Pediatric McCune-Albright Syndrome Medication
- Author: Bruce A Boston, MD; Chief Editor: Stephen Kemp, MD, PhD more...
Medication Summary
The focus of therapy in McCune-Albright syndrome is to decrease secretion of the hormones in question. At present, no therapy addresses the underlying molecular problem (ie, the inappropriate activation of the Gsa subunit).
Aromatase inhibitors
Class Summary
These agents are the mainstay of therapy in girls with persistent estradiol elevation.
Testolactone (Teslac)
Inhibits steroid aromatase activity, thus blocking the conversion of testosterone to estradiol, decreasing the production of estrogen in the autonomous follicular cyst. Although fairly effective, the large amount of medication required and the frequency of dosing make compliance difficult. Clinical studies using newer and more potent aromatase inhibitors are currently being proposed.
Anastrozole (Arimidex)
Highly selective aromatase inhibitor that significantly lowers serum estradiol concentrations by inhibiting the conversion of adrenally generated androstenedione to estrone. Daily dosing is convenient; case reports have shown good response; larger studies still needed.
Glucocorticoids
Class Summary
These agents are used for replacement therapy postadrenalectomy in infantile Cushing syndrome.
Hydrocortisone (Hydrocortone, Cortef)
DOC for glucocorticoid replacement because of mineralocorticoid activity and glucocorticoid effects.
Double or triple dose for febrile illnesses. Possible need for up to 10 times maintenance doses if under severe stress due to trauma, critical illness, or surgery.
Mineralocorticoids
Class Summary
These agents are used for replacement therapy postadrenalectomy in infantile Cushing syndrome. They act on fluid and electrolyte balance and enhance sodium reabsorption in the kidney, resulting in expanded extracellular fluid volume. They increase renal excretion of potassium and hydrogen ion.
Fludrocortisone (Florinef)
Partial replacement therapy for primary and secondary adrenocortical insufficiency.
Somatostatin analogues
Class Summary
These agents inhibit growth hormone (GH) secretion and adenoma growth in somatotroph adenomas. Octreotide, like natural somatostatin, inhibits GH secretion, insulin secretion and glucagon secretion. Following intravenous (IV) administration, basal serum GH, insulin, and glucagon levels are lowered. It also inhibits prolactin secretion via vasoactive intestinal peptide-mediated and thyrotropin releasing hormone–mediated secretion of prolactin. They are used in treatment of patients with acromegaly and hormone-secreting tumors.
Octreotide (Sandostatin)
Potent, long-acting analogue of somatostatin. This drug acts at the somatotroph to inhibit release of GH from the pituitary gland.
Dopamine receptor agonists
Class Summary
Bromocriptine and cabergoline have been used as adjunctive therapy to octreotide in the inhibition of GH release from somatotroph adenomas.
Bromocriptine (Parlodel)
Has been successful in further reducing GH levels in acromegalic patients treated with octreotide, although not generally a first-line therapy.
Cabergoline (Dostinex)
Has been successful in further reducing GH levels in acromegalic patients treated with or without octreotide, although not generally a first-line therapy.
Bisphosphonates
Class Summary
These agents are stable analogs of pyrophosphate and potent inhibitors of bone resorption and bone turnover. They are used to prevent the bone resorption and pain of polyostotic fibrous dysplasia
Pamidronate (Aredia)
Has been successful in treating the pain of polyostotic fibrous dysplasia; may have some benefit in increasing bone mineral density as well.
Alendronate (Fosamax)
Has been successful in treating the pain of polyostotic fibrous dysplasia; may have some benefit in increasing bone mineral density as well. Has the benefit of PO administration.
Estrogen receptor antagonists
Class Summary
This is a new therapy for girls with persistent estradiol elevation.
Tamoxifen (Nolvadex)
Competitively binds to estrogen receptor, producing a nuclear complex that decreases DNA synthesis and inhibits estrogen effects. Blocks the end-organ effects of abnormal estrogen exposure in prepubertal girls.
Antithyroid agents
Class Summary
These agents block production of thyroid hormone in functional thyroid nodules.
Methimazole (Tapazole)
Inhibits thyroid hormone by blocking oxidation of iodine in thyroid gland. Used to decrease the production of thyroid hormone in functional thyroid nodules associated with McCune-Albright syndrome. Unlike autoimmune-mediated hyperthyroidism, treatment is likely to be long-term. Consider more permanent solutions (ie, radioactive iodine, surgery).
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