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Haploidentical Transplantation with Modified Post-transplantation Cyclophosphamide for Patients with Primary Aplastic Anemia: A Multicenter Experience

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单位: [1]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Hematol,1095 Jie Fang Ave,Wuhan 430030,Hubei,Peoples R China [2]Wuhan 1 Hosp, Dept Hematol, Wuhan, Hubei, Peoples R China [3]Yichang Cent Peoples Hosp, Dept Hematol, Yichang, Hubei, Peoples R China [4]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Inst Organ Transplantat,Wuhan,Hubei,Peoples R China
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关键词: Haploidentical Transplantation Aplastic anemia Post-transplantation cyclophosphamide GVHD prophylaxis

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Aplastic anemia (AA) is a life-threatening hematological disorder that can be cured by hematopoietic stem cell transplantation. Haploidentical transplantation becomes an alternative choice for patients in the absence of a matched sibling donor. We used a retrospective study aimed to confirm the feasibility of busulfan-based modified post-transplantation cyclophosphamide (PTCY) strategy in haploidentical hematopoietic stem cell transplantation for AA patients. We analyzed the outcomes of 27 patients from 3 clinical centers who had undergone haploidentical transplantation between October 2018 and July 2020. The modified condition regimen consisted of anti-thymoglobulin/antilymphocyte globulin, fludarabine, busulfan and low-dose cyclophosphamide, and high-dose cyclophosphamide, mycophenolate mofetil (MMF) and tacrolimus were administered as graft versus host disease (GVHD) prophylaxis after transplantation. The median follow-up time was 370 (range 65-721) days. One patient developed primary graft failure, and successful engraftment was observed in 96.29% (95% confidence interval [CI], 93.45%-97.91%) of patients. The median times for neutrophil and platelet engraftment were 13 (range 11-18) days and 13 (range 11-28) days, respectively. The most common regimen-related toxicity was bladder toxicity, followed by stomatitis and gastrointestinal toxicity. The cumulative incidence of grade II-IV aGVHD was 25.93% (95% CI, 5.84%-52.64%), whereas the cumulative incidence of grade III-IV aGVHD was 7.4% (95% CI, 0%-52.16%). Chronic GVHD was observed in 3 patients by the end of follow-up. All 27 patients are alive, with a failure-free survival rate of 96.30% (95% CI, 6.49%-99.47%) and GVHD relapse-free survival rate of 88.89% (95% CI, 69.39%-96.28%). Virus reactivation was comparable, with rates of 53.54% for cytomegalovirus (CMV) reactivation and 41.57% for Epstein-Barr virus, but the CMV diseases and post-transplantation lymphoproliferative disorder were rare. Our study using haploidentical transplantation with modified PTCY demonstrated an encouraging result with prolonged survival and reduced complications for aplastic anemia patients. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.

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大类 | 3 区 医学
小类 | 3 区 血液学 3 区 免疫学 3 区 移植
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Q1 TRANSPLANTATION Q2 HEMATOLOGY Q2 IMMUNOLOGY

影响因子: 最新[2023版] 最新五年平均 出版当年[2019版] 出版当年五年平均 出版前一年[2018版]

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第一作者单位: [1]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Hematol,1095 Jie Fang Ave,Wuhan 430030,Hubei,Peoples R China
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通讯机构: [1]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Hematol,1095 Jie Fang Ave,Wuhan 430030,Hubei,Peoples R China [4]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Inst Organ Transplantat,Wuhan,Hubei,Peoples R China
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相关文献

[1]Post-transplantation cyclophosphamide as GVHD prophylaxis in allogenic hematopoietic stem cell transplantation: Recent advances and modification [2]Comparisons of Modified Post-Transplantation Cyclophosphamide and Granulocyte Colony-Stimulating Factor/Antithymocyte Globulin Regimens for Haploidentical Stem Cell Transplantation in Patients with Aplastic Anemia [3]Recombinant Human Thrombopoietin Promotes Platelet Engraftment in Severe Aplastic Anemia Patients following Treatment with Haploid Hematopoietic Stem Cell Transplantation using Modified Post-transplantation Cyclophosphamide [4]Comparisons Between modified PTCY and G-CSF/ATG Regimens for Haploidentical Transplantation in Patients with Aplastic Anemia. [5]Comparison of haploidentical-allogeneic hematopoietic stem cell transplantation and intensive immunosuppressive therapy for patients with severe aplastic anemia with an absolute neutrophil count of zero: a retrospective study [6]Interim Results of a Multi-Center Observational Study of Current Treatment Patterns for Patients with Severe Aplastic Anemia (SAA), Very Severe Aplastic Anemia (VSAA) and Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) in China [7]Genetic characterization of acquired aplastic anemia by targeted sequencing [8]Unrelated donor hematopoietic stem cell transplantation compared to immunosuppressive therapy plus eltrombopag as first-line treatment for adults with severe aplastic anemia [9]The outcome and predictive model of allogeneic hematopoietic stem cell transplantation among elderly patients with severe aplastic anemia from the Chinese Blood and Marrow Transplant Registry Group [10]Real-World Effectiveness and Safety of Eltrombopag in Patients with Aplastic Anemia: A Chinese Nationwide Survey

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