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International collaboration helps refine treatment for childhood leukemia

Vincristine and dexamethasone pulses can be safely eliminated from low-risk acute lymphoblastic leukemia treatment

Memphis, Tennessee, July 27, 2021

Doctor wearing a lab coat smiles at the camera.

Corresponding author Ching-Hon Pui, M.D., St. Jude Department of Oncology chair, is working with colleagues in China to develop better therapy for childhood acute lymphoblastic leukemia (ALL). 

Scientists at St. Jude Children’s Research Hospital are working with colleagues in China to develop better therapy for childhood acute lymphoblastic leukemia (ALL). Results from a large phase 3 noninferiority clinical trial definitively showed that vincristine and dexamethasone pulses can be eliminated in patients with low-risk disease. The findings were published today in The Lancet Oncology.  

Adding the chemotherapy vincristine plus a steroid (originally prednisone, and later dexamethasone) as pulse therapy for childhood ALL has been part of standard care since the 1970s. This is despite their being associated with neuropsychological side effects, neuropathy and other late effects. However, to date studies about the need for prolonged treatment with pulse therapy have been inconclusive.

“We wanted to study this issue to provide definitive conclusions about whether we can safely omit prolonged pulse therapy with these two drugs to improve quality of life for patients and lessen the burden to their family,” said corresponding author Ching-Hon Pui, M.D., St. Jude Department of Oncology chair. “That’s why doing this study through the Chinese Children’s Cancer Group was key: a definitive noninferiority randomized trial of a disease with a high cure rate such as ALL requires very large numbers of patients all treated consistently.”

Clinical trial provides clarity

Between January 2015 and February 2019, children with newly diagnosed ALL joined a randomized, open-label, phase 3 noninferiority study as part of the Chinese Children’s Cancer Group ALL-2015 protocol. This clinical trial randomized 6,108 patients, making it the largest clinical trial ever conducted in childhood acute lymphoblastic leukemia.

Patients in continuous remission for one year were stratified and randomized to receive or not receive seven pulses of vincristine plus dexamethasone during the second year of treatment. Using the noninferiority study design, researchers firmly established that pulse therapy can be safely omitted in the second year of care in patients with low-risk disease without affecting their five-year event-free survival or overall survival.

Omitting vincristine plus dexamethasone pulses after the first year of treatment in these children should reduce many acute and late effects of treatment as well as the burden on their families. Additional studies are needed to confirm whether this is true for patients with intermediate or high-risk ALL.

“These findings are very good news for patients and families because shortening this pulse therapy will substantially reduce neuropsychological side effects, emotional disturbances and many other neurological and metabolic late effects,” Pui said.

The study’s first authors are Wenyu Yang, Chinese Academy of Medical Sciences and Peking Union Medical College; Jiaoyang Cai and Shuhong Shen, Shanghai Children’s Medical Center; Ju Gao, West China Second University Hospital and Sichuan University; Jie Yu, Chongqing Medical University Affiliated Children’s Hospital; and Shaoyan Hu, Children’s Hospital of Soochow University. In addition to Pui, the paper’s co-senior authors are Cheng Cheng, St. Jude; Chi-kong Li, Hong Kong Children’s Hospital, The Chinese University of Hong Kong; Jingyan Tang, Shanghai Children’s Medical Center; and Xiaofan Zhu, Chinese Academy of Medical Sciences and Peking Union Medical College.

Additional authors of the study are Hua Jiang and Hui Zhang, Guangzhou Women and Children’s Medical Center; Yongjun Fang, Children’s Hospital of Nanjing Medical University; Changda Liang, Jiangxi Provincial Children’s Hospital; Xiuli Ju, Qilu Hospital of Shandong University; Xuedong Wu and Chunfu Li, Nanfang Hospital Southern Medical University; Xiaowen Zhai, Children’s Hospital of Fudan University; Xin Tian, KunMing Children’s Hospital; Ningling Wang, Anhui Medical University Second Affiliated Hospital; Aiguo Liu, Tongji Hospital of Tongji Medical College Huazhong University of Science and Technology; Hui Jiang, Children’s Hospital Affiliated to Shanghai Jiao Tong University; Runming Jin, Union Hospital of Tongji Medical College, Huazhong University of Science and Technology; Lirong Sun, Affiliated Hospital of Qingdao University; Minghua Yang, Xiangya Hospital Central South University; Alex WK Leung, Hong Kong Children’s Hospital, the Chinese University of Hong Kong; Kaili Pan, Xi’an Northwest Women’s and Children’s Hospital; Yingchi Zhang, Chinese Academy of Medical Sciences and Peking Union Medical College; Jing Chen, Shanghai Jiao Tong University; Yiping Zhu, West China Second University Hospital, Sichuan University; and Jun J. Yang, St. Jude.

The study was funded by the VIVA China Children’s Cancer Foundation, the National Natural Science Foundation of China, the fourth round of the Three-Year Public Health Action Plan (China), Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences, the National Cancer Institute (United States), St. Baldrick’s Foundation and ALSAC, the fundraising and awareness organization of St. Jude


St. Jude Children's Research Hospital

St. Jude Children's Research Hospital is leading the way the world understands, treats and cures childhood cancer and other life-threatening diseases. It is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children. Treatments developed at St. Jude have helped push the overall childhood cancer survival rate from 20% to 80% since the hospital opened more than 50 years ago. St. Jude freely shares the breakthroughs it makes, and every child saved at St. Jude means doctors and scientists worldwide can use that knowledge to save thousands more children. Families never receive a bill from St. Jude for treatment, travel, housing and food — because all a family should worry about is helping their child live. To learn more, visit or follow St. Jude on social media at @stjuderesearch.