Disease patterns and survival rate in children with metastatic soft‐tissue sarcoma: A report from the intergroup rhabdomyosarcoma study (IRS)‐I

The patterns of disease extent, response to treatment, and survival rates were examined in 129 children registered as metastatic (Group IV) soft-tissue sarcoma patients on the Intergroup Rhabdomyosarcoma Study (IRS)-I protocol from November 1972 through October 1978. The patients' ages at diagnosis ranged from 3 months to 21 years (median, 10.5 years); 72 were male patients and 57 were female patients (male-to-female ratio, 1.3:1). The tumor arose in an extremity in 34 patients, the head and neck (exclusive of the orbit) in 27, the genitourinary tract in 25, the retroperitoneum or pelvis in 14, the trunk in 12, the intrathoracic region in nine, the hepatobiliary system in four, the perineum in two, and the orbit in two patients. The histologic types of soft-tissue sarcoma confirmed by the pathology review committee were embryonal rhabdomyosarcoma in 68 patients, alveolar rhabdomyosarcoma in 41, undifferentiated sarcoma in 14, embryonal-botryoid in two, pleomorphic in one patient, and other sarcoma in three patients. Of 105 patients who were confirmed in Group IV and had complete information concerning sites of metastatic deposits, 53 patients presented with metastases restricted to only one anatomic region or tissue; the other 52 had diffuse metastases. The most common sites of distant tumor deposits were the lungs, bone marrow, and other soft tissues. After treatment with vincristine, dactinomycin, and cyclophosphamide with or without Adriamycin (Adria Laboratories, Inc., Columbus, OH) and radiation therapy, 65 (50%) of the 129 eligible patients achieved complete disappearance of all detectable tumor. The likelihood of achieving and maintaining a complete response for 2 years was highest among patients whose tumors originated in the genitourinary tract, retroperitoneum-pelvis, or the hepatobiliary or perineum regions. Thirteen of these 45 children (29%) are alive and disease-free at a minimum of 2 years after initiation of treatment. Only 10 of the other 84 (8%) are surviving disease-free at 2 years (P = 0.02). The presence of restricted rather than diffuse metastases at diagnosis (P = 0.02) was the only other characteristic significantly related to long-term complete remissions. One hundred and two patients (79%) died at a median of 47 weeks after diagnosis, 92 from sarcoma and ten from other causes. Prolonged disease-free survival was possible for some children with metastatic soft-tissue sarcoma arising in the genitourinary tract, retro-peritoneum-pelvis, or hepatobiliary and perineal sites, but it was unlikely for those whose tumor arose elsewhere.