The first 60 days: Neuroblastoma (paediatric)

Below, week by week, is what OnCo's record of Neuroblastoma (paediatric) says about the first two months: the order is typical, the timing is yours to ask about. Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease. Sections appear only where the record has something to say. Orientation, not medical advice.

Weeks 1 to 2

What happens now

Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.

Biomarkers your report may list

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)

Ask which of these were tested and what the results were; see the report reader for what each value means.

Tests and imaging linked to this cancer

SPECT/CT Ultrasound MIBG imaging and 131I-MIBG therapy

Weeks 1 to 3

Who is on your team

These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.

A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.

Weeks 2 to 6

Decisions coming up

Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.

1.Very-low / low risk (L1, MS)NCCN category COG/SIOPEN low-risk protocols (observation or surgery), COG ANBL1232 / SIOPEN LINES
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
2.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
3.High-risk NCI PDQ: Neuroblastoma Treatment (COG risk groups)
Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant.
Lorlatinib Monoclonal antibodies
4.High risk: inductionNCCN category COG ANBL1531 backbone, COG 2026
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 ANBL17P1
5.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
6.High risk: consolidationNCCN category COG standard (tandem); SIOPEN standard (BuMel), 2026
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
7.High risk: post-consolidationNCCN category Dinutuximab: FDA-approved standard; eflornithine: FDA-approved maintenance, 2026
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
8.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)ANBL1221
9.Survivorship
Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic.
Cardio-oncology

Every visit

Questions to ask at each visit

Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.

Newly diagnosed

What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
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?These results decide eligibility for targeted therapy, immunotherapy, and trials.
Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include Very-low and low risk, Intermediate risk, High risk.
Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.

High-risk

For my situation (high-risk), which of the standard options do you recommend and why?Guideline options include: Induction chemo → surgery → tandem transplant → RT → anti-GD2 + isotretinoin; lorlatinib if ALK-mutant.
Am I a candidate for Lorlatinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.

Very-low / low risk (L1, MS)

For my situation (very-low / low risk (l1, ms)), which of the standard options do you recommend and why?Guideline options include: Observation with serial imaging for asymptomatic L1/MS (many regress); surgery alone for resectable L1; short chemotherapy only for symptoms or progression.

Intermediate risk

For my situation (intermediate risk), which of the standard options do you recommend and why?Guideline options include: 2-8 cycles of moderate chemotherapy (carboplatin, etoposide, cyclophosphamide, doxorubicin) guided by response and biology; surgery; isotretinoin in some protocols.
Am I a candidate for Doxorubicin, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.

High risk: induction

For my situation (high risk: induction), which of the standard options do you recommend and why?Guideline options include: 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.
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?Knowing the expected toxicities helps you plan work, family, and supportive care.
How do the results of COG ANBL1531 and ANBL17P1 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.

High risk: local control

For my situation (high risk: local control), which of the standard options do you recommend and why?Guideline options include: 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.

High risk: consolidation

For my situation (high risk: consolidation), which of the standard options do you recommend and why?Guideline options include: Tandem autologous transplant (thiotepa-cyclophosphamide, then CEM) in North America (ANBL0532); single busulfan-melphalan transplant in Europe (HR-NBL1).
How do the results of COG ANBL0532 and SIOPEN HR-NBL1 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.

High risk: post-consolidation

For my situation (high risk: post-consolidation), which of the standard options do you recommend and why?Guideline options include: Anti-GD2 antibody (dinutuximab + GM-CSF + isotretinoin; dinutuximab beta in Europe, no IL-2) × 5-6 cycles; then eflornithine maintenance 2 years (US, 2023).
Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Eflornithine (DFMO), and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
How do the results of COG ANBL0032 and NMTRC003/003B (DFMO maintenance) apply to someone like me?Trial populations differ from individual patients; ask how closely you match.

Relapsed / refractory

For my situation (relapsed / refractory), which of the standard options do you recommend and why?Guideline options include: 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.
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?Knowing the expected toxicities helps you plan work, family, and supportive care.
How do the results of Naxitamab Study 201 and GD2-CART01 (Bambino Gesù phase 1/2) apply to someone like me?Trial populations differ from individual patients; ask how closely you match.

Survivorship

For my situation (survivorship), which of the standard options do you recommend and why?Guideline options include: Audiology (platinum, DFMO), endocrine and fertility follow-up, cardiac surveillance (anthracycline), second-malignancy screening, neurocognitive support; lifelong late-effects clinic.

Any stage

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?Trials are how the next standard of care is set; asking early keeps options open.
Would a second opinion at a high-volume centre change anything, and can you help arrange it?Rare or high-stakes decisions benefit from a centre that treats many similar patients.
What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Supportive care improves quality of life and helps patients complete treatment.
I read that “Relapsed high-risk disease”. How does that affect my plan?Open problems are where trials and second opinions matter most.
I read that “Long-term toxicity of intensive therapy”. How does that affect my plan?Open problems are where trials and second opinions matter most.

Weeks 3 to 8

Trials to ask about

Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.

From day one

Help that costs nothing

Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.

Any time

What to read next

Words this page and the cancer page use

MIBG Curie and SIOPEN scores: Neuroblastoma soaks up MIBG, a radioactive cousin of noradrenaline; the Curie and SIOPEN scores count how many body regions still light up on the scan, and a score that has not fallen enough after the first rounds of chemotherapy marks a child who is unlikely to be cured with the standard plan.
ADCC (antibody-dependent cellular cytotoxicity): In ADCC, an antibody flags a cell, and NK cells recognise the flag and kill it.
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.
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.
Segmental chromosomal aberrations and ploidy (neuroblastoma): In a child's neuroblastoma the pattern of chromosome damage is a crystal ball: tumours that have gained or lost whole chromosomes tend to regress or be cured, tumours with broken segments (11q loss, 1p loss, 17q gain) or with MYCN amplified relapse, and the pattern moves a child between low, intermediate and high-risk treatment.
Urinary catecholamine metabolites (VMA and HVA): Neuroblastoma cells make adrenaline-type hormones and spill their breakdown products, VMA and HVA, into urine; a simple urine test supports the diagnosis in nine out of ten children, and falling levels after treatment or rising ones in follow-up track the tumour without a scan.
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.
Event-free / disease-free survival (EFS, DFS, iDFS, RFS): In early-stage cancer: how long patients stay free of recurrence, progression, or death.
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.

Every term links to the glossary.