Topics in 10: Neuroblastoma
Topic overview
Drs Daniel von Allmen, Todd Ponsky and Rae Hanke come together to provide you the essentials on the diagnosis and management of Neuroblastoma.
Intro and outro tracks are adapted from "I dunno" by grapes, featuring J Lang, Morusque. Artist URL: ccmixter.org/files/grapes/16626
Neuroblastoma:
- Epidemiology:
- Most common extracranial solid tumor in children
- Only about 600 cases/ year in US
- Majority of cases in children under 1 year, but can be found through young adulthood
- Presentation:
- Prenatal: can be seen on ultrasound
- 2-3 year old: solid abdominal mass
- Metastasis: bony pain or neurologic symptoms from cord compression
- Work up:
- Abdominal ultrasound—solid mass
- Lead to labs:
- Always include urine or serum catecholamines (most diagnostic)
- CT or MRI—central abdominal or adrenal mass
- Next is MIBG to confirm neuroblastoma study and evaluate for metastasis
- MIBG negative in 10%, some will get PET if negative—eval for tumor uptake
- Pre-biopsy staging
- Based on International Neuroblastoma Risk Group Staging System (INRGSS) http://bit.ly/2RtEk1M
- Created to allow studies from different centers around the world to compare stage before surgery
- L1: localized
- L2: Localized with image defined risk factors L2
- M: Metastatic
- MS: for children <18 months with metastasis to bone marrow or skin
- Management:
- Case 1: Right adrenal mass that is MIBG positive, but no metastasis
- Primary resection of mass—laparotomy vs laparoscopic approach
- Take any obvious lymph nodes
- Case 2: Large mass from adrenal involving central abdominal vasculature
- Start with tissue for diagnosis: open, laparoscopic or core needle biopsy
- Look for biologic determinants
- NMYC status, 1p and 11q deletions (Shimada histology) http://bit.ly/2OZrTsA
- Allows for risk stratification of the patient
- Classified as very low, low, intermediate or high risk
- About half are very low/low risk and the other half high risk (with small percentage found to be intermediate risk)
- Most important prognostic determinants: NMYC positive and age > 18 months--often indicate high risk
- If high risk:
- Aggressive regimen including chemotherapy, peripheral stem cell transplant x 2, aggressive surgery with >90% resection (if safe), radiation, immunotherapy after chemotherapy, and potentially retinoic acid therapy
- If intermediate risk:
- Varying cycles of chemotherapy based on biologic risk factors
- Goal: neoadjuvant chemotherapy + surgery = 50% reduction in initial tumor size
- If low risk and diagnosed prenatally or after short postnatal period (< 6 months):
- Can be treated with aggressive observation (serial imaging and catecholamine level monitoring)
- Most will not require surgery
- If increasing in size or parental concern, most resected tumors will be low stage
- http://bit.ly/34ToHV7
- Case 3: Patient with metastasis
- 4 to 5 rounds of neoadjuvant chemo then reassess
- If metastasis and tumor respond, you can resect the primary tumor with goal of >90% resected (if safe)
- If metastasis are progressing, no surgery
- Case 4: MS disease
- Patients with primary site with metastatic to liver, skin or bone marrow (not bone) and <18 months
- Can aggressively observe
- If the tumor increases in size or if respiratory issues arise from increasing liver mass may need to resect
- Can biopsy skin lesions for diagnosis
Clinical pearls:
- Neuroblastoma is the most common extracranial solid tumor in children
- It often presents with an abdominal mass
- Work up involves abdominal ultrasound, blood work, MIBG, and cross sectional imaging
- Get tissue through open, laparoscopic or core needle biopsy
- Post-biopsy staging is stratified into very low, low, intermediate and high risk
- NMYC amplification and age > 18 months are poor prognostic factors
- Treatment is based on risk:
- Low risk—surgery or observation (age depending)
- Intermediate risk—chemotherapy and more limited surgical resection
- High risk—neoadjuvant chemo, surgery, radiation, peripheral stem cell transplant, immunotherapy
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