Background:

A multimodal approach is the standard treatment for desmoplastic small round cell tumor (DSRCT); however, many patients are diagnosed with inoperable disease, which leaves chemotherapy as the only treatment option. There are limited data on the effectiveness of palliative chemotherapy, especially when used after first-line treatment. Here, we evaluated the clinical outcomes of patients with DSRCT treated with multiple lines of chemotherapy.

Methods:

We reviewed medical records of 14 patients with pathologically confirmed DSRCT at Asan Medical Center between 2004 and 2018.

Results:

The median age at diagnosis was 25, with males comprising 92.9% of patients. All patients had inoperable disease at presentation and received chemotherapy as the initial treatment. Four patients (28.6%) were treated with surgery, and complete resection was achieved in 1 patient. Median overall survival (OS) was 23.9 months, and 1-, 2-, and 3-year survival rates were 92.9%, 48.6%, and 19.5%, respectively. In patients receiving first- (N = 14), second- (N = 10), and third-line (N = 8) chemotherapy, median time-to-progression was 9.9, 3.5, and 2.5 months, respectively, and the disease control rates were 100%, 88.9%, and 75.0%, respectively. Factors associated with longer OS in the univariable analysis were ⩽2 metastatic sites at presentation (27.0 vs 14.7 months; P = .024) and surgery with intended complete resection (43.5 vs 20.1 months; P = .027).

Conclusions:

Although advanced DSRCT may initially respond to chemotherapy after first-line treatment, the response becomes less durable as the disease progresses. Individualized treatment decisions focused on palliation should be made.

中文翻译：

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多线化疗在晚期促纤维增生性小圆细胞瘤中的作用和临床疗效

背景：

多模式方法是促纤维增生性小圆细胞瘤 (DSRCT) 的标准治疗方法；然而，许多患者被诊断出患有无法手术的疾病，这使得化疗成为唯一的治疗选择。关于姑息性化疗有效性的数据有限，尤其是在一线治疗后使用时。在这里，我们评估了接受多线化疗的 DSRCT 患者的临床结果。

方法：

我们回顾了 2004 年至 2018 年间在牙山医疗中心病理证实的 14 例 DSRCT 患者的病历。

结果：

诊断时的中位年龄为 25 岁，男性占患者的 92.9%。所有患者在就诊时都患有无法手术的疾病，并接受了化疗作为初始治疗。手术治疗4例（28.6%），1例完全切除。中位总生存期 (OS) 为 23.9 个月，1 年、2 年和 3 年生存率分别为 92.9%、48.6% 和 19.5%。在接受一线（N = 14）、二线（N = 10）和三线（N = 8）化疗的患者中，中位进展时间分别为 9.9、3.5 和 2.5 个月，并且疾病控制率分别为100%、88.9%和75.0%。单变量分析中与较长 OS 相关的因素是 2 个转移部位（27.0 对 14.7 个月；P= .024）和预期完全切除的手术（43.5 个月 vs 20.1 个月；P = .027）。

结论：

虽然晚期 DSRCT 最初可能在一线治疗后对化疗产生反应，但随着疾病的进展，反应变得不那么持久。应做出以姑息治疗为重点的个体化治疗决策。