Outcomes based on histopathologic response to preoperative chemotherapy in children with bilateral Wilms tumor: A prospective study (COG AREN0534)

Murali M. Chintagumpala, Texas Children's Cancer Center, Baylor College of Medicine, Houston, Texas.
Elizabeth J. Perlman, Department of Pathology, Ann and Robert H. Lurie Children's Hospital, Chicago, Illinois.
Brett Tornwall, Department of Biostatistics, College of Public Health & Health Professions and College of Medicine, University of Florida, Gainesville, Florida.
Yueh-Yun Chi, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California.
Yeonil Kim, Merck Research Laboratories, Merck & Company Inc, Rahway, New Jersey.
Fredric A. Hoffer, Department of Radiology, Fred Hutchison Cancer Center, University of Washington, Seattle, Washington.
John A. Kalapurakal, Department of Radiation Oncology, Northwestern Memorial Hospital, Northwestern University, Chicago, Illinois.
Anne B. Warwick, Department of Pediatrics, Uniformed Services University, Walter Reed National Military Medical Center, Washington, DC.
Robert C. Shamberger, Department of Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts.
Geetika Khanna, School of Medicine, Washington University of St Louis, St Louis, Missouri.
Thomas E. Hamilton, Department of Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts.
Kenneth W. Gow, Department of Surgery, University of Washington, Seattle, Washington.
Arnold C. Paulino, Department of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Eric J. Gratias, Children's Oncology Group, Philadelphia, Pennsylvania.
Elizabeth A. Mullen, Department of Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts.
James I. Geller, Division of Hematology/Oncology, Cincinnati Children's Hospital, Cincinnati, Ohio.
Conrad V. Fernandez, Pediatrics and Bioethics, IWK Health Center, Halifax, Nova Scotia, Canada.
Michael L. Ritchey, Department of Urology, Phoenix Children's Hospital, Phoenix, Arizona.
Paul E. Grundy, Department of Pediatrics and Oncology, University of Alberta Children's Hospital, Edmonton, Alberta, Canada.
Jeffrey S. Dome, Pediatric Surgery, Children's National Hospital and George Washington University School of Medicine and Health Sciences, Washington, DC.
Peter F. Ehrlich, Section of Pediatric Surgery, C.S. Mott Children's Hospital, University of Michigan, Ann Arbor, Michigan.

Document Type

Journal Article

Publication Date

4-5-2022

Journal

Cancer

DOI

10.1002/cncr.34219

Keywords

bilateral Wilms tumors; blastemal-type Wilms; histopathologic response; preoperative chemotherapy in Wilms tumors; risk stratification

Abstract

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.

Department

Pediatrics

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