An effective and chemotherapy-free strategy of all-trans retinoic acid and arsenic trioxide for acute promyelocytic leukemia in all risk groups (APL15 trial)

• Published in: Blood cancer journal (New York) Vol. 12; no. 11; pp. 158 - 8
• Main Authors: Wang, Huai-Yu, Gong, Sha, Li, Guo-Hui, Yao, Ya-Zhou, Zheng, Yin-Suo, Lu, Xiao-Hong, Wei, Su-Hua, Qin, Wei-Wei, Liu, Hai-Bo, Wang, Meng-Chang, Xi, Jie-Ying, Chen, Li-Mei, Zhang, Mei, Zhang, Xin-Xin, Zhang, Hui-Yun, Zhang, Cheng-Sheng, Wald, David N., Zhu, Hong-Hu, Liu, Li, He, Peng-Cheng
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 21.11.2022; Springer Nature B.V; Nature Publishing Group
• Subjects: 692/308/2779/777; 692/699/67/1990/283/1897; Arsenic; Arsenic Trioxide - therapeutic use; Arsenicals - therapeutic use; Biomedical and Life Sciences; Biomedicine; Cancer Research; Chemotherapy; Hematology; Humans; Leukemia; Leukemia, Promyelocytic, Acute - drug therapy; Oncology; Oxides - therapeutic use; Treatment Outcome; Tretinoin - therapeutic use
• ISSN: 2044-5385; 2044-5385
• DOI: 10.1038/s41408-022-00753-y

The combination of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) has been demonstrated to have comparable effectiveness or better to ATRA and chemotherapy (CHT) in non-high-risk acute promyelocytic leukemia (APL). However, the efficacy of ATRA-ATO compared to ATRA-ATO plus CHT in high-risk APL remains unknown. Here we performed a randomized multi-center non-inferiority phase III study to compare the efficacy of ATRA-ATO and ATRA-ATO plus CHT in newly diagnosed all-risk APL to address this question. Patients were assigned to receive ATRA-ATO for induction, consolidation, and maintenance or ATRA-ATO plus CHT for induction followed by three cycles of consolidation therapy, and maintenance therapy with ATRA-ATO. In the non-CHT group, hydroxyurea was used to control leukocytosis. A total of 128 patients were treated. The complete remission rate was 97% in both groups. The 2-year disease-free, event-free survival rates in the non-CHT group and CHT group in all-risk patients were 98% vs 97%, and 95% vs 92%, respectively ( P  = 0.62 and P  = 0.39, respectively). And they were 94% vs 87%, and 85% vs 78% in the high-risk patients ( P  = 0.52 and P  = 0.44, respectively). This study demonstrated that ATRA-ATO had the same efficacy as the ATRA-ATO plus CHT in the treatment of patients with all-risk APL.