Ahead of Print: High Response Rates and Prolonged Survival in Patients with CAPTEM

Background and Importance: Rarely, corticotrophic pituitary tumors take on an aggressive form characterized by rapid growth, invasion into local structures, compression of cranial nerves, and possible spread to distant sites. When conventional surgery, radiation therapy and hormones fail to control progression and symptoms, alternative therapies are needed. A novel chemotherapeutic regimen of capecitabine and temozolomide (CAPTEM), originally designed in our laboratory, demonstrated dramatic anti-neoplastic effects against corticotrophic pituitary tumors.

Clinical Presentation: Here we present a case series of four patients with aggressive, ACTH-producing pituitary tumors who had previously depleted all surgical, radiation, and hormonal therapies, and were then treated with CAPTEM. Dramatic clinical improvements in neurological deficits and Cushing’s symptoms were evident in all patients after treatment was initiated. Confirmed by radiographic imaging, two of four patients demonstrated complete regression of disease, one patient had a 75% regression, and the fourth patient has ongoing stable disease for over 4.5 years at the time of this writing. Immunohistochemical analysis of patient’s tumor samples showed low O6-methyguanyl methyltransferase (MGMT) expression and adequate levels of mismatch repair enzymes (MLH-1, MSH-2, MSH-6, and PMS-2), which are important for the in vivo efficacy of CAPTEM.

Conclusion: This is the first report of prolonged anti-tumor response and radiographic complete remissions to CAPTEM in patients with aggressive pituitary tumors who had exhausted all other therapies.

From: High Response Rates and Prolonged Survival in Patients With Corticotroph Pituitary Tumors and Refractory Cushing’s Disease From Capecitabine and Temozolomide (CAPTEM): A Case Series by Zacharia et al.

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