A study has reported that low-dose Lysodren (mitotane) bears the potential to help restore growth rates and pubertal development, and decrease body mass index (BMI) in children suffering with Cushing’s disease who are not eligible for pituitary surgery. This drug is associated with side effects that need close monitoring due to their toxic potential, the study mentioned. The study was titled as 'Mitotane restores growth and puberty in nine children with Cushing’s disease,' and was published in the journal Endocrine Connections. Lysodren exerts a direct toxic effect on the zona reticularis, which is the innermost layer of the adrenal cortex, responsible for producing cortisol.
Cushing's disease is caused by an ACTH-secreting pituitary tumor, known as pituitary adenoma, resulting in excess cortisol levels. Patients show high levels of cortisol, which adversely affects a child’s growth and puberty and interferes with many vital functions of the body. At present, the standard of care for pituitary adenoma treatment is transnasal transsphenoidal surgery, a minimally invasive procedure in which surgical access is made through the nose to remove the pituitary adenoma, with selective microadenomectomy, or removal of the adrenal gland. This is, however, not the best treatment option as between 25-50% of all patients can either not undergo this surgery or remain clinically unaffected by it.
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