Department of General Pediatrics, University Children's Hospital, Heidelberg, Germany.
Cell Transplant. 2010;19(5):629-38. doi: 10.3727/096368909X485058. Epub 2010 Jan 6.
Despite recent advances and promising results in children, liver cell transplantation (LCT) should still be regarded as an experimental therapy. Several substantial complications are known from animal studies and individual patients. However, safety data on liver cell infusion in children are scarce. We used LCT in four children of different ages (3 weeks to 11 years, 3-40 kg) and underlying diseases [acute liver failure (n = 1), urea cycle disorders (n = 2), and Crigler-Najjar syndrome (n = 1)]. Vital parameters, portal vein flow (PVF), portal vein pressure (PVP), and liver enzymes were measured every 5 min during cell application and hourly thereafter between applications. An application protocol with discontinuation rules depending on changes in PVF and PVP was developed and successfully applied. Application was feasible in all children despite the catastrophic overall condition of the patient with acute liver failure. No application-related changes in vital parameters were found, and none of the children experienced clinical signs of portal vein thrombosis, pulmonary embolism, or anaphylactic reactions. Time courses for changes in PVF, PVP, and liver enzymes were obtained. Thorough monitoring of portal vein pressure and duplex sonography according to a defined protocol is likely to increase safety of cell application in pediatric LCT.
尽管儿童肝细 胞移植(LCT)在近期取得了进展并取得了令人鼓舞的结果,但它仍应被视为一种实验性疗法。动物研究和个别患者都有一些严重的并发症。然而,儿童肝细 胞输注的安全性数据却很少。我们在四个不同年龄(3 周至 11 岁,3-40 公斤)和不同基础疾病(急性肝衰竭 1 例、尿素循环障碍 2 例、Crigler-Najjar 综合征 1 例)的儿童中使用了 LCT。在细胞应用过程中,每 5 分钟测量一次生命体征、门静脉流量(PVF)、门静脉压力(PVP)和肝酶,此后每小时测量一次。制定了一个应用方案,并根据 PVF 和 PVP 的变化制定了停止规则,并成功应用。尽管患有急性肝衰竭的患者病情严重,但所有儿童均能顺利应用。未发现生命体征与应用相关的变化,也未发现任何儿童出现门静脉血栓形成、肺栓塞或过敏反应的临床症状。还获得了 PVF、PVP 和肝酶变化的时间过程。根据既定方案对门静脉压力进行彻底监测和双重超声检查可能会提高儿科 LCT 中细胞应用的安全性。