Division of Pediatric Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.
Division of Urology, Department of Surgery, University of Colorado School of Medicine, the Children's Hospital Colorado, Aurora, Colorado.
Cancer. 2020 Dec 1;126(23):5156-5164. doi: 10.1002/cncr.33173. Epub 2020 Sep 14.
To the authors' knowledge, AREN0321 is the first prospective clinical study of pediatric and adolescent renal cell carcinoma (RCC). Goals of the study included establishing epidemiological, treatment, and outcome data and confirming that patients with completely resected pediatric RCC, including lymph node-positive disease (N1), have a favorable prognosis without adjuvant therapy.
From 2006 to 2012, patients aged <30 years with centrally reviewed pathology of RCC were enrolled prospectively.
A total of 68 patients were enrolled (39 of whom were male; median age of 13 years [range, 0.17-22.1 years]). Stage was classified according to the American Joint Committee on Cancer TNM stage seventh edition as stage I in 26 patients, stage II in 7 patients, stage III in 26 patients, and stage IV in 8 patients, and was not available in 1 patient. Sixty patients underwent resection of all known sites of disease, including 2 patients with stage IV disease. Surgery included radical nephrectomy (53 patients [81.5%]), partial nephrectomy (12 patients [18.5%]), and unknown (3 patients [4.4%]). Histology was TFE-associated RCC (translocation-type RCC; tRCC) in 40 patients, RCC not otherwise specified and/or other in 13 patients, papillary RCC in 9 patients, and renal medullary carcinoma (RMC) in 6 patients. Lymph node status was N0 in 21 patients, N1 in 21 patients (tRCC in 15 patients, RMC in 3 patients, papillary RCC in 2 patients, and not otherwise specified and/or other in 1 patient), and Nx in 26 patients. The 4-year event-free survival and overall survival rates were 80.2% (95% CI, 69.6%-90.9%) and 84.8% (95% CI, 75.2%-94.5%), respectively, overall and 87.5% (95% CI, 68.3%-100%) and 87.1% (95% CI, 67.6%-100%), respectively, for the 16 patients with N1M0 disease. Among patients presenting with metastases, 2 of 8 patients (2 of 5 patients with RMC) were alive (1 with disease) at the time of last follow-up, including 1 patient who was lost to follow-up (succinate dehydrogenase deficiency). The predominant RCC subtypes associated with mortality were tRCC and RMC.
Favorable short-term outcomes can be achieved without adjuvant therapy in children and adolescents with completely resected RCC, independent of lymph node status. A prospective study of patients with tRCC and RMC with M1 or recurrent disease is needed to optimize treatment.
据作者所知，AREN0321 是首例儿童和青少年肾细胞癌（RCC）的前瞻性临床研究。该研究的目的包括建立流行病学、治疗和结果数据，并确认完全切除的儿童 RCC 患者，包括淋巴结阳性疾病（N1），无需辅助治疗即可获得良好的预后。
从 2006 年到 2012 年，对经中心病理审查为 RCC 的年龄<30 岁的患者进行前瞻性入组。
共纳入 68 例患者（39 例为男性；中位年龄 13 岁[范围：0.17-22.1 岁]）。根据美国癌症联合委员会 TNM 分期第 7 版，分期为 I 期 26 例、II 期 7 例、III 期 26 例、IV 期 8 例，1 例分期不详。60 例患者行所有已知部位疾病的切除术，包括 2 例 IV 期疾病患者。手术包括根治性肾切除术（53 例[81.5%]）、部分肾切除术（12 例[18.5%]）和未知（3 例[4.4%]）。组织学为 TFE 相关 RCC（易位型 RCC；tRCC）40 例、非特指型 RCC 和/或其他型 13 例、乳头状 RCC 9 例、肾髓质癌（RMC）6 例。淋巴结状态为 N0 21 例、N1 21 例（tRCC 15 例、RMC 3 例、乳头状 RCC 2 例、非特指型和/或其他 1 例）、Nx 26 例。4 年无事件生存率和总生存率分别为 80.2%（95%CI：69.6%-90.9%）和 84.8%（95%CI：75.2%-94.5%），总体和 87.5%（95%CI：68.3%-100%）和 87.1%（95%CI：67.6%-100%），16 例 N1M0 疾病患者。在出现转移的患者中，8 例中有 2 例（5 例 RMC 中有 2 例）在最后一次随访时仍存活（1 例有疾病），其中 1 例失访（琥珀酸脱氢酶缺乏）。与死亡率相关的主要 RCC 亚型为 tRCC 和 RMC。
在完全切除的儿童和青少年 RCC 患者中，即使不进行辅助治疗也可获得良好的短期预后，而与淋巴结状态无关。需要对 tRCC 和 RMC 伴有 M1 或复发性疾病的患者进行前瞻性研究，以优化治疗。
