Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia

• DOI: 10.1073/pnas.0813280106
• Publication Date: 2009-02-19T02:40:55Z
• Authors: Jiong Hu; Yuan-Fang Liu; Chuan-Feng Wu; Fang Xu; Zhi-Xiang Shen; Yong-Mei Zhu; Jun-Min Li; Wei Tang; Wei-Li Zhao; Wen Wu; Hui-Ping Sun; Qiu-Sheng Chen; Bing Chen; Guang-Biao Zhou; Arthur Zelent; Samuel Waxman; Zhen-Yi Wang; Sai-Juan Chen; Zhu Chen
• Keywords: Arsenic Trioxide; Tretinoin

All-trans retinoic acid (ATRA)/arsenic trioxide (ATO) combination-based therapy has benefitted newly diagnosed acute promyelocytic leukemia (APL) in short-term studies, but the long-term efficacy and safety remained unclear. From April 2001, we have followed 85 patients administrated ATRA/ATO with a median follow-up of 70 months. Eighty (94.1%) entered complete remission (CR). Kaplan-Meier estimates 5-year event-free survival (EFS) overall (OS) for all were 89.2% +/- 3.4% 91.7% 3.0%, respectively, relapse-free (RFS) OS who achieved CR (n = 80) 94.8% 2.5% 97.4% 1.8%, respectively. Upon ATRA/ATO, prognosis was not influenced by initial white blood cell count, distinct PML-RARalpha types, or FLT3 mutations. The toxicity profile mild reversible. No secondary carcinoma observed, 24 months after last dose had urine arsenic concentrations well below limit. These results demonstrate high minimal treatment APL follow-up, suggesting potential frontline de novo APL.