Extracorporeal Photopheresis for Acute Graft Versus Host Disease

Study Overview
=================
Brief Summary
-----------------
The purpose of this research study is to evaluate the safety and feasibility of extracorporeal photopheresis (ECP) in the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in children.

Official Title
-----------------
Extracorporeal Photopheresis for Steroid-refractory Acute GVHD in Children and Young Adults: a Safety and Feasibility Study.

Conditions
-----------------
Graft Versus Host Disease, Cancer, Stem Cell Transplantation

Intervention / Treatment
-----------------
* Procedure: Extracorporeal Photopheresis


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Status-post allogeneic stem cell transplant (includes cord blood transplant, matched-unrelated-donor transplant and matched-related donor transplant) for any indication. HLA matching needs to be 4-6/6 by at least intermediate resolution for class I + II for cord blood and 5-6/6 for matched related or matched unrelated donors. Diagnosis of grade II-IV acute GVHD with histological confirmation of at least one organ (skin, gut, or liver) within the last 14 days. Grading of acute GVHD is per the standard Keystone criteria. Prior to enrollment, efforts should be made to rule out diagnoses that may mimic GVHD, such as drug rashes or GI infection. Patients that are being treated for acute GVHD and appear to be progressing to chronic GVHD are also eligible No improvement, or worsening, of acute GVHD after at least 4 days of IV methylprednisolone dosed at, at least 2.0mg/kg/day.. Weight >25.0kg. Adequate venous access. Exclusion Criteria: Evidence of veno-occlusive disease. Intubated patient. Patient receiving dialysis. Age > 30. Total bilirubin >15mg/dL

Ages Eligible for Study
-----------------
Maximum Age: 30 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Procedure: Extracorporeal Photopheresis|ECP will be performed using the UVAR® XTS™ photopheresis system (Therakos). Whole blood is drawn and separated by centrifuge to collect the buffy coat (lymphocyte solution). Methoxsalen (8-MOP) is added to the blood, and the final solution of cells is passed as a film, 1mm thick, through a disposable plastic device, exposed to a UVA light source (2J/cm2/cell) and then returned to the patient. ECP will be performed weekly on 2 consecutive days for 4 weeks. After 4 weeks the interval will be prolonged to every 2 weeks and ECP will be stopped after maximal response has been maintained for 2 weeks. The actual study finishes at d56 but ECP may be continued beyond that (off-study) at the discretion of the physician.|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the safety and feasibility of extracorporeal photopheresis (ECP) in the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in children. |  | To end of study | 
| To estimate the response rate of ECP in steroid-refractory acute GVHD in children. |  | To end of study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the immunological mechanisms of ECP in acute GVHD. |  | To end of study | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
graft versus host disease, ECP, extracorporeal photopheresis, allogeneic transplantation