Ependymoma
Ependymomas grow from the cells lining the fluid spaces of the brain and spinal cord. Removing the whole tumour, followed by precisely targeted radiotherapy, cures many children; the tumour has been sorted into molecular groups that behave very differently, and the field's work is to match the intensity of treatment to each group and to find the first effective drugs.
Overview
Ependymoma is a glial tumour arising along the ventricular system and spinal canal. The WHO 2021 classification replaced grade-based labels with molecular groups that carry prognostic weight: posterior fossa group A (PF-A, infants and young children, CpG hypermethylation with EZHIP overexpression and loss of H3K27me3, chromosome 1q gain marks high risk), posterior fossa group B (PF-B, older children and adults, favourable), supratentorial ZFTA (formerly RELA) fusion-positive (NF-kB driven), supratentorial YAP1 fusion-positive (young children, favourable), and spinal groups including myxopapillary ependymoma and MYCN-amplified spinal ependymoma. Few recurrent point mutations exist; the disease is largely driven by structural and epigenetic changes.
Treatment is surgical: gross total resection is the strongest modifiable prognostic factor, and second-look surgery for residual disease is standard practice. Post-operative conformal radiotherapy to the tumour bed, including in children as young as one year, was established by the St Jude RT1 and COG ACNS0121 studies and controls disease without the whole-brain exposure of earlier eras; proton therapy is increasingly used to spare cochlea, hypothalamus and healthy brain. Chemotherapy has a limited role: it is used to bridge infants to radiotherapy or to enable second surgery, and the COG trial ACNS0831 tested maintenance chemotherapy after radiotherapy without establishing it as a universal standard. There is no approved targeted drug.
The active fronts are molecular risk stratification (SIOP Ependymoma II, ACNS0831 successors), reducing radiotherapy in favourable groups such as PF-B and ST-YAP1, and finding therapies for PF-A and ZFTA tumours, where relapse is common and repeated surgery and re-irradiation are the mainstay. Epigenetic dependencies (EZHIP and PRC2 biology in PF-A, NF-kB signalling in ZFTA), CDK4/6 inhibition and immunotherapy against fusion neoantigens are in preclinical and early clinical work.
State of the art today
- Conformal and proton radiotherapy immediately after surgery, even in very young children, gives durable control with far less neurocognitive harm than older whole-brain approaches.
- Gross total resection remains the single most important treatment step; second-look surgery is standard when residual disease is seen.
- No drug has yet changed outcomes; epigenetic and fusion-directed strategies are the pipeline.
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.
- Molecular groups (PF-A, PF-B, ZFTA, YAP1, spinal) replaced grade as the basis of prognosis in WHO 2021 and are being used to de-escalate treatment for favourable groups.
Where it starts and where it drains
Gliomas infiltrate along white matter and can cross the corpus callosum, medulloblastoma sits in the cerebellum, and CNS lymphoma favours deep periventricular tissue; none spread through lymph nodes.
- Frontal lobe (glioblastoma commonest)
- Temporal lobe
- Corpus callosum (butterfly glioma)
- Lower-grade IDH-mutant glioma
- Cerebellum (medulloblastoma)
- Brainstem (diffuse midline glioma)Posterior fossa group A (PF-A; EZHIP, H3K27me3 loss; 1q gain high risk)
- Ventricles and ependymal lining (ependymoma)Posterior fossa group A (PF-A; EZHIP, H3K27me3 loss; 1q gain high risk) · Posterior fossa group B (PF-B; favourable) · Supratentorial, ZFTA fusion-positive · Supratentorial, YAP1 fusion-positive · Spinal ependymoma (including MYCN-amplified) · Myxopapillary ependymoma · Subependymoma
- Sella and pituitary (pituitary tumours, craniopharyngioma)
- Deep periventricular tissue (CNS lymphoma)
No conventional lymphatics: gliomas spread along white matter tracts and, rarely, through cerebrospinal fluid; medulloblastoma can seed the spine.
Same organ: Glioma & glioblastoma, Primary CNS lymphoma, Medulloblastoma, Paediatric low-grade glioma, Diffuse midline glioma, H3 K27-altered (including DIPG), Atypical teratoid/rhabdoid tumour (ATRT), Craniopharyngioma, Pituitary tumours (pituitary neuroendocrine tumours) and pituitary carcinoma
Roughly one in ten childhood brain tumours, most under five years of age; also occurs in adults, mainly in the spine (NCI PDQ).
