Aloini Maria Elena, Lardo Pina, Maggio Roberta, Matarazzo Iolanda, Berruti Alfredo, Stigliano Antonio
Department of Endocrinology, Department of Clinical and Molecular Medicine, Sant'Andrea University Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome, Rome 00189, Italy.
Department of Surgical and Medical Sciences and Translational Medicine, Sant'Andrea University Hospital, "Sapienza" University of Rome, Rome 00189, Italy.
JCEM Case Rep. 2025 Jul 31;3(9):luaf174. doi: 10.1210/jcemcr/luaf174. eCollection 2025 Sep.
Adrenocortical carcinoma (ACC) is an aggressive tumor; the 5-year overall survival rate for advanced disease is <15%. First-line therapy for advanced disease is mitotane, either as monotherapy or in combination with chemotherapy. Overall survival after these strategies is comparable when used in the appropriate subgroup of patients. Mitotane monotherapy is usually reserved for biologically low-risk tumors, with a reported partial response rate of 13% to 31%. Typically, responses are observed when mitotane plasma levels are >14 mg/L, but sometimes partial and complete responses have been reached with lower levels. No single clinical or pathological factor has been extensively validated to predict the response to mitotane monotherapy. We describe the case of a 45-year-old patient with metastatic, rapidly growing, unresectable ACC who had a remarkable response to mitotane monotherapy. After 4 months, a 45% reduction of the primary tumor and regression of lung metastases were observed, despite plasmatic mitotane levels of 5 mg/L. Mitotane may represent an effective therapy in selected cases of advanced ACC and despite plasma levels <14 mg/L.
肾上腺皮质癌（ACC）是一种侵袭性肿瘤；晚期疾病的5年总生存率低于15%。晚期疾病的一线治疗药物是米托坦，可单药治疗或与化疗联合使用。在合适的患者亚组中使用这些治疗策略后的总生存率相当。米托坦单药治疗通常适用于生物学低风险肿瘤，报道的部分缓解率为13%至31%。通常，当米托坦血浆水平>14mg/L时可观察到缓解，但有时血浆水平较低时也可达到部分缓解和完全缓解。尚未有单一的临床或病理因素被广泛验证可预测对米托坦单药治疗的反应。我们描述了一例45岁转移性、快速生长、不可切除的ACC患者，该患者对米托坦单药治疗有显著反应。4个月后，尽管血浆米托坦水平为5mg/L，但仍观察到原发肿瘤缩小45%且肺转移灶消退。米托坦可能是晚期ACC某些特定病例的有效治疗方法，即使血浆水平<14mg/L。
