Long-term outcomes of 172 children with severe aplastic anemia treated with rabbit antithymocyte globulin and cyclospori
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ORIGINAL ARTICLE
Long-term outcomes of 172 children with severe aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine Yang Lan 1 & Lixian Chang 1 & Meihui Yi 1 & Yuli Cai 1 & Jing Feng 1 & Yuanyuan Ren 1 & Chao Liu 1 & Xiaoyan Chen 1 & Shuchun Wang 1 & Ye Guo 1 & Aoli Zhang 1 & Lipeng Liu 1 & Jingliao Zhang 1 & Xiaofan Zhu 1 Received: 27 May 2020 / Accepted: 3 October 2020 # Springer-Verlag GmbH Germany, part of Springer Nature 2020
Abstract This study retrospectively analyzed the clinical outcome of 172 children with newly diagnosed severe aplastic anemia (SAA) between January 2008 and April 2018, who received rabbit antithymocyte globulin (ATG) and cyclosporine (CsA) as first-line treatment. The median age at diagnosis was 5 years (range, 1–14). The overall response rates were 22.7%, 45.3%, and 61% at 40 days, 3 months, and 6 months, respectively, after rabbit ATG. In multivariate analysis, mild disease severity was the only predictor of favorable response at 6 months (P = 0.006). In the present study, median follow-up period was 63 months (range, 1–135). The 5-year overall survival (OS) and failure-free survival (FFS) rates were 90.5% and 70.4%. Multivariate analysis showed that erythroid burst-forming units (BFU-E) > 2/105 bone marrow mononuclear cell (BMMNC) (P = 0.037) and time interval before IST ≤ 30 days (P = 0.017) were independent positive predictors for OS, meanwhile BFU-E > 2/105BMMNC (P = 0.029) was the only favorable prognostic factor for FFS. Keywords Antithymocyte globulin . Child . Immunosuppressive therapy . Predictor . Severe aplastic anemia
Abbreviations IST ATG CsA SAA HLA vSAA OS FFS BFU-E BMMNC PB BM
Immunosuppressive therapy Antithymocyte globulin Cyclosporine Severe aplastic anemia Human leukocyte antigen Very severe aplastic anemia Overall survival Failure-free survival Erythroid burst-forming units Bone marrow mononuclear cell Peripheral blood Bone marrow
Electronic supplementary material The online version of this article (https://doi.org/10.1007/s00277-020-04296-9) contains supplementary material, which is available to authorized users. * Xiaofan Zhu [email protected] 1
Division of Pediatric Blood Diseases Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, 288 Nanjing Road, Heping District, Tianjin 300020, China
aAA HSCs HSCT ALG AA ANC PLT ARC CR HGB NR PR IMDM FBS PNH CFU-GM AML MDS CFU-E NIH WBC ALC CFU-GEMM
Acquired aplastic anemia Hematopoietic stem cells Hematopoietic stem cell transplantation Antilymphocyte globulin Aplastic anemia Absolute neutrophil count Platelet Absolute reticulocyte count Complete response Hemoglobin Nonresponse Partial response Iscove’s Modified Dulbecco’s Medium Fetal bovine serum Paroxysmal nocturnal hemoglobinuria Granulocyte-macrophage colony-forming units Acute myelogenous leukemia Myelodysplastic syndrome Erythroid colony-forming units National
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