Multi-centre Retrospective Study to Describe the Use and Outcomes of ECP in Combination With New Treatment Protocols in Acute and Chronic GvHD

2019000902

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.

2021-04-13

2022-06-15

2022-06-15

Completed

319

Inclusion Criteria: Acute GvHD Patients 1. Patients who develop acute SR-GvHD after first HSCT and there is a minimum of 6 months follow up data in the database 2. Patients who initiate treatment with ECP or Ruxolitinib within 60 days of onset of SR aGvHD 3. Grade: II-IV only at time of treatment initiation 4. Patients who are ≥ 18 years at time of treatment initiation Chronic GvHD Patients 1. Patients who develop chronic SR-GvHD after first HSCT and there is with a minimum of 1 year follow up data in the database 2. Patients who initiate treatment with ECP or Ruxolitinib or Ibrutinib within I year of the onset of SR-cGvHD 3. Severity: moderate to severe only at time of treatment initiation 4. Patients who are ≥ 18 years at time of treatment initiation Exclusion Criteria: Acute GvHD 1. Patients on a clinical trial for GvHD for an interventional drug to treat GvHD in the retrospective period 2. Patient is pregnant or breastfeeding 3. Grade I at time of SR GvHD treatment initiation 4. Patients who receive ECP or new treatment as prophylaxis 5. Patients initiating ECP or new treatment later than 60 days from onset on SR-aGvHD 6. Patients \< 18 years at time of treatment initiation Chronic GvHD 1. Patients on a clinical trial for an interventional drug to treat GvHD in the retrospective period 2. Patient is pregnant or breastfeeding 3. Chronic GvHD : Severity mild at time of SR GvHD treatment initiation 4. Patients who receive ECP or new treatment as prophylaxis 5. Patients initiating ECP or new treatment after 1 year onset of SR-cGvHD 6. Patients \< 18 years at time of treatment initiation

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'RETROSPECTIVE'}, 'enrollmentInfo': {'count': 319, 'type': 'ACTUAL'}}

2024-03-28