TY - JOUR
T1 - Outcomes based on histopathologic response to preoperative chemotherapy in children with bilateral Wilms tumor
T2 - A prospective study (COG AREN0534)
AU - Chintagumpala, Murali M.
AU - Perlman, Elizabeth J.
AU - Tornwall, Brett
AU - Chi, Yueh Yun
AU - Kim, Yeonil
AU - Hoffer, Fredric A.
AU - Kalapurakal, John A.
AU - Warwick, Anne B.
AU - Shamberger, Robert C.
AU - Khanna, Geetika
AU - Hamilton, Thomas E.
AU - Gow, Kenneth W.
AU - Paulino, Arnold C.
AU - Gratias, Eric J.
AU - Mullen, Elizabeth A.
AU - Geller, James I.
AU - Fernandez, Conrad V.
AU - Ritchey, Michael L.
AU - Grundy, Paul E.
AU - Dome, Jeffrey S.
AU - Ehrlich, Peter F.
N1 - Funding Information:
This study was supported by grants U10CA180886, U10CA180899, U10CA098543, U10CA098413, and U24CA114766 from the National Cancer Institute, National Institutes of Health, to the Children's Oncology Group, and by the St Baldrick's Foundation.
Publisher Copyright:
© 2022 American Cancer Society.
PY - 2022/7/1
Y1 - 2022/7/1
N2 - Background: An objective of the Children's Oncology Group AREN0534 Study was to improve the survival of patients with bilateral Wilms tumors (BWT) by using preoperative chemotherapy of limited duration and tailoring postoperative therapy based on histopathologic response. The authors report outcomes based on postoperative histopathologic responses. Methods: Patients with BWT received treatment with vincristine, dactinomycin, and doxorubicin for 6 or 12 weeks followed by surgery. Postoperative therapy was prescribed based on the highest risk tumor according to the International Society of Pediatric Oncology classification and the Children's Oncology Group staging system. Results: Analyses were performed on data from 180 evaluable children. The 4-year event-free survival (EFS) and overall survival (OS) rates were 81% (95% CI, 74%-87%) and 95% (95% CI, 91%-99%), respectively. Seven patients who had completely necrotic tumors had a 4-year EFS rate of 100%. Of 118 patients who had tumors with intermediate-risk histopathology, the 4-year EFS and OS rates were 82% (95% CI, 74%-90%) and 97% (95% CI, 94%-100%), respectively. Fourteen patients who had blastemal-type tumors had 4-year EFS and OS rates of 79% (95% CI, 56%-100%) and 93% (95% CI, 79%-100%), respectively. Eighteen patients who had diffuse anaplasia had 4-year EFS and OS rates of 61% (95% CI, 35%-88%) and 72% (95% CI, 47%-97%), respectively; and the 4-year EFS and OS rates of 7 patients who had focal anaplasia were 71% (95% CI, 38%-100%) and 100%, respectively. There was no difference in the outcomes of patients who had different histopathologic subtypes within the intermediate-risk group (P =.54). Conclusions: A risk-adapted treatment approach for BWT results in excellent outcomes. This approach was not successful in improving the outcome of patients who had diffuse anaplasia.
AB - Background: An objective of the Children's Oncology Group AREN0534 Study was to improve the survival of patients with bilateral Wilms tumors (BWT) by using preoperative chemotherapy of limited duration and tailoring postoperative therapy based on histopathologic response. The authors report outcomes based on postoperative histopathologic responses. Methods: Patients with BWT received treatment with vincristine, dactinomycin, and doxorubicin for 6 or 12 weeks followed by surgery. Postoperative therapy was prescribed based on the highest risk tumor according to the International Society of Pediatric Oncology classification and the Children's Oncology Group staging system. Results: Analyses were performed on data from 180 evaluable children. The 4-year event-free survival (EFS) and overall survival (OS) rates were 81% (95% CI, 74%-87%) and 95% (95% CI, 91%-99%), respectively. Seven patients who had completely necrotic tumors had a 4-year EFS rate of 100%. Of 118 patients who had tumors with intermediate-risk histopathology, the 4-year EFS and OS rates were 82% (95% CI, 74%-90%) and 97% (95% CI, 94%-100%), respectively. Fourteen patients who had blastemal-type tumors had 4-year EFS and OS rates of 79% (95% CI, 56%-100%) and 93% (95% CI, 79%-100%), respectively. Eighteen patients who had diffuse anaplasia had 4-year EFS and OS rates of 61% (95% CI, 35%-88%) and 72% (95% CI, 47%-97%), respectively; and the 4-year EFS and OS rates of 7 patients who had focal anaplasia were 71% (95% CI, 38%-100%) and 100%, respectively. There was no difference in the outcomes of patients who had different histopathologic subtypes within the intermediate-risk group (P =.54). Conclusions: A risk-adapted treatment approach for BWT results in excellent outcomes. This approach was not successful in improving the outcome of patients who had diffuse anaplasia.
KW - bilateral Wilms tumors
KW - blastemal-type Wilms
KW - histopathologic response
KW - preoperative chemotherapy in Wilms tumors
KW - risk stratification
KW - Kidney Neoplasms/drug therapy
KW - Prospective Studies
KW - Humans
KW - Infant
KW - Wilms Tumor/drug therapy
KW - Vincristine
KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use
KW - Nephrectomy
KW - Anaplasia/pathology
KW - Neoplasm Staging
KW - Child
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U2 - 10.1002/cncr.34219
DO - 10.1002/cncr.34219
M3 - Article
C2 - 35383900
AN - SCOPUS:85127733482
SN - 0008-543X
VL - 128
SP - 2493
EP - 2503
JO - Cancer
JF - Cancer
IS - 13
ER -