- DNA methylation profilingEstablished
- AI in radiologyEstablished
- cfDNA fragmentomicsEstablished
- Colorectal cancer screening (colonoscopy, FIT, stool DNA, blood)Standard of care
- HCC surveillance in cirrhosis (ultrasound + AFP)Standard of care
- High-risk pancreatic surveillance (CAPS / PRECEDE)Established
- A 28-day national pathway for people with a positive multi-cancer blood test
- A breath test to rule out cancer in people with vague symptoms
- A cancer blood test for older people arriving at A&E with unexplained symptoms
- A legislated, publicly reported 28-day standard from urgent referral to diagnosis
- A live national dashboard of stage at diagnosis as the scorecard for early detection
- A single 'cancer check at 60' appointment bundling all screening tests
Background: Alpha-fetoprotein (AFP), Barrett's oesophagus, CA 19-9, Early detection, Faecal immunochemical test (FIT). Also on OnCo: Symptoms and red flags · Early detection roadmap.
Where the cases are
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
Maximal safe resection, second-look surgery for residual disease, then conformal or proton radiotherapy to the tumour bed (ACNS0121 approach); craniospinal irradiation only for disseminated disease.
Chemotherapy (vincristine, carboplatin, cyclophosphamide, etoposide-based) to delay radiotherapy or facilitate second surgery, per SIOP Ependymoma II and COG protocols.
Repeat resection and re-irradiation (focal or craniospinal) where feasible; no standard systemic therapy, so trial enrolment (including Pediatric MATCH-style molecular assignment) is recommended.
Subtypes & biomarkers
top- Posterior fossa group A (PF-A; EZHIP, H3K27me3 loss; 1q gain high risk)
- Posterior fossa group B (PF-B; favourable)
- Supratentorial, ZFTA fusion-positive
- Supratentorial, YAP1 fusion-positive
- Spinal ependymoma (including MYCN-amplified)
- Myxopapillary ependymoma
- Subependymoma
- Methylation-based molecular group
- H3K27me3 loss and EZHIP expression (PF-A)
- ZFTA or YAP1 fusion by FISH or sequencing
- Chromosome 1q gain and 6q loss (PF-A risk)
- MYCN amplification (spinal)
- Extent of resection on post-operative MRI
- Cerebrospinal-fluid cytology and spinal MRI for dissemination
Target prevalence in this cancer
- 2009Conformal radiotherapy safe and effective in young children
St Jude RT1 (Merchant, Lancet Oncology) shows tumour-bed irradiation controls disease with preserved cognition.
- 2011Two posterior fossa groups discovered
Witt and colleagues (Cancer Cell) separate PF-A and PF-B by expression.
- 2014C11orf95-RELA (ZFTA) fusion identified in supratentorial ependymoma
Parker and colleagues (Nature) link the fusion to NF-kB activation.
- 2015Nine molecular groups across the neuraxis
Pajtler and colleagues (Cancer Cell) define the methylation-based classification later adopted by WHO.
- 2019ACNS0121 confirms post-operative conformal radiotherapy
COG phase 2 (JCO 2019) reports outcomes by extent of resection and molecular group.
- 2021WHO 2021 adopts molecular groups for ependymoma
Open problems, and what is being done about each
PF-A and ZFTA tumours relapse often and have no effective drug; EZHIP/PRC2 and NF-kB dependencies are being tested preclinically and in early trials.
and how the field plans to fix it →What is being done about thisRecurrence and residual diseaseAvailable now- Survivorship care and late-effects surveillanceEstablished
- Liquid biopsy (ctDNA)Standard of care
- MRD / molecular residual disease testingEstablished
- Multiparameter flow cytometry MRDStandard of care
- NGS-based MRD (clonoSEQ and molecular MRD)Standard of care
- SignateraEstablished
In trials- NCI-COG Pediatric MATCH (APEC1621)Active
- ADAURAPositive
- CAMBRIA-1 & CAMBRIA-2Recruiting
- CIRCULATE-Japan (GALAXY / VEGA / ALTAIR)Active
- ctDNA monitoring in lymphoma (PhasED-seq, clonoSEQ)Emerging
- DYNAMICPositive
Ideas and roadmaps- A blood test for the pre-metastatic niche
- A bone marrow niche on a chip to study human dormancy
- A dedicated clinic for people whose blood test says the cancer is back
- A drug screen that only rewards killing sleeping cancer cells
- A national platform trial that every ctDNA-positive patient can join
- A national residual-disease weather service: serial blood tests for every curatively treated patient, pooled
Background: Circulating tumour DNA (ctDNA), Disseminated tumour cells (DTCs), Late recurrence, Minimal / molecular residual disease (MRD), MRD negativity (myeloma, 10⁻⁵ / 10⁻⁶). Also on OnCo: Treatment journeys · Survivorship planner.
