ECP Combination Study

Sponsor
European Society for Blood and Marrow Transplantation
Study ID
NCT05052385
Status
Completed

Conditions

  • Steroid Refractory GVHD

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    As per treating physician's decision - non interventional study
  • Extracorporeal photopheresis — DEVICE
    As per treating physician's decision - non interventional study
  • Ibrutinib — DRUG
    As per treating physician's decision - non interventional study

Study Details

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.

Key Dates

Start date
Apr 13, 2021
Status verified
Mar 2024
Primary completion
Jun 15, 2022
Completion
Jun 15, 2022

Study Design

Enrollment
319 participants (actual)

Arms

  • Arm: ECP only (aGVHD patients)
    Patients treated with ECP and other Standard Of Care treatments (SOC)
  • Arm: ECP and Ruxolitinib (aGVHD patients)
    Patients treated with ECP and Ruxolitinib
  • Arm: Ruxolitinib only (aGVHD patients)
    Patients treated with Ruxolitinib and other Standard Of Care treatments (SOC)
  • Arm: ECP only (cGVHD patients)
    Patients treated with ECP and other Standard Of Care treatments (SOC)
  • Arm: ECP and treatment combination (cGVHD patients)
    Patients treated with ECP and Ruxolitinib or Ibrutinib
  • Arm: Treatment combination only (cGVHD patients)
    Patients treated with Ibrutinib and/or Ruxolitinib and other Standard Of Care treatments (SOC)

Primary Outcome Measure

Overall response rate [ Time Frame: 3 months ]

Related Studies