The first 60 days: Atypical teratoid/rhabdoid tumour (ATRT)
ATRT is an aggressive brain tumour of babies and toddlers caused by loss of a single gene, SMARCB1, part of the machinery that opens and closes DNA. Intensive chemotherapy with stem-cell rescue, and radiotherapy where age allows, now cure a meaningful share of children who once had little chance, and drugs aimed at the epigenetic consequence of SMARCB1 loss (EZH2 inhibitors) are in trials. Below, week by week, is what OnCo's record of Atypical teratoid/rhabdoid tumour (ATRT) 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.
Maximal safe resection followed by an intensive multimodal protocol: ACNS0333-style induction, high-dose chemotherapy with autologous stem-cell rescue, and age-adapted focal radiotherapy; or the EU-RHAB regimen with intraventricular methotrexate. Enrolment in SIOPE ATRT01 or a COG successor where available.
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.
- PathologistNamed in the standard of care for: Germline SMARCB1 or SMARCA4 alteration.
- RadiologistNamed in the standard of care for: Germline SMARCB1 or SMARCA4 alteration.
- SurgeonNamed in the standard of care for: Newly diagnosed, any age.
- Medical oncologistNamed in the standard of care for: Newly diagnosed, any age, Relapsed or refractory.
- Clinical oncologist (radiotherapy)Named in the standard of care for: Newly diagnosed, any age, Relapsed or refractory.
- Transplant and cell therapy teamNamed in the standard of care for: Newly diagnosed, any age.
- Palliative and supportive care teamNamed in the standard of care for: Relapsed or refractory.
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.
Genetic counselling and testing of parents and siblings; surveillance imaging for synchronous or second rhabdoid tumours in the kidney and soft tissue.
No standard; EZH2 inhibition with tazemetostat was explored in trials and compassionate use until the drug was withdrawn from all markets in March 2026; aurora kinase A inhibition, re-irradiation where feasible; early palliative care.
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 Loss of SMARCB1nuclear staining by immunohistochemistry, SMARCB1 or SMARCA4 sequencing, somatic and germline, Methylation subgroup, Metastatic stage on MRI and cerebrospinal-fluid cytology, Age), 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 ATRT-TYR, ATRT-SHH, ATRT-MYC.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Newly diagnosed, any age
- For my situation (newly diagnosed, any age), which of the standard options do you recommend and why?Guideline options include: Maximal safe resection followed by an intensive multimodal protocol: ACNS0333-style induction, high-dose chemotherapy with autologous stem-cell rescue, and age-adapted focal radiotherapy; or the EU-RHAB regimen with intraventricular methotrexate. Enrolment in SIOPE ATRT01 or a COG successor where available.
- Am I a candidate for Methotrexate, Cyclophosphamide, Cisplatin or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Germline SMARCB1 or SMARCA4 alteration
- For my situation (germline smarcb1 or smarca4 alteration), which of the standard options do you recommend and why?Guideline options include: Genetic counselling and testing of parents and siblings; surveillance imaging for synchronous or second rhabdoid tumours in the kidney and soft tissue.
Relapsed or refractory
- For my situation (relapsed or refractory), which of the standard options do you recommend and why?Guideline options include: No standard; EZH2 inhibition with tazemetostat was explored in trials and compassionate use until the drug was withdrawn from all markets in March 2026; aurora kinase A inhibition, re-irradiation where feasible; early palliative care.
- Am I a candidate for Tazemetostat, 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 DNA methylation profiling, Proton therapy, NCI-COG Pediatric MATCH (APEC1621)?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 “Infants too young for radiotherapy and children with metastatic or ATRT-MYC disease still do poorly; SIOPE ATRT01 and COG successors are testing intensified and subgroup-directed therapy”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Long-term neurocognitive and endocrine cost of intensive therapy in the first years of life; proton therapy and radiation-sparing arms aim to reduce 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.
- Atypical teratoid/rhabdoid tumour (ATRT): the full pageATRT is an aggressive brain tumour of babies and toddlers caused by loss of a single gene, SMARCB1, part of the machinery that opens and closes DNA. Intensive chemotherapy with stem-cell rescue, and radiotherapy where age allows, now cure a meaningful share of children who once had little chance, and drugs aimed at the epigenetic consequence of SMARCB1 loss (EZH2 inhibitors) are in trials.
- 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.
- 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.
- Hereditary cancer syndromes: About 5-10% of cancers arise from an inherited gene fault.
Every term links to the glossary.