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Hematopoietic stem cell transplantation without in vivo T-cell depletion for pediatric aplastic anemia: A single-center experience.
Pediatr Transplant. 2018 08; 22(5):e13204.PT

Abstract

For young patients, HLA-MRD HSCT is the first-line treatment of SAA. However, due to China's birth control policy, few patients could find suitable sibling donors and HLA-MUD. More and more transplantation centers have used Haplo-D as the donor source for young adult and pediatric patients. However, studies with larger amount of pediatric patients are rare. We retrospectively analyzed the data of children with AA who were treated with allogeneic HSCT and compared the therapeutic efficacy of Haplo-HSCT and MRD/MUD group. A total of 62 patients were enrolled. Implantation was successfully performed in 58 patients. There was no significant difference in the time for reconstruction of hematopoietic function between patients in the two groups. Thirty-two had grade I-IV aGVHD with incidence of 51.61%. The incidence of aGVHD was 79.41% for patients in the Haplo-HSCT, significantly higher than that of 17.86% for patients in the MRD/MUD group (P < .01). However, the incidence of cGVHD was not significantly different between patients in the two groups (26.47% vs 10.71%, P = .09), the incidence of CMV infection was 28.57% and 52.94% for patients in the MRD/MUD and Haplo group, respectively, showing no significant difference (P = .053). The incidence of EBV infection was 47.06% for patients in the Haplo group and 28.57% for patients in the MRD/MUD group, showing no significant difference (P = .11). However, the 3- and 5-year cumulative OS and FFS rates showed statistically significant difference in the two groups, P = .012 and .045, respectively. Compared to Haplo-HSCT, MRD/MUD is more economic. In this study, we achieved good Haplo transplantation results. The incidences of cGVHD and CMV/EBV were not significantly different between Haplo group and MRD/MUD group. Although OS and FFS of the Haplo group were not as good as those of the MRD/MUD group, it is still acceptable as an alternative treatment under emergency.

Authors+Show Affiliations

Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Emergency Department, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China. Hematology Oncology Center, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, China.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

29744996

Citation

Li, Sidan, et al. "Hematopoietic Stem Cell Transplantation Without in Vivo T-cell Depletion for Pediatric Aplastic Anemia: a Single-center Experience." Pediatric Transplantation, vol. 22, no. 5, 2018, pp. e13204.
Li S, Wang B, Fu L, et al. Hematopoietic stem cell transplantation without in vivo T-cell depletion for pediatric aplastic anemia: A single-center experience. Pediatr Transplant. 2018;22(5):e13204.
Li, S., Wang, B., Fu, L., Pang, Y., Zhu, G., Zhou, X., Ma, J., Su, Y., Qin, M., & Wu, R. (2018). Hematopoietic stem cell transplantation without in vivo T-cell depletion for pediatric aplastic anemia: A single-center experience. Pediatric Transplantation, 22(5), e13204. https://doi.org/10.1111/petr.13204
Li S, et al. Hematopoietic Stem Cell Transplantation Without in Vivo T-cell Depletion for Pediatric Aplastic Anemia: a Single-center Experience. Pediatr Transplant. 2018;22(5):e13204. PubMed PMID: 29744996.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Hematopoietic stem cell transplantation without in vivo T-cell depletion for pediatric aplastic anemia: A single-center experience. AU - Li,Sidan, AU - Wang,Bin, AU - Fu,Lingling, AU - Pang,Yilin, AU - Zhu,Guanghua, AU - Zhou,Xuan, AU - Ma,Jie, AU - Su,Yan, AU - Qin,Maoquan, AU - Wu,Runhui, Y1 - 2018/05/10/ PY - 2018/03/07/accepted PY - 2018/5/11/pubmed PY - 2018/11/8/medline PY - 2018/5/11/entrez KW - aplastic anemia KW - haploidentical donor KW - hematopoietic stem cell transplantation KW - pediatric SP - e13204 EP - e13204 JF - Pediatric transplantation JO - Pediatr Transplant VL - 22 IS - 5 N2 - For young patients, HLA-MRD HSCT is the first-line treatment of SAA. However, due to China's birth control policy, few patients could find suitable sibling donors and HLA-MUD. More and more transplantation centers have used Haplo-D as the donor source for young adult and pediatric patients. However, studies with larger amount of pediatric patients are rare. We retrospectively analyzed the data of children with AA who were treated with allogeneic HSCT and compared the therapeutic efficacy of Haplo-HSCT and MRD/MUD group. A total of 62 patients were enrolled. Implantation was successfully performed in 58 patients. There was no significant difference in the time for reconstruction of hematopoietic function between patients in the two groups. Thirty-two had grade I-IV aGVHD with incidence of 51.61%. The incidence of aGVHD was 79.41% for patients in the Haplo-HSCT, significantly higher than that of 17.86% for patients in the MRD/MUD group (P < .01). However, the incidence of cGVHD was not significantly different between patients in the two groups (26.47% vs 10.71%, P = .09), the incidence of CMV infection was 28.57% and 52.94% for patients in the MRD/MUD and Haplo group, respectively, showing no significant difference (P = .053). The incidence of EBV infection was 47.06% for patients in the Haplo group and 28.57% for patients in the MRD/MUD group, showing no significant difference (P = .11). However, the 3- and 5-year cumulative OS and FFS rates showed statistically significant difference in the two groups, P = .012 and .045, respectively. Compared to Haplo-HSCT, MRD/MUD is more economic. In this study, we achieved good Haplo transplantation results. The incidences of cGVHD and CMV/EBV were not significantly different between Haplo group and MRD/MUD group. Although OS and FFS of the Haplo group were not as good as those of the MRD/MUD group, it is still acceptable as an alternative treatment under emergency. SN - 1399-3046 UR - https://www.unboundmedicine.com/medline/citation/29744996/Hematopoietic_stem_cell_transplantation_without_in_vivo_T_cell_depletion_for_pediatric_aplastic_anemia:_A_single_center_experience_ L2 - https://doi.org/10.1111/petr.13204 DB - PRIME DP - Unbound Medicine ER -