Background

Adrenocortical carcinoma (ACC) is a rare and aggressive disease that is often diagnosed at an advanced stage. There is no standard treatment for metastatic ACC; EDP-M (etoposide, doxorubicin, and cisplatin plus mitotane) is one treatment option. A randomized controlled trial (FIRM-ACT) evaluating the efficacy of EDP-M showed progression-free survival (PFS) was 5.0 months, overall survival (OS) was 14.8 months, the response rate was 19%, and adrenal insufficiency occurred in 3.4% of patients. However, the efficacy and safety of this regimen in Asia are not fully reported.

Methods

We retrospectively analyzed 43 patients diagnosed with metastatic ACC at the National Cancer Center Hospital between 1997 and 2020. We evaluated PFS, OS, and response in 17 patients who received EDP-M as first-line therapy.

Results

The median age at treatment initiation was 45 years (range 18–74). Eight patients (47%) had autonomous hormone production, including six patients with hypercortisolism. The best response of partial response and stable disease was seen in two (12%) and ten (59%) patients, respectively. The median PFS was 6.2 months [95% confidence interval (CI): 4.3–10.0]. The median OS was 15.4 months (95% CI 11.6–not reached). Three patients received only one cycle due to adverse effects associated with hypercortisolism. Grade 3/4 adverse events associated with adrenal insufficiency occurred in three (17%) cases, resulting in EDP-M discontinuation.

Conclusions

The EDP-M regimen had similar PFS to that observed in FIRM-ACT. Adrenal insufficiency was more frequent in the current study, but this could be managed with supportive endocrinological care such as cortisol replacement.

中文翻译：

---

转移性肾上腺皮质癌中与依托泊苷、多柔比星和顺铂加米托坦治疗相关的临床管理和结果：单一机构经验

背景

肾上腺皮质癌 (ACC) 是一种罕见的侵袭性疾病，通常在晚期才被诊断出来。转移性 ACC 没有标准治疗方法；EDP​​-M（依托泊苷、多柔比星和顺铂加米托坦）是一种治疗选择。一项评估 EDP-M 疗效的随机对照试验（FIRM-ACT）显示，无进展生存期（PFS）为 5.0 个月，总生存期（OS）为 14.8 个月，缓解率为 19%，肾上腺功能不全发生率为 3.4 % 的患者。然而，该方案在亚洲的疗效和安全性尚未得到充分报道。

方法

我们回顾性分析了 1997 年至 2020 年间在国家癌症中心医院诊断为转移性 ACC 的 43 名患者。我们评估了 17 名接受 EDP-M 作为一线治疗的患者的 PFS、OS 和反应。

结果

开始治疗的中位年龄为 45 岁（范围 18-74 岁）。8 名患者 (47%) 有自主激素产生，包括 6 名皮质醇增多症患者。部分反应和疾病稳定的最佳反应分别见于两名 (12%) 和十名 (59%) 患者。中位 PFS 为 6.2 个月 [95% 置信区间 (CI)：4.3–10.0]。中位 OS 为 15.4 个月（95% CI 11.6——未达到）。由于与皮质醇增多症相关的不良反应，三名患者仅接受了一个周期。与肾上腺功能不全相关的 3/4 级不良事件发生在三例 (17%) 病例中，导致 EDP-M 停药。

结论

EDP​​-M 方案的 PFS 与 FIRM-ACT 中观察到的相似。肾上腺功能不全在当前研究中更为常见，但这可以通过皮质醇替代等支持性内分泌治疗来控制。