Temozolomide treatment of a malignant pheochromocytoma and an unresectable MAX-related paraganglioma

Anticancer Drugs. 2018 Jan;29(1):102-105. doi: 10.1097/CAD.0000000000000570.

Abstract

Pheochromocytomas (PCCs) and paragangliomas (PGLs) are neuroendocrine tumors with a strong genetic background. The mainstay of treatment for PCC/PGLs is surgery. However, for unresectable lesions, no curative treatment is currently available. Temozolomide (TMZ) has been shown to determine radiological and biochemical response in malignant PCC/PGLs. We report two cases of PCC/PGLs treated with TMZ. Case 1 is a 51-year-old man with local and distant recurrence (liver and bone metastases) of right adrenal PCC. Case 2 is a 54-year-old woman with a PCC/PGL syndrome caused by a mutation in MAX gene (c.171+1G>A), operated on for bilateral adrenal PCC and presenting with a large unresectable abdominal PGL. Both patients presented hypertension due to catecholamine hypersecretion. TMZ determined radiological response according to RECIST criteria, reduction of urinary catecholamine levels, and controlled hypertension in both patients. Furthermore, the current study demonstrates, for the first time, that MAX-related PGLs are responsive to TMZ.

Publication types

  • Case Reports

MeSH terms

  • Adrenal Gland Neoplasms / drug therapy*
  • Antineoplastic Agents, Alkylating / therapeutic use
  • Basic Helix-Loop-Helix Leucine Zipper Transcription Factors / genetics
  • Dacarbazine / analogs & derivatives*
  • Dacarbazine / therapeutic use
  • Female
  • Humans
  • Male
  • Middle Aged
  • Paraganglioma / drug therapy*
  • Paraganglioma / genetics
  • Pheochromocytoma / drug therapy*
  • Temozolomide

Substances

  • Antineoplastic Agents, Alkylating
  • Basic Helix-Loop-Helix Leucine Zipper Transcription Factors
  • MAX protein, human
  • Dacarbazine
  • Temozolomide