Mitotane concentrations influence outcome in patients with advanced adrenocortical carcinoma

Soraya Puglisi, Anna Calabrese, Vittoria Basile, Filippo Ceccato, Carla Scaroni, Barbara Altieri, Silvia Della Casa, Paola Loli, Rosario Pivonello, Maria Cristina De Martino, Letizia Canu, Marco Russo, Giuseppe Badalamenti, Massimo Torlontano, Antonio Stigliano, Francesco Ferraù, Giorgio Arnaldi, Laura Saba, Alessandra Quirino, Paola PerottiPaola Berchialla, Massimo Terzolo

Research output: Contribution to journalArticlepeer-review


Mitotane is the main option of treatment for advanced adrenocortical carcinoma (ACC). However, limited evidence is available regarding the impact of plasma mitotane levels on patient outcome. To address this question, we retrospectively analyzed patients with advanced ACC treated with mitotane for ≥3 months, with ≥3 measurements of plasma mitotane reported in the Lysosafe Online® database (HRA Pharma, France), followed at 12 tertiary centers in Italy from 2005 to 2017. We identified 80 patients, initially treated with mitotane alone (56.2%) or plus chemotherapy (43.8%). The preference toward combination therapy was given to de novo stage IV ACC and younger patients. After the first line of treatment, 25% of valid cases experienced clinical benefit (14.5% objective response, 10.5% stabilization of disease) and 75% progression, without differences between the groups of treatment. Patients with progression had a lower time in the target range (TTR) of plasma mitotane and an unfavorable outcome. Death occurred in 76.2% of cases and multivariate analysis showed that clinical benefit after first treatment and longer TTR were favorable predictors of overall survival (OS). In conclusion, the present findings support the importance of mitotane monitoring and strengthen the concept of a therapeutic window for mitotane.

Original languageEnglish
Article number740
Issue number3
Publication statusPublished - Mar 2020


  • Adrenal cancer
  • Mitotane
  • Prognosis
  • Recurrence
  • Survival

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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