Department of Radiology, Sapienza University of Rome, Viale Regina Elena, Rome, Italy.
Urology. 2013 Aug;82(2):382-6. doi: 10.1016/j.urology.2013.04.015. Epub 2013 Jun 22.
To collect and analyze clinical and pathological features of primitive neuroectodermal tumor (PNET)/Ewing sarcoma (EWS), a rare tumor occurring most commonly in bone and soft tissues of young people, which rarely occurs as a primary renal neoplasm and exhibits highly aggressive biological behavior.
All cases of PNET/EWS published from 1975 to February 2012 were collected. When available, clinical and pathological data were extracted for each case. Survivals were estimated with the Kaplan-Meier method and compared with the log-rank test with 95% confidence interval (CI).
A total of 116 cases were found. All patients had clinical symptoms as first presentation of disease such as pain (54%), hematuria (29%), and bulky renal mass (28%). Sixty-six percent of patients had stage IV disease at diagnosis. Median disease-free survival (DFS) was 5.0 months (95% CI 2.4-7.6). The probability to be alive at 18 months was 60% and 85% for patients with metastatic disease (M1) or not (M0) at diagnosis, respectively. Median overall survival (OS) was 24 months (95% CI 4.5-15.1) in patients with M1 disease, whereas it was not reached in patients with M0 disease (P <.001). In patients with M0 disease, 50% received neoadjuvant chemotherapy and the 12-month OS was 93% compared to 75% of untreated patients (P = .092). In patients with M1 disease who underwent treatment, the median progression-free survival (PFS) was 22.0 months (95% CI 17.9-26.1) with a clinical benefit in 74% of cases.
Our findings suggest that PNET/EWS is a rare aggressive tumor affecting principally young people, with a poor prognosis for patients with M1 disease; chemotherapy is an effective strategy in M1 disease and probably also in M0 disease.
收集和分析原始神经外胚层肿瘤（PNET）/尤文肉瘤（EWS）的临床和病理特征，这是一种罕见的肿瘤，主要发生在年轻人的骨骼和软组织中，很少作为原发性肾肿瘤发生，并表现出高度侵袭性的生物学行为。
收集了 1975 年至 2012 年 2 月期间发表的所有 PNET/EWS 病例。在有条件的情况下，从每个病例中提取临床和病理数据。采用 Kaplan-Meier 法估计生存率，并采用对数秩检验进行比较，置信区间为 95%。
共发现 116 例患者。所有患者均以疾病首发症状就诊，如疼痛（54%）、血尿（29%）和巨大肾肿块（28%）。66%的患者在诊断时为 IV 期疾病。无疾病进展生存（DFS）的中位数为 5.0 个月（95% CI 2.4-7.6）。诊断时患有转移疾病（M1）或无转移疾病（M0）的患者，18 个月时的存活率分别为 60%和 85%。患有 M1 疾病的患者中位总生存（OS）为 24 个月（95% CI 4.5-15.1），而 M0 疾病的患者未达到（P<0.001）。在 M0 疾病的患者中，50%接受了新辅助化疗，12 个月的 OS 为 93%，而未接受治疗的患者为 75%（P=0.092）。在接受治疗的 M1 疾病患者中，中位无进展生存（PFS）为 22.0 个月（95% CI 17.9-26.1），74%的患者有临床获益。
我们的研究结果表明，PNET/EWS 是一种罕见的侵袭性肿瘤，主要影响年轻人，M1 疾病患者预后较差；化疗是 M1 疾病的有效治疗策略，可能也是 M0 疾病的有效策略。
