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The problem
Intervention: Arsenic trioxide (ATO) in combination with all-trans retinoic acid (ATRA) replacing all three conventional intensification phases of chemotherapy
Who it was tested with: 27 children with newly diagnosed acute promyelocytic leukemia in Japan.
Experience from people who deliver programmes: stories, case studies and lessons learned. Valuable for understanding how and why, and for spotting what to test next, but not a measure of effect.
"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 study prospectively measured outcomes and safety in a defined pediatric population receiving ATO-based therapy, with outcome measurements at baseline, during treatment cycles, and at 3-year and 5-year follow-up points.
Single-arm prospective study without concurrent control group limits ability to attribute outcomes solely to the ATO intervention. Small sample size (n=27). No comparison of long-term toxicities or developmental outcomes between ATO and conventional chemotherapy arms. Limited follow-up data beyond 5 years.
Across countries and designs
1 study · 1 country