High-risk neuroblastoma
Prepared with OnCo (onco.cc/prep/neuroblastoma-high-risk/). 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
23 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 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?
- 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 (induction), which of the standard options do you recommend and why?
- 6.Am I a candidate for Cyclophosphamide, Topotecan, Cisplatin or related drugs, and what side effects should I expect?
- 7.How do the results of COG ANBL1531 apply to someone like me?
- 8.For my situation (consolidation), which of the standard options do you recommend and why?
- 9.Am I a candidate for Busulfan, Melphalan (including hepatic delivery system), Thiotepa or related drugs, and what side effects should I expect?
- 10.How do the results of COG ANBL0532 and SIOPEN HR-NBL1 apply to someone like me?
- 11.For my situation (local control), which of the standard options do you recommend and why?
- 12.For my situation (post-consolidation), which of the standard options do you recommend and why?
- 13.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?
- 14.How do the results of COG ANBL0032 and NMTRC003/003B (DFMO maintenance) apply to someone like me?
- 15.For my situation (relapsed or refractory), which of the standard options do you recommend and why?
- 16.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?
- 17.How do the results of Naxitamab Study 201 and GD2-CART01 (Bambino Gesù phase 1/2) apply to someone like me?
- 18.For my situation (survivorship), which of the standard options do you recommend and why?
- 19.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)?
- 20.Would a second opinion at a high-volume centre change anything, and can you help arrange it?
- 21.What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
- 22.I read that “About half of children relapse and relapsed disease is rarely cured”. How does that affect my plan?
- 23.I read that “MYCN has no direct inhibitor; eflornithine and lorlatinib act around it”. How does that affect my plan?
The words I may hear
- 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.
Tests and results to bring
Biomarker results to ask for: MYCN amplification, ALK mutation or amplification, 11q loss, 1p loss and 17q gain, TERT rearrangement and ATRX loss (telomere maintenance), MIBG avidity and Curie or SIOPEN score, End-of-induction response (INRC), Marrow minimal residual disease by GD2 synthase or PHOX2B PCR, Urinary catecholamine metabolites.
Scans and tests linked to this cancer: 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
- Induction: 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. (Cyclophosphamide, Topotecan, Cisplatin, Etoposide, Doxorubicin, Vincristine, COG ANBL1531, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, MIBG imaging and 131I-MIBG therapy)
- Consolidation: Busulfan-melphalan (HR-NBL1) or tandem thiotepa-cyclophosphamide then carboplatin-etoposide-melphalan (ANBL0532) with autologous stem cell rescue. (Busulfan, Melphalan (including hepatic delivery system), Thiotepa, Carboplatin, Etoposide, Autologous stem cell transplant (high-dose therapy), Tandem autologous transplant, COG ANBL0532, SIOPEN HR-NBL1)
- Local control: Radiotherapy to the primary site bed and residual MIBG-avid metastases after transplant. (IMRT / IGRT (modern external beam), Proton therapy)
- Post-consolidation: Dinutuximab with GM-CSF and isotretinoin for five cycles (ANBL0032; interleukin-2 dropped after HR-NBL1), then two years of eflornithine maintenance. (Dinutuximab (ch14.18) / dinutuximab beta, Sargramostim, Aldesleukin (high-dose IL-2), COG ANBL0032, Caution: IL-2 added to anti-GD2 therapy, Eflornithine (DFMO), NMTRC003/003B (DFMO maintenance))
- Relapsed or refractory: 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. (Dinutuximab (ch14.18) / dinutuximab beta, Irinotecan (and liposomal irinotecan), Temozolomide, Anti-GD2 antibody + irinotecan-temozolomide (chemoimmunotherapy), Naxitamab, Naxitamab Study 201, 131I-MIBG (iobenguane I-131) therapy, Lorlatinib, GD2-CART01 (Bambino Gesù phase 1/2))
- Survivorship: Hearing, cardiac, renal, endocrine, fertility and second-cancer follow-up for life. (Cardio-oncology, Late effects and survivorship toxicity, Oncofertility and fertility preservation)
From the standard of care recorded for this cancer; which apply depends on your stage and biomarkers. Ask which the team recommends and why.