The first 60 days: High-risk neuroblastoma
High-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse. Below, week by week, is what OnCo's record of High-risk neuroblastoma says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Ask which of these were tested and what the results were; see the report reader for what each value means.
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.
- RadiologistNamed in the standard of care for: Induction, Relapsed or refractory.
- SurgeonNamed in the standard of care for: Induction, Consolidation.
- Medical oncologistNamed in the standard of care for: Induction, Consolidation, Local control, Post-consolidation and 2 more.
- Clinical oncologist (radiotherapy)Named in the standard of care for: Induction, Local control, Relapsed or refractory.
- Transplant and cell therapy teamNamed in the standard of care for: Induction, Consolidation, Local control, Post-consolidation.
- Palliative and supportive care teamNamed in the standard of care for: Induction, Survivorship.
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.
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.
Five or six cycles of cyclophosphamide-topotecan, cisplatin-etoposide and cyclophosphamide-doxorubicin-vincristine (COG) or rapid COJEC (SIOPEN); iodine-131 MIBG and, for ALK-mutant disease, lorlatinib within ANBL1531; surgery after induction.
Busulfan-melphalan (HR-NBL1) or tandem thiotepa-cyclophosphamide then carboplatin-etoposide-melphalan (ANBL0532) with autologous stem cell rescue.
Radiotherapy to the primary site bed and residual MIBG-avid metastases after transplant.
Dinutuximab with GM-CSF and isotretinoin for five cycles (ANBL0032; interleukin-2 dropped after HR-NBL1), then two years of eflornithine maintenance.
Irinotecan-temozolomide with dinutuximab (ANBL1221) or naxitamab with GM-CSF; iodine-131 MIBG for avid disease; lorlatinib for ALK-mutant disease; GD2 CAR T-cells in trials.
Hearing, cardiac, renal, endocrine, fertility and second-cancer follow-up for life.
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 or amplification, 11q loss, 1p loss and 17q gain, TERT rearrangement and ATRX loss, MIBG avidity and Curie or SIOPEN score), 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 Stage M neuroblastoma over 18 months without MYCN amplification, MYCN-amplified neuroblastoma at any age or stage, ALK-mutated or ALK-amplified high-risk neuroblastoma.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Induction
- For my situation (induction), which of the standard options do you recommend and why?Guideline options include: Five or six cycles of cyclophosphamide-topotecan, cisplatin-etoposide and cyclophosphamide-doxorubicin-vincristine (COG) or rapid COJEC (SIOPEN); iodine-131 MIBG and, for ALK-mutant disease, lorlatinib within ANBL1531; surgery after induction.
- Am I a candidate for Cyclophosphamide, Topotecan, Cisplatin 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 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Consolidation
- For my situation (consolidation), which of the standard options do you recommend and why?Guideline options include: Busulfan-melphalan (HR-NBL1) or tandem thiotepa-cyclophosphamide then carboplatin-etoposide-melphalan (ANBL0532) with autologous stem cell rescue.
- Am I a candidate for Busulfan, Melphalan (including hepatic delivery system), Thiotepa 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 ANBL0532 and SIOPEN HR-NBL1 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Local control
- For my situation (local control), which of the standard options do you recommend and why?Guideline options include: Radiotherapy to the primary site bed and residual MIBG-avid metastases after transplant.
Post-consolidation
- For my situation (post-consolidation), which of the standard options do you recommend and why?Guideline options include: Dinutuximab with GM-CSF and isotretinoin for five cycles (ANBL0032; interleukin-2 dropped after HR-NBL1), then two years of eflornithine maintenance.
- Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Sargramostim, Aldesleukin (high-dose IL-2) 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 ANBL0032 and NMTRC003/003B (DFMO maintenance) apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Relapsed or refractory
- For my situation (relapsed or refractory), which of the standard options do you recommend and why?Guideline options include: Irinotecan-temozolomide with dinutuximab (ANBL1221) or naxitamab with GM-CSF; iodine-131 MIBG for avid disease; lorlatinib for ALK-mutant disease; GD2 CAR T-cells in trials.
- Am I a candidate for Dinutuximab (ch14.18) / dinutuximab beta, Irinotecan (and liposomal irinotecan), Temozolomide 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: Hearing, cardiac, renal, endocrine, fertility and second-cancer follow-up for life.
Any stage
- Are there clinical trials I could join, for example of COG ANBL1531, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, GD2-CART01 (Bambino Gesù phase 1/2)?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 “About half of children relapse and relapsed disease is rarely cured”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “MYCN has no direct inhibitor; eflornithine and lorlatinib act around it”. How does that affect my plan?Open problems are where trials and second opinions matter most.
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.
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- High-risk neuroblastoma: the full pageHigh-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- 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.
- Late effects and survivorship toxicity: Health problems appearing months or decades after treatment ends: heart damage, infertility, second cancers, lymphoedema, dry mouth, memory problems, weak bones.
Every term links to the glossary.