Biomarkers in Predicting Response in Patients With Graft-Versus-Host Disease Undergoing Extracorporeal Photophoresis

PRIMARY OBJECTIVE:

I. To show that extracorporeal photopheresis (ECP)increases skin and gut homing T regulatory
(T-reg) cells in patients with GVHD clinically responding to ECP.

SECONDARY OBJECTIVES:

I. Response rates of GVHD with extracorporeal photopheresis(ECP)as measured by NIH response
criteria

II. Incidence of T-reg cell frequency(%)with various NIH subtypes of chronic
graft-versus-host disease (GVHD)

III. Incidence of T-reg homing subsets(%)with various NIH subtypes of chronic
graft-versus-host disease (GVHD)

OUTLINE:

Patients undergo ECP twice a week for 4 weeks and then twice a week every 2 weeks for 8
weeks.

After completion of study treatment, patients are followed up at 2, 4, and 6 months.

Inclusion Criteria:

- Patients with any NIH subtype of chronic GVHD that is being treated with ECP

- Karnofsky Performance Scale (KPS) > 60% at time of study enrollment

- Life expectancy > 3 months

- Steroid dose not greater than 2 mg/kg prednisone equivalent at time of study
enrollment

- If patient has steroid refractory GVHD (defined as worsening of GVHD after 3 days of 2
mg/kg prednisone equivalent or no improvement after 7 days of 2 mg/kg prednisone
equivalent), time interval from start of steroids to initiation of ECP should not be >
14 days

- No use of an investigational agent within 2 weeks of starting ECP

- No uncontrolled bacterial, fungal or viral disease (therapy for cytomegalovirus [CMV]
viremia is permitted)

- No evidence of relapse or progression of underlying disease (molecular evidence of
relapse/progression or mixed chimerism is permitted)

- Women of childbearing potential (WOCBP) should be willing to use 2 forms of
contraception; male patients should be willing to use contraception

- Voluntary written informed consent before performance of any study-related procedure
not part of normal medical care, with the understanding that consent may be withdrawn
by the subject at any time without prejudice to future medical care

Exclusion Criteria:

- Female patients who are breastfeeding or pregnant

- Patients known to be human immunodeficiency virus (HIV) positive

- Bronchiolitis obliterans as the sole indication of ECP

- Serious medical or psychiatric illness likely to interfere with participation in this
clinical study

- Mechanical ventilation, renal replacement therapy, admitted in intensive care until at
time of enrollment

- Stage 4 gastrointestinal GVHD as per Seattle-Glucksberg criteria