A Randomized Phase II Study for the Evaluation of Extracorporeal Photopheresis (ECP) in Combination With Corticosteroids for the Initial Treatment of Acute Graft-Versus-Host Disease (GVHD)
ECP uses ultraviolet A radiation to treat lymphocytes. Although the exact reason why ECP
may be beneficial is not completely understood, researchers believe that the lymphocytes
treated this way are less likely to continue causing GVHD.
In this study, researchers want to evaluate whether adding another treatment to standard
therapy with corticosteroids, in this case ECP, will improve the response to therapy,
duration of therapy, and survival. After the diagnosis of acute GVHD, you will be randomly
selected (at the toss of a coin) to be in one of 2 treatment groups. One group will receive
treatment with corticosteroids (like methylprednisolone or prednisone) alone, and the other
will receive ECP treatments in addition to the corticosteroids. ECP treatments are explained
In order to have ECP, you will need a special central venous catheter similar to the one you
have now. A central venous catheter is a sterile flexible tube that will be placed into a
large vein while you are under local anesthesia. Your physician will explain this procedure
to you in more detail, and you will be required to sign a separate consent form for this
Blood is drawn by a machine called "photopheresis machine". This blood goes into a bowl
inside the machine, where it is spun and separated into white cells (called buffy coat), red
cells, and platelets. The red cells and platelets are promptly returned to you, while the
white cells in the buffy coat undergo the process of ECP. The buffy coat will come out of
the bowl and will be mixed with a substance called methoxsalen, that will make lymphocytes
more sensitive to the effects of light. After mixing with methoxsalen, the buffy coat goes
in to chamber where it is exposed to ultraviolet A radiation, and from there back to you.
This process is done gradually, in cycles, and takes about 3 hours.
You will have up to 4 of these treatments weekly for the first 14 days of therapy, then 3
treatments weekly from Day 15-28, and after that 2 treatments weekly until Day 60, which is
the end of the study. After Day 60, your doctor will decide whether ECP is worth continuing,
and also the frequency of treatments. It is not necessary to be in the study to continue to
receive ECP treatments. If you respond to the treatment, your corticosteroids will be
reduced slowly to prevent the GVHD from coming back.
The length of corticosteroid therapy will depend on how GVHD responds to the treatment. You
will follow a corticosteroid therapy tapering schedule according to the suggested
guidelines. You will continue to receive tacrolimus or cyclosporine, whichever you have been
using as GVHD prevention, throughout the study independent of what treatment group you are
While you are getting treatment in this study, every week you will have a physical exam and
blood (about 1 tablespoon) will be drawn for routine tests. If your doctor thinks it is
necessary, blood may be drawn more often.
You will be considered off-study after 6 months of treatment completion. You will be taken
off study if the disease gets worse, you do not respond to treatment , you are unable to
comply with study requirements, you refuse to continue participation in the study, or
intolerable side effects occur.
This is an investigational study. ECP has been approved by the FDA for the treatment of a
certain type of lymphomas of the skin and is commercially available. Its use in patients
with GVHD is considered to be investigational. Up to 80 patients will take part in this
study. All will be enrolled at MD Anderson.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
First analysis after first 20 patients; Acute GVHD will be scored every week for 8 weeks.
Amin Alousi, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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