Safety and Effectiveness of Rasburicase in the Control of Hyperuricemia in Pediatric Patients with Non-Hodgkin’s Lymphoma and Acute Leukemia: An Open-Label, Single-Arm, Multi-center, Interventional Study
Tianyou Wang,
Xiaofan Zhu,
Yumei Chen,
Shuhong Shen,
Yongmin Tang,
Jingying Zhang,
Yingyi He,
Hui Zhang,
Ju Gao,
Jianpei Fang,
Rong Liu,
Xiaoyan Wu,
Jinchuan Sun,
Minlu Zhang
Affiliations
Tianyou Wang
Department of Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education; Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health
Xiaofan Zhu
Department of Hematology, Chinese Academy of Medical Sciences, Hematology Hospital
Yumei Chen
Department of Hematology, Chinese Academy of Medical Sciences, Hematology Hospital
Shuhong Shen
Department of Hematology, Shanghai Children’s Medical Center
Yongmin Tang
Department/Center of Hematology-Oncology, Children’s Hospital of Zhejiang University School of Medicine, Pediatric Leukemia Diagnostic and Therapeutic Technology Research Center of Zhejiang Province, National Clinical Research Center for Child Health
Jingying Zhang
Department/Center of Hematology-Oncology, Children’s Hospital of Zhejiang University School of Medicine, Pediatric Leukemia Diagnostic and Therapeutic Technology Research Center of Zhejiang Province, National Clinical Research Center for Child Health
Yingyi He
Department of Pediatric Hematology/Oncology, Guangzhou Women and Children’s Medical Center
Hui Zhang
Department of Pediatric Hematology/Oncology, Guangzhou Women and Children’s Medical Center
Ju Gao
Department of Hematology, West China Second University Hospital
Jianpei Fang
Department of Hematology, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
Rong Liu
Department of Hematology, Children’s Hospital Capital Institute of Pediatrics
Xiaoyan Wu
Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Abstract Introduction Despite rasburicase's proven efficiency in Caucasians, Japanese, and Koreans, studies evaluating the safety and effectiveness of rasburicase in Chinese pediatric patients with non-Hodgkin’s lymphoma (NHL) and acute leukemia (AL) in particular are lacking. Objective The aim was to evaluate the safety and effectiveness of rasburicase in Chinese pediatric patients with NHL and AL. Methods In this phase IV, open-label, non-randomized, single-arm, multi-center, interventional study (NCT04349306), children newly diagnosed with NHL or AL who received 0.20 mg/kg/day of rasburicase were included. The primary objective was to assess the safety of rasburicase by the incidence of adverse events (AEs). The secondary objective was to determine the effectiveness of rasburicase in the control of hyperuricemia. Results Out of 50 patients, 25 reported a total of 76 treatment-emergent adverse events (TEAEs), including eight TEAEs of grade ≥ 3 in 12 patients. A drug-related serious AE was reported in one patient, and there was no incidence of death. The response rate in the intent-to-treat population was 100.0% (95% confidence interval 82.4–100.0) in patients (n = 19) with baseline uric acid level of > 8.0 mg/dL. Similarly, the response rate was 86.2% (n = 25) among 29 patients (60.4%) with baseline uric acid levels of ≤ 8.0 mg/dL. The maximum mean percentage decrease of plasma uric acid level in the overall patients was 96.9%. Conclusion Rasburicase was well tolerated and effective in controlling hyperuricemia in Chinese pediatric patients with NHL and AL.
