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First-line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy.
Clin Transplant. 2018 02; 32(2)CT

Abstract

We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = .806). The failure-free survival was significantly inferior in patients receiving IST than in those undergoing transplantation from an HID (52.6 ± 10.5% vs 89.3 ± 5.8, P = .008). In univariate and multivariate analysis, the choice of first-line immunosuppressive therapy was the only adverse predictor for failure-free survival. At the last follow-up, completely normal blood count was observed in 11 of 20 (55.0%) and 24 of 25 (96.0%) live cases in IST and HID-HSCT cohort (P = .003). These suggest that HSCT from a haploidentical donor could be considered as first-line treatment in children who lack a matched related donor, especially in experienced transplantation centers.

Authors+Show Affiliations

Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Department of Pediatrics, Peking University People's Hospital, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.Department of Pediatrics, Peking University People's Hospital, Beijing, China.Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.

Pub Type(s)

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

Language

eng

PubMed ID

29297952

Citation

Cheng, Yifei, et al. "First-line Choice for Severe Aplastic Anemia in Children: Transplantation From a Haploidentical Donor Vs Immunosuppressive Therapy." Clinical Transplantation, vol. 32, no. 2, 2018.
Cheng Y, Xu Z, Zhang Y, et al. First-line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy. Clin Transplant. 2018;32(2).
Cheng, Y., Xu, Z., Zhang, Y., Wu, J., Wang, F., Mo, X., Chen, Y., Han, W., Jia, J., Wang, Y., Zhang, X., Huang, X., Zhang, L., & Xu, L. (2018). First-line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy. Clinical Transplantation, 32(2). https://doi.org/10.1111/ctr.13179
Cheng Y, et al. First-line Choice for Severe Aplastic Anemia in Children: Transplantation From a Haploidentical Donor Vs Immunosuppressive Therapy. Clin Transplant. 2018;32(2) PubMed PMID: 29297952.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - First-line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy. AU - Cheng,Yifei, AU - Xu,Zhengli, AU - Zhang,Yuanyuan, AU - Wu,Jun, AU - Wang,Fengrong, AU - Mo,Xiaodong, AU - Chen,Yuhong, AU - Han,Wei, AU - Jia,Jinsong, AU - Wang,Yu, AU - Zhang,Xiaohui, AU - Huang,Xiaojun, AU - Zhang,Leping, AU - Xu,Lanping, Y1 - 2018/01/03/ PY - 2017/12/11/accepted PY - 2018/1/4/pubmed PY - 2019/5/29/medline PY - 2018/1/4/entrez KW - aplastic anemia KW - haploidentical transplantation KW - immunosuppressive therapy JF - Clinical transplantation JO - Clin Transplant VL - 32 IS - 2 N2 - We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = .806). The failure-free survival was significantly inferior in patients receiving IST than in those undergoing transplantation from an HID (52.6 ± 10.5% vs 89.3 ± 5.8, P = .008). In univariate and multivariate analysis, the choice of first-line immunosuppressive therapy was the only adverse predictor for failure-free survival. At the last follow-up, completely normal blood count was observed in 11 of 20 (55.0%) and 24 of 25 (96.0%) live cases in IST and HID-HSCT cohort (P = .003). These suggest that HSCT from a haploidentical donor could be considered as first-line treatment in children who lack a matched related donor, especially in experienced transplantation centers. SN - 1399-0012 UR - https://www.unboundmedicine.com/medline/citation/29297952/First_line_choice_for_severe_aplastic_anemia_in_children:_Transplantation_from_a_haploidentical_donor_vs_immunosuppressive_therapy_ L2 - https://doi.org/10.1111/ctr.13179 DB - PRIME DP - Unbound Medicine ER -