospital, Padova, Italy.
S RavichandranNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
A KandraNovartis Pharma AG, Basel, Switzerland.
B M K BillerNeuroendocrine Clinical Center, Massachusetts General Hospital, Boston, MA, USA.
Centre Hospitalier de l’Université de Montréal · CAHospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo · BRInstituto Nacional de Neurología y Neurocirugía · MXMarche Polytechnic University · ITMassachusetts General Hospital · USMünchen Klinik · DENovartis (Switzerland) · CHNovartis (United States) · USUniversity of Padua · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

AdolescentAdultAgedFemaleHumansMaleMiddle AgedPituitary ACTH HypersecretionProspective StudiesSomatostatinTreatment OutcomeYoung AdultpasireotideSomatostatinClinical trialCushing’s diseaseLong-termPasireotidePhase III

Identifiers

PMID28597198
PMCPMC5486525
OpenAlexW2624390869

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.