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Encouraging Early Outcomes of Treatment With Arsenic Trioxide Combined With Chemotherapy for Alveolar
Xiaomin Peng1, Xilin Xiong1, Yang Li1
1Department of Pediatric Hematology/Oncology, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, China.
Background:
Alveolar rhabdomyosarcoma (ARMS) is a subtype of rhabdomyosarcoma characterized by its aggressive behavior and poor prognosis, highlighting the need for novel treatment options. Arsenic trioxide (ATO) has been shown to specifically inhibit tumor growth and the metastasis of ARMS in vitro by acting on the hedgehog pathway. Here we report on a pilot clinical study to evaluate the activity of an ATO-combined chemotherapy approach for the treatment of ARMS patients.
Methods:
We designed a therapeutic schedule of an ATO-combined chemotherapy, incorporating comprehensive management according to the Intergroup Rhabdomyosarcoma Study Group protocol. ATO was administered at 0.16 mg/kg per day over 8 h via an IV for 10 days combined with a chemotherapeutic regimen of vincristine, actinomycin, and cyclophosphamide (VAC regimen) on the third day, which was repeated every 21 days. A total of eight cycles of ATO-combined chemotherapy were applied throughout the entire scheme.
Results:
A total of three refractory/recurrent and one untreated ARMS patient, three male and one female, with a median age of 5.8 years (range, 5.1 to 12.5 years), were enrolled in the present study. All patients were sensitive to combined chemotherapy with ATO and achieved partial or complete remission during treatment. Except for reversible gastrointestinal reaction and myelosuppression, no other adverse events were observed during the process of treatment.
Conclusions:
The combined chemotherapy of ATO and the VAC regimen exhibited beneficial activities against ARMS in pediatrics and was well tolerated, but prospective large-scale clinical trials are warranted to determine the long-term efficacy, optimal courses, and late toxicity in this population.
Insights
Arsenic trioxide (ATO) combined with chemotherapy shows promise for treating alveolar rhabdomyosarcoma (ARMS) in children. This pilot study found ATO and VAC regimen well-tolerated, achieving remission in pediatric ARMS patients.
Area of Science:
- Pediatric Oncology
- Medical Chemistry
- Clinical Pharmacology
Background:
- Alveolar rhabdomyosarcoma (ARMS) is an aggressive pediatric cancer with a poor prognosis.
- Novel therapeutic strategies are urgently needed for ARMS treatment.
- Arsenic trioxide (ATO) demonstrates in vitro efficacy against ARMS by targeting the hedgehog pathway.
Purpose of the Study:
- To evaluate the clinical activity of a novel arsenic trioxide (ATO)-combined chemotherapy regimen for ARMS patients.
- To assess the safety and tolerability of this combined therapeutic approach.
Main Methods:
- A pilot study was conducted using a therapeutic schedule combining ATO with the vincristine, actinomycin, and cyclophosphamide (VAC) regimen.
- ATO was administered intravenously at 0.16 mg/kg/day for 10 days, with VAC chemotherapy initiated on day 3.
- The treatment cycle was repeated every 21 days for a total of eight cycles.
Main Results:
- Four pediatric ARMS patients (3 refractory/recurrent, 1 untreated) were enrolled.
- All patients achieved partial or complete remission during treatment with the ATO-combined chemotherapy.
- Adverse events were limited to reversible gastrointestinal reactions and myelosuppression, indicating good tolerability.
Conclusions:
- The combination of ATO and the VAC regimen demonstrates beneficial activity and is well-tolerated in pediatric ARMS patients.
- Further prospective, large-scale clinical trials are necessary to establish long-term efficacy and optimal treatment protocols.
- Long-term toxicity and late adverse events require further investigation in larger cohorts.
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