Peer-reviewed article
Safety and efficacy of arsenic trioxide in intensification therapy for newly diagnosed childhood acute promyelocytic leukemia: results from the JPLSG AML-P13 study.
Takahashi H, Tanaka S, Yuza Y, Iijima-Yamashita Y, Hasegawa D et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study tested arsenic trioxide (ATO) combined with retinoic acid as a treatment for childhood acute promyelocytic leukemia in 27 Japanese children. The treatment achieved very high survival rates (96.3% event-free survival, 100% overall survival) and reduced the need for toxic chemotherapy drugs. The main safety concern was heart rhythm changes in some treatment cycles, but no long-term heart, kidney, or other organ damage was observed up to 5 years after treatment ended.
Key points
- 3-year event-free survival rate was 96.3% and overall survival was 100% in 27 children with newly diagnosed acute promyelocytic leukemia
- Prolonged QTc interval or cardiac conduction disorders occurred in 20 out of 63 intensification cycles
- Durations of leukocytopenia, neutropenia, and G-CSF treatment were significantly shorter compared to previous conventional chemotherapy approaches
- No cardiac, metabolic, renal, cutaneous, or neurological symptoms were reported during 5-year follow-up after protocol completion
- ATO treatment allowed replacement of conventional cytotoxic chemotherapy intensification phases, potentially reducing exposure to toxic agents
Limitations
The study included only 27 children and is a single-arm prospective study without a concurrent control group, making it difficult to attribute outcomes solely to ATO or compare directly with other treatments. Long-term follow-up beyond 5 years and effects on growth, development, or fertility in childhood survivors are not reported.
Abstract
Arsenic trioxide (ATO) in combination with all-trans retinoic acid (ATRA) has been shown to be effective in both adult and pediatric patients with acute promyelocytic leukemia (APL). Addition of ATO to conventional chemotherapy could lead to a reduction in the doses of cytotoxic agents, but the long-term safety of ATO is not fully understood, especially in children. The Japan Children's Cancer Group conducted a risk-stratified prospective study to investigate safety and efficacy of ATO in children with newly diagnosed APL by replacing all three intensification phases with ATO. The 3-year event-free survival and overall survival rates in 27 children were 96.3% (95% CI 76.5%-99.5%) and 100% (95% CI 87.2%-100%), respectively. Prolonged QTc interval or other cardiac conduction disorders of any grade were observed in 20 out of the 63 intensification cycles. The durations of leukocytopenia, neutropenia, and G-CSF treatment were significantly shorter in this study than in a previous Japanese study that used conventional cytotoxic chemotherapy. Furthermore, no cardiac, metabolic, renal, cutaneous, or neurological symptoms were reported for up to 5 years after completion of the protocol therapy. The JPLSG AML-P13 study demonstrated excellent outcomes and safety of ATO in children with APL.
Findings the Hub has extracted
Each claim below is labelled by its type of design and links back to this report.
- Intervention
- Arsenic trioxide (ATO) in combination with all-trans retinoic acid (ATRA) replacing all three conventional intensification phases of chemotherapy
- Population
- 27 children with newly diagnosed acute promyelocytic leukemia in Japan
Key finding
3-year event-free survival 96.3% (95% CI 76.5%-99.5%)
H et al. · Europe PMC · 4 Sept 2025