Lixian Chang, PhD1,2*, Ju GAO3*, Xiaoying Lei4*, Yingyi He5*, Shuquan Zhuang6*, Chunhuai Li7*, Kaizhi Weng8*, Lingzhen Wang9*, Xia Guo3*, Qihui Liu4*, Pengfei Wang5*, Yong Zhuang10*, Mei Yan11*, Wei Liu12*, Hui Chen13*, Min Zhang14*, Shuhong Shen15, Xiaofan Zhu, MD1,16, Xiuli Ju10*, Li Zhang1,16* and Zhuo Wang17*
1State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin, China
2Tianjin Institutes of Health Science, Tianjin, AL, China
3West China Second University Hospital, Sichuan University, Sichuan, China
4Children's Hospital of Chongqing Medical University, Chongqing, China
5Department of Pediatric Hematology/Oncology,Guangzhou Women and Children's Medical Center,Guangzhou Medical University, Guangzhou, China
6Department of Pediatric,Quanzhou First Hospital Affiliated to Fujian Medical University, Quanzhou, China
7Department of pediatric hematology, the First Hospital of Jilin University, Jilin, China
8Department of Pediatric Hematology，Rheumatology and Nephrology, Zhangzhou Municipal Hospital of Fujian Province, Zhangzhou, China
9the affiliated hospital of Qingdao University, Qingdao, China
10Department of Pediatrics, Qilu Hospital of Shandong University, Jinan, China
11The First Affiliated Hospital of Xinjiang Medical University, Urumqi, China
12Children's Hospital Affiliated to Zhengzhou University, Zhengzhou, China
13Tianjin Children's Hospital, Tianjin, China
14Northwest women's and children's Hospital, Xi'an, China
15Department of Hematology , Shanghai Children’s Medical Center, School of Medicine, Shanghai Jiao Tong University, Shanghai, China
16Tianjin Institutes of Health Science, Tianjin, China
17Department of Hematology, Shanghai Children's Medical Center, School of Medicine,Shanghai Jiao Tong University, Shanghai, China
The standard therapy for childhood acute promyelocytic leukemia (APL) involves the combination of all-trans retinoic acid (ATRA) with arsenic trioxide (ATO) or Compound Huangdai tablet (Realgar-Indigo Naturalis formula, RIF). However, the effectiveness and safety of combining RIF with ATRA in a larger population of pediatric APL patients remains undocumented. Additionally, there is variability in the oral timing and dosage of RIF in different studies, which should be standardized.
We conducted a multicenter clinical trial (ChiCTR-OIC-16010014) in China. Individuals newly diagnosed with APL were categorized into two risk groups based on their initial white blood cell (WBC) count. Those with a WBC count exceeding 5×10^9/L received hydroxycarbamide at a dosage ranging from 20 to 50 mg/kg per day until their WBC levels dropped to 10×10^9/L or lower. The primary outcomes assessed were event-free survival (EFS) and overall survival (OS) over six years.
We recruited 200 patients diagnosed with APL. After a median follow-up time of 43.2 months, it was observed that the six-year OS rate was 100% in the low-risk cohort and 97.6% in the high-risk cohort. The six-year EFS rate was found to be 98.3% in the low-risk group and 97.6% in the high-risk group. It was observed that plasma levels of arsenic remained stable after the administration of RIF at a dosage of 60 mg/kg/d for seven days and returned to baseline levels within fourteen days after discontinuation of RIF administration. Furthermore, treatment strategies aimed at controlling WBC counts to maintain levels at or below 30×10^9/L were found to reduce the incidence of induced differentiation syndrome (DS) or alleviate its associated symptoms.
In conclusion, The CCCG-APL-2017 regimen, which combines RIF with ATRA, demonstrates both efficacy and safety in treating children with APL. A dosage of 60mg/kg/d of RIF is considered sufficient for the management of pediatric APL.
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