Kidney Preservation and Wilms Tumor Development in Children with Diffuse Hyperplastic Perilobar Nephroblastomatosis: A Report from the Children’s Oncology Group Study AREN0534

Ehrlich PF, Tornwall B, Chintagumpala MM, Chi YY, Hoffer FA, Perlman EJ, Kalapurakal JA, Warwick A, Shamberger RC, Khanna G, Hamilton TE, Gow KW, Paulino AC, Gratias EJ, Mullen EA, Geller JI, Fernandez CV, Dome JS. Kidney Preservation and Wilms Tumor Development in Children with Diffuse Hyperplastic Perilobar Nephroblastomatosis: A Report from the Children’s Oncology Group Study AREN0534. Ann Surg Oncol. 2022 May;29(5):3252-3261. doi: 10.1245/s10434-021-11266-6. Epub 2022 Jan 24. PubMed PMID: 35072864; PubMed Central PMCID: PMC9254258.

Diffuse hyperplastic perilobar nephroblastomatosis (DHPLN) represents a unique category of nephroblastomatosis. Treatment has ranged from observation to multiple regimens of chemotherapy. Wilms tumors (WT) develop in 100% of untreated patients and between 32–52% of those treated. Renal preservation rates have not been previously reported. An aim of the Children’s Oncology Group (COG) study AREN0534 was to evaluate prospectively the efficacy of chemotherapy in preserving renal units and preventing WT development in children with DHPLN. Patients were enrolled through COG protocol AREN03B2 with central radiological review. DHPLN was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved with a thick rind capping all of one or both kidneys. Treatment was with vincristine and dactinomycin (EE4A) with cross –sectional imaging at weeks 6 and 12. If the patient’s disease was stable or decreasing, treatment was continued for 19 weeks. Renal preservation, WT development rates at one year and overall survival (OS) are reported.