Trial Information

The Natural History of Ocular Graft-Versus Host Disease

OBJECTIVE:

The study objectives are to study the development of ocular graft-versus-host disease (GVHD)
in participants treated with stem cell transplantation (SCT) at the NIH, including exam
findings prior to SCT, changes in ocular signs and symptoms following SCT and response to
standard therapy in participants identified with dry eye and ocular GVHD; to develop
clinical outcome measures for early onset and more advanced ocular GVHD and provide
estimates of progression rates for these outcomes; to analyze tear fluid, impression
cytology (IC) and serum samples from participants undergoing SCT, to identify biomarkers or
other findings which may predict the onset of ocular GVHD or correlate with disease
progression or response to therapy and to establish a cohort of participants with ocular
GVHD in anticipation of future clinical trials.

STUDY POPULATION:

Up to 50 adult participants undergoing SCT at the NIH will be enrolled.

DESIGN:

This prospective, natural history study will follow participants through 12 months post-SC,
with the option to extend follow-up for up to five years.

OUTCOME MEASURES:

The primary objective outcome variable is ocular surface staining using fluorescein and
lissamine green dyes, as measured by the Oxford criteria, with the scale ranging from 0 to
15 in each eye. The primary subjective outcome measure is the Ocular Surface Disease Index
(OSDI), ranging from 0 to 100. Secondary outcome variables include best-corrected visual
acuity (BCVA), tear film osmolarity, Schirmer's tear testing with anesthesia, meibomian
gland function and tear break-up time. In addition, tear fluid biomarkers and/or IC will be
studied as secondary outcome variables to determine whether changes in biomarker levels
(tear fluid) or cell densities and morphologies (IC) correlate with the development or
progression of ocular GVHD.