Paediatric Gastroenterology and Hepatology, University Children's Hospital Tübingen, Tübingen, Germany.
SSD Paediatric Gastroenterology, Ospedale Infantile Regina Margherita, Torino, Italy.
J Pediatr Gastroenterol Nutr. 2024 Jun;78(6):1364-1373. doi: 10.1002/jpn3.12211. Epub 2024 Apr 16.
Paediatric acute liver failure (PALF) is a life-threatening disease. Management aims to support hepatic regeneration or to bridge to liver transplantation. High-volume plasmapheresis (HVP) removes protein-bound substances, alleviates inflammation, and improves survival in adult acute liver failure. However, experience with HVP in PALF is limited. Aim of this study is to report on feasibility, safety, efficacy and outcomes of HVP in PALF.
Retrospective observational study in children with PALF. HVP was performed upon identification of negative prognostic indicators, in toxic aetiology or multiorgan failure (MOF). Exchanged volume with fresh-frozen plasma corresponded to 1.5-2.0 times the patient's estimated plasma volume. One daily cycle was performed until the patient met criteria for discontinuation, that is, liver regeneration, liver transplantation, or death.
Twenty-two children with PALF (body weight 2.5-106 kg) received 1-7 HVP cycles. No bleeding or procedure-related mortality occurred. Alkalosis, hypothermia and reduction in platelets were observed. Haemolysis led to HVP termination in one infant. Seven children (32%) survived with their native livers, 13 patients (59%) underwent liver transplantation. Two infants died due to MOF. Overall survival was 86%. International normalization ratio (INR), alanine aminotransaminases (ALT), bilirubin and inotropic support were reduced significantly (p < 0.05) after the first HVP-cycle (median): INR 2.85 versus 1.5; ALT 1280 versus 434 U/L; bilirubin 12.7 versus 6.7 mg/dL; norepinephrine dosage 0.083 versus 0.009 µg/kg/min. Median soluble-interleukin-2-receptor dropped significantly following HVP (n = 7): 2407 versus 950 U/mL (p < 0.02).
HVP in PALF is feasible, safe, improves markers of liver failure and inflammation and is associated with lowering inotropic support. Prospective and controlled studies are required to confirm efficacy of HVP in PALF.
儿科急性肝衰竭（PALF）是一种危及生命的疾病。治疗的目的是支持肝再生或桥接肝移植。大体积血浆置换（HVP）可清除蛋白结合物质，减轻炎症，并提高成人急性肝衰竭的存活率。然而，PALF 中 HVP 的经验有限。本研究旨在报告 HVP 在 PALF 中的可行性、安全性、疗效和结果。
对 PALF 患儿进行回顾性观察性研究。在出现预后不良指标、中毒性病因或多器官衰竭（MOF）时进行 HVP。与患者估计的血浆体积相比，交换的新鲜冷冻血浆量为 1.5-2.0 倍。每天进行一个周期，直到患者符合停止治疗的标准，即肝再生、肝移植或死亡。
22 名 PALF 患儿（体重 2.5-106kg）接受了 1-7 次 HVP 循环。无出血或与操作相关的死亡率。观察到碱中毒、低体温和血小板减少。一名婴儿因溶血导致 HVP 终止。7 名儿童（32%）在保留自身肝脏的情况下存活，13 名患者（59%）接受了肝移植。两名婴儿因 MOF 死亡。总体生存率为 86%。国际标准化比值（INR）、丙氨酸氨基转移酶（ALT）、胆红素和正性肌力支持在第一次 HVP 循环后显著降低（p<0.05）（中位数）：INR 从 2.85 降至 1.5；ALT 从 1280 降至 434U/L；胆红素从 12.7 降至 6.7mg/dL；去甲肾上腺素剂量从 0.083 降至 0.009μg/kg/min。7 名患者的可溶性白细胞介素-2 受体在 HVP 后显著下降（n=7）：2407 降至 950U/mL（p<0.02）。
PALF 中的 HVP 是可行的、安全的，可改善肝衰竭和炎症标志物，并与降低正性肌力支持相关。需要进行前瞻性和对照研究以确认 HVP 在 PALF 中的疗效。
