Int J Med Sci
2024; 21(6):1027-1036.
doi:10.7150/ijms.94012 This issueCite
Research Paper
Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research
Meng Yuan1#, Chenguang Jia1#, Jie Ma1#, Man Zhang1#, Guanghua Zhu1, Bin Wang1, Jie Zheng1, Maoquan Qin1✉, Runhui Wu1✉, Sidan Li2✉
1. 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; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China 2. Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. # These authors contributed equally to this work and should be considered co-first authors.
✉ Corresponding authors: Sidan Li, Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. E-mail: lisidan2006com. Runhui Wu, 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; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China. E-mail: runhuiwucom. Maoquan Qin, 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; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China. E-mail: 18123956com.More
Citation:
Yuan M, Jia C, Ma J, Zhang M, Zhu G, Wang B, Zheng J, Qin M, Wu R, Li S. Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research. Int J Med Sci 2024; 21(6):1027-1036. doi:10.7150/ijms.94012. https://www.medsci.org/v21p1027.htm
MRD-HSCT is the first-line therapy for children with SAA, while it is not easy to find a compatible donor due to the Chinese one-child policy. IST has a high recurrence rate, a risk of clonal transformation. Thus, Haplo-HSCT, as a first-line treatment, has gradually attracted clinicians' attention. To evaluate the efficacy of Haplo-HSCT in children with SAA, we performed a retrospective study (2006.06-2021.01) of 210 patients with AA who received HSCT or IST in Beijing Children's Hospital. The OS and FFS rates were analyzed to evaluate the efficacy of Haplo-HSCT and IST. We found that from 2006 to 2021, 3- and 5-year cumulative survival rates were both 85.3% in the first-line Haplo group, 98.1% and 96.8% in the first-line IST group, both 85.7% in the ATG group (P = 0.866), both 100% in the ATG + TPO group (P = 0.016), and 99.1% and 97.2% in the ATG + eltrombopag group (P = 0.056). 3- and 5-year cumulative FFS rates were both 85.3% in the first-line Haplo-HSCT group and 67.5% and 66.2% in the first-line IST group (P = 0.033). Therefore, we believe that Haplo-HSCT can be a first-line treatment for paediatric SAA.
Keywords: aplastic anemia, hematopoietic stem cell transplantation, immunosuppressive therapy, children
Citation styles
APA
Yuan, M., Jia, C., Ma, J., Zhang, M., Zhu, G., Wang, B., Zheng, J., Qin, M., Wu, R., Li, S. (2024). Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research. International Journal of Medical Sciences, 21(6), 1027-1036. https://doi.org/10.7150/ijms.94012.
ACS
Yuan, M.; Jia, C.; Ma, J.; Zhang, M.; Zhu, G.; Wang, B.; Zheng, J.; Qin, M.; Wu, R.; Li, S. Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research. Int. J. Med. Sci. 2024, 21 (6), 1027-1036. DOI: 10.7150/ijms.94012.
NLM
Yuan M, Jia C, Ma J, Zhang M, Zhu G, Wang B, Zheng J, Qin M, Wu R, Li S. Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research. Int J Med Sci 2024; 21(6):1027-1036. doi:10.7150/ijms.94012. https://www.medsci.org/v21p1027.htm
CSE
Yuan M, Jia C, Ma J, Zhang M, Zhu G, Wang B, Zheng J, Qin M, Wu R, Li S. 2024. Haploidentical hematopoietic stem cell transplantation as a first-line treatment for paediatric severe aplastic anemia: a single-center research. Int J Med Sci. 21(6):1027-1036.
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