Multi-centre Retrospective Study to Describe the Use and Outcomes of ECP in Combination With New Treatment Protocols in Acute and Chronic GvHD
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 319
- 试验地点
- 38
- 主要终点
- Overall response rate
研究概览
简要总结
Extracorporeal photopheresis (ECP) offers an alternative to standard immunosuppression and shows an immunomodulatory rather than an immunosuppressive effect, which is associated with less toxicities and side effects. Additionally ECP has been shown to allow tapering of steroids and immunosuppressant agents which should be a goal of GvHD therapy.
ECP has been used for the management of GvHD since first described in 1994 and as its use has continued over the decades. The treatment was incorporated into a number of guidelines as a second line therapy in steroid refractory or steroid dependent GvHD patients. As well as being used in addition and after steroids, it is also used in combination with CNI Inhibitors, MMF and other immunosuppressant agents.
However, despite the current widespread use of ECP in the treatment of patients with GvHD, clinical data from randomized studies is limited and small prospective and retrospective trials are the main evidence base .This is also the case for other commonly used immunosuppressant agents, which have been used in GvHD since ECP was introduced.
The systematic review concluded that ECP is an effective therapy for oral, skin, and liver SR-cGVHD, with modest activity in lung and gastrointestinal SR-cGVHD.
In the USA Ibrutinib is the only FDA approved agent for second line cGvHD therapy once steroid therapy has failed and Ruxolitinib had been approved in the USA for the treatment of steroid refractory GvHD.
While studies have shown the effectiveness and safety of ECP in GvHD treatment, there is limited data to show how it is being used in combination with the recently approved agents.
Using existing registry data targeting centres where the newer agents are being used and enhancing the capture of treatment data we believe we can undertake a larger scale study, which will include the new treatment protocols. The aim of the current study is to improve the evidence basis on the potential benefit of ECP use as treatment of GVHD.
详细描述
This is a Registry Based Study (RBS) designed to collect data on the treatment behaviour of acute and chronic GvHD after HSCT. The data collection will be based on the EBMT registry, which so far consists of two questionnaires (Forms A and B), mainly covering the primary disease diagnostics, the status before and at HSCT, the type of HSCT (donor status, preparative regimen etc) and the survival status. With a new questionnaire Form C, which will be similar in design as the current forms used in the registry, we aim at collecting more information and additional data on GvHD characteristics and treatment (schedule, combination, disease states) for both chronic and acute GvHD EBMT will work with the selected sites to facilitate the collection of additional data as specified in section 4.
The data collected will all be retrospective and include up to 3 years of data covering 2017 onwards, from onset of GvHD that has failed to respond to steroids with a minimum data follow up of 6 months for acute and 1 year for chronic. Centres will be asked to select patients that meet the inclusion criteria and fill in Form C retrospectively. The amount of additional data required will depend on whether the centre selected fills in the more detailed Form B or the more minimum data set in Form A.
Criteria for centre selection will be based on:
- Centres that have expressed a willingness to participate in the study through a feasibility questionnaire that was sent out prior to the study or via Email confirmation
- Centres who have responded through the feasibility questionnaire
- Centres where there is prior knowledge of use of both Ruxolitinib/Ibrutinib and ECP or have responded as such in the feasibility questionnaire
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Acute GvHD Patients
- •Patients who develop acute SR-GvHD after first HSCT and there is a minimum of 6 months follow up data in the database
- •Patients who initiate treatment with ECP or Ruxolitinib within 60 days of onset of SR aGvHD
- •Grade: II-IV only at time of treatment initiation
- •Patients who are ≥ 18 years at time of treatment initiation
- •Chronic GvHD Patients
- •Patients who develop chronic SR-GvHD after first HSCT and there is with a minimum of 1 year follow up data in the database
- •Patients who initiate treatment with ECP or Ruxolitinib or Ibrutinib within I year of the onset of SR-cGvHD
- •Severity: moderate to severe only at time of treatment initiation
- •Patients who are ≥ 18 years at time of treatment initiation
排除标准
- •Patients on a clinical trial for GvHD for an interventional drug to treat GvHD in the retrospective period
- •Patient is pregnant or breastfeeding
- •Grade I at time of SR GvHD treatment initiation
- •Patients who receive ECP or new treatment as prophylaxis
- •Patients initiating ECP or new treatment later than 60 days from onset on SR-aGvHD
- •Patients < 18 years at time of treatment initiation
- •Chronic GvHD
- •Patients on a clinical trial for an interventional drug to treat GvHD in the retrospective period
- •Patient is pregnant or breastfeeding
- •Chronic GvHD : Severity mild at time of SR GvHD treatment initiation
- •Patients who receive ECP or new treatment as prophylaxis
- •Patients initiating ECP or new treatment after 1 year onset of SR-cGvHD
- •Patients < 18 years at time of treatment initiation
研究组 & 干预措施
ECP and treatment combination (cGVHD patients)
Patients treated with ECP and Ruxolitinib or Ibrutinib
干预措施: Ibrutinib (Drug)
ECP only (aGVHD patients)
Patients treated with ECP and other Standard Of Care treatments (SOC)
干预措施: Extracorporeal photopheresis (Device)
ECP and Ruxolitinib (aGVHD patients)
Patients treated with ECP and Ruxolitinib
干预措施: Ruxolitinib (Drug)
ECP and Ruxolitinib (aGVHD patients)
Patients treated with ECP and Ruxolitinib
干预措施: Extracorporeal photopheresis (Device)
Ruxolitinib only (aGVHD patients)
Patients treated with Ruxolitinib and other Standard Of Care treatments (SOC)
干预措施: Ruxolitinib (Drug)
ECP only (cGVHD patients)
Patients treated with ECP and other Standard Of Care treatments (SOC)
干预措施: Extracorporeal photopheresis (Device)
ECP and treatment combination (cGVHD patients)
Patients treated with ECP and Ruxolitinib or Ibrutinib
干预措施: Ruxolitinib (Drug)
ECP and treatment combination (cGVHD patients)
Patients treated with ECP and Ruxolitinib or Ibrutinib
干预措施: Extracorporeal photopheresis (Device)
Treatment combination only (cGVHD patients)
Patients treated with Ibrutinib and/or Ruxolitinib and other Standard Of Care treatments (SOC)
干预措施: Ruxolitinib (Drug)
Treatment combination only (cGVHD patients)
Patients treated with Ibrutinib and/or Ruxolitinib and other Standard Of Care treatments (SOC)
干预措施: Ibrutinib (Drug)
结局指标
主要结局
Overall response rate
时间窗: 3 months
Partial or Complete response according to NIH/Glucksberg classification) at 3 months for acute GvHD since start of targeted\* treatment for SR-GvHD
Overall response rate (Partial or Complete response according to NIH/Glucksberg classification) at 6 months for chronic GvHD since start of targeted* treatment for SR-GvHD
时间窗: 6 months
次要结局
- Efficacy of ECP(Up to one year)
- Safety of ECP(Up to one year)