- Clinical Trials
- Research News
- Industry Trends
- Agency Actions
- Drug Safety Issues
- Approvals, Launches, & New Indications
- Health Care Reform
New Study Data: Leukemia May Be Curable Without Chemotherapy
Retinoic acid plus arsenic trioxide eradicates acute promyelocytic disease (July 11)
All-trans retinoic acid (ATRA) with chemotherapy is the standard of care for acute promyelocytic leukemia (APL), providing cures rates that exceed 80%. In a new phase III study published in the New England Journal of Medicine, however, researchers suggest that APL may be curable with ATRA plus arsenic trioxide rather than chemo.
Patients with low- to intermediate-risk APL (white blood cell count, = 10×109/L) were randomly assigned to receive either ATRA plus arsenic trioxide for induction and consolidation therapy or standard ATRA plus idarubicin induction therapy followed by three cycles of consolidation therapy with ATRA plus chemotherapy, and maintenance therapy with ATRA and low-dose chemotherapy.
After a median follow-up period of 34.4 months, complete remission was achieved in all 77 patients treated with ATRA plus arsenic trioxide compared with 75 of 79 patients (95%) given the chemotherapy-based standard of care (P = 0.12).
Two-year event-free survival rates were 97% in the ATRA/arsenic trioxide group and 86% in the ATRA/chemotherapy group (P < 0.001 for noninferiority and P = 0.02 for superiority of ATRA plus arsenic trioxide). Overall survival was also better in the ATRA/arsenic trioxide group (P = 0.02).
ATRA plus arsenic trioxide was associated with less hematologic toxicity and fewer infections than the chemotherapy combination, but with more hepatic toxicity.
“Although longer follow-up will be needed to draw firm conclusions, our results support previously reported clinical and experimental evidence indicating that ATRA and arsenic trioxide act synergistically to eradicate APL,” the authors said.
Source: NEJM; July 11, 2013.