ICEECE2012 Poster Presentations Adrenal cortex (113 abstracts)
Efficacy of long-term treatment with retinoic acid in patients with Cushing’s disease
F. Pecori Giraldi 2, , A. Ambrogio 2 , M. Andrioli 1 , F. Sanguin 3 , I. Karamouzis 4 , S. Corsello 5 , C. Scaroni 3 , E. Arvat 4 , A. Pontecorvi 5 & F. Cavagnini 1
1Istituto Auxologico Italiano, Milan, Italy; 2University of Milan, Milan, Italy; 3University of Padua, Padua, Italy; 4University of Turin, Turin, Italy; 5Catholic University of Rome, Rome, Italy.
Cushing’s disease, i.e. cortisol excess due to an ACTH-secreting pituitary adenoma, is a rare disorder with considerable morbidity and mortality. Current therapeutic strategies include pituitary surgery, radiation, adrenalectomy and medical treatment with steroidogenesis inhibitors, as drugs aimed at the pituitary adenoma are as yet under investigation. Experimental data showed that retinoic acid restrains ACTH secretion by tumoral corticotrophes thus we decided to evaluate the efficacy of retinoic acid treatment in patients with Cushing’s disease.
Methods: Seven patients with Cushing’s disease (two men, five postmenopausal women) were started on 10 mg retinoic acid daily and dosage increased up to 80 mg/day for 6–12 months. ACTH, UFC and cortisol as well as clinical features of hypercortisolism were evaluated at baseline, during retinoic acid administration and after drug withdrawal.
Results: A marked decrease in UFC levels was observed in five patients: mean UFC levels on retinoic acid were 47–79% of baseline values and normalization in UFC was achieved in four patients. Plasma ACTH decreased by 10–35% in four responsive patients, including patients in whom UFC normalized while levels increased up to 130% of pretreatment values in one patient. Serum cortisol was unchanged during retinoic acid treatment except for a 40% decrease observed in one patient. Blood pressure, glycaemia and symptoms of hypercortisolism, e.g. facial plethora, ameliorated to a variable extent on treatment. Patients reported mild adverse effects, e.g. xerophthalmia, arthralgias.
Conclusions: Prolonged treatment with retinoic acid proved beneficial and well tolerated in five out of seven patients with Cushing’s disease. This represents a novel, promising approach to medical treatment with Cushing’s disease.
This study was funded by AIFA grant #FARM78F3YR.
Declaration of interest: The authors declare that there is no conflict of interest that could be perceived as prejudicing the impartiality of the research project.
Funding: This work was supported, however, funding details unavailable.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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