The first 60 days: Paediatric low-grade glioma
Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain. Below, week by week, is what OnCo's record of Paediatric low-grade glioma 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: Resectable tumour.
- SurgeonNamed in the standard of care for: Resectable tumour.
- Medical oncologistNamed in the standard of care for: Resectable tumour, Unresectable or progressive, BRAF V600E, Relapsed or refractory, BRAF fusion or V600E, Unresectable, no targetable alteration or targeted drug unavailable.
- Clinical oncologist (radiotherapy)Named in the standard of care for: Unresectable, no targetable alteration or targeted drug unavailable.
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.
Maximal safe resection; gross total resection is curative in most and no adjuvant therapy is given. Observation for stable residual disease.
Dabrafenib plus trametinib first line (TADPOLE: higher response rate and longer progression-free survival than carboplatin-vincristine; FDA approval March 2023 for patients aged one year and over).
Carboplatin and vincristine, or weekly vinblastine, to defer radiotherapy; focal conformal or proton radiotherapy is reserved for older children and for progression after systemic options.
Tovorafenib (FIREFLY-1; FDA accelerated approval April 2024 for patients aged six months and over), or a MEK inhibitor such as selumetinib in trials.
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 KIAA1549-BRAF fusion, BRAF V600E, NF1 germline status, FGFR1 mutation or fusion, CDKN2A deletion), 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 Pilocytic astrocytoma, Ganglioglioma and pleomorphic xanthoastrocytoma, NF1-associated optic pathway glioma.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Resectable tumour
- For my situation (resectable tumour), which of the standard options do you recommend and why?Guideline options include: Maximal safe resection; gross total resection is curative in most and no adjuvant therapy is given. Observation for stable residual disease.
Unresectable or progressive, BRAF V600E
- For my situation (unresectable or progressive, braf v600e), which of the standard options do you recommend and why?Guideline options include: Dabrafenib plus trametinib first line (TADPOLE: higher response rate and longer progression-free survival than carboplatin-vincristine; FDA approval March 2023 for patients aged one year and over).
- Am I a candidate for Dabrafenib + trametinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of TADPOLE (CDRB436G2201) apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Relapsed or refractory, BRAF fusion or V600E
- For my situation (relapsed or refractory, braf fusion or v600e), which of the standard options do you recommend and why?Guideline options include: Tovorafenib (FIREFLY-1; FDA accelerated approval April 2024 for patients aged six months and over), or a MEK inhibitor such as selumetinib in trials.
- Am I a candidate for Tovorafenib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of FIREFLY-1 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Unresectable, no targetable alteration or targeted drug unavailable
- For my situation (unresectable, no targetable alteration or targeted drug unavailable), which of the standard options do you recommend and why?Guideline options include: Carboplatin and vincristine, or weekly vinblastine, to defer radiotherapy; focal conformal or proton radiotherapy is reserved for older children and for progression after systemic options.
- Am I a candidate for Carboplatin, Vincristine, Vinblastine, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Tovorafenib, Dabrafenib + trametinib, FIREFLY-1, DNA methylation profiling?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 “How long to continue MAPK inhibitors and whether tumours regrow on stopping; intermittent dosing and stop rules are being studied in FIREFLY-2 and the COG selumetinib trials”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Long-term effects of RAF and MEK inhibition on growth plates, skin and heart in children who may take them for years; registries and trial follow-up are collecting these data”. 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.
- Paediatric low-grade glioma: the full pagePaediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain.
- 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.
- 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.
- 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.