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临床试验/NCT05663827
NCT05663827已完成3 期

Ruxolitinib Add-on in Steroid-refractory Graft-vs-host Disease After Allogeneic Stem Cell Transplantation: a Single Institutional Experience

Yi-Lun Wang1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2019年1月1日最近更新:
适应症
干预措施
相关药物

试验速览

阶段
3 期
状态
已完成
发起方
入组人数
6
试验地点
1
主要终点
GI symptoms secondary to GVHD

研究概览

简要总结

Steroid-refractory graft-vs-host disease (SR-GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation (HSCT). We sought to evaluate the effect and safety of ruxolitinib (RUX) add-on in the treatment of patients with SR-GVHD.

详细描述

Nowadays, second-line treatment for SR-GVHD differs between centers and depends on the physician's experience. The agents commonly used as second and further lines of therapy include anti-thymocyte globulin, mycophenolate mofetil, calcineurin inhibitor, or extracorporeal photopheresis. However, the response varied. This study highlighted that corticosteroid (CS) is not a panacea for treating GVHD, and some patients with remission still experience further relapse. Additionally, the identification of multiple infections adds complexity.

Hope was supposed to come in 2019 after the Food and Drug Administration (FDA) approved RUX for the therapy of SR-GVHD in adult and pediatric patients > 12 years. RUX add-on should be withheld as the risks outweigh the benefits, especially for patients who developed scleroderma and bronchiolitis obliterans. RUX can be added on as needed for the treatment of GVHD, especially in patients receiving DLI as relapse therapy with the conversion from mixed chimerism to complete chimerism. Despite some studies showing the efficacy of RUX across affected organs, some other data bring the opposite results. Thus we conducted this study to reinforce the efficacy of RUX as an add-on therapy for SR-GVHD.

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 Months 至 30 Years(Child, Adult)
性别
All
接受健康志愿者

入选标准

  • Patients diagnosed with hematologic disorder

排除标准

  • Those who are not applicable of receiving Ruxolitinib as immunodeficiency-modulation therapy
  • Those who are actively encountering acute infectious illness

研究组 & 干预措施

Ruxolitinib add-on group

Experimental

Once diagnosed with steroid-refractory GVHD, after discussion with family, as per their willing, Ruxolitinib will be administered as add-on therapy. Its dose depends on participants' age and body weight. Usually a dosage of 5mg once per day will be applied as initiation and titrated in accordance with clinical response.

干预措施: Ruxolitinib (Drug)

结局指标

主要结局

GI symptoms secondary to GVHD

时间窗: 3 years

Diarrhea event exceeds a frequency of 5 times a day

Skin symptoms secondary to GVHD

时间窗: 3 years

Skin rash extends more than 25% of body surface area

次要结局

  • Overall survival(3 years)

研究者

发起方
Yi-Lun Wang
申办方类型
Other
责任方
Sponsor Investigator
主要研究者

Yi-Lun Wang

Principal investigator, Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Memorial Hospital

Chang Gung Memorial Hospital

研究点 (1)

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