Reduced Intensity Stem Cell Transplant in Children and Young Adults Utilizing Photopheresis, Fludarabine, and Busulfan
This study tests the feasibility of a reduced intensity preparative regimen for stem cell
transplant including extracorporeal photopheresis (ECP), busulfan, and fludarabine in
patients with leukemia, lymphoma, and certain non-malignant diseases. The current reduced
intensity protocol includes busulfan, fludarabine, and anti-thymocyte immunoglobulin. ECP
is currently used in diseases such as chronic GVHD and cutaneous T cell lymphoma. The
mechanism of ECP has not been defined. It is hypothesized that exposure of white blood
cells to ultraviolet light with 8-methoxypsoralen initiates an apoptotic cellular cascade.
Apoptotic cells are recognized and removed by the reticuloendothelial system, initiating the
secretion of anti-inflammatory cytokines and the reduction of proinflammatory cytokines.
Antigen presenting cells then regulate immune responses through the induction of tolerance.
Here we incorporate the use of ECP, fludarabine, and busulfan in the preparative regimen,
followed by ECP as prophylaxis for acute graft versus host disease. We hypothesize that
photopheresis is safe and feasible, and patients will have similar rates of engraftment with
less GVHD as those treated with current reduced intensity protocols. The use of ECP prior
to transplant provides immunosuppression promoting host engraftment. Furthermore, the
introduction of ECP following transplant may be able to induce tolerance thereby reducing
rates of GVHD.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To determine the feasibility of using photopheresis as the backbone of a reduced intensity transplant regimen to reduce transplant related mortality and acute and chronic graft versus host disease (GVHD)
Jennifer Schneiderman, MD, MS
Ann & Robert H Lurie Children's Hospital of Chicago
United States: Institutional Review Board
|Children's Memorial Hospital||Chicago, Illinois 60614|