Whether radiotherapy can be omitted or reduced in PF-B, YAP1 and completely resected favourable tumours; SIOP Ependymoma II and COG successors are stratifying by group.
Late effects of radiotherapy in very young children; proton therapy is expected to reduce them and is being tracked in registries.
and how the field plans to fix it →What is being done about thisSide effects and quality of lifeAvailable now- IMRT / IGRT (modern external beam)Standard of care
- Proton therapyEstablished
- Survivorship care and late-effects surveillanceEstablished
- Cardio-oncologyEstablished
- Exercise & lifestyle oncologyEstablished
- Geriatric assessmentEstablished
In trialsIdeas and roadmaps- A cheap old tablet to restore appetite
- A coordinated FLASH radiotherapy evidence programme with shared dose-rate standards
- A dedicated programme for cachexia and treatment toxicity research
- A dietitian in every gastrointestinal and head and neck tumour board
- A funded programme of organ-preservation trials to avoid radical surgery
- A lifelong late-effects registry linked to every treatment for adult survivors
Background: CTCAE toxicity grading (grade 3-4 adverse events), De-escalation, escalation and response-adapted therapy, Immune-related adverse events (irAEs), Quality of life, Toxicity grade. Also on OnCo: Side effects, symptom first · Immune-related side effects · Toxicity compare · Survivorship planner.
Adult spinal and intracranial ependymoma lack dedicated trials and borrow from paediatric evidence.
Trials
topRecruiting now (live from ClinicalTrials.gov)
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Landmark trials in OnCo
Expert centres
topCentres linked to this cancer in OnCo
- via Proton therapy
- via Proton therapy
- via Proton therapy
- via DNA methylation profiling
- via Proton therapy
- via Proton therapy
- Aarhus University HospitalAarhus, DKvia Proton therapy, IMRT / IGRT (modern external beam)
- American Society for Radiation OncologyArlington, VA, USvia Proton therapy, IMRT / IGRT (modern external beam)
- via DNA methylation profiling, Proton therapy
- Centre Antoine LacassagneNice, FRvia Proton therapy, IMRT / IGRT (modern external beam)
- Children's Oncology Group (COG)Monrovia, CA, USvia this cancer, NCI-COG Pediatric MATCH (APEC1621)
- European Society for Radiotherapy and OncologyBrussels, BEvia Proton therapy, IMRT / IGRT (modern external beam)
- German Cancer Research Center (DKFZ)Heidelberg, DEvia this cancer, DNA methylation profiling
- Hokkaido University HospitalSapporo, JPvia Proton therapy, IMRT / IGRT (modern external beam)
- Institute of Oncology LjubljanaLjubljana, SIvia IMRT / IGRT (modern external beam), Carboplatin
- via Proton therapy, IMRT / IGRT (modern external beam)
- via Proton therapy, IMRT / IGRT (modern external beam)
- Royal Adelaide HospitalAdelaide, AUvia Proton therapy, IMRT / IGRT (modern external beam)
- via Proton therapy, IMRT / IGRT (modern external beam)
- via this cancer, Proton therapy
- Zhejiang Cancer HospitalHangzhou, CNvia Proton therapy, IMRT / IGRT (modern external beam)
- via Proton therapy
- Alliance for Clinical Trials in OncologyChicago, IL, USvia Carboplatin
- Apollo Hospitals (Apollo Cancer Centres)Chennai, INvia Proton therapy
- via Proton therapy
- Centre François BaclesseCaen, FRvia Proton therapy
- Centre Oscar LambretLille, FRvia IMRT / IGRT (modern external beam)
- Chang Gung Memorial HospitalTaoyuan, TWvia Proton therapy
- via IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- Children's Hospital of PhiladelphiaPhiladelphia, PA, USvia Proton therapy
- Cleveland Clinic Abu DhabiAbu Dhabi, AEvia Proton therapy
- Comprehensive Cancer Center Freiburg (CCCF)Freiburg im Breisgau, DEvia IMRT / IGRT (modern external beam)
- via Proton therapy
- Erasmus MC Cancer InstituteRotterdam, NLvia Proton therapy
- via DNA methylation profiling
- Geneva University Hospitals (HUG)Geneva, CHvia IMRT / IGRT (modern external beam)
- German Breast Group (GBG)Neu-Isenburg, DEvia Carboplatin
- Groote Schuur Hospital / University of Cape TownCape Town, ZAvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- Hacettepe University Cancer InstituteAnkara, TRvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- Ho Chi Minh City Oncology HospitalHo Chi Minh City, VNvia IMRT / IGRT (modern external beam)
- Hunan Cancer HospitalChangsha, CNvia IMRT / IGRT (modern external beam)
- Innovative Therapies for Children with Cancer (ITCC)Villejuif, FRvia NCI-COG Pediatric MATCH (APEC1621)
- Institut BergoniéBordeaux, FRvia IMRT / IGRT (modern external beam)
