Treatment Overview
Receiving a diagnosis of neuroblastoma is an overwhelming experience for any family. As a cancer that primarily affects infants and young children, it brings a wave of fear and uncertainty that disrupts normal life. The symptoms, which can range from a painless lump to abdominal swelling, bone pain, or dark circles around the eyes, are often distressing for both the child and the caregivers. Treatment is essential not only to control the tumor and relieve these physical symptoms but to prevent the cancer from spreading to vital organs like the bones or liver.
Because neuroblastoma behaves very differently from child to child, treatment plans are highly specific. Some tumors in infants may disappear on their own, while others in older children can be aggressive and widespread. Doctors classify the disease into low, intermediate, or high-risk groups to determine the intensity of therapy needed. A treatment plan is carefully tailored based on the child’s age, the tumor’s stage, and specific genetic markers found within the cancer cells (American Cancer Society, 2024).
Overview of treatment options for Neuroblastoma
The main goal of treatment is to eliminate the cancer cells and prevent them from returning. For low-risk cases, surgery alone may be sufficient. However, for intermediate and high-risk neuroblastoma, a multimodal approach is standard. This typically involves a combination of surgery to remove the tumor, radiation therapy to kill remaining cells, and powerful medications to treat disease throughout the body.
Medication is the backbone of treatment for higher-risk cases. It is used to shrink the tumor before surgery, destroy cancer cells that have spread, and “mop up” any microscopic disease left behind. High-dose chemotherapy followed by stem cell rescue is a common strategy for aggressive disease. Newer approaches also incorporate immunotherapy and retinoid therapy to improve survival rates and reduce the risk of relapse.
Medications used for Neuroblastoma
Doctors utilize a combination of drug classes to attack the cancer from multiple angles.
Chemotherapy is the primary treatment for most intermediate and high-risk cases. Combinations of drugs are usually given intravenously to ensure they reach cancer cells anywhere in the body. Common medications include cyclophosphamide, cisplatin, doxorubicin, and vincristine. These drugs are often administered in cycles, allowing the body time to recover between doses. Clinical experience suggests that using a combination of agents is more effective than a single drug at shrinking the tumor.
Immunotherapy has become a standard part of treatment for high-risk neuroblastoma. Monoclonal antibodies, such as dinutuximab, are designed to target specific proteins found on the surface of neuroblastoma cells. This therapy is typically given after a stem cell transplant to help the body’s immune system find and destroy any remaining cancer cells.
Retinoids are another crucial class of medication used in the maintenance phase. Isotretinoin is related to Vitamin A and is typically taken orally after chemotherapy or transplant is complete. Studies show that retinoid therapy can help prevent the cancer from returning by forcing the cancer cells to mature into normal cells (National Cancer Institute, 2023).
How these medications work
The medications used for neuroblastoma target the cancer’s ability to grow and hide.
Chemotherapy disrupts the cell cycle, primarily targeting fast-dividing cancer cells by damaging their DNA or inhibiting replication material synthesis.
Immunotherapy, using drugs like dinutuximab, flags hidden neuroblastoma cells (via GD2 surface markers) for attack by the immune system’s white blood cells.
Retinoids work by promoting differentiation, encouraging immature neuroblastoma cells to mature into non-cancerous nerve cells that cease dividing, preventing tumor formation.
Side effects and safety considerations
The intensive nature of neuroblastoma treatment requires close monitoring for side effects.
Chemotherapy frequently causes nausea, vomiting, hair loss, and fatigue. A major safety concern is bone marrow suppression, leading to low blood counts and high infection risk. Cisplatin specifically risks hearing loss; doxorubicin requires heart monitoring.
Immunotherapy may cause nerve pain, blood vessel fluid leakage, or allergic reactions during infusion, often requiring pain management. Retinoids commonly cause dry skin and chapped lips, but can also affect liver function.
Parents must seek immediate medical care if a child develops a fever, as it may signal a life-threatening infection due to low white blood cell counts.
Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.
References
- American Cancer Society. https://www.cancer.org
- National Cancer Institute. https://www.cancer.gov
- Mayo Clinic. https://www.mayoclinic.org
- St. Jude Children’s Research Hospital. https://www.stjude.org
Medications for Neuroblastoma
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Neuroblastoma.