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Group 3 and group 4 medulloblastoma: the decisions you may face

5 treatment settings, 4 with more than one named option. Each section lays out the options the standard of care names, what each is for, the trials behind them with their recorded results, the side effects and cautions on record, and questions to ask. Built from the cancer page's standard-of-care rows; nothing here is advice for your case.

Other settings

Average risk (aged 3 or over, no metastases, residual under 1.5 square centimetres)

Maximal safe resection, 23.4 Gy craniospinal radiotherapy with tumour-bed boost (ACNS0331 showed 18 Gy is inferior), then cisplatin, vincristine and cyclophosphamide or lomustine.

The options, in plain words
Proton therapyEstablished

Radiation using protons, which stop inside the tumour instead of passing through, so tissue behind it gets no dose.

  • No exit dose; lower integral dose
  • Reduced second cancers in children

IMRT and IGRT shape the radiation beam to the tumour's outline from multiple angles and check the patient's position with a scan before every session, so surrounding organs receive less dose. Fewer, larger doses are now standard in breast and prostate cancer, but a low-dose bath still spreads across normal tissue.

  • Conformal dose, fewer side effects
  • Hypofractionation saves visits

Cisplatin is the original platinum chemotherapy, discovered by accident in 1965; it cures testicular cancer and makes radiation work better in cervical and head and neck cancer.

A plant-derived chemotherapy from the Madagascar periwinkle that has been in almost every childhood leukaemia and lymphoma regimen since the 1960s.

Cyclophosphamide is an alkylating chemotherapy that damages DNA in dividing cells. It is part of CHOP for lymphoma, AC for breast cancer and VAC for childhood sarcomas, clears lymphocytes before CAR-T and prevents graft-versus-host disease after transplant; bladder bleeding, infertility and secondary leukaemia are its harms.

An old chemotherapy pill used when glioblastoma comes back, and the control arm most new glioblastoma drugs must beat.

The evidence behind it
  • Average-risk medulloblastoma, age 3-21: involved-field (tumour bed) vs whole posterior fossa boost; and, in children aged 3-7, 18 Gy vs 23.4 Gy craniospinal irradiation
    Show survival figures (1)

    Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

    • Involved-field boost non-inferior (5-year EFS 82.5% vs 80.5%); 18 Gy CSI inferior to 23.4 Gy (71.4% vs 82.9%).
    5-year event-free survival: boost volume (%): Involved-field boost 82.5 vs Posterior fossa boost 80.5 · HR 0.97 · source
The main trade-offs on record
  • Cost
  • Range uncertainty
  • Limited randomised evidence in adults
  • Low-dose bath to normal tissue
  • Motion management
  • Dose reduce or avoid for CrCl below 60 (carboplatin is the alternative).
  • Fatal if given intrathecally: label all syringes.
Questions to ask about this decision
  1. Between Proton therapy, IMRT / IGRT (modern external beam), Cisplatin and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. How closely do I match the people in COG ACNS0331, and does that change what the results mean for me?
    Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
  4. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  5. Does your recommendation follow the current guideline (NCI PDQ: Childhood Medulloblastoma and Other CNS Embryonal Tumors Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  6. For my situation (average risk (aged 3 or over, no metastases, residual under 1.5 square centimetres)), which of the standard options do you recommend and why?
    Why: Guideline options include: Maximal safe resection, 23.4 Gy craniospinal radiotherapy with tumour-bed boost (ACNS0331 showed 18 Gy is inferior), then cisplatin, vincristine and cyclophosphamide or lomustine.
  7. Am I a candidate for Cisplatin, Vincristine, Cyclophosphamide or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  8. How do the results of COG ACNS0331 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Add these to your appointment list, or take the full question set for this cancer.

Advanced, first line

High risk (metastatic, residual disease or MYC amplification)

36 Gy craniospinal radiotherapy with boost, daily carboplatin during radiotherapy for group 3 (ACNS0332), then cisplatin, vincristine and cyclophosphamide.

The options, in plain words

Carboplatin is a platinum chemotherapy that crosslinks DNA; it is part of the standard pre-surgery regimen for triple-negative breast cancer.

IMRT and IGRT shape the radiation beam to the tumour's outline from multiple angles and check the patient's position with a scan before every session, so surrounding organs receive less dose. Fewer, larger doses are now standard in breast and prostate cancer, but a low-dose bath still spreads across normal tissue.

  • Conformal dose, fewer side effects
  • Hypofractionation saves visits
Proton therapyEstablished

Radiation using protons, which stop inside the tumour instead of passing through, so tissue behind it gets no dose.

