Sharma Shruti, Lal Sadhna B, Sachdeva Manupdesh, Bhatia Anmol, Varma Neelam
Division of Paediatric Gastroenterology, Hepatology & Nutrition, Post Graduate Institute of Medical Education & Research, Chandigarh, 160012, India.
Department of Hematology, Post Graduate Institute of Medical Education & Research, Chandigarh, 160012, India.
J Clin Exp Hepatol. 2020 May-Jun;10(3):201-210. doi: 10.1016/j.jceh.2019.10.001. Epub 2019 Oct 23.
Acute-on-chronic liver failure (ACLF) results in very high mortality in children. We aimed to evaluate the role of granulocyte colony-stimulating factor (GCSF) on short-term outcome of children with ACLF in a nontransplant unit.
Children (aged > 1 year) diagnosed with ACLF over a 15 month period were randomised. Group A was given GCSF therapy along with standard medical care (SMC - details in supplementary data) and group B was given only SMC. The outcome was evaluated as survival at 30 and 60 days of therapy.
Thirty-one children with ACLF were enrolled, with a mean age of 6.92 ± 4.3yrs. A total of 15 patients were randomised to group A and 16 to group B. The overall mortality was 54.83%. The intervention group showed survival rates of 80%, 66.67% and 53.3%, whereas the control group had survival rates of 43.75%, 37.5% and 37.5% at 14, 30 and 60 days, respectively. A significant survival benefit was noted on day 14 (p = 0.043) of therapy in group A with significant difference in Child-Turcotte-Pugh (CTP) and pediatric end-stage liver disease (PELD) scores in the two groups. After an initial rise in group A, the granulocyte counts fell to become comparable in the two groups by day 30 and 60, indicating that the effect of GCSF therapy wears off over time. There was no significant difference in the overall survival, median/mean CTP, PELD and MCS (Modified Cliff sequential organ failure assesment (SOFA)) scores on day 30 and 60. Mean (%) CD 34 + cells level showed a rise on day 7 in group A but was statistically insignificant.
The present study shows that GCSF therapy at 5 mcg/kg/day for 5 days seems to be ineffective in improving the survival outcome on day 30 and 60 of therapy. Studies with larger number of children enrolled and longer duration of therapy are required. (CTRI/2017/11/010420).
急性慢性肝衰竭(ACLF)导致儿童死亡率极高。我们旨在评估粒细胞集落刺激因子(GCSF)在非移植单位对ACLF儿童短期预后的作用。
对在15个月期间诊断为ACLF的儿童(年龄>1岁)进行随机分组。A组在接受标准医疗护理(SMC - 补充数据中有详细说明)的同时给予GCSF治疗,B组仅接受SMC治疗。以治疗30天和60天的生存率评估预后。
共纳入31例ACLF儿童,平均年龄6.92±4.3岁。15例患者被随机分配到A组,16例到B组。总死亡率为54.83%。干预组在第14、30和60天的生存率分别为80%、66.67%和53.3%,而对照组分别为43.75%、37.5%和37.5%。治疗第14天A组有显著的生存获益(p = 0.043),两组的Child-Turcotte-Pugh(CTP)和儿童终末期肝病(PELD)评分有显著差异。A组粒细胞计数最初升高后,到第30天和60天两组变得相当,表明GCSF治疗的效果随时间减弱。第30天和60天的总生存率、中位/平均CTP、PELD和MCS(改良Cliff序贯器官衰竭评估(SOFA))评分无显著差异。A组平均(%)CD 34 +细胞水平在第7天有所升高,但无统计学意义。
本研究表明,每天5 mcg/kg持续5天的GCSF治疗似乎对改善治疗第30天和60天的生存结局无效。需要开展纳入更多儿童且治疗时间更长的研究。(CTRI/2017/11/010420)