Neuroblastoma (paediatric)
Prepared with OnCo (onco.cc/prep/neuroblastoma/). Orientation, not medical advice; your team knows your case.
My details
What I know, what is unclear, changes to discuss
Saved in this browserMy questions
27 on the sheet- 1.What is my exact diagnosis, stage, and grade, and which tests established them?
- 2.Which biomarkers have been tested on my tumour (for example MYCN amplification, ALK mutation, Age, stage, ploidy, Segmental chromosome aberrations, INRG stage and image-defined risk factors), and what were the results?
- 3.Which subtype is my cancer, and does that change the recommended treatment?
- 4.Is germline (inherited) genetic testing recommended for me or my family?
- 5.For my situation (high-risk), which of the standard options do you recommend and why?
- 6.Am I a candidate for Lorlatinib, and what side effects should I expect?
- 7.For my situation (very-low / low risk (l1, ms)), which of the standard options do you recommend and why?
- 8.For my situation (intermediate risk), which of the standard options do you recommend and why?
- 9.Am I a candidate for Doxorubicin, and what side effects should I expect?
- 10.For my situation (high risk: induction), which of the standard options do you recommend and why?
- 11.Am I a candidate for 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, Dinutuximab (ch14.18) / dinutuximab beta or related drugs, and what side effects should I expect?
- 12.How do the results of COG ANBL1531 apply to someone like me?
- 13.For my situation (high risk: local control), which of the standard options do you recommend and why?
- 14.For my situation (high risk: consolidation), which of the standard options do you recommend and why?
- 15.How do the results of COG ANBL0532 and SIOPEN HR-NBL1 apply to someone like me?
- 16.For my situation (high risk: post-consolidation), which of the standard options do you recommend and why?
- 17.Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Eflornithine (DFMO), and what side effects should I expect?
- 18.How do the results of COG ANBL0032 and NMTRC003/003B (DFMO maintenance) apply to someone like me?
- 19.For my situation (relapsed / refractory), which of the standard options do you recommend and why?
- 20.Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Naxitamab, 131I-MIBG (iobenguane I-131) therapy or related drugs, and what side effects should I expect?
- 21.How do the results of Naxitamab Study 201 and GD2-CART01 (Bambino Gesù phase 1/2) apply to someone like me?
- 22.For my situation (survivorship), which of the standard options do you recommend and why?
- 23.Are there clinical trials I could join, for example of Armoured, logic-gated & next-gen CARs, COG ANBL1531, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib?
- 24.Would a second opinion at a high-volume centre change anything, and can you help arrange it?
- 25.What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
- 26.I read that “Relapsed high-risk disease”. How does that affect my plan?
- 27.I read that “Long-term toxicity of intensive therapy”. How does that affect my plan?
The words I may hear
- Event-free / disease-free survival (EFS, DFS, iDFS, RFS): In early-stage cancer: how long patients stay free of recurrence, progression, or death.
- MYCN amplification: Extra copies of the MYCN oncogene, found in about 20% of neuroblastomas, mark the most aggressive disease and define high risk at any age.
- INRG staging and risk groups: INRG staging is the international system that sorts neuroblastoma into four risk groups, from the lowest (often observed, sometimes regressing on its own) to high risk (about half of patients), using age under 18 months, spread, MYCN amplification, 11q status, ploidy and histology.
- ADCC (antibody-dependent cellular cytotoxicity): In ADCC, an antibody flags a cell, and NK cells recognise the flag and kill it.
- RACE for Children Act: A US law that makes drug companies test new targeted cancer drugs in children whenever the drug's target matters in a childhood cancer, instead of letting them skip children because their cancers are rare.
- Autologous stem cell transplant (ASCT): High-dose chemotherapy (melphalan in myeloma, BEAM in lymphoma) that would permanently destroy the bone marrow, made survivable by giving the patient back their own previously collected stem cells, which engraft in 10-14 days.
- Cytokine release syndrome (CRS): A flood of inflammatory signals when immune cells are activated en masse, causing fever, low blood pressure, and sometimes organ failure.
Tests and results to bring
Biomarker results to ask for: MYCN amplification, ALK mutation, Age, stage, ploidy, Segmental chromosome aberrations, INRG stage and image-defined risk factors, Age (<18 months), MYCN amplification (FISH), ALK mutation/amplification, 11q aberration, 1p deletion, ploidy, segmental chromosomal aberrations, INPC histology, Urinary catecholamines (VMA/HVA), 123I-MIBG Curie score / 18F-MFBG PET, Bone marrow minimal residual disease (PHOX2B, TH qPCR), GD2 expression (near-universal).
Scans and tests linked to this cancer: SPECT/CT, Ultrasound, MIBG imaging and 131I-MIBG therapy.
Bring copies of scan reports, pathology and blood results, and a list of every medicine and supplement.
The treatments I may be offered
- Very-low / low risk (L1, MS): Observation with serial imaging for asymptomatic L1/MS (many regress); surgery alone for resectable L1; short chemotherapy only for symptoms or progression. (INRG staging and risk groups, Ultrasound)
- Intermediate risk: 2-8 cycles of moderate chemotherapy (carboplatin, etoposide, cyclophosphamide, doxorubicin) guided by response and biology; surgery; isotretinoin in some protocols. (Doxorubicin, Cytotoxic chemotherapy, INRG staging and risk groups)
- High-risk: Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant. (Lorlatinib, Monoclonal antibodies)
- High risk: induction: 5-6 cycles (COG: topotecan-cyclophosphamide × 2 then cisplatin-etoposide, cyclophosphamide-doxorubicin-vincristine; SIOPEN: rapid COJEC); stem-cell harvest; ANBL1531 adds 131I-MIBG (randomised) or lorlatinib (ALK); ANBL17P1 adds dinutuximab to induction. (COG ANBL1531, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, Dinutuximab (ch14.18) / dinutuximab beta, Doxorubicin)
- High risk: local control: Surgical resection of primary after induction (gross total where safe); external-beam radiotherapy 21.6 Gy to primary site (boost to residual) and MIBG-avid metastatic sites; proton therapy where available. (IMRT / IGRT (modern external beam), Proton therapy)
- High risk: consolidation: Tandem autologous transplant (thiotepa-cyclophosphamide, then CEM) in North America (ANBL0532); single busulfan-melphalan transplant in Europe (HR-NBL1). (Tandem autologous transplant, Autologous stem cell transplant (high-dose therapy), COG ANBL0532, SIOPEN HR-NBL1)
- High risk: post-consolidation: Anti-GD2 antibody (dinutuximab + GM-CSF + isotretinoin; dinutuximab beta in Europe, no IL-2) × 5-6 cycles; then eflornithine maintenance 2 years (US, 2023). (Dinutuximab (ch14.18) / dinutuximab beta, COG ANBL0032, Eflornithine (DFMO), NMTRC003/003B (DFMO maintenance), Caution: IL-2 added to anti-GD2 therapy)
- Relapsed / refractory: Irinotecan-temozolomide + dinutuximab or naxitamab (ANBL1221); naxitamab + GM-CSF for marrow/bone disease; 131I-MIBG for MIBG-avid disease; lorlatinib for ALK; GD2 CAR-T (Italy, trials); DFMO-based maintenance; palliative radiotherapy. (Dinutuximab (ch14.18) / dinutuximab beta, Naxitamab, Naxitamab Study 201, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, GD2-CART01 (Bambino Gesù phase 1/2), Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy))
- Survivorship: Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic. (Cardio-oncology)
From the standard of care recorded for this cancer; which apply depends on your stage and biomarkers. Ask which the team recommends and why.