Fecal Microbiota Transplantation for Treatment of Refractory Graft Versus Host Disease-a Pilot Study
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 15
- 主要终点
- Efficacy of FMT in the treatment of refractory GVHD on day 7
研究概览
简要总结
The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory graft-versus-host-disease (GVHD) of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
详细描述
Graft-versus-host-disease (GVHD) is a major complication after hematopoietic stem cell transplantation (HSCT). Gut is the most vulnerable target organ of acute GVHD. Patients who have a gastrointestinal acute GVHD received a first-line standard treatment of corticosteroids. For patients who do not respond or progress after an initial response have a high mortality. Therefore, the investigation of effective second line therapy for these patients are in need. The study evaluates safety and efficacy of fecal microbiota transplantation (FMT) for the treatment of refractory GVHD of the gut. FMT might be a beneficial treatment in this clinical situation with a poor prognosis and limited therapeutic options.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •3-18 years of age
- •Allo-SCT patients with acute steroid-resistant GI-related GVHD grade III-IV. Steroid-resistant GI-related GVHD will be defined as lack of improvement (same stage) or worsening of GI symptoms after 7 days of steroid therapy (≥ 2 ml/kg of IV methylprednisolone)
- •No definite contraindication for gastrointestinal endoscopy
- •Signature of informed consent by the legal guardians of patients
排除标准
- •Prior inclusion to an interventional study
- •Previous Allo-SCT
- •Known multi-drug resistance carriage prior to stool collection
- •Severe colitis of any etiology or a history of inflammatory bowel disease (IBD)
- •Uncontrolled infection (hemodynamic instability, ongoing high fever or bacteremia within 3 days after antibiotics administration)
- •Active GI bleeding
- •Absolute neutrophil count < 500 cells/microL
- •Absolute platelet count< 10 x 109 /L
- •Patients who cannot give informed consent
研究组 & 干预措施
HSCT patients with refractory GVHD
Patients will accept FilmArray Gastrointestinal (GI) panel test before pre-treatment of HSCT and 28±3 days post-HSCT. Patients will receive 50ml fecal microbiota from unrelated healthy donors through nasojejunal tube and monitored under gastroscopy. Patients receiving FMT treatment will be followed for at least 6 months. The ideal follow up time is 2 year. Stool and blood samples will be serially collected and tested (before pre-treatment, 1/3/6/12 months after FMT).
干预措施: Fecal Microbiota Transplantation (Biological)
结局指标
主要结局
Efficacy of FMT in the treatment of refractory GVHD on day 7
时间窗: 7 days following FMT
Participants will be evaluated on days 7 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 7 days.
Efficacy of FMT in the treatment of refractory GVHD on day 28
时间窗: 28 days following FMT
Participants will be evaluated on days 28 following FMT. The volume and frequency of daily diarrhoea will be continuous observed and recorded in 28 days.
次要结局
- Treatment-related AEs and SAEs(up to 28 days following FMT)
- GVHD severity(up to 28 days following FMT)
- Implantation rate(through study completion, an average of 6 months)
- Survival rate(through study completion, an average of 6 months)
- Change in biomarkers(up to 28 days following FMT)
- Number of patients with infectious disorders(through study completion, an average of 6 months)
- Quality of Life(up to 6 months following FMT)
研究者
Cao Qing
Director of Infectious Diseases
Shanghai Children's Medical Center