Dataset Information

Medical Therapy with Pasireotide in Recurrent Cushing's Disease: Experience of Patients Treated for At Least 1?Year at a Single Center.

ABSTRACT: Subcutaneous (SC) injection of pasireotide, a somatostatin analog, is approved for the treatment of adults with Cushing's disease (CD) for whom pituitary surgery was unsuccessful or is not an option. We highlight the symptomatic and biochemical improvement of six patients with recurrent CD treated with pasireotide SC at a single center for at least 1?year. Patients were treated either through commercial use (n?=?5) or through the Phase 3 trial (n?=?1; http://ClinicalTrials.gov identifier, NCT00434148; study number, B2305). Most patients (n?=?5) were female, and the mean age at diagnosis was 35.8?years. All patients demonstrated biochemical control at 1?year of treatment. Three of the five real-world patients followed for more than 1?year remain on pasireotide SC and are controlled. Two patients discontinued pasireotide SC; one patient because of persistently elevated urinary-free cortisol levels and gallstones, and the other because of treatment for an unrelated brain tumor. Symptomatic improvement varied, but all patients demonstrated weight loss. Nausea and mild, transient injection-site reactions were the most frequently reported adverse events. Although glycated hemoglobin (HbA1c) increased after treatment initiation, four of five patients maintained HbA1c levels ?7.0% while receiving pasireotide SC and concomitant individualized diabetes medication, if necessary. In patients who discontinued pasireotide SC, HbA1c levels decreased within 6?weeks. This report documents real-world use of pasireotide SC and indicates its effectiveness as a long-term treatment option for patients with CD. Although hyperglycemia was observed in most patients, it was managed with appropriate monitoring and treatment and was reversible upon discontinuation of pasireotide SC.

SUBMITTER: Yedinak CG

PROVIDER: S-EPMC5327352 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Medical Therapy with Pasireotide in Recurrent Cushing's Disease: Experience of Patients Treated for At Least 1 Year at a Single Center.

Yedinak Chris G CG Hopkins Sarah S Williams Jessica J Ibrahim Aly A Cetas Justin Schultz JS Fleseriu Maria M

Frontiers in endocrinology 20170227

Subcutaneous (SC) injection of pasireotide, a somatostatin analog, is approved for the treatment of adults with Cushing's disease (CD) for whom pituitary surgery was unsuccessful or is not an option. We highlight the symptomatic and biochemical improvement of six patients with recurrent CD treated with pasireotide SC at a single center for at least 1 year. Patients were treated either through commercial use (<i>n</i> = 5) or through the Phase 3 trial (<i>n</i> = 1; http://ClinicalTrials.gov iden ...[more]

PMID: 28289402

Similar Datasets

Project description:This study aimed to assess the effectiveness and safety of tocilizumab use for the treatment of active steroid-resistant Graves' orbitopathy (GO). A retrospective longitudinal study was conducted by reviewing the medical records at a single center between November 2009 and December 2018. A total of 114 patients with steroid-resistant Graves' orbitopathy were examined and treated with tocilizumab, of which 54 adults met the inclusion criteria. No concomitant medication for the treatment of orbitopathy was used. The main primary outcomes included changes from baseline in the Clinical Activity Score (CAS) and thyrotropin receptor antibody (TRAb) levels throughout therapy with tocilizumab. The absolute responses to treatment were defined as the achievement of CAS ≤ 1 and TRAb ≤ 10 U/L. A composite ophthalmic score including CAS, proptosis, eyelid retraction, and diplopia was used to evaluate individual improvement in GO. Adverse drug reactions were also assessed. Analysis of the patient's CAS and TRAb levels showed meaningful reductions during tocilizumab treatment. Differences between values at baseline and subsequent time points were statistically significant (p < 0.001 for all comparisons). The absolute CAS response (CAS = 0 or 1) was achieved in 74% (37/50) of patients after the fourth dose of tocilizumab (at week 16), with a TRAb response being achieved in 55% (23/42) of patients. The relative CAS response (reduction ≥ 2 points) was achieved in 90.9% of patients (40/44) after the first dose of tocilizumab (at week 4). Measurements of proptosis (reduction ≥ 2 mm in 78% of patients, 42/54) and eyelid retraction (reduction ≥ 2 mm in 75%, 33/44), and the prevalence of diplopia (improvement in 68%, 19/28) were significantly reduced after the last dose of tocilizumab (p < 0.001 for all comparisons). GO improved in 98% (53/54) of patients when at least two criteria of the composite evaluation were required. Four patients exhibited disease recurrence, defined as an increase in CAS of ≥2 points in the six months following the date of inactivation. Most adverse drug reactions were mild or moderate in severity. In conclusion, our data suggest that a course of at least 4 months (one monthly dose) of tocilizumab therapy provides a significant benefit to patients with active moderate-to-severe steroid-resistant GO.

