Definition:
- Malignant tumor arising from neural crest cells (sympathetic nervous system precursors).
- Most common extracranial solid tumor in children.
Epidemiology
- Usually diagnosed in children <5 years.
- Slight male predominance.
- Can arise anywhere along the sympathetic chain (adrenal gland most common).
Clinical Features
- Abdominal mass (most common), often palpable and firm
- Systemic: fever, weight loss, malaise
- Catecholamine excess: ↑ urine HVA (homovanillic acid) and VMA (vanillylmandelic acid)
- Metastatic disease may present with: bone pain, periorbital ecchymosis (“raccoon eyes”), hepatomegaly, spinal cord compression
Staging & Prognosis
- INRG staging based on image-defined risk factors (IDRFs)
- Prognosis varies:
- <18 months with localized disease → excellent
- Older children or metastatic disease → high-risk, poorer prognosis
- Genetics: MYCN amplification → high-risk, aggressive tumor
Treatment & Pharmacist Role
1. Surgery
- Maximal safe resection if feasible
2. Chemotherapy
- Low-/intermediate-risk: cyclophosphamide, doxorubicin, vincristine
- High-risk: multi-agent chemo: cyclophosphamide, vincristine, doxorubicin, cisplatin, etoposide, ifosfamide
- Pharmacist considerations:
- Dose adjustments for age, renal, hepatic function
- Monitor for myelosuppression, cardiotoxicity (doxorubicin), nephrotoxicity (cisplatin/ifosfamide), neuropathy (vincristine)
3. Radiotherapy
- Often used in high-risk or residual disease
4. Immunotherapy / Targeted Therapy
- Anti-GD2 monoclonal antibodies (e.g., dinutuximab) for high-risk disease
- Supportive care for neuropathic pain and infusion reactions
5. Stem Cell Transplant
- Autologous HSCT in high-risk neuroblastoma after induction chemo
High-Yield Pharmacist Pearls
- TLS prophylaxis often needed in high-burden tumors (rasburicase preferred)
- Monitor cumulative anthracycline dose (doxorubicin) for cardiotoxicity
- Renal and hearing monitoring for cisplatin/ifosfamide
- Growth factor support may be required during intensive chemo
- Long-term: monitor for secondary malignancies and organ toxicity