Thorvaldson Linda, Remberger Mats, Winiarski Jacek, Omazic Brigitta, Fischler Björn, Sundin Mikael
Division of Pediatrics, Department of Clinical Science, Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
Department of Oncology-Pathology, Karolinska Institutet, Stockholm, Sweden.
Pediatr Transplant. 2016 Feb;20(1):96-104. doi: 10.1111/petr.12623. Epub 2015 Oct 31.
Hepatic dysfunction is common after allogeneic hematopoietic stem cell transplantation (HSCT). The aim of this retrospective study was to determine the risk factors, frequency, and outcome of hepatic complications post-HSCT in children. Two hundred and thirty-seven cases of allogeneic HSCT in children were included. Data on biochemical liver function at start of HSCT, at +1, +3, +6, and +9 months, and at each subsequent yearly follow-up were extracted. Patients were stratified into groups with hepatocellular (none and mild, and moderate to severe) and hepatobiliary (none and present) dysfunction. Statistical analysis included variables such as diagnosis, age, conditioning regimen, and HLA type.
One hundred and fifty-six (66%) patients displayed hepatocellular dysfunction post-HSCT. In most cases transient, but 32% had a persistent abnormality three yr post-HSCT. Risk factors were chronic GVHD (OR 4.20, p = 0.003) and donor HLA-A01 (OR 2.97, p = 0.02). HLA-DQB103 decreased the risk (OR 0.35, p = 0.02). Hepatobiliary dysfunction was less frequent (12%) but carried a poor prognosis. aGVHD grade II-IV (OR 2.7, p = 0.02) and long-term TPN (OR 3.25, p = 0.01) increased the risk.
GVHD is an important risk factor for liver dysfunction post-HSCT. Specific HLA types may also contribute as a risk factor, while others seem to have a protective effect.
异基因造血干细胞移植（HSCT）后肝功能障碍很常见。这项回顾性研究的目的是确定儿童HSCT后肝脏并发症的危险因素、发生率及预后。纳入了237例儿童异基因HSCT病例。提取了HSCT开始时、+1、+3、+6和+9个月时以及随后每年随访时的生化肝功能数据。患者被分为肝细胞功能障碍组（无、轻度、中度至重度）和肝胆功能障碍组（无、存在）。统计分析包括诊断、年龄、预处理方案和HLA类型等变量。
156例（66%）患者在HSCT后出现肝细胞功能障碍。大多数情况下为短暂性，但32%的患者在HSCT后3年仍有持续性异常。危险因素为慢性移植物抗宿主病（OR 4.20，p = 0.003）和供体HLA-A01（OR 2.97，p = 0.02）。HLA-DQB103降低了风险（OR 0.35，p = 0.02）。肝胆功能障碍发生率较低（12%），但预后较差。急性移植物抗宿主病II-IV级（OR 2.7，p = 0.02）和长期全胃肠外营养（OR 3.25，p = 0.01）增加了风险。
移植物抗宿主病是HSCT后肝功能障碍的重要危险因素。特定的HLA类型也可能作为危险因素起作用，而其他一些类型似乎具有保护作用。
