For patients with advanced adrenocortical carcinoma, cabozantinib shows promising efficacy, according to a study published online April 9 in The Lancet Oncology.

Matthew T. Campbell, M.D., from The University of Texas MD Anderson Cancer Center in Houston, and colleagues conducted a single-arm, Phase II trial in adults with advanced adrenocortical carcinoma who were not candidates for surgery with curative intent and had an estimated life expectancy of at least three months, as well as an Eastern Cooperative Oncology Group performance status of 0 to 2.

Patients were given oral cabozantinib 60 mg daily with the option of dose reduction for management of adverse events. Eighteen patients were enrolled between March 1, 2018, and May 31, 2021, with a median follow-up of 36.8 months.

The researchers found that 13 of the patients (72.2 percent) had progression-free survival at four months, and median progression-free survival was six months. One patient is still receiving treatment. Eleven patients (61 percent) had treatment-related adverse events of grade 3 or worse.

The most common grade 3 adverse events were lipase elevation, elevated γ-glutamyl transferase concentrations, elevated alanine aminotransferase concentrations, hypophosphatemia, and hypertension (17, 11, 11, 11, and 11 percent, respectively). Grade 4 hypertension occurred in one patient (6 percent). There were no treatment-related deaths reported.

"Cabozantinib in advanced adrenocortical carcinoma showed promising activity and a manageable safety profile," the authors write. "The possible immunomodulatory effect of cabozantinib, if proven, could have potential for combination therapy with immunotherapy in patients with adrenocortical carcinoma."

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