Results of Treatment for Patients With Multicentric or Bilaterally Predisposed Unilateral Wilms Tumor (AREN0534): A report from the Children's Oncology Group.

Autor: Ehrlich PF; Section of Pediatric Surgery, CS Mott Children's Hospital, University of Michigan, Ann Arbor, Michigan, USA., Chi YY; Department of Biostatistics, University of Florida, Gainesville, Florida, USA., Chintagumpala MM; Department of Pediatrics and Oncology, Texas Children's Hospital Cancer Center at Baylor College of Medicine, Houston, Texas, USA., Hoffer FA; Department of Radiology, Fred Hutchison Cancer Center, University of Washington, Seattle, Washington, USA., Perlman EJ; Division of Pediatrics and Oncology, Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, USA., Kalapurakal JA; Department of Radiation Oncology, Northwestern Memorial Hospital, Northwestern University, Chicago, Illinois, USA., Tornwall B; Department of Biostatistics, University of Florida, Gainesville, Florida, USA., Warwick A; Department of Oncology, Walter Reed National Military Medical Center, Washington, District of Columbia, USA., Shamberger RC; Department of Pediatric Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts, USA., Khanna G; Department of Pediatric Radiology, Washington University of St Louis, St Louis, Missouri, USA., Hamilton TE; Department of Pediatric Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts, USA., Gow KW; Department of Surgery, University of Washington, Seattle, Washington, USA., Paulino AC; Department of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Gratias EJ; Children's Oncology Group, Philadelphia, Pennsylvania, USA., Mullen EA; Department of Pediatric Surgery, Boston Children's Hospital and Dana Farber Cancer Center, Boston, Massachusetts, USA., Geller JI; Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA., Grundy PE; Department of Pediatrics, University of Alberta Children's Hospital, Edmonton, Alberta, Canada., Fernandez CV; Department of Pediatrics, IWK Children's Hospital, Halifax, Nova Scotia, Canada.; Department of Bioethics, IWK Children's Hospital, Halifax, Nova Scotia, Canada., Dome JS; Children's National Hospital, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.
Jazyk: angličtina
Zdroj: Cancer [Cancer] 2020 Aug 01; Vol. 126 (15), pp. 3516-3525. Date of Electronic Publication: 2020 May 27.
DOI: 10.1002/cncr.32958
Abstrakt: Background: A primary objective of Children's Oncology Group study AREN0534 (Treatment for Patients With Multicentric or Bilaterally Predisposed, Unilateral Wilms Tumor) was to facilitate partial nephrectomy in 25% of children with bilaterally predisposed unilateral tumors (Wilms tumor/aniridia/genitourinary anomalies/range of developmental delays [WAGR] syndrome; and multifocal and overgrowth syndromes). The purpose of this prospective study was to achieve excellent event-free survival (EFS) and overall survival (OS) while preserving renal tissue through preoperative chemotherapy, completing definitive surgery by 12 weeks from diagnosis, and modifying postoperative chemotherapy based on histologic response.
Methods: The treating institution identified whether a predisposition syndrome existed. Patients underwent a central review of imaging studies through the biology and classification study AREN03B2 and then were eligible to enroll on AREN0534. Patients were treated with induction chemotherapy determined by localized or metastatic disease on imaging (and histology if a biopsy had been undertaken). Surgery was based on radiographic response at 6 or 12 weeks. Further chemotherapy was determined by histology. Patients who had stage III or IV disease with favorable histology received radiotherapy as well as those who had stage I through IV anaplasia.
Results: In total, 34 patients were evaluable, including 13 males and 21 females with a mean age at diagnosis of 2.79 years (range, 0.49-8.78 years). The median follow-up was 4.49 years (range, 1.67-8.01 years). The underlying diagnosis included Beckwith-Wiedemann syndrome in 9 patients, hemihypertrophy in 9 patients, multicentric tumors in 10 patients, WAGR syndrome in 2 patients, a solitary kidney in 2 patients, Denys-Drash syndrome in 1 patient, and Simpson-Golabi-Behmel syndrome in 1 patient. The 4-year EFS and OS rates were 94% (95% CI, 85.2%-100%) and 100%, respectively. Two patients relapsed (1 tumor bed, 1 abdomen), and none had disease progression during induction. According to Response Evaluation Criteria in Solid Tumor 1.1 criteria, radiographic responses included a complete response in 2 patients, a partial response in 21 patients, stable disease in 11 patients, and progressive disease in 0 patients. Posttherapy histologic classification was low-risk in 13 patients (including the 2 complete responders), intermediate-risk in 15 patients, and high-risk in 6 patients (1 focal anaplasia and 5 blastemal subtype). Prenephrectomy chemotherapy facilitated renal preservation in 22 of 34 patients (65%).
Conclusions: A standardized approach of preoperative chemotherapy, surgical resection within 12 weeks, and histology-based postoperative chemotherapy results in excellent EFS, OS, and preservation of renal parenchyma.
(© 2020 American Cancer Society.)
Databáze: MEDLINE
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