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Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor.
Biol Blood Marrow Transplant. 2019 05; 25(5):975-980.BB

Abstract

We compared the outcomes of immunosuppressive therapy (IST) with those of T cell-replete haploidentical donor hematopoietic stem cell transplantation (haplo-HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 49 patients with SAA who received frontline IST (n = 29) or frontline haplo-HSCT (n = 20) between 2012 and 2016 were analyzed retrospectively. Fourteen patients responded after the first IST, and 1 patient responded after the second IST in the frontline IST group; 12 patients underwent salvage HSCT after IST failure. Sixteen of the 20 patients who underwent frontline haplo-HSCT survived without treatment failure. The 3-year overall survival of the frontline IST group was comparable to that of the frontline haplo-HSCT group (79.3 ± 7.5% versus 85.0 ± 8.0%; χ2 = 0.110; P = .740). The 3-year failure-free survival was lower in the frontline IST group compared with the frontline haplo-HSCT group (35.9 ± 10.9% versus 80.0 ± 8.9%; χ2 = 4.089; P = .043). Five patients of the IST group who underwent salvage HSCT achieved long survival without event. The event-free survival was lower in the salvage HSCT group compared with the haplo-HSCT group (41.7 ± 14.2% versus 80.0 ± 8.9%; χ2 = 3.992; P = .046), and the incidences of acute GVHD, grade II-IV acute GVHD, chronic GVHD, and severe infection were comparable between the 2 groups. Our results suggest that frontline haplo-HSCT may be a better treatment than IST for children and adolescents with SAA who lack an HLA age-matched familial donor.

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Authors+Show Affiliations

Institute of Hematology of Henan Provincial People's Hospital, Henan Provincial People's Hospital, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China.

Department of Hematology, People's Hospital of Zhengzhou University, Henan, China. Electronic address: zhuzm1964@163.com.

MeSH

AdolescentAnemia, AplasticChildGraft vs Host DiseaseHematopoietic Stem Cell TransplantationHumansImmunosuppressionRetrospective StudiesSalvage TherapySurvival AnalysisTransplantation, HaploidenticalTreatment Outcome

Pub Type(s)

Comparative Study

Journal Article

Language

eng

PubMed ID

30658223

Citation

Yang, Shiwei, et al. "Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children With Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 25, no. 5, 2019, pp. 975-980.

Yang S, Yuan X, Ma R, et al. Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor. Biol Blood Marrow Transplant. 2019;25(5):975-980.

Yang, S., Yuan, X., Ma, R., Jiang, L., Guo, J., Zang, Y., Shi, J., Yang, J., Lei, P., Liu, Z., Zhang, Y., & Zhu, Z. (2019). Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 25(5), 975-980. https://doi.org/10.1016/j.bbmt.2019.01.017

Yang S, et al. Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children With Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor. Biol Blood Marrow Transplant. 2019;25(5):975-980. PubMed PMID: 30658223.

* Article titles in AMA citation format should be in sentence-case

TY - JOUR T1 - Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor. AU - Yang,Shiwei, AU - Yuan,Xiaoli, AU - Ma,Rongjun, AU - Jiang,Li, AU - Guo,Jianmin, AU - Zang,Yuzhu, AU - Shi,Jie, AU - Yang,Jing, AU - Lei,Pingchong, AU - Liu,Zhongwen, AU - Zhang,Yin, AU - Zhu,Zunmin, Y1 - 2019/01/15/ PY - 2018/10/10/received PY - 2019/01/09/accepted PY - 2019/1/19/pubmed PY - 2020/3/21/medline PY - 2019/1/19/entrez KW - Haploidentical transplantation KW - Hematopoietic stem cell KW - IST KW - Severe aplastic anemia SP - 975 EP - 980 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol. Blood Marrow Transplant. VL - 25 IS - 5 N2 - We compared the outcomes of immunosuppressive therapy (IST) with those of T cell-replete haploidentical donor hematopoietic stem cell transplantation (haplo-HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 49 patients with SAA who received frontline IST (n = 29) or frontline haplo-HSCT (n = 20) between 2012 and 2016 were analyzed retrospectively. Fourteen patients responded after the first IST, and 1 patient responded after the second IST in the frontline IST group; 12 patients underwent salvage HSCT after IST failure. Sixteen of the 20 patients who underwent frontline haplo-HSCT survived without treatment failure. The 3-year overall survival of the frontline IST group was comparable to that of the frontline haplo-HSCT group (79.3 ± 7.5% versus 85.0 ± 8.0%; χ2 = 0.110; P = .740). The 3-year failure-free survival was lower in the frontline IST group compared with the frontline haplo-HSCT group (35.9 ± 10.9% versus 80.0 ± 8.9%; χ2 = 4.089; P = .043). Five patients of the IST group who underwent salvage HSCT achieved long survival without event. The event-free survival was lower in the salvage HSCT group compared with the haplo-HSCT group (41.7 ± 14.2% versus 80.0 ± 8.9%; χ2 = 3.992; P = .046), and the incidences of acute GVHD, grade II-IV acute GVHD, chronic GVHD, and severe infection were comparable between the 2 groups. Our results suggest that frontline haplo-HSCT may be a better treatment than IST for children and adolescents with SAA who lack an HLA age-matched familial donor. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/30658223/Comparison_of_Outcomes_of_Frontline_Immunosuppressive_Therapy_and_Frontline_Haploidentical_Hematopoietic_Stem_Cell_Transplantation_for_Children_with_Severe_Aplastic_Anemia_Who_Lack_an_HLA_Matched_Sibling_Donor_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(19)30066-7 DB - PRIME DP - Unbound Medicine ER -