Long-term outcome of children with acute promyelocytic leukemia: a randomized study of oral versus intravenous arsenic by SCCLG-APL group.

Autor: Huang DP; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Yang LC; Department of Pediatrics, Xiangya Hospital, Central South University, Changsha, Hunan, China., Chen YQ; Department of Pediatric Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China., Wan WQ; Department of Pediatrics, Second Xiangya Hospital, Central South University, Changsha, Hunan, China., Zhou DH; Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Mai HR; Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen, Guangdong, China., Li WL; Department of Hematology, Hunan Children's Hospital, Changsha, Hunan, China., Yang LH; Department of Pediatrics, Zhujiang Hospital, Southern Medical University, Guangzhou, Guangdong, China., Lan HK; Department of Pediatrics, Zhujiang Hospital, Southern Medical University, Guangzhou, Guangdong, China., Chen HQ; Department of Pediatrics, Third Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Guo BY; Department of Pediatrics, First Affiliated Hospital of Xiamen University, Xiamen, Fujian, China., Zhen ZJ; Department of Pediatrics, Sun Yat-sen University Cancer Center, Guanzhou, Guangdong, China., Liu RY; Department of Pediatrics, Huizhou Central People's Hospital, Huizhou, Guangdong, China., Chen GH; Department of Pediatrics, First People's Hospital of Huizhou, Huizhou, Guangdong, China., Feng XQ; Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China., Liang C; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Wang LN; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Li Y; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Luo JS; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Fan Z; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Luo XQ; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China., Li B; Biostatistics Team, Clinical Trials Unit, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guandong, China., Tang YL; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China. tangylai@mail.sysu.edu.cn., Zhang XL; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China. zhangxli@mail.sysu.edu.cn., Huang LB; Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China. huanglb3@mail.sysu.edu.cn.
Jazyk: angličtina
Zdroj: Blood cancer journal [Blood Cancer J] 2023 Dec 05; Vol. 13 (1), pp. 178. Date of Electronic Publication: 2023 Dec 05.
DOI: 10.1038/s41408-023-00949-w
Abstrakt: Realgar-Indigo naturalis formula (RIF), an oral traditional Chinese medicine mainly containing Realgar (As 4 S 4 ), is highly effective in treating adult acute promyelocytic leukemia (APL). However, the treatment efficacy and safety of RIF have not been verified in pediatric patients. SCCLG-APL group conducted a multicenter randomized non-inferiority trial to determine whether intravenous arsenic trioxide (ATO) can be substituted by oral RIF in treating pediatric APL. Of 176 eligible patients enrolled, 91 and 85 were randomized to ATO and RIF groups, respectively. Patients were treated with the risk-adapted protocol. Induction, consolidation, and 96-week maintenance treatment contained all-trans-retinoic acid and low-intensity chemotherapy, and either ATO or RIF. The primary endpoint was 5-year event-free survival (EFS). The secondary endpoints were adverse events and hospital days. After a median 6-year follow-up, the 5-year EFS was 97.6% in both groups. However, the RIF group had significantly shorter hospital stays and lower incidence of infection and tended to have less cardiac toxicity. All 4 relapses occurred within 1.5 years after completion of maintenance therapy. No long-term arsenic retentions were observed in either group. Substituting oral RIF for ATO maintains treatment efficacy while reducing hospitalization and adverse events in treating pediatric APL patients, which may be a future treatment strategy for APL.
(© 2023. The Author(s).)
Databáze: MEDLINE
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