  • No exit dose; lower integral dose
  • Reduced second cancers in children

Cisplatin is the original platinum chemotherapy, discovered by accident in 1965; it cures testicular cancer and makes radiation work better in cervical and head and neck cancer.

A plant-derived chemotherapy from the Madagascar periwinkle that has been in almost every childhood leukaemia and lymphoma regimen since the 1960s.

Cyclophosphamide is an alkylating chemotherapy that damages DNA in dividing cells. It is part of CHOP for lymphoma, AC for breast cancer and VAC for childhood sarcomas, clears lymphocytes before CAR-T and prevents graft-versus-host disease after transplant; bladder bleeding, infertility and secondary leukaemia are its harms.

The evidence behind it

No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.

The main trade-offs on record
  • Dose by Calvert formula using GFR (see the calculators).
  • Low-dose bath to normal tissue
  • Motion management
  • Cost
  • Range uncertainty
  • Limited randomised evidence in adults
  • Dose reduce or avoid for CrCl below 60 (carboplatin is the alternative).
  • Fatal if given intrathecally: label all syringes.
Questions to ask about this decision
  1. Between Carboplatin, IMRT / IGRT (modern external beam), Proton therapy and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  4. Does your recommendation follow the current guideline (NCI PDQ: Childhood Medulloblastoma and Other CNS Embryonal Tumors Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (high risk (metastatic, residual disease or myc amplification)), which of the standard options do you recommend and why?
    Why: Guideline options include: 36 Gy craniospinal radiotherapy with boost, daily carboplatin during radiotherapy for group 3 (ACNS0332), then cisplatin, vincristine and cyclophosphamide.
  6. Am I a candidate for Carboplatin, Cisplatin, Vincristine or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Add these to your appointment list, or take the full question set for this cancer.

Other settings

Infants under three

Intensive chemotherapy with methotrexate, then high-dose consolidation with stem cell rescue, to avoid or delay craniospinal radiotherapy.

The options, in plain words

The 1948 antifolate that produced the first chemotherapy remissions in childhood leukaemia and the first cure of a solid tumour; still essential in ALL, lymphoma, osteosarcoma and CNS lymphoma.

Cyclophosphamide is an alkylating chemotherapy that damages DNA in dividing cells. It is part of CHOP for lymphoma, AC for breast cancer and VAC for childhood sarcomas, clears lymphocytes before CAR-T and prevents graft-versus-host disease after transplant; bladder bleeding, infertility and secondary leukaemia are its harms.

A plant-derived chemotherapy from the Madagascar periwinkle that has been in almost every childhood leukaemia and lymphoma regimen since the 1960s.

Carboplatin is a platinum chemotherapy that crosslinks DNA; it is part of the standard pre-surgery regimen for triple-negative breast cancer.

Thiotepa is an old chemotherapy with a modern job: at high doses it prepares patients for stem cell transplants, and it is still instilled into the bladder or body cavities for superficial bladder cancer and malignant effusions.

Autologous stem cell transplant collects the patient's own blood-forming stem cells, gives melphalan or another chemotherapy at a dose that would otherwise destroy the marrow, then returns the cells to rebuild it. It is standard consolidation in myeloma and for relapsed lymphoma, though CAR-T has displaced it in early-relapsing large B-cell lymphoma.

  • Permits dose intensity impossible otherwise
  • Decades of outcome data
  • Widely available
The evidence behind it

No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.

The main trade-offs on record
  • High-dose methotrexate requires normal renal function, hydration, urine alkalinisation and leucovorin rescue with level monitoring.
  • Fatal if given intrathecally: label all syringes.
  • Dose by Calvert formula using GFR (see the calculators).
  • Treatment-related mortality ~1-2% (myeloma) to higher in children
  • Infertility, second malignancies, prolonged cytopenias
  • Being challenged by CAR-T and MRD-guided deferral
Questions to ask about this decision
  1. Between Methotrexate, Cyclophosphamide, Vincristine and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  4. Does your recommendation follow the current guideline (NCI PDQ: Childhood Medulloblastoma and Other CNS Embryonal Tumors Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (infants under three), which of the standard options do you recommend and why?
    Why: Guideline options include: Intensive chemotherapy with methotrexate, then high-dose consolidation with stem cell rescue, to avoid or delay craniospinal radiotherapy.
  6. Am I a candidate for Methotrexate, Cyclophosphamide, Vincristine or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Add these to your appointment list, or take the full question set for this cancer.

