Other approaches

As with adult RCC, there is no standard treatment for unresectable metastatic disease in children. The response to radiation therapy is poor, and chemotherapy is not effective. Immunotherapy with agents such as interferon-alpha and interleukin-2 may have some effect on cancer control.[54,55] Spontaneous regression of pulmonary metastasis rarely occurs with resection of the primary tumor.

Several targeted therapies (e.g., sorafenib, sunitinib, bevacizumab, temsirolimus, pazopanib, axitinib, and everolimus) have been approved for use in adults with RCC. The SIOP-Renal Tumor Study Group (RTSG) retrospectively reported their experience of 31 patients (aged 0–18 years) with RCC, 21 of whom had translocation-positive tumors. Sunitinib was often administered as the first choice (recommended in the SIOP-RTSG 2016 protocol) and predominantly resulted in stable disease (53%). Overall, 15 of 31 patients died of their disease.[56] Case reports of pediatric and adolescent patients with TFE3 translocation–positive RCC suggest responsiveness to multiple tyrosine kinase inhibitors.[35,57,58]; [59][Level of evidence C1] Disease regression and improvement in symptoms have been reported with the use of cabozantinib in pediatric patients with translocation-positive RCC expressing MET.[60] Any RCC that is positive for TFE3 and lacks a translocation should be tested for ALK expression and translocation. Recognition of this subtype may lead to consideration of ALK inhibitor therapy.[45]

For more information about the use of targeted therapies, see Renal Cell Cancer Treatment.