TY - JOUR
T1 - A prospective study of pediatric and adolescent renal cell carcinoma
T2 - A report from the Children's Oncology Group AREN0321 study
AU - the Children's Oncology Group (COG) Renal Tumor Committee
AU - Geller, James I.
AU - Cost, Nicholas G.
AU - Chi, Yueh Yun
AU - Tornwall, Brett
AU - Cajaiba, Mariana
AU - Perlman, Elizabeth J.
AU - Kim, Yeonil
AU - Mullen, Elizabeth A.
AU - Glick, Richard D.
AU - Khanna, Geetika
AU - Daw, Najat C.
AU - Ehrlich, Peter
AU - Fernandez, Conrad V.
AU - Dome, Jeffrey S.
N1 - Funding Information:
Supported by grants U10CA180886, U10CA180899, U10CA098543, U10CA098413, and U24CA196173 from the National Cancer Institute of the National Institutes of Health and the St. Baldrick's Foundation. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
Publisher Copyright:
© 2020 American Cancer Society
PY - 2020/12/1
Y1 - 2020/12/1
N2 - Background: 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. Methods: From 2006 to 2012, patients aged <30 years with centrally reviewed pathology of RCC were enrolled prospectively. Results: 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. Conclusions: 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.
AB - Background: 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. Methods: From 2006 to 2012, patients aged <30 years with centrally reviewed pathology of RCC were enrolled prospectively. Results: 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. Conclusions: 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.
KW - adjuvant therapy
KW - nephrectomy
KW - pediatric renal cell carcinoma
KW - renal medullary carcinoma
KW - translocation renal cell carcinoma
UR - http://www.scopus.com/inward/record.url?scp=85090948968&partnerID=8YFLogxK
U2 - 10.1002/cncr.33173
DO - 10.1002/cncr.33173
M3 - Article
C2 - 32926409
AN - SCOPUS:85090948968
SN - 0008-543X
VL - 126
SP - 5156
EP - 5164
JO - Cancer
JF - Cancer
IS - 23
ER -