Woods Deborah, Hayashi Robert J, Binkley Michael M, Sparks Gianna W, Hulbert Monica L
Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri.
Department of Neurology, Washington University School of Medicine, St. Louis, Missouri.
Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26635. Epub 2017 May 19.
Children and adolescents with sickle cell disease (SCD) are at high risk of strokes and are frequently treated with red blood cell (RBC) transfusions. The goal is to suppress hemoglobin (Hb) S while minimizing transfusion-induced iron overload. RBCs may be given via simple transfusion, manual exchange transfusion (MET), or erythrocytapheresis (aRBCX). Chronic transfusion practices vary among institutions.
This single-institution, retrospective cohort study compares Hb S control and therapy complication rates between MET and aRBCX in a cohort of children and adolescents with SCD and stroke during a 5-year period from 2008 through 2012. Duration and mode of transfusion therapy, achievement of Hb S suppression goal, iron burden by ferritin levels, and catheter complications were evaluated.
Thirty-seven children were included in analysis. The prevalence of catheter complications was 75% in aRBCX recipients compared with 0% in MET recipients (P < 0.001). There was no significant difference between modalities in achieving Hb S suppression or ferritin goals, but those receiving aRBCX had a greater likelihood of discontinuing chelation therapy. Among aRBCX recipients, adherence to >90% of transfusion appointments was associated with achieving Hb S suppression goals.
aRBCX may have increased complication risks compared with MET for chronic transfusion therapy in SCD. Risks and benefits of aRBCX and MET should be considered when selecting a chronic transfusion modality. Transfusion therapy modalities should be compared in prospective studies for stroke prevention in children with SCD.
患有镰状细胞病（SCD）的儿童和青少年中风风险很高，经常接受红细胞（RBC）输血治疗。目标是抑制血红蛋白（Hb）S，同时尽量减少输血引起的铁过载。红细胞可通过简单输血、手工换血输血（MET）或红细胞单采术（aRBCX）进行输注。不同机构的慢性输血方案各不相同。
这项单机构回顾性队列研究比较了2008年至2012年期间一组患有SCD和中风的儿童和青少年中，MET和aRBCX之间的Hb S控制情况和治疗并发症发生率。评估了输血治疗的持续时间和方式、Hb S抑制目标的实现情况、铁蛋白水平反映的铁负荷以及导管并发症。
37名儿童纳入分析。接受aRBCX治疗的患者导管并发症发生率为75%，而接受MET治疗的患者为0%（P<0.001）。在实现Hb S抑制或铁蛋白目标方面，两种方式之间没有显著差异，但接受aRBCX治疗的患者更有可能停止螯合疗法。在接受aRBCX治疗的患者中，遵守超过90%的输血预约与实现Hb S抑制目标相关。
对于SCD的慢性输血治疗，与MET相比，aRBCX可能增加并发症风险。选择慢性输血方式时应考虑aRBCX和MET的风险与益处。应在前瞻性研究中比较输血治疗方式对SCD儿童中风的预防效果。
