Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months.
研究概览
简要总结
The aim of the study is to evaluate the use of Granulocyte Colony Stimulating Factor (GCSF) on the clinical and biochemical outcome of type 3 biliary atresia post kasai.
详细描述
Biliary atresia (BA) is a devastating disease manifest early in infancy characterized by bile duct injury and extrahepatic biliary obstruction, leading to cirrhosis in the majority of infants.
Although BA is a rare disease, occurring in ~1 in 5600 to 1 in 18,000 infants worldwide, it is considered the most common indication for liver transplantation in children.
However, despite a 50-60% rate of initial jaundice clearance, liver transplantation by 2 years of age is necessary for long term survival in many of the post-Kasai patients.
GCSF cytokine that stimulates neutrophil and hematopoietic stem cell (HSC) production and mobilization from the bone marrow, and has served as a complementary agent to bone marrow stem cell therapy for patients with congenital or acquired diseases of bone marrow suppression.
Granulocyte colony-stimulating factor (G-CSF) mobilizes CD34+(cluster of differentiation34) cells, these CD34+ cells increase hepatocyte growth factor inducing the proliferation of hepatic progenitor cells within 7 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 20 Days 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Infants with initial diagnosis of biliary atresia with biliary atresia score > 23.927 will be allocated for Kasai porto-enterostomy with intra-operative cholangiogram reaching type 3 biliary atresia anatomy as a final diagnosis.
排除标准
- •Major cardiac, renal, pulmonary, neurological malformations or illnesses.
- •Hemoglobinopathies, such as sickle cell anemia
- •Active systemic infection.
- •White blood cell count > 20,000 cells/mm
- •Platelet count < 40,000 cells/mm3 or ≥ 800,000 cells/mm
- •Purpura fulminans or unexplained vascular thrombotic conditions.
研究组 & 干预措施
biliary atresia with GCSF
20 infants with the final diagnosis of biliary atresia type 3 (supported by the liver histology and intra-operative finding) for GCSF after Kasai operation will receive GCSF.
干预措施: Granulocyte Colony-Stimulating Factor (Drug)
结局指标
主要结局
Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months.
时间窗: baseline
Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months will be analyzed using generalized linear mixed effects model (GLMM) with a logistic link function for the covariates as fixed effects, with study sites as random effect to control for site variability in management practices and patient characteristics. The covariates are potential confounders for poor outcome, such as age at the time of Kasai, presence of ductal plate malformation, cholangitis and possibly CMV IgM(cytomegalovirus immune globulin M antibodies) positivity.
次要结局
- Differences in the proportion of subjects with cholangitis at 6 months.(base line)