What is the role of pharmacologic therapy in the treatment of hyperthyroidism in McCune-Albright syndrome (MAS)?

Updated: Jan 17, 2019
  • Author: Gabriel I Uwaifo, MD; Chief Editor: George T Griffing, MD  more...
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Answer

As a rule, hyperthyroidism in the setting of MAS is treated with the same medication options as regular hyperthyroidism, including thionamides (eg, propylthiouracil) and methimazole.

Hyperthyroidism due to functional thyroid follicular adenomas can be treated medically. Antithyroid medications can be used to decrease thyroid hormone production. Unlike Graves disease, hyperthyroidism secondary to a GNAS1 mutation is unlikely to go into remission. Therefore, patients probably should use antithyroid drugs indefinitely. A more permanent treatment of the hyperthyroidism, including radioiodine therapy or thyroidectomy, should be considered if a diagnosis of MAS is confirmed.

Hyperthyroidism usually occurs in the context of toxic multinodular goiter. Notably, hyperthyroidism secondary to toxic multinodular goiter is the second most common endocrinopathy in MAS, after precocious puberty. Although radioiodine can be effective in controlling hyperthyroidism, it is a less popular option, because high doses or repeated administration may be necessary. Obvious issues arise with regard to the safety of radioiodine in children, especially in view of the potential for benign and malignant thyroid nodules to develop after radioiodine therapy.


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