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单倍体亲缘异基因造血干细胞移植治疗SAA 1例

The haploid genetic allogeneic hematopoietic stem cell transplantation for severe aplastic anemia: one case report

来源期刊: 广州医药 | 28-30 发布时间:2021-11-30 收稿时间:2025/11/13 17:00:28 阅读量:31
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关键词:
再生障碍性贫血重型造血干细胞移植单倍体亲缘供者
Aplastic anemiaSevereHematopoietic stem cell transplantationHaploid Related donors
DOI:
10.3969/j.issn.1000-8535.2016.03.010
收稿时间:
2016-01-06 
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引用总数:
0  
目的 探讨单倍体亲缘异基因造血干细胞移植治疗重型再生障碍性贫血(SAA)的可行性。方法 对1例诊断SAA 4年余,先后经CsA治疗、脐血移植治疗均无效并反复输注红细胞、血小板的12岁男性患者进行单倍体亲缘异基因造血干细胞移植,供者为其胞兄,高分辨HLA基因型5/10相合,预处理方案为BU+CTX+ATG:BU 3.2 mg/kg×2 d,CTX 50 mg/kg×4 d,ATG 2.5 mg/kg×4 d。干细胞来源为G-CSF动员的骨髓+外周造血干细胞,共计输注单个核细胞(MNC)4.055×108/kg(受者体重),CD 34 2.331×106/kg。GVHD预防:-1 d采用与受者HLA部分相合的第三方脐带血细胞,术后联合应用环孢素A、短程氨甲碟呤、霉酚酸酯。结果 造血缓慢重建,术后22天(+22 d)ANC>0.5×109/L,术后3月血小板脱离输注。+26天DNA指纹图全部表现为供者基因型。+40天血型转为供者型“O”型。+29 d出现急性移植物抗宿主病aGVHD(胃肠型,Ⅲ度),+31 d、+34 d及+42 d予巴利昔单抗20 mg静滴,+40 d、+44 d、+63 d输注间充质干细胞,患者急性GVHD逐渐控制。期间曾出现肺部感染、口腔黏膜炎及巨细胞病毒血症,经抗感染后可控制。现随访3年,血象正常稳定,Kamofsky评分100分。结论 单倍体亲缘异基因造血干细胞移植治疗SAA,对无相合供者(包括亲缘或非亲缘)且强效免疫抑制治疗失败的患者,可考虑进行,GVHD和感染为主要并发症,需根据患者病情采用相应措施。
Objective To investigate the feasibility of haploid genetic allogeneic hematopoietic stem cell transplantation in the treatment of severe aplastic anemia(SAA) in our hospital. Methods A 12-year-old patient with acquired SAA for 4 years showed no response to CsA and cord blood transplant treatment and was transfusion-dependent. Lacking an HLA-identical sibling donor, the patient was treated with HSCT from his brother 5/10 matched at the generic level. Theconditioning regimen was BU+CTX+ATG:BU 3.2 mg/kg×2 d,CTX 50 mg/kg×4 d,ATG 2.5 mg/kg×4 d. Stem cells were the source of G-CSF mobilization of bone marrow and peripheral blood stem cells, dose of stem cells infused: mononuclear cells (MNC) 4.055×108/kg (body weight of subject), CD34 2.331×106/kg. Prevention of GVHD: -1 d Third-party umbilical cord blood cells which were HLA partially matched were used. Postoperative joint use included cyclosporine A, short-course methotrexate, mycophenolate mofetil. Results Hematopoiesis was slowly rebuilding, 22 d after surgery (+22 d) ANC> 0.5×109/L, after three months departing from transfusion of platelets. +26 d suggesting that the DNA fingerprints showed donor genotypes. +40 d into donor blood type “O” type. + 29 d occurred acute GVHD (GI type, Ⅲ degrees), + 31 d, + 34 d + 42 d infusion of basiliximab 20mg, + 40 d, + 44 d, + 63 d infusion of mesenchymal stem cells. Gradually acute GVHD was controlled in the patient, who had lung infections, oral mucositis and cytomegalovirus viremia, could be controlled with anti-infective. Now followed up for 3 years, hemogram change has been normal and stable. Kamofsky score was 100 points. Conclusion It may be considered to have haploid genetic allogeneic hematopoietic stem cell transplantation for treatment of SAA, for those patients who have non-matched donor (including relatives and non-relatives) and potent immunosuppressive therapy failure. GVHD and infection are major complications. Need to adopt appropriate measures in accordance with the patient's condition.
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