Abstract
Metastatic neuroblastoma is an aggressive malignancy with a poor prognosis. Recent findings have shown that sorafenib decreases cell viability and increases apoptosis in human neuroblastoma cell lines. We report an experience of compassionate use of sorafenib in children with treatment‐refractory neuroblastoma. Sorafenib showed transient anti‐tumor activity in all four patients without adverse effects. However, progression was observed after a short stabilization phase. While sorafenib showed minimal anti‐tumor activity in our patients, it might still be effective in patients with neuroblastoma in an earlier stage.
Highlights
Children with high-risk neuroblastoma have poor long- term survival despite intensive multimodal treatment [1, 2]
Sorafenib has been evaluated in pediatric patients with solid tumors or leukemia in two phase I studies in combination with other agents [8, 9] and, thereafter, in a Children’s Oncology Group phase I trial as single-agent therapy [10]
The efficacy of sorafenib in children with neuroblastoma is unclear, we considered that compassionate use of sorafenib might be of value in end-stage neuroblastoma patients with acquired resistance to conventional chemotherapy
Summary
Children with high-risk neuroblastoma have poor long- term survival despite intensive multimodal treatment [1, 2]. Recent findings have shown that sorafenib decreases cell viability and increases apoptosis in human neuroblastoma cell lines in association with downregulation of ERK, AKT, RAF-MEK, and JAK2-STAT3 survival pathways [5, 6]. Sorafenib has been evaluated in pediatric patients with solid tumors or leukemia in two phase I studies in combination with other agents [8, 9] and, thereafter, in a Children’s Oncology Group phase I trial as single-agent therapy [10]. Hypertension, diarrhea, rash, fatigue, and increased serum levels of alanine aminotransferase (ALT)/aspartate aminotransferase (AST) were the most common sorafenib-related toxicities. These toxicities and the pharmacokinetic profile were similar to adult studies. The recommended phase II dose of sorafenib was 200 and 150 mg/m2 every 12 h for children with solid tumors and leukemia, respectively
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