Targeted therapy in pediatric malignancies treatment: Pediatric Oncology and Haematology Research Institute experience

Abstract

Actually targeted drugs became a therapeutic component for adult patients with malignancies and merge into clinical therapeutic recommendations. Targeted drugs in pediatric oncology had been using for 10-15 years and this experience under systematization and analysis. In the current issue 12-year experience of targeted therapy of Pediatric Oncology and Haematology Research Institute of N.N.Blokhin National Cancer Research Center is presented. Authors estimated efficacy and toxicity of rituximab, bortezomib, sorafenib and everolimus in 108 patients with advanced stages of mature B-cell non-Hodgkins lymphomas (B-NHL), relapsed/refractory acute lymphoblastic leukemia (ALL) and refractory osteosarcoma. Rituximab plus standard chemotherapy of advanced stages of B-NHL increased 10-years event-free survival up to 93.8±3%. Bortezomib plus anti-relapsed chemotherapy protocol (COG AALL07P1) reached minimal residual disease - negative status in 64.3% patients, but relapse-free survival was 24.5±19.4% (median 28±3.3 months). Sorafenib and everolimus were used for refractory osteosarcoma treatment, response rate was 100%, progression-free survival more than 6 months was 46% (median follow up 7±1.2 months). Toxicity profile was acceptable.Our data demonstrate a fact of significant improvement of advanced stages B-NHL treatment results by rituximab use, but relapsed/refractory ALL and osteosarcoma need treatment improvement.

Keywords

• targeted therapy
• non-hodgkins lymphomas
• acute lymphoblastic leukemia
• osteosarcoma
• children