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

Vanderbilt-Ingram Cancer Center164 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

164

Conditions

Chronic Myeloproliferative Disorders

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (5)

Stanford University

Palo Alto, California, United States

Norwestern Memorial Hospital

Chicago, Illinois, United States

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

M. D. Anderson Cancer Center

Houston, Texas, United States

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Outcomes

Primary Outcomes

Number of Patients That Develop Ocular GVHD While on Study in the Two Arms (Ocular Cyclosporine (Restasis) vs. Placebo)

Data analysis will be performed on an intention-to-treat basis. Logistic regression will be used to estimate the odds ratio and 95% confidence interval of the two treatment groups with the adjustment of baseline covariates. Outcome comparisons for categorical/dichotomous variables will be assessed by 2 test or Fisher's exact test where expected cell frequencies were \<5; continuous variables will be compared by X/2 test or by Mann-Whitney U test where the data are strongly skewed.

Time frame: Up to 2 years after transplantation

Secondary Outcomes

Numerical Score of Ocular Surface Disease Index (OSDI) and Development or Lack of Ocular GVHD (by Ophthalmologic Examination) as Measured by Odds Ratio in a Linear Regression Model.

OSDI is a patient reported questionnaire consisting of 12 questions which are scored from 0 to 4. The total scored is computed and depending on the number of questions answered scores are computed which are then categorized into mild, moderate or severe dry eye symptoms. This tool should adequately reflect the morbidity of the dry eye symptoms.

Time frame: 1 year after transplant