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首页> 外文期刊>British Journal of Haematology >Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT
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Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT

机译：特发性小儿再生障碍性贫血中前期无关和匹配的同胞干细胞移植的相似结果。代表英国儿科BMT工作组，儿科疾病工作组和EBMT重症再生障碍性贫血工作组进行的研究

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We explored the feasibility of unrelated donor haematopoietic stem cell transplant (HSCT) upfront without prior immunosuppressive therapy (IST) in paediatric idiopathic severe aplastic anaemia (SAA). This cohort was then compared to matched historical controls who had undergone first-line therapy with a matched sibling/family donor (MSD) HSCT (n=87) or IST with horse antithymocyte globulin and ciclosporin (n=58) or second-line therapy with unrelated donor HSCT post-failed IST (n=24). The 2-year overall survival in the upfront cohort was 96 +/- 4% compared to 91 +/- 3% in the MSD controls (P=0.30) and 94 +/- 3% in the IST controls (P=0.68) and 74 +/- 9% in the unrelated donor HSCT post-IST failure controls (P=0.02).The 2-year event-free survival in the upfront cohort was 92 +/- 5% compared to 87 +/- 4% in MSD controls (P=0.37), 40 +/- 7% in IST controls (P=0.0001) and 74 +/- 9% in the unrelated donor HSCT post-IST failure controls (n=24) (P=0.02). Outcomes for upfront-unrelated donor HSCT in paediatric idiopathic SAA were similar to MSD HSCT and superior to IST and unrelated donor HSCT post-IST failure. Front-line therapy with matched unrelated donor HSCT is a novel treatment approach and could be considered as first-line therapy in selected paediatric patients who lack a MSD.
机译：我们探讨了在不进行事先免疫抑制治疗（IST）的情况下，不相关供体造血干细胞移植（HSCT）在小儿特发性再生障碍性贫血（SAA）中的可行性。然后将该队列与匹配的历史对照进行比较，这些对照曾接受过一线治疗并接受了兄弟姐妹/家庭供体（MSD）HSCT（n = 87）或IST接受了马抗胸腺细胞球蛋白和环孢素（n = 58）或二线治疗不相关的捐助者HSCT失败后的IST（n = 24）。前期队列的2年总生存率为96 +/- 4％，而MSD对照者为91 +/- 3％（P = 0.30）和IST对照者为94 +/- 3％（P = 0.68）和无关的供体HSCT IST失败后对照中的74 +/- 9％（P = 0.02）。前期队列的2年无事件生存率为92 +/- 5％，而87 +/- 4％在MSD对照中（P = 0.37），在IST对照中为40 +/- 7％（P = 0.0001），在无关的供体HSCT IST失败后对照中为74 +/- 9％（n = 24）（P = 0.02） 。小儿特发性SAA中前期无关供体HSCT的结果与MSD HSCT相似，优于IST和IST后失败的无关供体HSCT。匹配的无关供体HSCT的一线治疗是一种新颖的治疗方法，可以被视为缺乏MSD的部分儿科患者的一线治疗。

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