Eculizumab Bridging before Bone Marrow Transplant for Marrow Failure Disorders Is Safe and Does Not Limit Engraftment.
Eculizumab Bridging before Bone Marrow Transplant for Marrow Failure Disorders Is Safe and Does Not Limit Engraftment.
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DOI：
10.1016/j.bbmt.2018.07.032
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发表时间：
2018-12-01
期刊：
影响因子：
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通讯作者：
Brodsky, Robert A
中科院分区：
文献类型：
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作者：
DeZern, Amy E;Jones, Richard J;Brodsky, Robert A
阵发性夜间血红蛋白尿（PNH）通常继发于其他骨髓衰竭（BMF）疾病，尤其是再生障碍性贫血（AA）。患有AA/PNH综合征的患者可能需要eculizumab治疗，以减少血管内溶血和血栓形成的风险，并对严重的BMF进行异体干细胞移植。有人担心eculizumab可能会对这些患者的移植结果产生不利影响。这是一项回顾性的单中心研究，研究对象是在移植前立即接受eculizumab治疗的严重AA (SAA)/PNH患者。描述了植入和感染结果的指标。8例患有SAA/PNH和PNH相关症状的患者接受eculizumab治疗，然后进行移植。所有移植均成功，在调理前无与C5阻塞相关的不良事件。所有患者的PNH和SAA均已治愈。Eculizumab对于需要移植的PNH克隆患者是安全有效的。这有时需要在骨髓移植前“桥接”患者，即使使用HLA匹配的非亲属或单倍体相同的供体也不会对结果产生不利影响。
Paroxysmal nocturnal hemoglobinuria (PNH) often develops secondary to other bone marrow failure (BMF) disorders, especially aplastic anemia (AA). Patients with the AA/PNH syndrome may require treatment with both eculizumab to reduce intravascular hemolysis and the risk of thrombosis and allogeneic stem cell transplant for the severe BMF. There has been concern that eculizumab could adversely affect the outcomes for transplant in these patients. This is a retrospective, single-center study of severe AA (SAA)/PNH patients treated with eculizumab immediately before the start of conditioning for transplant. Metrics of engraftment and infectious outcomes are described. Eight patients with SAA/PNH and PNH-related symptoms were treated with eculizumab and then proceeded to transplant. All were successfully transplanted without adverse events related to C5 blockage before conditioning. All were also cured of their both PNH and SAA. Eculizumab is safe and efficacious in patients with PNH clones who require transplant. This is sometimes required to "bridge" patients before bone marrow transplantation and does not appear to adversely impact outcomes even when using HLA matched unrelated or haploidentical donors.