Extracranial malignant rhabdoid tumors in childhood: the Childrens Hospital Los Angeles experience.

BACKGROUND

Extracranial malignant rhabdoid tumor (MRT) is a rare, aggressive, pediatric malignancy with a historically poor outcome. Recent efforts to intensify treatment for MRT have resulted in isolated reports of long-term survival.

METHODS

The authors conducted a retrospective review of consecutive patients with MRT at Childrens Hospital Los Angeles over the 20 years from 1983 to 2003.

RESULTS

Fourteen children were diagnosed with MRT over the 20-year study period. The median age at presentation was 22.5 months (range, 0.5-108 months). Five patients had renal primary tumors, and 9 patients had extrarenal tumors. Eleven of 14 patients had stage III or IV disease at diagnosis. Five patients (35.7%) were long-term survivors. The time to disease progression was rapid (mean, 3.6 months). There were no recurrences or deaths beyond 10 months after diagnosis. All survivors received multimodal therapy, including both chemotherapy and surgery with or without radiation. In addition, 2 patients received high-dose chemotherapy with hematopoietic stem cell rescue (HSCT) after neoadjuvant chemotherapy and local tumor control. Both of those patients were long-term survivors. There were no survivors after disease recurrence or progression.

CONCLUSIONS

Patients with localized disease and complete surgical resection were most likely to survive long-term. Consolidation with HSCT may benefit selected patients with advanced disease stage. International collaboration and further understanding of the biology of this disease is necessary to improve the survival of children with MRT.