Desmoplastic Small Round Cell Tumors

Desmoplastic small round cell tumor (DSRCT) is an aggressive tumor that typically begins in the abdomen or pelvis. Considered a pediatric cancer, this rare type of soft tissue sarcoma is diagnosed in about 20 adolescents in the United States each year. The most common symptoms include pain or a mass in the abdomen.

Memorial Sloan Kettering (MSK) pathologists William Gerald and Juan Rosai first described DSRCT in 1989. They then characterized the unique genetic defect associated with the cancer, the EWS-WT1 transcription factor, along with MSK pathologist Marc Ladanyi.

In the years since, MSK’s Department of Pediatrics has cared for approximately 100 patients with DSRCT — more than any other hospital in the country. Our team has led the field in developing treatment strategies for this disease. We have also published the largest reports of patients treated with different methods, including chemotherapy, surgery, and radiation therapy.

Diagnosis

DSRCT is so rare that it is often overlooked as the root of disease. Genomic testing — in which a patient’s tumor sample is analyzed on a molecular level — can reveal the unique genetic marker found only in DSRCT. This can help doctors make a correct diagnosis. Although DSRCT most commonly occurs in the abdominal cavity, it can appear in any part of the body. We have seen patients with DSRCT that began in the chest, bones, and even brain.

The disease can spread widely throughout the body. Our experts often examine patients’ bone marrow and perform imaging studies — such as CT, MRI, and PET — to determine how far the cancer has spread.

Treatment

MSK’s pediatrics team treats patients with DSRCT using many approaches. Chemotherapy is usually given first to shrink the tumor and to prevent new ones from forming. After several months of chemotherapy, surgery is performed to remove the cancerous areas. For many patients, radiation therapy is also used to kill any remaining cancer cells in the body.

Research

MSK leads the world’s only two clinical trials for patients with DSRCT.

  • For newly diagnosed patients, the standard chemotherapy regimen is combined with the drugs irinotecan, temozolomide, and bevacizumab to increase effectiveness. This approach is based upon basic-science research carried out at MSK showing that DSRCT is heavily driven by the VEG-F receptor, which is bevacizumab’s target.
    https://www.mskcc.org/cancer-care/clinical-trials/10-091
     
  • We are also investigating an innovative therapy that uses the 8H9 antibody to deliver an extremely high dose of radiation directly to a tumor without damaging normal cells. Patients may qualify for this trial as part of their initial therapy or if their cancer recurs.
    https://www.mskcc.org/cancer-care/clinical-trials/09-090

MSK is deeply proud of our expertise in caring for young people with DSRCT and other very rare cancers. MSK’s Pediatrics team is hopeful that further research will improve outcomes and set new standards of care for patients — not just here at MSK but everywhere.