Temozolomide with irinotecan, bevacizumab, re-irradiation or high-dose chemotherapy; almost never curative; early-phase trials.

The options, in plain words

The only chemotherapy proven to extend life in glioblastoma, given during and after radiation. It works best when the tumour has switched off a repair gene called MGMT.

Irinotecan is a topoisomerase-blocking chemotherapy central to bowel and pancreatic cancer regimens (FOLFIRI, FOLFIRINOX, NALIRIFOX) and to salvage therapy in childhood sarcomas; it carries the same warhead as the deruxtecan ADC payloads.

Bevacizumab is a humanised antibody that soaks up VEGF-A, the signal tumours use to grow new blood vessels. Approved in 2004, it partners chemotherapy in colorectal, ovarian, cervical, lung, kidney and liver cancer and glioblastoma, and adds to trifluridine/tipiracil in late-line bowel cancer; hypertension, protein in the urine and bleeding are its characteristic side effects.

Autologous stem cell transplant collects the patient's own blood-forming stem cells, gives melphalan or another chemotherapy at a dose that would otherwise destroy the marrow, then returns the cells to rebuild it. It is standard consolidation in myeloma and for relapsed lymphoma, though CAR-T has displaced it in early-relapsing large B-cell lymphoma.

  • Permits dose intensity impossible otherwise
  • Decades of outcome data
  • Widely available

IMRT and IGRT shape the radiation beam to the tumour's outline from multiple angles and check the patient's position with a scan before every session, so surrounding organs receive less dose. Fewer, larger doses are now standard in breast and prostate cancer, but a low-dose bath still spreads across normal tissue.

  • Conformal dose, fewer side effects
  • Hypofractionation saves visits
The evidence behind it

No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.

The main trade-offs on record
  • Take on an empty stomach or at bedtime to reduce nausea; PJP prophylaxis during concurrent chemoradiation.
  • UGT1A1*28 homozygotes: consider a lower starting dose (neutropenia). Cholinergic syndrome: atropine.
  • Treatment-related mortality ~1-2% (myeloma) to higher in children
  • Infertility, second malignancies, prolonged cytopenias
  • Being challenged by CAR-T and MRD-guided deferral
  • Low-dose bath to normal tissue
  • Motion management
Questions to ask about this decision
  1. Between Temozolomide, Irinotecan (and liposomal irinotecan), Bevacizumab and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  4. Does your recommendation follow the current guideline (NCI PDQ: Childhood Medulloblastoma and Other CNS Embryonal Tumors Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (relapsed), which of the standard options do you recommend and why?
    Why: Guideline options include: Temozolomide with irinotecan, bevacizumab, re-irradiation or high-dose chemotherapy; almost never curative; early-phase trials.
  6. Am I a candidate for Temozolomide, Irinotecan (and liposomal irinotecan), Bevacizumab, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Add these to your appointment list, or take the full question set for this cancer.

Special situations

Survivorship

One path named

Neurocognitive, endocrine, hearing, vascular and second-tumour follow-up for life.

The path, in plain words

Organised follow-up for the 18 million US and 50+ million global cancer survivors: watching for recurrence and second cancers, managing long-term side effects such as heart damage, infertility, neuropathy and fatigue, and helping people return to work and life.

  • Evidence-based screening prevents or detects late effects (e.g. breast MRI after chest radiation)
  • Nurse-led and primary-care models are as safe as specialist follow-up for low-risk survivors
  • Childhood survivor guidelines are mature and international (IGHG)
The evidence behind it

No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.

The main trade-offs on record
  • Care plans alone do not change outcomes
  • Fragmentation between oncology and primary care
  • Adult late-effects guidelines less developed than paediatric
Questions to ask about this decision
  1. Is Survivorship care and late-effects surveillance the only reasonable path for me, or is there a trial, a different sequence or a wait-and-see option?
    Why: A single standard does not mean a single choice; timing and trials are decisions too.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  4. Does your recommendation follow the current guideline (NCI PDQ: Childhood Medulloblastoma and Other CNS Embryonal Tumors Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (survivorship), which of the standard options do you recommend and why?
    Why: Guideline options include: Neurocognitive, endocrine, hearing, vascular and second-tumour follow-up for life.

Add these to your appointment list, or take the full question set for this cancer.

How to read this page. Options and results come from OnCo records with their sources; the settings are the standard-of-care rows on the cancer page, and the lines of therapy are on the sequencing grid. Where a setting names one path, the choice is usually about timing, trials and where to be treated: see expert centres. OnCo is orientation, not medical advice.