切换至 "中华医学电子期刊资源库"

中华细胞与干细胞杂志(电子版) ›› 2019, Vol. 09 ›› Issue (04) : 242 -246. doi: 10.3877/cma.j.issn.2095-1221.2019.04.009

所属专题: 文献

综述

脐带血干细胞移植治疗非恶性血液病进展
王苹1, 翟晓文1,()   
  1. 1. 201102 上海,复旦大学附属儿科医院血液科
  • 收稿日期:2019-06-21 出版日期:2019-08-01
  • 通信作者: 翟晓文

Umbilical cord blood stem cell transplantation for non-malignant hematological diseases

Ping Wang1, Xiaowen Zhai1,()   

  1. 1. Department of Hematology/oncology, Children's Hospital of Fudan University, Shanghai 201102, China
  • Received:2019-06-21 Published:2019-08-01
  • Corresponding author: Xiaowen Zhai
  • About author:
    Corresponding author: Zhai Xiaowen, Email:
引用本文:

王苹, 翟晓文. 脐带血干细胞移植治疗非恶性血液病进展[J/OL]. 中华细胞与干细胞杂志(电子版), 2019, 09(04): 242-246.

Ping Wang, Xiaowen Zhai. Umbilical cord blood stem cell transplantation for non-malignant hematological diseases[J/OL]. Chinese Journal of Cell and Stem Cell(Electronic Edition), 2019, 09(04): 242-246.

异基因造血干细胞移植(allo-HSCT)是治愈多种非恶性病的有效方法。脐带血干细胞(UCB)具有免疫原性低、人类白细胞抗原不合耐受性好、移植物抗宿主反应发生率低以及获取相对快捷等特点,可作为非恶性血液疾病患者allo-HSCT的来源。本文简要综述脐血干细胞移植在原发性免疫缺陷病、遗传性骨髓衰竭、遗传代谢病以及自身免疫性疾病等非恶性血液疾病的治疗效果。

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective method to cure non-malignant hematological diseases. Umbilical cord blood stem cells (UCB) are characterized by weak immunogenicity, good tolerance human leukocyte antigen (HLA) mismatch, low incidence of graft-versus-host disease and relatively fast availability. UCB can be used as the graft of allo-HSCT for patients with non-malignant hematological diseases. This article reviews the efficacy of the umbilical cord blood stem cell transplantation (UCBT) in patients with primary immunodeficiency, hereditary bone marrow failure, hereditary metabolic diseases and autoimmune diseases.

