High dose alkylator therapy for extracranial malignant rhabdoid tumors in children
Rajkumar Venkatramani1, Poone Shoureshi, Jemily Malvar
1Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, Los Angeles, California; Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California.
Background:
Extracranial malignant rhabdoid tumor (MRT) is a rare pediatric cancer with a poor prognosis. The kidney is the most common site. Isolated reports have shown improvements in patient survival, but no specific treatment regimen has shown efficacy over others.
Procedure:
Retrospective review of patients diagnosed with extracranial MRT at Children's Hospital Los Angeles between 1983 and 2012.
Results:
The median age at presentation for the 21 patients was 13 months (range, 0-108 months). Ten patients had renal primary tumors. The median time to progression was 4 months (range, 0.4-7 months). The 5-year event free survival (EFS) and overall survival (OS) of the entire cohort was 38 ± 10.6%. After 2002, patients diagnosed with extracranial MRT were administered a chemotherapy regimen of vincristine, doxorubicin and high dose cyclophosphamide (VDC). The OS for the patients diagnosed before and after 2002 were 20 ± 12% and 54 ± 15%, respectively. Of the 13 patients who received VDC containing regimen, eight patients achieved a complete radiological remission; five of these patients are long-term survivors. Four patients who received autologous bone marrow transplantation were alive at last follow-up. All patients with unresectable primary tumors died. Patients who had disease progression or relapse did not survive.
Conclusions:
Patients with extracranial MRT have a poor prognosis. Treatment with high dose alkylator therapy followed by consolidation with high dose chemotherapy and autologous bone marrow transplant for those patients in radiographic complete remission appears to have a beneficial effect on survival.
Insights
Extracranial malignant rhabdoid tumors (MRT) in children are rare and aggressive. A chemotherapy regimen including vincristine, doxorubicin, and cyclophosphamide (VDC) improved survival rates, especially when followed by autologous bone marrow transplant.
Area of Science:
- Pediatric Oncology
- Cancer Research
- Medical Science
Background:
- Extracranial malignant rhabdoid tumor (MRT) is a rare pediatric cancer with a poor prognosis.
- The kidney is the most common primary site for this aggressive malignancy.
- Current treatment strategies lack established efficacy, highlighting the need for improved therapeutic approaches.
Purpose of the Study:
- To evaluate the prognostic factors and treatment outcomes for patients with extracranial MRT.
- To assess the impact of a specific chemotherapy regimen on patient survival.
- To identify potential therapeutic strategies for improving outcomes in this rare cancer.
Main Methods:
- Retrospective review of 21 patients diagnosed with extracranial MRT between 1983 and 2012.
- Analysis of patient demographics, tumor characteristics, treatment regimens, and survival data.
- Comparison of outcomes for patients treated before and after the introduction of a VDC chemotherapy regimen.
Main Results:
- The median age at diagnosis was 13 months, with 10 patients having renal primary tumors.
- The overall 5-year event-free survival (EFS) and overall survival (OS) was 38%.
- Patients treated with vincristine, doxorubicin, and high-dose cyclophosphamide (VDC) after 2002 showed improved OS (54%) compared to earlier cohorts (20%). Eight of 13 patients receiving VDC achieved complete remission, with five being long-term survivors.
Conclusions:
- Extracranial MRT is associated with a poor prognosis, underscoring the need for effective treatments.
- High-dose alkylator therapy, such as VDC, followed by consolidation chemotherapy and autologous bone marrow transplant in complete remission, appears to improve survival.
- Further research into optimized treatment protocols is crucial for enhancing outcomes in pediatric extracranial MRT.
