Division of Gastroenterology, University Hospital Leipzig, Leipzig, Germany.
Clinical Trial Centre, University of Leipzig, Leipzig, Germany.
Bone Marrow Transplant. 2019 Nov;54(11):1738-1746. doi: 10.1038/s41409-019-0464-x. Epub 2019 Feb 26.
Allogeneic hematopoietic stem cell transplantation is the only curative option for a variety of diseases. Despite advances, it is associated with considerable morbidity and mortality, often involving liver complications. Liver disease can be characterized using ultrasound-based liver stiffness measurement. To assess its prognostic value, consecutive patients undergoing allogeneic hematopoietic stem cell transplantation were prospectively evaluated in a single-center study. Endpoints included liver event-free survival and all-cause mortality at 1 year. Competing risk and Cox-regression were used for analysis. We evaluated 106 patients (42 female, age 57) and observed 33 life-threatening events (14 died) including 16 liver complications at 100 days. At 1 year, 36 patients had died, 20 with disease relapse. The hazard ratios for liver-related complications at 100 days were 3.2 (95% CI: 1.8-14.6, p = 0.0022) and 4.4 (95% CI: 1.6-11.9, p = 0.0042) for elevated transient elastography (n = 11) and shear-wave velocity (n = 31), respectively. Results were analogous for all-cause mortality at 1 year. Prior stem cell therapy and elevated gamma glutamyltransferase were also associated with outcome. This demonstrates that elastography is a promising and viable tool for risk prediction and should be included in upcoming multi-center trials to establish new means of guiding treatment and prophylaxis.
同种异体造血干细胞移植是治疗多种疾病的唯一根治方法。尽管取得了进展,但它仍与相当大的发病率和死亡率相关,常伴有肝脏并发症。肝脏疾病可以通过基于超声的肝脏硬度测量来进行特征描述。为了评估其预后价值,在一项单中心研究中前瞻性评估了连续接受同种异体造血干细胞移植的患者。终点包括 1 年时的肝脏无事件生存率和全因死亡率。使用竞争风险和 Cox 回归进行分析。我们评估了 106 例患者(42 例女性,年龄 57 岁),并观察到 33 例危及生命的事件(14 例死亡),包括 100 天内的 16 例肝脏并发症。在 1 年时,36 例患者死亡,其中 20 例死于疾病复发。100 天时与肝脏相关并发症相关的风险比为 3.2(95%CI:1.8-14.6,p=0.0022)和 4.4(95%CI:1.6-11.9,p=0.0042),分别用于升高的瞬时弹性成像(n=11)和剪切波速度(n=31)。1 年时的全因死亡率结果也类似。先前的干细胞治疗和谷氨酰转肽酶升高也与结局相关。这表明弹性成像具有预测风险的潜力,应包含在即将进行的多中心试验中,以建立新的治疗和预防指导手段。