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Background: Despite improvement in relapse free survival (RFS) in recurrent Wilms tumor, the 4-year RFS rate for patients treated with vincristine/dactinomycin/doxorubicin is approximately 40%. Ifosfamide, carboplatin and etoposide are commonly used in relapsed solid tumors, but have significant toxicities and require hospital admission for administration. Our patient focused on quality of life, and this led to exploration of other treatment options. Objective: Describe a case of multiply relapsed Wilms tumor with management on oral irinotecan/temozolomide for improved quality of life and remission for almost 1 year. Design/Method: Case Report Results: The patient is an 11-year-old female diagnosed with Wilms tumor at 6 years of age in Honduras who received approximately 6 months of chemotherapy with Vincristine and a nephrectomy. Staging in Honduras was unknown. Due to financial difficulties, she was unable to continue treatment and family immigrated to the United States. One year after initial diagnosis, imaging revealed large chest mass and intracardiac masses. Biopsy confirmed recurrent Wilms and she received modified NWTS/AREN0534 regimen. Patient responded well to this regimen and then underwent surgical resections and whole lung radiation. At 20 months off therapy, she was found to have a lung nodule, confirmed to be recurrent Wilms tumor. As this was her second recurrence, both patient and family chose a chemotherapy regimen allowing for the best quality of life. She received Irinotecan 90mg/m2/dose PO on days 1-5 and Temozolomide 40mg/m2/dose PO on days 1-5, 8-12, 15-19. She received the 2-drug regimen for 10, 28-day cycles without toxicities or hospitalizations, allowing her to attend school. At the end of cycle 10, she had a third recurrence. Irinotecan is a camptothecin prodrug shown to have activity against adult solid tumors. Topotecan of the same drug class also showed activity in children with favorable histology Wilms tumor with a 48% response rate in heavily pretreated patients. Temozolomide (TMZ) has activity against adult solid tumors with promising results in xenograft models of pediatric solid tumors. O6-methylguanine-DNA methyltransferase (MGMT) is a DNA repair enzyme which inhibits the anti-tumor effect of alkylating agents, like TMZ. Negative MGMT protein expression increases the sensitivity to alkylating agents and can predict the response to temozolomide. Our patient was MGMT negative. Conclusion: Irinotecan/Temozolomide should continue to be explored as an option for relapsed/refractory solid tumors as it can achieve remission for a substantial period while allowing for improved quality of life.


Oncology | Pediatrics


Presented at the American Society of Pediatric Hematology/Oncology Conference; Pittsburgh, PA; May 3-7, 2022.

Treatment of Recurrent Wilms Tumor with Irinotecan/Temozolomide



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