The first 60 days: Medulloblastoma
Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less. Below, week by week, is what OnCo's record of Medulloblastoma 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.
- SurgeonNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp).
- Medical oncologistNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), High risk (metastatic, residual, anaplastic, MYC), Infants (<3 years), Relapsed.
- Clinical oncologist (radiotherapy)Named in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), Infants (<3 years), Relapsed.
- Transplant and cell therapy teamNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), High risk (metastatic, residual, anaplastic, MYC), Infants (<3 years).
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.
Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.
CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols.
Resection, CSI 23.4 Gy with boost to 54 Gy (proton where available), then cisplatin/vincristine/cyclophosphamide or lomustine-based chemotherapy (ACNS0331); WNT tumours receive reduced CSI in trials.
- 4.Relapsed
Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.
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 Molecular subgroup, MYC / MYCN amplification, TP53 mutation, Metastatic stageby MRI and CSF cytology, Extent of resection), 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 WNT-activated, SHH-activated, TP53-wild-type, SHH-activated, TP53-mutant.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp)
- For my situation (average risk (≥3 years, m0, <1.5 cm² residual, no myc amp)), which of the standard options do you recommend and why?Guideline options include: Resection, CSI 23.4 Gy with boost to 54 Gy (proton where available), then cisplatin/vincristine/cyclophosphamide or lomustine-based chemotherapy (ACNS0331); WNT tumours receive reduced CSI in trials.
- Am I a candidate for Cisplatin, Vincristine, Cyclophosphamide or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
High risk (metastatic, residual, anaplastic, MYC)
- For my situation (high risk (metastatic, residual, anaplastic, myc)), which of the standard options do you recommend and why?Guideline options include: CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols.
- Am I a candidate for Carboplatin, Cisplatin, Cyclophosphamide, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Infants (<3 years)
- For my situation (infants (<3 years)), which of the standard options do you recommend and why?Guideline options include: Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.
- Am I a candidate for Methotrexate, Cyclophosphamide, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed
- For my situation (relapsed), which of the standard options do you recommend and why?Guideline options include: Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.
- Am I a candidate for Temozolomide, Irinotecan (and liposomal irinotecan), Bevacizumab or related drugs, 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 Proton therapy, Vismodegib, 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 “Group 3 MYC-amplified and SHH TP53-mutant tumours: survival under 50%”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Relapse after craniospinal irradiation is rarely curable; it is where new approaches are most needed”. 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.
- Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid TumorsPhase 2 · active · NCT03709680PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS
- Childhood Cancer Survivor Study (CCSS)Phase observational · active · NCT01120353Retrospective cohort with prospective follow-up of five-year survivors of childhood cancer diagnosed 1970-1999 at 31 North American centres, with sibling controls
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Medulloblastoma: the full pageMedulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less.
- 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.
- Blood-brain barrier (BBB): The tight seal around brain blood vessels that keeps most drugs out, one of the two main reasons brain cancer is so hard to treat.
- Li-Fraumeni syndrome (germline TP53): Li-Fraumeni syndrome is an inherited fault in the TP53 gene giving a lifetime cancer risk near 100% in women and ~75% in men, with sarcomas, breast cancer, brain tumours, adrenal cancer and leukaemias often in childhood.
Every term links to the glossary.