WHAT IS DSRCT?
Desmoplastic Small Round Cell Tumor (DSRCT) is a rare soft tissue sarcoma that typically presents in the abdomen and is thought to originate from the peritoneum (lining of the abdominal cavity) . It is highly aggressive and usually presents with multiple abdominal tumors and often, metastatic disease at the time of original diagnosis (meaning that it has spread beyond the primary tumor to other parts of the body). It was first described in medical literature in 1989, and it is estimated to have at least 100 diagnoses a year worldwide. DSRCT is most common in adolescent and young adult males (~90% of cases), but cases have been reported in both males and females from 2 years to 75 years of age. DSRCT is considered a pediatric cancer because the tumors are made up of primitive, undifferentiated cells. The tumor looks under the microscope like other childhood cancers like Wilms tumor and Ewings sarcoma. DSRCT is characterized by abnormal fusion of the Ewings gene (EWS) and Wilms tumor gene (WT1) to create a cancer causing fusion gene, EWS-WT1.
Treatment for DSRCT includes high-dose multiagent induction chemotherapy using drugs similar to Ewings sarcoma (P6 or VIDE protocol), aggressive surgical resection (often with hyperthermic intraperitoneal chemotherapy (HIPEC), chemotherapy during radiation therapy, then maintenance chemotherapy. Sometimes high dose chemotherapy and stem cell transplant has been used. Many DSRCT patients have disease that is resistant to chemotherapy and radiation. While surgery that removes more than 90% of tumor is proven to prolong life, it rarely achieves a lasting cure. Hospitalization resulting from toxicity or side effects of the chemotherapy regimen is common. For some patients the needed surgical intervention is extensive and can result in permanent damage to the colon, rectum, and/or bladder, removal of the spleen and other organs, and damage to kidney, urinary or other organ function. Further, many patients have metastasized disease in the liver, lungs or mediastinum that is not resectable. IF a patient can achieve a very low or no evidence of disease status (NED), they have a better chance of long term survival. There are a few known survivors with 12+ years without recurrence.
A 2013 article resulting from the nearly 10 year old DSRCT project at Christus Stehlin Foundation asserts that there are two distinct populations of DSRCT with drastic differences in growth patterns. This is important because it explains why some patients (like my Cole) have gone years with little to no disease progression (reflecting slow growth pattern) unlike others who have rapid, aggressive progression (an example, a DSRCT patient who had 300% tumor growth in 6 weeks between scans).
While most DSRCT publications report a grave prognosis, it is important to realize that there are a spectrum of experiences among DSRCT patients that are not always reflected in the research literature. Many patients diagnosed in recent years are treated at top cancer centers in the United States by sarcoma specialists who have experience treating DSRCT, and as a result, they have improved outcomes. Overall DSRCT statistics are improving as a result. A 2012 article (Hayes-Jordan & Pappo) reported a 71% 3-year survival for patients who have complete resection surgery with HIPEC. Patients treated with standard protocol (debulking along chemotherapy and radiation) have a 62% 3-year survival rate, and patients treated without surgical intervention have a 29% 3-year survival rate. In 2005, DSRCT was reported to have less than 15% 3-year survival. Aggressive surgical intervention and multi-modal therapy has improved DSRCT outcomes dramatically.
Memorial Sloan Kettering Cancer Center, a top facility for DSRCT surgery and treatment provides an updated disease summary HERE.
ABOUT CHILDHOOD CANCER
Cancer is the leading cause of death by disease for kids under 15. Cancer causes more deaths in children than asthma, diabetes, cystic fibrosis, congenital abnormalities and pediatric AIDS combined.
About 1 in 285 children will be diagnosed with cancer. Almost 14,000 kids are diagnosed each year, about 35,000 children are currently being treated, and an estimated 1500 will die in 2014 (in the United States).
Only about $197 million of the National Cancer Institute’s $5 billion budget goes to pediatric cancer research. That’s less than 4%!
There are more than 300,000 survivors of childhood cancer in the United States. Due to the treatments that saved their lives, 2/3rds of these survivors have at least 1 chronic health problem. One quarter of all survivors suffer from a severe health condition as a result of treatment. One quarter have 3 or more chronic health problems for the duration of their lifetimes, like heart & lung problems, infertility, alterations in growth/development and impaired cognitive ability. Many suffer ‘late effects’ of treatment, side effects that don’t appear for months or even years after receiving the treatment.
Most standard treatments for pediatric cancer carry a high risk of toxicity to healthy organs and tissues, along with a risk of developing secondary cancers. Only 1 new drug has been approved for pediatric use in 20 years. Many of the therapies used in pediatrics have not been extensively studied for their long term effects on the growing body.
Though most common among children, sarcomas only account for about 1% of all cancer diagnoses. While 4 of 10 people will have cancer in their lifetimes, the incidence of sarcoma is 1 in 250. 1 of 3 sarcoma patients will die from their disease. Due to the rarity of sarcoma, it is important for sarcoma patients to seek treatment at a specialized cancer treatment center with experience treating their specific diagnosis.