Rhabdoid tumor

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:69077OMIM:609322C49.9
Who is this for?
Show terms as
1Active trials24Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Rhabdoid tumor is a rare and aggressive type of cancer that most often affects young children, particularly infants and toddlers. The name comes from the word 'rhabdoid,' which describes how the cancer cells look under a microscope — they resemble a type of muscle cell. These tumors can grow in several parts of the body, but they most commonly appear in the kidneys (called malignant rhabdoid tumor of the kidney, or MRTK), the brain and spine (called atypical teratoid/rhabdoid tumor, or AT/RT), and soft tissues throughout the body. When the tumor occurs in multiple locations or is linked to a genetic change, it may be called rhabdoid tumor predisposition syndrome (RTPS). Rhabdoid tumors grow very quickly and can spread to other parts of the body, including the lungs, liver, and brain. Because of this, they are considered one of the most challenging childhood cancers to treat. Symptoms depend on where the tumor is located but often include a lump or swelling, pain, neurological changes, and general signs of illness like fever and weight loss. Treatment typically involves a combination of surgery to remove the tumor, chemotherapy, and sometimes radiation therapy. Newer approaches, including targeted therapies and clinical trials, are being studied. Despite advances in treatment, outcomes remain difficult, especially for very young children. Early diagnosis and care at a specialized cancer center are critical for the best possible outcome.

Also known as:

Key symptoms:

A lump or mass in the abdomen, kidney area, or soft tissueHeadaches or vomiting (if the tumor is in the brain)Seizures or changes in behaviorWeakness or paralysis on one side of the bodyDifficulty walking or loss of balanceBlood in the urine (if the kidney is affected)Unexplained weight lossPersistent feverSwollen belly or abdomenExtreme tiredness or low energyVision or hearing changesIrritability in infantsBulging fontanelle (soft spot) in babies

Clinical phenotype terms (24)— hover any for plain English
HemiplegiaHP:0002301Neoplasm of the liverHP:0002896Renal neoplasmHP:0009726Internal hemorrhageHP:0011029
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Dec 2025Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells

Seattle Children's Hospital — PHASE1

TrialRECRUITING
Jan 2019Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

National Cancer Institute (NCI) — NA

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Rhabdoid tumor.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Phase 11 trial
Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells
Phase 1
Actively Recruiting
PI: Colleen Annesley, MD (Seattle Children's Hospital) · Sites: Seattle, Washington · Age: 126 yrs

Specialists

24 foundView all specialists →
AM
Amar Gajjar, MD
MEMPHIS, TN
Specialist
PI on 8 active trials
DP
Donald W Parsons
Specialist
PI on 1 active trial17 Rhabdoid tumor publications
DM
David Steffin, MD
HOUSTON, TX
Specialist
PI on 3 active trials
TM
Thomas Cash, MD
BIRMINGHAM, AL
Specialist
PI on 3 active trials
SM
Suzanne Forrest, MD
Specialist
PI on 1 active trial
MI
Meredith S Irwin
CINCINNATI, OH
Specialist
PI on 1 active trial
RM
Rajen Mody
ANN ARBOR, MI
Specialist
PI on 2 active trials
KM
Kim E. Nichols, MD
Memphis, Tennessee
Specialist

Rare Disease Specialist

PI on 2 active trials
CM
Catherine Albert, MD
Specialist
PI on 3 active trials
JM
Jordan Milner, MD
GAINESVILLE, FL
Specialist
PI on 5 active trials
CM
Chris DeRenzo, MD
MEMPHIS, TN
Specialist
PI on 1 active trial
CM
Colleen Annesley, MD
SEATTLE, WA
Specialist
PI on 5 active trials
MM
Muna Qayed, MD
ATLANTA, GA
Specialist
PI on 2 active trials
KW
Katherine Warren
Specialist
PI on 1 active trial
SM
Scot C. Remick, MD
SCARBOROUGH, ME
Specialist
PI on 9 active trials
MM
Mariko D. DeWire-Schottmiller, MD
CINCINNATI, OH
Specialist
PI on 1 active trial
IM
Ipsen Medical
Specialist
PI on 1 active trial
MD
Mary F Wedekind Malone, D.O.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 3 active trials
HM
Hugh Reyburn, MD
Specialist
PI on 2 active trials

Treatment Centers

8 centers
⚗️ Trial Site

National Institutes of Health Clinical Center

📍 Bethesda, Maryland

👤 Payal P Khincha, M.D.

👤 Christopher Grunseich, M.D.

⚗️ Trial Site

Oregon Health and Science University

📍 Portland, Oregon

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to Rhabdoid tumor.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Rhabdoid tumorForum →

No community posts yet. Be the first to share your experience with Rhabdoid tumor.

Start the conversation →

Latest news about Rhabdoid tumor

Disease timeline:

New trial: Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

Phase NA trial recruiting.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What type of rhabdoid tumor does my child have, and where exactly is it located?,Has genetic testing been done on the tumor and my child's blood, and what did it show?,What treatment plan do you recommend, and what are the goals — cure or disease control?,Are there any clinical trials that my child might be eligible for?,What side effects should we watch for at home, and when should we go to the emergency room?,Should other family members be tested for a genetic mutation?,What support services — such as social work, psychology, or palliative care — are available to our family?

Common questions about Rhabdoid tumor

What is Rhabdoid tumor?

Rhabdoid tumor is a rare and aggressive type of cancer that most often affects young children, particularly infants and toddlers. The name comes from the word 'rhabdoid,' which describes how the cancer cells look under a microscope — they resemble a type of muscle cell. These tumors can grow in several parts of the body, but they most commonly appear in the kidneys (called malignant rhabdoid tumor of the kidney, or MRTK), the brain and spine (called atypical teratoid/rhabdoid tumor, or AT/RT), and soft tissues throughout the body. When the tumor occurs in multiple locations or is linked to a g

At what age does Rhabdoid tumor typically begin?

Typical onset of Rhabdoid tumor is infantile. Age of onset can vary across affected individuals.

Are there clinical trials for Rhabdoid tumor?

Yes — 1 recruiting clinical trial is currently listed for Rhabdoid tumor on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Rhabdoid tumor?

24 specialists and care centers treating Rhabdoid tumor are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.