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A 25-year-old woman presents with a thyroid nodule. Biopsy shows medullary thyroid carcinoma. Calcitonin is markedly elevated. Genetic testing reveals a RET pathogenic variant. Her 5-year-old son tests positive for the same variant and is referred for prophylactic thyroidectomy.

AD; RET proto-oncogene (gain-of-function)

  • Genotype-phenotype correlation determines management
  • Specific RET codon variants predict disease severity and timing

Subtypes:

FeatureMEN2A (~95%)MEN2B (~5%)
MTCNearly 100%Nearly 100% (earlier, more aggressive)
Pheochromocytoma~50%~50%
Parathyroid hyperplasia20-30%Rare
Mucosal neuromasNoYes (lips, tongue, eyelids)
Marfanoid habitusNoYes
Intestinal ganglioneuromatosisNoYes
  • Medullary thyroid carcinoma (MTC): nearly 100%; arises from parafollicular C cells; calcitonin is the tumor marker
  • Pheochromocytoma: bilateral in ~50%; screen before any surgery
  • Prophylactic thyroidectomy based on RET variant:
    • Highest risk (M918T/MEN2B): thyroidectomy within first 6 months of life
    • High risk (C634R): thyroidectomy by age 5
    • Moderate risk: thyroidectomy can be guided by calcitonin levels
  • RET genetic testing
  • Calcitonin and CEA for MTC screening
  • Plasma/urine metanephrines and catecholamines for pheochromocytoma
  • Always screen for pheochromocytoma BEFORE surgery
  • Risk-stratified prophylactic thyroidectomy
  • Annual biochemical screening for pheochromocytoma
  • Calcium/PTH monitoring for hyperparathyroidism (MEN2A)
  • Genetic testing of at-risk family members

"Piter Pan's papa pheels thyr'd and mucousy": MEN1 = Pituitary, pancreas, parathyroid. MEN2A = parathyroid, pheo, thyroid. MEN2B = pheo, thyroid, mucosal neuromas.

Mutations in RET are associated with ThyRoid cancer: RET is a proto-oncogene (gain-of-function), unlike most cancer predisposition genes which are tumor suppressors (loss-of-function). 50% of MEN2B occurs de novo.

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