Atypical teratoid rhabdoid tumors (ATRT) are rare, aggressive tumors of the central nervous system. They occur primarily in the cerebellum (the part of the brain that controls movement and balance) and the brain stem (the part of the brain that controls
basic body functions).
- ATRT represents 1 to 2 percent of childhood brain tumors.
- The condition usually appears by 3 years old. Occasionally, it occurs in older children.
- It is a part of a larger group of malignant tumors called rhabdoid tumors, which can occur outside the brain (such as a rhabdoid tumor of the kidney).
- ATRT may be localized to one part of the brain. It can also spread to other locations in the brain, spine, or body.
At Dana-Farber/Boston Children's Cancer and Blood Disorders Center, we treat children with ATRR in our Brain Tumor Center. Our pediatric experts are leaders in treating children with
rare diseases such as ATRT.
Causes and Symptoms of Childhood Atypical Teratoid Rhabdoid Tumor (ATRT)
As a parent, you undoubtedly want to know what may have caused your child's tumor. It's important to remember that nothing you could have done or avoided doing would have prevented the tumor from developing.
In most cases, atypical teratoid rhabdoid tumors are associated with a specific mutation (INI1) that can occur spontaneously or be inherited. More than 90 percent of cases of ATRT are related to this mutation and not inherited. However, the cause of this
abnormality is not known.
Atypical teratoid rhabdoid tumors can cause various symptoms in children, depending on their size and location. Keep in mind that the symptoms may resemble other more common conditions or medical problems. It is important to consult your child's physician
if you have concerns.
ATRT symptoms can come on quickly, in a matter of days or weeks, due to the fast-growing nature of these tumors. Symptoms may include:
- Headache, generally upon awakening in the morning
- Hydrocephalus, where there is a buildup of fluid in the brain, can increase head size in infants or headaches and vomiting in older children
- Nausea and vomiting, often worse in the morning and improve throughout the day
- Lethargy and fatigue
- Trouble with balance and coordination
How We Diagnose Childhood Atypical Teratoid Rhabdoid Tumor (ATRT)
Doctors typically diagnosis ATRT with physical and neurological exams, advanced imaging studies, biopsy, and lumbar puncture.
There may be other diagnostic tests that your doctor will discuss with you, depending on your child's situation.
After completing all necessary tests, our experts meet to review and discuss what they have learned about your child's condition. Then we will be able to discuss the results and outline the best possible treatment options.
How We Treat Childhood Atypical Teratoid Rhabdoid Tumor (ATRT)
Your child's physician will determine a specific course of ATRT treatment based on your child’s unique needs. ATRT treatments include:
- Surgery: We typically recommend surgery first to remove as much of the tumor as possible.
- Chemotherapy: Chemotherapy treatment involves medications that interfere with a cancer cell’s ability to grow or reproduce. Doctors use it to shrink tumors and destroy remaining
cancer cells. Intrathecal chemotherapy delivers medicine directly to the spinal fluid and central nervous system to treat the tumor.
- Radiation therapy: Radiation therapy involves using high-energy waves to damage or shrink tumors. We are careful to recommend radiation to children under 3 years
old. If your doctor recommends it, we will likely use limited field radiation at a reduced intensity. Children over 3 years old receive radiation therapy as a standard treatment.
- Stem cell transplant: This approach allows children with aggressive ATRT to receive high doses of chemotherapy. It involves transfusing healthy bone
marrow cells into your child if intensive chemotherapy damages their marrow.
There is no standard treatment for recurrent ATRT. Clinical trials provide children with recurrent ATRT access to the latest experimental therapies.
At Dana-Farber/Boston Children’s, we provide ongoing care and support through our extensive pediatric cancer survivorship programs, including the Stop & Shop
Family Pediatric Neuro-Oncology Outcomes Clinic for pediatric brain tumor survivors. These services address health and social issues,
ranging from motor function evaluation and physical therapy to return-to-school and learning programs.
Research and Clinical Trials for Childhood Atypical Teratoid Rhabdoid Tumor (ATRT)
Because ATRT is so rare, these tumors are difficult to study. As a result, our researchers collaborate with other institutions around the country to improve our current understanding of this condition and offer new treatments to children with ATRT.
Researchers at Dana-Farber/Boston Children’s are currently working with the Children’s Hospital of Philadelphia and the Broad Institute of MIT and Harvard to learn more about the molecular characteristics of atypical teratoid rhabdoid tumors.
In addition, researchers in our Childhood Brain Tumor Center collaborate with physicians and researchers that treat other types of rhabdoid tumors to study the effectiveness of current treatments and establish new, more standardized therapies for all
kinds of rhabdoid tumors.
Through clinical trials, our researchers are testing new, targeted treatments that may help improve the survival rate for ATRT. Learn more about clinical trials.
If you have any questions about clinical trials, your child’s medical team can provide you with additional information.
In addition to launching our own clinical trials, we also offer trials available through collaborative groups such as the Children's Oncology Group (COG) and
the Pacific Pediatric Neuro-Oncology Consortium (PNOC). We are also the New England Phase I Center of the Children's Oncology Group. If your child has a progressive
or recurrent tumor, she may be eligible for experimental therapies available through these groups or from one of our independent clinical investigators.
Long-term Outcomes for Children with Atypical Teratoid Rhabdoid Tumor (ATRT)
ATRT is challenging to cure, and, unfortunately, the outcomes remain poor. Older children with ATRT tend to do better than younger children. Through the studies done at Dana-Farber/Boston Children's, we have improved the cure rate from 10 to 50 percent.
Childhood Atypical Teratoid Rhabdoid Tumor (ATRT) Treatment Team
At Dana-Farber/Boston Children's, our team of brain tumor specialists includes neuro-oncologists, neurosurgeons, and neurologists. See a complete list of the specialists in our Childhood Brain Tumor Center.