表1 UCBT治疗非恶性血液疾病疗效总结
1
Ballen KK, Gluckman E, Broxmeyer HE. Umbilical cord blood transplantation: the first 25 years and beyond[J]. Blood, 2013, 122(4): 491-498.
2
Forman SJ, Negrin RS, Antin JH, et al. Thomas' hematopoietic cell transplantation: Stem cell transplantation, fifth edition[M]. Oxford, England: John Wiley & Sons, Ltd, 2015:437-455.
3
杨曦, 赵晓东. 原发性免疫缺陷病分类(2017伦敦版)解读[J]. 中华儿科杂志, 2018, 56(9):648-650.
4
Pai SY, Logan BR, Griffith LM, et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009[J]. N Engl J Med, 2014, 371(5):434-446.
5
Gaspar HB, Qasim W, Davies EG, et al. How I treat severe combined immunodeficiency[J]. Blood, 2013, 122(23):3749-3758.
6
Fernandes JF, Rocha V, Labopin M, et al. Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?[J]. Blood, 2012, 119(12):2949-2955.
7
Morio T, Atsuta Y, Tomizawa D, et al. Outcome of unrelated umbilical cord blood transplantation in 88 patients with primary immunodeficiency in Japan[J]. Br J Haematol, 2011, 154(3):363-372.
8
Díaz De Heredia C, Ortega JJ, Díaz MA, et al. Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies[J]. Bone Marrow Transplant, 2008, 41(7):627-633.
9
Cole T, Pearce MS, Cant AJ, et al. Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation[J]. J Allergy Clin Immunol, 2013, 132(5):1150-1155.
10
Tewari P, Martin PL, Mendizabal A, et al. Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high Long-Term donor chimerism and excellent survival in chronic granulomatous disease[J]. Biol Blood Marrow Transplant, 2012, 18(9):1368-1377.
11
Gozdzik J, Pituch-Noworolska A, Skoczen SA, et al. Allogeneic haematopoietic stem cell transplantation as therapy for chronic granulomatous Disease-Single centre experience[J]. J Clin Immunol, 2011, 31(3):332-337.
12
Jaing TH, Lee WI, Cheng PJ, et al. Successful unrelated donor cord blood transplantation for chronic granulomatous disease[J]. Int J Hematol, 2010, 91(4):670-672.
13
Lin CJ, Wang SC, Ku CL, et al. Successful unrelated cord blood stem cell transplantation in an x-linked chronic granulomatous disease patient with disseminated BCG-induced infection[J]. Pediatr Neonatol, 2015, 56(5):346-350.
14
Soncini E, Slatter MA, Jones LB, et al. Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth[J]. Br J Haematol, 2009, 145(1):73-83.
15
Al-Dhekri H, Al-Mousa H, Ayas M, et al. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type 1: a single center experience[J]. Biol Blood Marrow Transplant, 2011, 17(8):1245-1249.
16
Sakaguchi H, Nakanishi K, Kojima S. Inherited bone marrow failure syndromes in 2012[J]. Int J Hematol, 2013, 97(1):20-29.
17
Macmillan ML, Wagner JE. Haematopoeitic cell transplantation for Fanconi anaemia-when and how?[J]. Br J Haematol, 2010, 149(1):14-21.
18
de la Fuente J, Dokal I. Dyskeratosis congenita:advances in the understanding of the telomerase defect and the role of stem cell transplantation[J]. Pediatr Transplant, 2007, 11(6):584-594.
19
Dietz AC, Orchard PJ, Baker KS, et al. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita[J]. Bone Marrow Transplant, 2011, 46(1):98-104.
20
De Latour RP. Transplantation for bone marrow failure: current issues[J]. Hematology Am Soc Hematol Educ Program, 2016 (1):90-98.
21
Broxmeyer HE. Enhancing the efficacy of engraftment of cord blood for hematopoietic cell transplantation[J]. Transfus Apher Sci, 2016, 54(3):364-372.
22
Gluckman E, Rocha V, Ionescu I, et al. Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival[J]. Biol Blood Marrow Transplant, 2007, 13(9):1073-1082.
23
Buchbinder D, Hsieh L, Mahajerin A, et al. Successful treatment of secondary graft failure following unrelated cord blood transplant with hematopoietic growth factors in a pediatric patient with Fanconi anemia[J]. Pediatr Transplant, 2015, 19(7):E181-E184.
24
Jaing TH, Chen SH, Yang CP, et al. Successful hematopoietic reconstitution by unrelated donor cord blood transplantation in children with fanconi anemia: report of 3 cases[J]. J Pediatr Hematol Oncol, 2014, 36(8):E553-E555.
25
Prasad VK, Kurtzberg J. Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions[J]. Br J Haematol, 2010, 148(3):356-372.
26
Prasad VK, Kurtzberg J. Transplant outcomes in mucopolysaccharidoses[J]. Semin Hematol, 2010, 47(1):59-69.
