Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder

Randomized Placebo Controlled Double Blind Study of Restasis Versus Placebo in Primary Prevention of Ocular GVHD After Allogeneic Stem Cell Transplantation

RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.

PURPOSE: This randomized phase I trial is studying how well cyclosporine eye drops work in preventing graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.

OBJECTIVES:

Primary

• To assess the efficacy of cyclosporine ophthalmic emulsion (Restasis®) in the prevention of ocular graft-versus-host disease in patients who have undergone allogeneic stem cell transplantation for hematologic malignancies or bone marrow failure disorders.

Secondary

• To correlate the Ocular Surface Disease Index with clinical ophthalmologic examination.

OUTLINE: This is a multicenter study. Patients are stratified according to age, type of transplant (related vs unrelated), and intensity of transplant (ablative vs other). Patients are randomized to 1 of 2 treatment arms.

• Arm I: Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
• Arm II: Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.

Patients in both arms may also receive artificial tear drops at least twice daily as clinically necessary.

Inclusion criteria:

• Age greater than or equal to 18 years at time of enrollment
• Day 80-120 after first allogeneic stem cell transplant for hematologic malignancies or -marrow failure disorder at time of study enrollment
• Signed informed consent
• Willing to adhere to protocol requirements

Exclusion criteria:

• history of non-compliance
• diagnosis of ocular GVHD at time of study enrollment
• documented dry eye prior to onset of stem cell transplant
• significant non- GVHD ocular problems that precludes participation in study
• life expectancy of lesser than 6 months at time of enrollment (viz. grade 4 acute GVHD, florid progression or relapse of underlying disease)
• history of documented ocular infections prior to stem cell transplant or during transplant (i.e. history of herpetic keratitis)
• females who are pregnant or breastfeeding