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首页> 美国卫生研究院文献>Children >Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia
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Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

机译：成功的造血干细胞移植与β-地中海贫血患者为严重的血糖贫血

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摘要

The first-line treatment for severe aplastic anemia (SAA) patients is hematopoietic stem cell transplantation (HSCT), with full-matched related donors considered the most suitable. We report a case of SAA in which the patient successfully underwent HSCT from a donor with β-thalassemia minor. The patient in this case underwent HSCT from a human leukocyte antigen (HLA)-matched younger brother with β-thalassemia minor. A 7-year-old girl was referred to our facility following a 6-month history of easy bruising and pallor. Laboratory examinations showed pancytopenia and hypocellular bone marrow with cellularity of <5%. She was diagnosed with acquired SAA, and HLA typing of her family members was performed. Her younger brother was an HLA-matched sibling but had β-thalassemia minor. Since his hemoglobin levels were maintained at 10–11 d/dL, he was considered a suitable HSCT donor. The conditioning regimen included fludarabine, cyclophosphamide, and anti-thymocyte globulin. The CD34+ and CD3+ cell counts were 6.6 × 106/kg and 0.48 × 108/kg, respectively. White blood cell engraftment was evident on day +11. Regimen-associated toxicities, such as anorexia and enteritis, were mild; no infections occurred, and no symptoms of acute graft-versus-host disease (GVHD) were observed. The 30-day follow-up bone marrow examination revealed normocellular marrow with 80%–90% cellularity. Acute or chronic GVHD has not been reported, and good performance status has been observed throughout the 5 years after HSCT. β-thalassemia minor patients can be considered as bone marrow donors for SAA patients.
机译：严重血栓性贫血（SAA）患者的第一线治疗是造血干细胞移植（HSCT），具有全匹配的相关捐赠者认为最适合。我们举报了SAA的情况，其中患者从β-地中海贫血症的供体中成功接受了HSCT。在这种情况下，患者从人白细胞抗原（HLA）的患者接受了HSCT - β-Thalassemia未成年人。一个7岁的女孩在一个易瘀伤和苍白的历史6个月的历史之后被提交给我们的设施。实验室考试显示PancyTopenia和细胞间骨髓，细胞性<5％。她被诊断出患有收购的SAA，并进行了HLA的键入她的家庭成员。她的弟弟是一个匹配的兄弟姐妹，但有β-thalassemia未成年人。由于他的血红蛋白水平保持在10-11d / dl，因此他被认为是合适的HSCT供体。调理方案包括氟酰胺，环磷酰胺和抗胸腺细胞球蛋白。 CD34 +和CD3 +细胞计数分别为6.6×106 / kg和0.48×108 / kg。白细胞植入在第+11天是明显的。方案相关的毒性，如厌食症和肠炎，温和;没有发生感染，并且没有观察到急性移植物与宿主疾病（GVHD）的症状。 30天的后续骨髓检查显示常压骨髓，具有80％-90％的细胞性。尚未报告急性或慢性GVHD，并在HSCT后5年后观察到良好的性能状态。 β-血症症次要患者可作为SAA患者的骨髓供体。

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