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Hereditary leiomyomatosis and renal cell cancer (HLRCC)

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A young woman with numerous painful skin nodules (leiomyomas) and uterine fibroids requiring early hysterectomy is found to have an aggressive renal tumor. Genetic testing reveals a FH variant.

AD; FH (fumarate hydratase)

  • Krebs cycle enzyme; tumor suppressor
  • Cutaneous leiomyomas (painful skin nodules)
  • Uterine leiomyomas (fibroids, often early/severe)
  • Renal cell carcinoma (type 2 papillary, aggressive)
    • Often unilateral, solitary
    • Highly aggressive - metastatic even when small
  • Reed syndrome (cutaneous + uterine leiomyomas)
  • Clinical suspicion from multiple cutaneous leiomyomas (skin biopsy confirms), early/severe uterine leiomyomas, or a type 2 papillary RCC
  • Tumors and leiomyomas show loss of FH activity; supportive immunohistochemistry includes loss of FH staining and nuclear accumulation of 2-succinocysteine (2SC)
  • Confirm with germline FH sequencing and deletion/duplication analysis (biallelic FH variants instead cause fumarase deficiency, a severe neurometabolic disorder)
  • Annual renal imaging (MRI preferred)
  • Aggressive treatment of any renal tumor (even small)
  • Dermatologic and gynecologic surveillance
  • No nephron-sparing surgery (due to aggressive nature)

HLRCC predisposes to "FUMAR": Fibroid Uterus (uterine leiomyoma, ~100%), Moles (skin leiomyoma, ~75%), Adrenal nodules (a rarer, less-established FH-related finding; FH is also a recognized pheochromocytoma/paraganglioma gene), Renal cell carcinoma (~15%, type 2 papillary). The defining HLRCC triad is cutaneous leiomyomas, uterine leiomyomas, and type 2 papillary RCC.

"Fibroids and Fee-o (pheo) in FH": FH = fumarate hydratase. Your kidney helps regulate hydration/volume status, so RCC is associated with fumarate hydratase deficiency.

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