A Randomized, Controlled, Double-Masked, Clinical Trial of Autologous Serum Eye Drops for Severe Ocular Chronic Graft-versus-Host Disease (GVHD) in Hematopoietic Stem Cell Transplant (HSCT) Patients
Condition: Graft vs Host Disease
MeSH terms: Bronchiolitis Obliterans Syndrome; Graft vs Host Disease
Intervention: ASEDs; Saline
Intervention description: Experimental Intervention; Control Intervention
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Enrollment: 18.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Graft Versus Host Disease; Hematopoietic Stem Cell Transplant

Summary:

      Some eye diseases can be helped by eye drops made from a person's own blood. These eye drops
      are called autologous serum eye drops, or ASEDs. ASEDs have been studied in only a few people
      with graft vs. host disease (GVHD) affecting the eye and were found to be helpful with few
      side effects.

      The purpose of this study was to determine whether ASEDs are safe and more effective than
      control (normal saline) and can help with eye symptoms in people with severe chronic eye
      GVHD.

      Each participant in this study was to have blood drawn to prepare ASEDs specifically for the
      participant. Each participant was scheduled to receive ASEDs for 3 months and placebo eye
      drops (salt water) for 3 months. Participants did not know when they were receiving the ASEDs
      and when they were receiving placebo eye drops.
    

Eligibility criteria:

        Inclusion Criteria

          1. Participant must be 18 years of age or older.

          2. Participant must understand and sign the protocol's informed consent document.

          3. Participant must have severe ocular Graft-versus-host Disease (GVHD) in one
             (unilateral) or both (bilateral) eyes with the following characteristics in the study
             eye:

               1. Combined score of modified Oxford punctate keratopathy grading and National
                  Institutes of Health (NIH)/National Eye Institute (NEI) visual analogue scale of
                  ≥ 4, and

               2. Composite assessment scale (CAS) score of ≥ 3, and

               3. Schirmer's tear test without anesthesia of ≤ 5 mm, and

               4. Not responsive to standard medical treatment for at least three months prior to
                  randomization. Standard medical treatment includes cyclosporine (Restasis®)
                  ophthalmic emulsion (if tolerated), steroid drops (unless contraindicated),
                  lubricating drops and ointments.

          4. Participant is enrolled in an NIH study at the National Cancer Institute (NCI) or
             National Heart, Lung and Blood Institute (NHLBI).

          5. Participant is willing and able to supply an adequate amount of blood to create the
             autologous serum eye drops (ASEDs).

        Exclusion Criteria

          1. Participant is unable to comply with study procedures or follow-up visits.

          2. Participant is seropositive with positive nucleic acid confirmatory tests for human
             immunodeficiency virus-1/2 (HIV-1/2), human T lymphotropic virus-I/II (HTLV-I/II),
             hepatitis C virus (HCV), and/or hepatitis B virus (HBV) without confirmed history of
             vaccination.

          3. Participant has GVHD proliferative keratopathy, uveitis or GVHD retinopathy in either
             eye.

          4. Participant has an active ocular infection in either eye.

          5. Participant has an allergy to dilating or anesthetic eye drops.

          6. Participant has used Boston Scleral Lens (or similar lenses) in either eye or has used
             ASEDs in either eye within the past two months. Participants who have used the Boston
             Scleral Lens (or similar lenses) or ASEDs in either eye who did not respond to
             treatment and have stopped using them for at least two months are eligible.