27
Boelens JJ, Aldenhoven M, Purtill D, et al. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning[J]. Blood, 2013, 121(19):3981-3987.
28
Boelens JJ, Rocha V, Aldenhoven M, et al. Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome[J]. Biol Blood Marrow Transplant, 2009, 15(5):618-625.
29
Aldenhoven M, Jones SA, Bonney D, et al. Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines[J]. Biol Blood Marrow Transplant, 2015, 21(6):1106-1109.
30
Boelens JJ, Wynn RF, O'Meara A, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe:a risk factor analysis for graft failure[J]. Bone Marrow Transplant, 2007, 40(3):225-233.
31
Miller WP, Rothman SM, Nascene D, et al. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report[J]. Blood, 2011, 118(7):1971-1978.
32
Prasad VK, Mendizabal A, Parikh SH, et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes[J]. Blood, 2008, 112(7):2979-2989.
33
Van Den Broek B, Page K, Paviglianiti A, et al. Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies[J]. Blood Adv, 2018, 2(1):49-60.
34
Engelen M, Kemp S, Poll-The BT. X-linked adrenoleukodystrophy: pathogenesis and treatment[J]. Curr Neurol Neurosci Rep, 2014, 14(10):486.
35
Beltran-Quintero ML, Bascou NA, Poe MD, et al. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months[J]. Orphanet J Rare Dis, 2019, 14(1):46.
36
Escolar ML, Poe MD, Provenzale JM, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease[J]. N Engl J Med, 2005, 352(20):2069-2081.
37
Anna, Monica, Bianco, et al. Genetics of inflammatory bowel disease from multifactorial to monogenic forms[J]. World J Gastroenterol, 2015, 21(43):12296-12310.
38
Kotlarz D, Beier R, Murugan D, et al. Loss of interleukin-10 signaling and infantile inflammatory bowel disease: implications for diagnosis and therapy[J]. Gastroenterology, 2012, 143(2):347-355.
39
Engelhardt KR, Shah N, Faizura-Yeop I, et al. Clinical outcome in IL-10-and IL-10 receptor–deficient patients with or without hematopoietic stem cell transplantation[J]. J Allergy Clin Immunol, 2013, 131(3):825-830.
40
Pigneur B, Escher J, Elawad M, et al. Phenotypic characterization of very early-onset IBD due to mutations in the IL10, IL10 receptor alpha or beta gene: a survey of the GENIUS working group[J]. Inflamm Bowel Dis, 2013, 19(13):2820-2828.
41
Huang Z, Peng K, Li X, et al. Mutations in interleukin-10 receptor and clinical phenotypes in patients with very early onset inflammatory bowel disease[J]. I Inflamm Bowel Dis, 2017, 23(4):578-590
[1] 周容, 张亚萍, 廖宇, 程晓萍, 管玉龙, 潘广玉, 闫杰, 王贤芝, 苟中山, 潘登科, 李巅远. 超声在基因编辑猪-猴异种并联式心脏移植术中的应用价值[J/OL]. 中华医学超声杂志(电子版), 2024, 21(06): 617-623.
[2] 谢雨彤, 蒋良福, 池征璘, 李志杰, 高伟阳, 闫合德. 手指皮肤脱套伤修复的研究进展[J/OL]. 中华损伤与修复杂志(电子版), 2024, 19(05): 440-442.
[3] 陈进宏. 腹腔镜活体供肝获取规范与创新[J/OL]. 中华普通外科学文献(电子版), 2024, 18(05): 324-324.
[4] 胡宁宁, 赵延荣, 王栋, 王胜亮, 郭源. FMNL3与肝细胞癌肝移植受者预后的相关性研究[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 283-288.
[5] 邹永康, 石雍, 徐贤刚, 张帅民, 刘衍, 杨生鹏, 叶啟发, 陈根, 张毅. 肾移植术后手术切口米根霉感染伴菌血症一例并文献复习[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 289-292.
[6] 仲福顺, 余露, 范晓礼, 叶啟发. 肝移植治疗肝上皮样血管内皮瘤一例[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 293-297.
[7] 刘冉佳, 崔向丽, 周效竹, 曲伟, 朱志军. 儿童肝移植受者健康相关生存质量评价的荟萃分析[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 302-309.
[8] 贺健, 张骊, 王洪海, 蒋文涛. 肝移植术后脾功能亢进转归及治疗研究进展[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 310-314.
[9] 郭倩男, 史嘉玮, 董念国. T细胞不同代谢方式在移植排斥反应中的研究进展[J/OL]. 中华移植杂志(电子版), 2024, 18(05): 315-320.
[10] 傅红兴, 王植楷, 谢贵林, 蔡娟娟, 杨威, 严盛. 间充质干细胞促进胰岛移植效果的研究进展[J/OL]. 中华细胞与干细胞杂志(电子版), 2024, 14(06): 351-360.
[11] 傅斌生, 冯啸, 杨卿, 曾凯宁, 姚嘉, 唐晖, 刘剑戎, 魏绪霞, 易慧敏, 易述红, 陈规划, 杨扬. 脂肪变性供肝在成人劈离式肝移植中的应用[J/OL]. 中华肝脏外科手术学电子杂志, 2024, 13(06): 789-794.
[12] 中华医学会器官移植学分会. 肝移植术后缺血性胆道病变诊断与治疗中国实践指南[J/OL]. 中华肝脏外科手术学电子杂志, 2024, 13(06): 739-748.
[13] 魏志鸿, 刘建勇, 吴小雅, 杨芳, 吕立志, 江艺, 蔡秋程. 肝移植术后急性移植物抗宿主病的诊治(附四例报告)[J/OL]. 中华肝脏外科手术学电子杂志, 2024, 13(06): 846-851.
[14] 中华医学会器官移植学分会, 中华医学会外科学分会外科手术学学组, 中华医学会外科学分会移植学组, 华南劈离式肝移植联盟. 劈离式供肝儿童肝移植中国临床操作指南[J/OL]. 中华肝脏外科手术学电子杂志, 2024, 13(05): 593-601.
[15] 刘军, 丘文静, 孙方昊, 李松盈, 易述红, 傅斌生, 杨扬, 罗慧. 在体与离体劈离式肝移植在儿童肝移植中的应用比较[J/OL]. 中华肝脏外科手术学电子杂志, 2024, 13(05): 688-693.
阅读次数
全文


摘要


AI


AI小编
你好!我是《中华医学电子期刊资源库》AI小编,有什么可以帮您的吗?