Introduction: Renal cell carcinoma (RCC) is a very rare pediatric renal tumor. Robust evidence to guide treatment is lacking and knowledge on targeted therapies and immunotherapy is mainly based on adult studies. Currently, the International Society of Pediatric Oncology-Renal Tumor Study Group (SIOP-RTSG) 2016 UMBRELLA protocol recommends sunitinib for metastatic or unresectable RCC.
This retrospective study describes the effects of tyrosine kinase inhibitors (TKI), anti-programmed cell death 1 (PD-(L)1) monoclonal antibodies, and immunotherapeutic regimens in advanced-stage and relapsed pediatric RCC.
Of the 31 identified patients (0-18 years) with histologically proven RCC, 3/31 presented with TNM stage I/II, 8/31 with TNM stage III, and 20/31 with TNM stage IV at diagnosis. The majority were diagnosed with translocation type RCC (MiT-RCC) (21/31) and the remaining patients mainly presented with papillary or clear-cell RCC. Treatment in a neoadjuvant or adjuvant setting, or upon relapse or progression, included mono- or combination therapy with a large variety of drugs, illustrating center specific choices in most patients. Sunitinib was often administered as first choice and predominantly resulted in stable disease (53%). Other frequently used drugs included axitinib, cabozantinib, sorafenib, and nivolumab; however, no treatment seemed more promising than sunitinib. Overall, 15/31 patients died of disease, 12/31 are alive with active disease, and only four patients had a complete response. The sample size and heterogeneity of this cohort only allowed descriptive statistical analysis.
This study provides an overview of a unique series of clinical and treatment characteristics of pediatric patients with RCC treated with targeted therapies.
Cancer medicine. 2023 Dec 22 [Epub]
Julia Sprokkerieft, Justine N van der Beek, Filippo Spreafico, Barbara Selle, Estelle Thebaud, Tanzina Chowdhury, Jesper Brok, Gábor Ottóffy, Xiaofei Sun, Gema L Ramírez Villar, Garik Sagoyan, Heidi Segers, Dimitrios Doganis, Annalisa Serra, Lauriane Lemelle, Norbert Graf, Arnauld C Verschuur, Godelieve A M Tytgat, Marry M van den Heuvel-Eibrink
Department of Pediatric Oncology, Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Pediatric Oncology Unit, Department of Medical Oncology and Hematology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy., Hopp Children's Cancer Center Heidelberg (KiTZ) & Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ) and German Cancer Consortium (DKTK), Heidelberg, Germany., Hôtel Dieu, CHU de Nantes, Nantes, France., Pediatric Oncology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Department of Pediatric Hematology and Oncology, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark., Department of Pediatrics, Medical School, University of Pécs, Pécs, Hungary., Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guanghzou, China., Department of Pediatric Oncology, Hospital Universitario Virgen del Rocío, Seville, Spain., N.N. Blokhin National Medical Research Center of Oncology, Moscow, Russia., Department of Pediatric Hemato-Oncology, University Hospitals Leuven and Catholic University Leuven, Leuven, Belgium., Oncology Department, P&A Kyriakou Children's Hospital, Athens, Greece., Dipartimento di Onco-Ematologia e Medicina Trasfusionale, IRCCS Ospedale Bambino Gesù, Roma, Italy., SIREDO Oncology Center (Care, Innovation and Research for Children and AYA with Cancer), Institut Curie, Paris, France., Department of Pediatric Oncology and Hematology, Saarland University, Homburg, Germany., Department of Pediatric Hematology-Oncology, Hôpital d'Enfants de la Timone, APHM, Marseille, France.