Abstract
A phase III study has demonstrated that 6-month pasireotide treatment induced disease control with good safety in 15–26% of patients with Cushing’s disease (CD). The aim of the current study was to evaluate the 6-month efficacy and safety of pasireotide treatment according to the real-world evidence. Thirty-two CD patients started pasireotide at the dose of 600 µg twice a day (bid) and with the chance of up-titration to 900 µg bid, or down-titration to 450 or 300 µg bid, on the basis of urinary cortisol (UC) levels or safety. Hormonal, clinical and metabolic parameters were measured at baseline and at 3-month and 6-month follow-up, whereas tumour size was evaluated at baseline and at 6-month follow-up. At baseline, 31 patients had very mild to moderate disease and 1 patient had very severe disease. Five (15.6%) patients discontinued treatment for adverse events; the remaining 27 patients (26 with very mild to moderate disease and 1 with very severe disease), reached 6-month follow-up. Considering the group of patients with very mild to moderate disease, responsiveness, defined by the normalization (<1 the upper limit of normal range, ULN) or near normalization (>1 and ≤1.1 ULN) of UC levels, was registered in 21 patients (full control in 19 and near control in 2), corresponding to 67.7% and 80.8% according to an “intention-to-treat” or “per-protocol” methodological approach, respectively. Weight, body mass index, waist circumference, as well as total and LDL-cholesterol significantly decreased, whereas fasting plasma glucose and glycated haemoglobin significantly increased. Hyperglycaemia was documented in 81.2%, whereas gastrointestinal disturbances in 40.6% of patients. In conclusion, in the real-life clinical practice, pasireotide treatment normalizes or nearly normalizes UC in at least 68% of patients with very mild to moderate disease, with consequent improvement in weight, visceral adiposity and lipid profile, despite the occurrence or deterioration of diabetes in the majority of cases, confirming the usefulness of this treatment in patients with milder disease and without uncontrolled diabetes.
Highlights
These authors contributed : Rosario Pivonello and Giorgio ArnaldiCushing’s disease (CD) is a rare but severe endocrine disease caused by chronic endogenous glucocorticoid excess due to a corticotroph pituitary tumour [1,2,3,4]
At baseline; these patients had, increased midnight serum cortisol and/or LNSC, together with lack of suppression at the low-dose dexamethasone suppression test and a clinical picture suggestive of disease activity, and a previous diagnosis of CD confirmed by the pathological evidence of corticotroph pituitary tumour at the previous surgery
The results of the current study demonstrated that pasireotide treatment generally at the dose of 450–900 μg bid administered subcutaneously was able to induce disease remission, in terms of control of cortisol secretion, in at least 67.7% of patients with very mild to moderate disease
Summary
These authors contributed : Rosario Pivonello and Giorgio ArnaldiCushing’s disease (CD) is a rare but severe endocrine disease caused by chronic endogenous glucocorticoid excess due to a corticotroph pituitary tumour [1,2,3,4]. Unità Operativa di Endocrinologia, Dipartimento di Medicina, DIMED, Università di Padova, Padova, Italy hypertension, insulin resistance, glucose intolerance or diabetes mellitus (DM) and dyslipidemia, which, together with thromboembolic diathesis, contribute to determine an increased cardiovascular risk; skeletal damage, mainly characterized by osteopenia or osteoporosis, and often complicated with vertebral fractures; susceptibility to infections, which may worsen in generalized sepsis; and neuropsychiatric disorders, which may affect either cognitive or emotional function and episodically induce suicide [1,2,3,4,5] This cohort of clinical manifestations and comorbidities is responsible for the increase in mortality or deterioration of quality of life [2, 4, 6,7,8,9,10]. Pituitary surgery is the primary therapy in the majority of patients with CD, but it is associated with initial remission rate ranging from 25 to
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
