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Cushing's disease is a rare systemic and disabling disease due to oversecretion of adrenocorticotrophic hormone (ACTH) resulting in excess cortisol levels. Diagnosis and treatment are difficult; despite the availability of various pharmaceutical treatment options, there is an ongoing, unmet need for even more effective treatment. Areas covered: The present review aims at providing an overview of available drugs and presenting new developments. Focusing on the pituitary as a target, the review covers compounds targeting pituitary cell signaling or cell cycle control such as heat shock protein inhibitors (e.g. silibinin), histone deacetylase inhibitors (trichostatin A, vorinostat), kinase inhibitors (gefitinib, seliciclib), and others (such as triptolide, AT-101). Levoketoconazole and osilodrostat are in clinical testing and inhibit steroidogenesis. Blockade of ACTH receptor binding at the adrenal level is explained as a theoretical drug target. Inhibition of binding of the glucocorticoid receptor in the peripheral tissue plays a minor role due to its lack of biomonitoring options. Expert opinion: In our opinion, further research and drug development of pituitary-directed targets are necessary. Combination therapies may exert synergistic effects and allow for smaller and better tolerated doses, but more experience and data are needed to guide such treatment schemes.
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Name: Expert opinion on pharmacotherapy
Alongside various physical symptoms, patients with Cushing's disease and Cushing's syndrome display a wide variety of neuropsychiatric and cognitive symptoms, which are indicative of involvement of th...
To investigate whether low-dose mitotane (up to 2 g/day) could be a temporary therapeutic alternative to transsphenoidal surgery (TSS) in pediatric Cushing's disease (CD).
In most clinical series of Cushing's disease (CD), over 80% of patients are women, many of whom are of reproductive age. The year following pregnancy may be a common time to develop CD. We sought to e...
Cushing's syndrome (CS) or hypercortisolism results from disruption of the hypothalamus-pituitary-adrenal (HPA) axis with the resultant increase in the circulating serum and urinary cortisol levels an...
Despite advances in diagnostic and therapeutic approach, Cushing's disease (CD) presents a challenging situation for the treating physician.
The study treatment period is 15 days in length and includes patients with pituitary Cushing's disease who are candidates for surgical intervention as well as and patients who have recurr...
This is a non-interventional, multinational, multi-center post-marketing study, to further document the safety and efficacy of pasireotide s.c. administered in routine clinical practice in...
The study aims to confirm long-term efficacy and safety of LCI699 for the treatment of patients with Cushing's disease. It is a pivotal trial intended to support the registration of LCI69...
Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study will assess the long-term safety and efficacy ...
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disea...
The science concerned with the benefit and risk of drugs used in populations and the analysis of the outcomes of drug therapies. Pharmacoepidemiologic data come from both clinical trials and epidemiological studies with emphasis on methods for the detection and evaluation of drug-related adverse effects, assessment of risk vs benefit ratios in drug therapy, patterns of drug utilization, the cost-effectiveness of specific drugs, methodology of postmarketing surveillance, and the relation between pharmacoepidemiology and the formulation and interpretation of regulatory guidelines. (Pharmacoepidemiol Drug Saf 1992;1(1); J Pharmacoepidemiol 1990;1(1))
Therapies using arts or directed at the senses.
Sampling of blood levels of the adrenocorticotropic hormone (ACTH) by withdrawal of blood from the inferior petrosal sinus. The inferior petrosal sinus arises from the cavernous sinus and runs to the internal jugular vein. Sampling of blood at this level is a valuable tool in the differential diagnosis of Cushing disease, Cushing syndrome, and other adrenocortical diseases.
Diminished or failed response of an organism, disease or tissue to the intended effectiveness of a chemical or drug. It should be differentiated from DRUG TOLERANCE which is the progressive diminution of the susceptibility of a human or animal to the effects of a drug, as a result of continued administration.
Progressive diminution of the susceptibility of a human or animal to the effects of a drug, resulting from its continued administration. It should be differentiated from DRUG RESISTANCE wherein an organism, disease, or tissue fails to respond to the intended effectiveness of a chemical or drug. It should also be differentiated from MAXIMUM TOLERATED DOSE and NO-OBSERVED-ADVERSE-EFFECT LEVEL.
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