Overall survival and event free survival in patients with high-risk neuroblastoma after targeted therapy
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| Year of publication | 2022 |
| Type | Conference abstract |
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| Description | Neuroblastoma belongs to the most common extracranial solid tumors of childhood age. In children diagnosed with high-risk neuroblastoma, the effectiveness of the treatment is low, and the therapy often fails. Study aimed to evaluate the effectiveness and safety of targeted therapy in pediatric patients with refractory or relapsed neuroblastoma. The group analyzed consisted of 11 pediatric patients diagnosed with high-risk neuroblastoma at the Department of Paediatric Oncology, University Hospital Brno. Treatment of the patients was carried out in three phases: first-line treatment, second-line treatment, and targeted treatment. The retrospective data collection was performed from September 2020 to July 2021. Targeted therapy included dinutuximab beta, sunitinib malate, propranolol, vinblastine succinate, crizotinib, lorlatinib, ribociclib succinate, ceritinib, nivolumab, nintedanib esilate and regorafenib. The final tumor response to the targeted therapy was complete response in 9%, stable disease in 27%, and progressive disease in 64% of patients. Compared to the historical cohort (survival in relapsed and refractory neuroblastoma two years after diagnosis 10-15%), all patients experienced objective treatment response, increased progression time, and overall survival. Tolerance to the targeted therapy was good. No patient experienced life-threatening toxicity, maximum toxicity was haematological grade IV in one patient, but it spontaneously resolved after discontinuation of the targeted therapy. Targeted therapy can prevent death in 54% of patients with relapsed and refractory neuroblastoma using precision medicine procedures within two years of diagnosis. The results suggest that neuroblastoma is a very aggressive tumor that is very difficult to treat, however, the precision medicine approach and targeted therapies may become new modern perspectives for treating high-risk tumors. |
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