1. Oral arsenic and retinoic acid for high-risk acute promyelocytic leukemia.
- Author
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Ma YF, Lu Y, Wu Q, Lou YJ, Yang M, Xu JY, Sun CH, Mao LP, Xu GX, Li L, Huang J, Wang HY, Lou LJ, Meng HT, Qian JJ, Yu WJ, Wei JY, Li ZY, Zhu XL, Yan XY, Chen SN, Jin J, and Zhu HH
- Subjects
- Humans, Adult, Tretinoin therapeutic use, Arsenic Trioxide therapeutic use, Arsenic Trioxide adverse effects, Treatment Outcome, Antineoplastic Combined Chemotherapy Protocols adverse effects, Oxides therapeutic use, Leukemia, Promyelocytic, Acute drug therapy, Arsenic therapeutic use, Arsenicals adverse effects
- Abstract
Acute promyelocytic leukemia (APL) has become curable over 95% patients under a complete chemo-free treatment with all-trans retinoic acid (ATRA) and arsenic trioxide in low-risk patients. Minimizing chemotherapy has proven feasible in high-risk patients. We evaluated oral arsenic and ATRA without chemotherapy as an outpatient consolidation therapy and no maintenance for high-risk APL. We conducted a multicenter, single-arm, phase 2 study with consolidation phases. The consolidation therapy included Realgar-Indigo naturalis formula (60 mg/kg daily in an oral divided dose) in a 4-week-on and 4-week-off regimen for 4 cycles and ATRA (25 mg/m
2 daily in an oral divided dose) in a 2-week-on and 2-week-off regimen for 7 cycles. The primary end point was the disease-free survival (DFS). Secondary end points included measurable resident disease, overall survival (OS), and safety. A total of 54 participants were enrolled at seven centers from May 2019. The median age was 40 years. At the median follow-up of 13.8 months (through April 2022), estimated 2-year DFS and OS were 94% and 100% in an intention-to-treat analysis. All the patients achieved complete molecular remission at the end of consolidation phase. Two patients relapsed after consolidation with a cumulative incidence of relapse of 6.2%. The majority of adverse events were grade 1-2, and only three grade 3 adverse events were observed. Oral arsenic plus ATRA without chemotherapy was active as a first-line consolidation therapy for high-risk APL.Trial registration: chictr.org.cn number, ChiCTR1900023309., (© 2022. The Author(s).)- Published
- 2022
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