- Institut National d'Oncologie, RabatRabat, MAvia IMRT / IGRT (modern external beam)
- Institut Salah AzaïezTunis, TNvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- via DNA methylation profiling
- via IMRT / IGRT (modern external beam)
- International Extranodal Lymphoma Study GroupBellinzona, CHvia IMRT / IGRT (modern external beam)
- IRCCS Humanitas Research HospitalRozzano (Milan), ITvia IMRT / IGRT (modern external beam)
- Istanbul University Institute of OncologyIstanbul, TRvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- Juravinski Cancer Centre / Escarpment Cancer Research InstituteHamilton, ON, CAvia IMRT / IGRT (modern external beam)
- Kenyatta National HospitalNairobi, KEvia IMRT / IGRT (modern external beam)
- via Proton therapy
- Korle Bu Teaching HospitalAccra, GHvia IMRT / IGRT (modern external beam)
- Lagos University Teaching HospitalLagos, NGvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- Leiden University Medical CenterLeiden, NLvia Proton therapy
- Maastricht UMC+ Comprehensive Cancer CenterMaastricht, NLvia Proton therapy
- via IMRT / IGRT (modern external beam)
- via Proton therapy
- via Proton therapy
- via Proton therapy
- National Cancer Center Hospital EastKashiwa, Chiba, JPvia Proton therapy
- National Cancer Center KoreaGoyang, KRvia Proton therapy
- National Cancer Centre SingaporeSingapore, SGvia Proton therapy
- National Cancer Institute (NIH)Bethesda, MD, USvia NCI-COG Pediatric MATCH (APEC1621)
- via IMRT / IGRT (modern external beam)
- National Institute of Oncology, HungaryBudapest, HUvia IMRT / IGRT (modern external beam)
- National Taiwan University HospitalTaipei, TWvia Proton therapy
- via Proton therapy
- Ocean Road Cancer InstituteDar es Salaam, TZvia IMRT / IGRT (modern external beam)
- via Proton therapy
- via IMRT / IGRT (modern external beam)
- via DNA methylation profiling
- via Proton therapy
- Rajiv Gandhi Cancer Institute and Research CentreNew Delhi, INvia IMRT / IGRT (modern external beam)
- Rambam Health Care CampusHaifa, ILvia IMRT / IGRT (modern external beam)
- Rigshospitalet – Copenhagen University HospitalCopenhagen, DKvia IMRT / IGRT (modern external beam)
- Ruijin Hospital, Shanghai Jiao Tong UniversityShanghai, CNvia Proton therapy
- Shizuoka Cancer CenterNagaizumi, Shizuoka, JPvia Proton therapy
- via this cancer
- Siriraj Hospital, Mahidol UniversityBangkok, THvia IMRT / IGRT (modern external beam)
- via DNA methylation profiling
- via Proton therapy
- Tata Medical Center, KolkataKolkata, INvia IMRT / IGRT (modern external beam)
- Tawam HospitalAl Ain, AEvia IMRT / IGRT (modern external beam)
- Tel Aviv Sourasky Medical CenterTel Aviv, ILvia IMRT / IGRT (modern external beam)
- The Clatterbridge Cancer Centre NHS Foundation TrustLiverpool, GBvia Proton therapy
- via IMRT / IGRT (modern external beam)
- TROG Cancer ResearchNewcastle, NSW, AUvia IMRT / IGRT (modern external beam)
- UMC Utrecht Cancer CenterUtrecht, NLvia IMRT / IGRT (modern external beam)
- via Proton therapy
- via Proton therapy
- via Proton therapy
- via Proton therapy
- University of Malaya Medical CentreKuala Lumpur, MYvia IMRT / IGRT (modern external beam)
- University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer CenterBaltimore, MD, USNCI comprehensivevia Proton therapy
- via IMRT / IGRT (modern external beam)
- via Proton therapy
- via DNA methylation profiling
- UZ Leuven / Leuven Cancer InstituteLeuven, BEvia Proton therapy
- Velindre Cancer CentreCardiff, GBvia IMRT / IGRT (modern external beam)
- via IMRT / IGRT (modern external beam)
- via Proton therapy
Questions to ask
topQuestions to ask your oncologist about Ependymoma
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example Methylation-based molecular group, H3K27me3 loss and EZHIP expression, ZFTA or YAP1 fusion by FISH or sequencing, Chromosome 1q gain and 6q loss, MYCN amplification), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include Posterior fossa group A, Posterior fossa group B, Supratentorial, ZFTA fusion-positive.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Newly diagnosed intracranial ependymoma, age one year and over
- For my situation (newly diagnosed intracranial ependymoma, age one year and over), which of the standard options do you recommend and why?Why: Guideline options include: Maximal safe resection, second-look surgery for residual disease, then conformal or proton radiotherapy to the tumour bed (ACNS0121 approach); craniospinal irradiation only for disseminated disease.
