ULTRA-RARE CANCERPediatric Brain TumorWHO 5th Edition Classification

Embryonal Tumor with Multilayered Rosettes (ETMR)

Ultra-Rare Malignancy (< 1 case per 1,000,000 / year) • Clinical Staging, Genomic Targets & Vetted Specialists

An ultra-rare, highly malignant embryonal brain tumour of very young children, defined by amplification of the C19MC microRNA cluster at chromosome 19q13.42 and strong LIN28A expression. It arises most often in the cerebral hemispheres but also in the brainstem and cerebellum.

Who It Affects

Children, usually under 4 years; slight female predominance

Annual Incidence

Fewer than 1 per million children per year

Clinical Evidence Review

Last audited: 2026-08-13 against NCCN, ESMO, and WHO 5th ed. guidelines.

Clinical Presentation & Hallmark Symptoms

Presenting signs most frequently observed across clinical case series

Seizures
Rapidly increasing head circumference
Persistent vomiting and irritability
Focal weakness or asymmetric movement
Regression of milestones

Genomic Profiling & Defining Molecular Lesions

Critical diagnostic fusions, somatic mutations, and therapeutic targets

Rare malignancies frequently depend on distinct oncogenic drivers rather than conventional environmental carcinogens. Comprehensive Next-Generation Sequencing (NGS comprehensive panel) and FISH/IHC are mandatory to establish the true diagnosis and screen for basket trial agents.

C19MC amplification (19q13.42)LIN28A overexpressionDICER1 - variant form

Standard-of-Care Treatment Protocol

Frontline and multimodal strategies established under international consensus guidelines

1

Maximal safe resection - extent of resection is a strong prognostic factor

Administered in specialized high-volume oncology programs with subspecialty pathology and organ-preservation protocols.

2

Intensive induction chemotherapy

Administered in specialized high-volume oncology programs with subspecialty pathology and organ-preservation protocols.

3

High-dose chemotherapy with stem cell rescue

Administered in specialized high-volume oncology programs with subspecialty pathology and organ-preservation protocols.

4

Radiotherapy in children old enough to receive it safely

Administered in specialized high-volume oncology programs with subspecialty pathology and organ-preservation protocols.

5

Clinical trial enrolment wherever available

Administered in specialized high-volume oncology programs with subspecialty pathology and organ-preservation protocols.

Clinical Trial Advisory: For rare and ultra-rare malignancies, enrollment in an active clinical trial or expanded-access program is widely considered the preferred standard of care by ASCO and NCCN panels.

Prognosis & Disease Trajectory

Objective clinical outlook without false reassurance

Generally poor, with median survival historically under 12 months. Outcomes are meaningfully better in children who achieve gross total resection and are treated on intensive protocols at specialist centres.

Note: Statistics reflect cohort averages. Individual outcomes depend heavily on performance status, resectability, biomarker expression, and timely access to specialized tertiary care.

Active Research, Biomarkers & Clinical Trials

Novel investigational agents, phase I/II trials, and international rare disease consortia

LIN28A/let-7 axis targeting, ALK and IGF1R inhibition, and refined risk-adapted protocols are the principal research directions.

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Expert Clinicians

Verified Specialists for Embryonal Tumor with Multilayered Rosettes

Browse All Oncologists

Dr. Maryam Fouladi

Co-Director, Pediatric Neuro-Oncology Program

20+ yrs exp

Nationwide Children’s Hospital

Columbus, USA

Pediatric brain tumorsETMRClinical trials
Vetted DirectoryConsult Specialist

Dr. Chan Mei Yoke

Senior Consultant, Pediatric Oncology

15+ yrs exp

KK Women’s and Children’s Hospital (KKH)

Singapore, Singapore

Pediatric oncologyRare brain tumorsETMR
Vetted DirectoryConsult Specialist

Prof. Stefan Pfister

Director, Hopp Children’s Cancer Center

15+ yrs exp

Hopp Children’s Cancer Center / Heidelberg University

Heidelberg, Germany

Pediatric neuro-oncologyGenomicsETMR
Vetted DirectoryConsult Specialist

Dr. Sameer Bakshi

Professor of Medical Oncology

20+ yrs exp

AIIMS, New Delhi

New Delhi, India

Pediatric and adolescent oncologyRare CNS tumorsETMR
Vetted DirectoryConsult Specialist
Hospital Network

Designated Cancer Centers with Dedicated Programs

Search All 72 NCI Centers

NCI-Designated Comprehensive Cancer Center

World-leading rare tumor board & pediatric solid tumor protocols

Proton TherapyCAR-T Cell TherapyRobotic Surgery
1,000+ Active Clinical TrialsView Center Profile

NCI-Designated Comprehensive Cancer Center

Largest specialized rare cancer and sarcoma multidisciplinary program

Proton TherapyCAR-T Cell TherapyCyberKnife SBRT
1,200+ Active Clinical TrialsView Center Profile

NCI-Designated Comprehensive Cancer Center

Pioneering genomic molecular tumor boards & rare histologies

Proton TherapyCAR-T Cell TherapyPhase I Unit
1,100+ Active Clinical TrialsView Center Profile

NCI-Designated Comprehensive Cancer Center

High-volume surgical oncology & rare endocrine/neuroendocrine expertise

Proton TherapyCAR-T Cell TherapyCyberKnife SBRT
600+ Active Clinical TrialsView Center Profile

Frequently Asked Clinical Questions

Authoritative guidance on diagnosis, tumor boards, genomic markers, and care options

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