Efficacy and safety of pralsetinib in multiple endocrine neoplasia type 2-associated pheochromocytoma: a case report

A 34-year-old male developed bilateral recurrent pheochromocytomas 10 years after laparoscopic adrenal-sparing surgery for pheochromocytomas. Based on his clinical manifestations and germline REarranged during Transfection (RET) variant, the patient was ultimately diagnosed with multiple endocrine neoplasia type 2B. Based on drug susceptibility testing results from organoid-guided precision therapy, the patient underwent secondary adrenalectomy for the right lesion and received oral pralsetinib to control the left ones. Over 16 months of pralsetinib therapy, we found that the patient achieved sustained therapeutic benefits, specifically characterized by symptomatic relief, significant reduction in hormone levels, and shrinkage of the left adrenal masses. These findings indicate that pralsetinib is effective and safe for treating pheochromocytomas associated with RET missense mutation, but further clinical practices are warranted to confirm its efficacy and safety profile.