Infants under one year or unresectable residual
- For my situation (infants under one year or unresectable residual), which of the standard options do you recommend and why?Why: Guideline options include: Chemotherapy (vincristine, carboplatin, cyclophosphamide, etoposide-based) to delay radiotherapy or facilitate second surgery, per SIOP Ependymoma II and COG protocols.
- Am I a candidate for Vincristine, Carboplatin, Cyclophosphamide or related drugs, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Recurrent
- For my situation (recurrent), which of the standard options do you recommend and why?Why: Guideline options include: Repeat resection and re-irradiation (focal or craniospinal) where feasible; no standard systemic therapy, so trial enrolment (including Pediatric MATCH-style molecular assignment) is recommended.
- How do the results of NCI-COG Pediatric MATCH (APEC1621) apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Any stage
- Are there clinical trials I could join, for example of DNA methylation profiling, Proton therapy, NCI-COG Pediatric MATCH (APEC1621)?Why: 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?Why: 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?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “PF-A and ZFTA tumours relapse often and have no effective drug; EZHIP/PRC2 and NF-kB dependencies are being tested preclinically and in early trials”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Whether radiotherapy can be omitted or reduced in PF-B, YAP1 and completely resected favourable tumours; SIOP Ependymoma II and COG successors are stratifying by group”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.
Direct links plus the targets, companies, and technologies of this cancer's products.
technologies
11targets
2drugs
4institutions
4pathways
3terms
1trials
1bottlenecks
2Latest papers
topQuery for this cancer: (TITLE:"Ependymoma" OR ABSTRACT:"Ependymoma" OR TITLE:"Childhood ependymoma" OR ABSTRACT:"Childhood ependymoma" OR TITLE:"Posterior fossa ependymoma" OR ABSTRACT:"Posterior fossa ependymoma" OR TITLE:"Supratentorial ZFTA fusion ependymoma" OR ABSTRACT:"Supratentorial ZFTA fusion ependymoma" OR TITLE:"Myxopapillary ependymoma" OR ABSTRACT:"Myxopapillary ependymoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Ependymoma, not a curated reading list.
Pages like this
not linked directly; found by shared links- CancerAtypical teratoid/rhabdoid tumour (ATRT)
Shares NCI-COG Pediatric MATCH (APEC1621), St. Jude Children's Research Hospital, Late effects and survivorship toxicity, SIOP Europe – European Society for Paediatric Oncology and the tags nci-coverage, paediatric, cns.
- CancerCraniopharyngioma
Shares St. Jude Children's Research Hospital, Late effects and survivorship toxicity, Inflammation & NF-κB, Paediatric low-grade glioma and the tags nci-coverage, paediatric, cns.
- CancerDiffuse midline glioma, H3 K27-altered (including DIPG)
Shares NCI-COG Pediatric MATCH (APEC1621), Late effects and survivorship toxicity, SIOP Europe – European Society for Paediatric Oncology, Paediatric low-grade glioma and the tags nci-coverage, paediatric, cns.
- TrialCOG ACNS0331
Shares Late effects and survivorship toxicity, Vincristine, Cyclophosphamide, Medulloblastoma and the tags nci-coverage, paediatric, cns.
- TrialTADPOLE (CDRB436G2201)
Shares Paediatric low-grade glioma, Vincristine, Carboplatin and the tags nci-coverage, paediatric, cns.
- TrialFIREFLY-1
Shares Paediatric low-grade glioma and the tags nci-coverage, paediatric, cns.
- TrialACTION
Shares Rare and paediatric cancers without markets and the tags nci-coverage, paediatric, cns.
- CancerGerm cell tumours of childhood and adolescence (extracranial and CNS)
Shares Late effects and survivorship toxicity, SIOP Europe – European Society for Paediatric Oncology, Etoposide, Survivorship care and late-effects surveillance and the tags nci-coverage, paediatric.