A Study of Extracorporeal Photopheresis With UVADEX® in the Setting of a Standard Myeloablative Conditioning Regimen in Related or Unrelated Donor Hematopoietic Stem Cell Transplantation for the Prevention of Graft Versus Host Disease
This study is to test the concept that using ECP treatment prior to and after an allogeneic
bone marrow transplant (BMT) or peripheral blood stem cell (PBSC) transplant will prevent
the development of GvHD. This study is not designed to detect a specific treatment effect.
However, some statements about the outcome of the study are possible. A sample size of n =
21 patients could detect a statistically significant difference between the expected rate of
GvHD in an untreated population, 60%, and our hypothesized rate, 30%, for the
matched-unrelated recipients. This calculation is based on a one-sample, two-sided
chi-square test at the 5% level of significance with 80% power.
Patients will receive ECP from day -10 and day -8 before transplant and then from day of
engraftment absolute neutrophil count (ANC>500) until day 90 after transplant. Patients who
enter the study will receive a BMT or PBSC transplant from a donor who is matched unrelated
(8/10 to 10/10 match). Rates of acute GvHD and chronic GvHD that occur in patients are
50-70% for the matched-unrelated donor transplant.
The choice of sample size is 21 patients. The analysis will determine if there are favorable
trends for a treatment effect. Comparison on survival, and rates of acute and chronic GvHD
will be made with historical controls who have undergone similar myeloablative transplant
from an unrelated donor.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Presence/absence of grade II-IV acute Graft versus Host Disease (aGVHD)
The primary efficacy variable is the presence/absence of grade II-IV acute GvHD within the first 100 days after transplantation
100 days after transplant
Sunil Abhyankar, MD
University of Kansas
United States: Food and Drug Administration
|University of Kansas Medical Center||Kansas City, Kansas 66160-7353|