Treatment of Steroid-refractory Acute and Chronic Graft-versus-host Disease With Inhibitor of Janus Kinases
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Overall response rate
研究概览
简要总结
Steroid-refractory acute GVHD (srGVHD) is one of the causes of mortality after allogeneic stem cell transplantation, while steroid-refractory chronic GVHD significantly increases morbidity, aggravates quality of life and may also impact survival. Currently there is no standard treatment of srGVHD. One of the most promising agents is Janus kinase (JAK) inhibitor ruxolitinib, which in the retrospective study demonstrated excellent response rate and survival of patients with either acute or chronic srGVHD. This study prospectively evaluates the efficacy of ruxolitinib in srGVHD patients.
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
- 1 Year 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of graft-versus-host disease established based on tissue biopsy
- •Steroid-refractory acute or chronic graft-versus-host disease according to EBMT/ELN criteria (T. Ruutu et al, 2014)
- •Age 1 to 70 years
- •Karnofsky index >30%.
- •Ability for oral drug intake
- •Life expectancy > 1 month
- •Signed informed consent
排除标准
- •Severe organ dysfunction: AST or ALT >5 upper normal limits (excluding cases related to liver GVHD), creatinine >2 upper normal limits
- •Requirement for vasopressor support at the time of enrollment
- •Uncontrolled bacterial or fungal infection at the time of enrollment
- •Pregnancy
- •Somatic or psychiatric disorder making the patient (or legal guardian) unable to sign informed consent
研究组 & 干预措施
Ruxolitinib treatment
Ruxolitinib 10 mg bid for adults and children with body weight > 40 kg, 0.15 mg/kg bid for children with body weight < 40 kg.
干预措施: Ruxolitinib Oral Tablet (Drug)
结局指标
主要结局
Overall response rate
时间窗: 84 days
Partial response for acute GVHD is defined as the improvement of at least one stage in the severity of aGVHD in one organ without deterioration in any other organ. A response had to last for at least 3 weeks. Partial response for chronic GVHD was defined as reduction in GVHD National Institute of Health (NIH) severity score at east for one organ without deterioration in any other organ. A complete response was defined as the absence of any symptoms related to GVHD. Overall response is defined as presence of partial or complete response
次要结局
- 6-month overall survival(6 months)
- Toxicity based NCI CTC grades(6 months)
- Infectious complications, including analysis of severe bacterial, fungal and viral infections incidence(6 months)
- GVHD relapse incidence after complete response(6 months)
- Relapse incidence of underlying hematologic malignancy(12 months)
- Quality of life measured by FACT BMT ver. 4 questionnaire in adults(6 months)
- Quality of life measured by PedQL Stem Cell transplant module ver.1.0 questionnaire in children(6 months)
研究者
Ivan S Moiseev
Head of oncology, hematology and transplantation department for adults
St. Petersburg State Pavlov Medical University