Abstract
While recent increases in our understanding of the biology of neuroblastoma have allowed for more precise risk stratification and improved outcomes for many patients, children with high-risk neuroblastoma continue to suffer from frequent disease relapse, and despite recent advances in our understanding of neuroblastoma pathogenesis, the outcomes for children with relapsed neuroblastoma remain poor. These children with relapsed neuroblastoma, therefore, continue to need novel treatment strategies based on a better understanding of neuroblastoma biology to improve outcomes. The discovery of new tumor targets and the development of novel antibody- and cell-mediated immunotherapy agents have led to a large number of clinical trials for children with relapsed neuroblastoma, and additional clinical trials using molecular and genetic tumor profiling to target tumor-specific aberrations are ongoing. Combinations of these new therapeutic modalities with current treatment regimens will likely be needed to improve the outcomes of children with relapsed and refractory neuroblastoma.
Highlights
With new treatment strategies, including immunotherapy and therapies directed against tumor-specific molecular aberrations, the outcomes of children with high-risk neuroblastoma have significantly improved over the past two decades
Targeted Therapy—Targeted agent used in non-targeted fashion; Molecularly Guided Therapy—Targeted agent used in targeted patient population based on molecular aberration or phenotype; NET—norepinephrine transporter; SSTR—somatostatin receptor; XRT—radiation therapy; ODC—ornithine decarboxylase, HIFU—high-intensity focused ultrasound, HDAC—histone deacetylase; MIBG—meta-iodobenzylguanidine; CAR-T cells—chimeric antigen receptor T cells; Anaplastic Lymphoma Kinase (ALK)—anaplastic lymphoma kinase; NK cells—natural killer cells
Recent advances in our understanding of the biology of neuroblastoma and of novel strategies to target tumor-specific pathways and antigens have led to a dramatic increase in the number of available treatment options for these patients and give hope that, in the future, novel treatment regimens will increase the responses of tumors to upfront therapy, limit the overall chances of relapse, and, in those hopefully rare cases of relapse, provide safe and effective therapies to eradicate neuroblastoma tumors
Summary
With new treatment strategies, including immunotherapy and therapies directed against tumor-specific molecular aberrations, the outcomes of children with high-risk neuroblastoma have significantly improved over the past two decades. Despite these recent successes, many of these children continue to suffer from refractory or relapsed disease, and to date, there are no established curative treatment options for many of these patients. The reported 5-year overall survival rate for children after the initial relapse of neuroblastoma is 20% [3,4] These outcomes were dependent on both the time of relapse and the initial patient tumor stage [3,4,5], suggesting that a subset of patients with recurrent neuroblastoma can be cured with appropriate treatment and emphasizing the need for additional treatment options for these patients. Recent data have suggested that more aggressive treatment of these patients to achieve a complete or nearly complete response, followed by additional consolidation or maintenance therapy, can lead to durable disease remission and even cure [10], suggesting that the goals of treatment for many children with relapsed and refractory neuroblastoma may need to be modified
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