Treatment of Childhood Neuroblastoma

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Due to the diversity and complexity of neuroblastoma (NBL) treatment options may vary depending on the stage and grade of cancer, cancer spread and age of the children. In fact, the various treatment options for NBL include surgery, chemotherapy, and immunotherapy.


Due to the diversity of tumor location in neuroblastoma (NBL), grade, and stage of diagnosis, treatment options include simple observation, chemotherapy, surgery, radiation therapy, immunotherapy, stem cell transplantation. Ho wever, low-risk NBL patients tend to have local tumors, and some (infants) are more likely to have spontaneous tumor regression. Therefore, children with small tumors (less than 5 cm) may start imaging every 6 to 12 weeks to monitor tumor growth, thus avoiding surgery in young infants​1​.

Some of the treatment options for NBL are:


Doctors operate large, localized tumors in late infancy. In symptomatic children, doctors give limited chemotherapy without surgical procedures or radiation therapy.

Because NBL is a heterogeneous disease in terms of tumor biology as well as clinical behaviour, pediatric oncologists should pay attention to reducing complications that may occur during surgery depending on the stage and age of the disease. The medium-risk group presents with localized metastases, i.e. in the bone marrow or lymph nodes. A doctor usually treats it only with chemotherapy and, if possible, with surgery.

Surgery plays an important role in the treatment of neuroblastoma. A doctor can achieve treatment of local disorders through surgery. Sometimes surgery is a must to make a diagnosis. After chemotherapy, doctors often do a secondary surgery to remove the tumor​1​.


The high-risk group has the worst prognosis and has extensive metastatic disease in the bone, bone marrow, liver, and lung. They undergo induction chemotherapy to reduce the burden of the tumor at both the primary and metastatic sites, followed by maximal surgical resection, stem cell transplantation, and myeloablative chemotherapy. The doctor then gives the patient a combination of maintenance chemotherapy and immunotherapy​1​.


Researchers are currently evaluating immunotherapy with anti-GD2 chimeric mAb followed by high-dose chemotherapy in high-risk NBL as a treatment for the multidrug-resistant disease. Monoclonal antibodies, such as dinutuximab, that bind to the carbohydrate molecule (GD2) on the surface of many neuroblastoma cells are useful as immunotherapy drugs to treat NBL. Dinutuzimab treatment improves the 2-year survival rate in patients with high-risk neuroblastoma from 46% to 66%​1​.

Immune checkpoint inhibitors

Doctors use immune checkpoint inhibitors increasingly to treat various types of refractory cancers as targets for novel immunotherapy. They target activation of circulating T lymphocytes (CTLs) by inhibiting the programmed cell death-1 (PD-1) pathway, which includes targets such as CTL-associated protein 4 (CTLA4), PD-1 and PD-L1. In fact, several preliminary studies have confirmed that PD-L1 is expressed in high-risk NBL cells​2​.

CAR-T therapy

CAR-T has been shown to be effective against B-cell malignancies. Undoubtedly, methods for NBL treatment are mainly investigated for more efficient production of CAR-T with higher therapeutic effect using GD2 as the target antigen. However, traditional methods of preparing CAR-T have the disadvantages of high cost, complexity, and low efficiency. Consequently, in recent years, simpler, cheaper, and more efficient methods of manufacturing CAR-T using CRISPR/Cas 9 technology have been attracting attention that can be used to treat NBL​2​.


  1. 1.
    Mahapatra S, Challagundla K. statpearls. Published online July 17, 2021.
  2. 2.
    Nakagawara A, Li Y, Izumi H, Muramori K, Inada H, Nishi M. Neuroblastoma. Japanese Journal of Clinical Oncology. Published online January 25, 2018:214-241. doi:10.1093/jjco/hyx176