Adrenocortical carcinoma (ACC) is a lethal disease with poor prognosis and lack of effective therapeutic options. Systemic treatment is often employed to treat patients with advanced ACC, but outcomes are disappointing. During the last decade, some of the causative genetic mutations in sporadic ACCs have been identified. Molecular analysis has had a significant impact on the understanding of the pathogenetic mechanism of ACC development and the evaluation of prognostic and predictive markers. Preclinical investigations and clinical trials of tyrosine kinase inhibitors and anti-angiogenic compounds have been initiated to seek target therapy of ACCs. This review summarizes the current view of molecular alterations involved in the pathophysiology of adrenocortical carcinogenesis. The rationale for testing targeted therapies of ACC is also presented.
© 2013 John Wiley & Sons Ltd.