Project description:Amyloidosis of the gastrointestinal tract, with biopsy-proven disease, is rare. We reviewed a series of patients who presented with biopsy-proven gastrointestinal amyloidosis and report their clinical characteristics, treatments, and survival. This is a retrospective review of data prospectively collected from January 1998 to December 2011 in a tertiary referral center; 2,334 patients with all types of amyloidosis were evaluated during this period. Seventy-six patients (3.2%) had biopsy-proven amyloid involvement of the gastrointestinal tract. Their median age was 61 years (range, 34-79). Systemic amyloidosis with dominant gastrointestinal involvement was present in 60 (79%) patients, whereas the other 16 (21%) patients had amyloidosis localized to the gastrointestinal tract without evidence of an associated plasma cell dyscrasia or other organ involvement. Of the 60 systemic cases, 50 (83%) had immunoglobulin light-chain, five (8%) had familial lysozyme, three (5%) had wild-type transthyretin, and two (3%) had mutant transthyretin amyloidosis. The most frequent symptoms for all patients were weight loss in 33 (45%) and gastrointestinal bleeding in 27 (36%). Incidental identification of amyloidosis on routine endoscopic surveillance played a role in the diagnosis of seven patients with systemic immunoglobulin light-chain, and four patients with immunoglobulin light-chain localized to the gastrointestinal tract. Amyloid protein subtyping was performed in 12 of the cases of localized disease, and all had lambda light chain disease. Of the 50 patients with systemic immunoglobulin light-chain amyloidosis, 45 were treated with anti-plasma cell therapy. The median survival has not been reached for this group. For the 16 patients with localized gastrointestinal amyloidosis, supportive care was the mainstay of treatment; none received anti-plasma cell therapy. All 16 are alive at a median follow-up of 36 months (range, 1-143). Patients with biopsy-proven gastrointestinal amyloidosis often present with weight loss and bleeding. In localized cases, all that underwent typing were due to lambda light chain amyloidosis and none progressed to systemic disease during the period of follow-up. Most patients with systemic disease had immunoglobulin light-chain, and their tolerance of therapy and median survival were excellent. Although a rare manifestation of amyloidosis, staining for amyloid should be considered in patients undergoing gastrointestinal biopsy who have unexplained chronic gastrointestinal symptoms.

Project description:BackgroundPatients with a rectal foreign body (RFB) are still a rare entity in general surgery departments but with an increasing incidence over the last years. This case is sometimes difficult to treat, and due to a lack of standardized treatment options, the aim of the study was to present our clinical experiences with the diagnostic and therapeutic approach to RFBs and a review of the currently available literature.Materials and methodsData were collected retrospectively from the patient's records of 20 patients who were treated due to an RFB between 2006 and 2016. Patient's demographics, circumstances of insertion, inserted objects, clinical presentation, laboratory and imaging results, as well as surgical treatment and duration of hospital stay were analyzed. Additionally, a review of the literature was performed with the search items "rectal foreign body" and "surgical therapy". Because many publications were just case reports, we did not perform a meta-analysis or a systematic review.ResultsTwenty-two cases in 20 patients (80% male) presented to the emergency room. The mean age was 38.5±13.7 years. In 68.2% of the cases, the cause of RFB was due to sexual preferences. The following objects were inserted: six dildos, three vibrators, two bottles, one glass, one deodorant, one apple, one fever thermometer, multiple glass fragments and razor blades in one patient and six unknown objects. For 18 RFBs, manual peranal removal without anesthesia was possible in the emergency room, but two patients required intravenous analgesia. Two patients were transferred to the operating room and the foreign body was removed via the anus under general anesthesia. Open surgery with a laparotomy was necessary for two complicated cases. One patient was in need of surgery due to a vacuum generated by the RFB, whereas the second patient suffered from a sigmoid perforation. In all cases, there was no morbidity or mortality.ConclusionIn most cases, the removal of an RFB can be performed peranally in the emergency room without further complications, therefore representing the therapy of choice for RFB. Only in cases with perforation, acute abdomen, or failed peranal approaches, surgery is indicated to remove the foreign body.

Dataset Information

Medical Therapy with Pasireotide in Recurrent Cushing's Disease: Experience of Patients Treated for At Least 1?Year at a Single Center.

Publications

Medical Therapy with Pasireotide in Recurrent Cushing's Disease: Experience of Patients Treated for At Least 1 Year at a Single Center.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets