Department of Surgical Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
Department of General and Digestive Surgery, Hautepierre Hospital, Strasbourg University, Strasbourg, France.
Surg Oncol. 2019 Jun;29:107-112. doi: 10.1016/j.suronc.2019.04.002. Epub 2019 Apr 15.
Despite being associated with a very poor prognosis, long-term survivors across all series of Desmoplastic Small Round Cell Tumor (DSRCT) have been reported.
To analyze patients 'characteristics associated with a prolonged survival after DSRCT diagnosis.
All consecutive patients treated for DSRCT in nine French expert centers between 1991 and 2018 were retrospectively analyzed. Patients with a follow-up of less than 2 years were excluded and cure defined as being disease-free at least 5 years.
100 pts were identified (median age 25 years, 89% male). 27 had distant metastases at diagnosis and 80 pts underwent upfront chemotherapy (CT). 71 pts were operated, 20 pts without prior CT). Surgery was macroscopically complete (CC0/1) in 50 pts. Hyperthermic intraperitoneal Chemotherapy (HIPEC) was administered during surgery in 15 pts 54 pts had postoperative CT and 26 pts had postoperative whole abdomino-pelvic RT (WAP-RT). After a median follow-up of 103 months (range 23-311), the median overall survival (OS) was 25 months. The 1- year, 3-year and 5-year OS rates were 90%, 35% and 4% respectively. 5 patients were considered cured after a median disease-free interval of 100 months (range 22-139). Predictive factors of cure were female sex (HR = 0.49, p = 0.014), median PCI<12 (HR = 0.32, p = 0.0004), MD Anderson stage I (HR = 0.25, p < 0.0001), CC0/1 (HR = 0.34, p < 0.0001), and WAP-RT (HR = 0.36, p = 0.00013). HIPEC did not statistically improve survival.
Cure in DSRCT is possible in 5% of patients and is best achieved combining systemic chemotherapy, complete cytoreductive surgery and WAP-RT. Despite aggressive treatment, recurrence is common and targeted therapies are urgently needed.
尽管弥漫性固有型小圆细胞肿瘤（DSRCT）的预后非常差，但所有系列报道中都存在长期存活的患者。
分析与 DSRCT 诊断后延长生存时间相关的患者特征。
回顾性分析 1991 年至 2018 年期间在 9 个法国专家中心接受 DSRCT 治疗的所有连续患者。排除随访时间少于 2 年的患者，并将治愈定义为至少 5 年无疾病。
共确定了 100 例患者（中位年龄 25 岁，89%为男性）。27 例患者诊断时有远处转移，80 例患者接受了一线化疗（CT）。71 例患者接受了手术，其中 20 例患者未接受 CT 治疗。50 例患者的手术达到了宏观完全切除（CC0/1）。15 例患者在手术中接受了腹腔内热灌注化疗（HIPEC），54 例患者在术后接受了 CT 治疗，26 例患者接受了全腹盆腔放疗（WAP-RT）。中位随访 103 个月（范围 23-311）后，中位总生存期（OS）为 25 个月。1 年、3 年和 5 年 OS 率分别为 90%、35%和 4%。5 例患者在无疾病间隔中位数为 100 个月（范围 22-139）后被认为治愈。治愈的预测因素为女性（HR=0.49，p=0.014）、中位数 PCI<12（HR=0.32，p=0.0004）、MD 安德森分期 I（HR=0.25，p<0.0001）、CC0/1（HR=0.34，p<0.0001）和 WAP-RT（HR=0.36，p=0.00013）。HIPEC 并未在统计学上改善生存。
在 5%的患者中可以实现 DSRCT 的治愈，最好的方法是结合全身化疗、完全细胞减灭性手术和 WAP-RT。尽管采用了积极的治疗方法，但复发很常见，迫切需要靶向治疗。
