June 2017
Volume 58, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2017
A Randomized Placebo Controlled Double Masked Clinical Trial of Autologous Serum Eye Drops for severe Ocular Graft Versus Host Disease (GVHD)
Author Affiliations & Notes
  • Manuel B Datiles
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Rachel Bishop
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Janine Clayton
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Steven Pavletic
    National Cancer Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Kirsten Williams
    National Cancer Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Daniel Fowler
    National Cancer Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Minoo Battiwalla
    National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Sandra Mitchell
    Clinical Center, National Institutes of Health, Bethesda, Maryland, United States
  • John Barrett
    National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Richard Childs
    National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Cathy Conry Cantilena
    Department of Transfusion Medicine, Clinical Center, National Institutes of Health, Bethesda, Maryland, United States
  • George Grimes
    Pharmacy Department, Clinical Center, National Institutes of Health, Bethesda, Maryland, United States
  • Angel Garced
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Lauren Curtis
    National Cancer Institute, National Institutes of Health, Bethesda, Maryland, United States
  • Awilda Holland
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Frederick Ferris
    NEI-NIH , National Eye Institute-Nat Inst Health, Bethesda, Maryland, United States
  • Footnotes
    Commercial Relationships   Manuel Datiles, None; Rachel Bishop, None; Janine Clayton, None; Steven Pavletic, None; Kirsten Williams, None; Daniel Fowler, None; Minoo Battiwalla, None; Sandra Mitchell, None; John Barrett, None; Richard Childs, None; Cathy Cantilena, None; George Grimes, None; Angel Garced, None; Lauren Curtis, None; Awilda Holland, None; Frederick Ferris, None
  • Footnotes
    Support  This project has been funded in whole or in part with Federal funds from the National Eye Institute, National Institutes of Health, Department of Health and Human Services, under Contract Nos. HHSN263201200001C and HHSN263201700001C.
Investigative Ophthalmology & Visual Science June 2017, Vol.58, 4809. doi:
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      Manuel B Datiles, Rachel Bishop, Janine Clayton, Steven Pavletic, Kirsten Williams, Daniel Fowler, Minoo Battiwalla, Sandra Mitchell, John Barrett, Richard Childs, Cathy Conry Cantilena, George Grimes, Angel Garced, Lauren Curtis, Awilda Holland, Frederick Ferris; A Randomized Placebo Controlled Double Masked Clinical Trial of Autologous Serum Eye Drops for severe Ocular Graft Versus Host Disease (GVHD). Invest. Ophthalmol. Vis. Sci. 2017;58(8):4809.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To determine whether Autologous Serum Eye Drops (ASED) are more effective than control (normal saline) in the treatment of severe chronic Ocular Graft Versus Host Disease (GVHD) in Hematopoietic Stem Cell Transplant (HSCT) patients unresponsive to standard medical treatment.

Methods : We conducted a Phase 2, randomized, double-masked, controlled, cross-over, single-center study to investigate ASEDs in participants with severe chronic ocular GVHD not responsive to standard steroid drops. Participants were randomized to either ASEDs (A) or placebo (saline drops) (S) for 3 months and then participants were switched to the other group for another 3 months (Group 1 A/S, Group 2 S/A). The primary outcome was a ≥50% reduction in composite Oxford punctate keratopathy grading and the NIH/NEI visual analogue scale (VAS) in the study eye from baseline to Month 3. Secondary outcomes measures included Schirmer’s tests without anesthetic, Van Bjisterveld and Oxford grading ocular surface staining tests, Tear Lab tear osmolarity testing, OSDI and NEI VFQ-25 quality of life testing, Lee Chronic GHVD Symptom Bother Self-report Scale, NIH Chronic GVHD Individual Organ Score, visual acuity and intraocular pressure testing.

Results : A total of 18 participants were enrolled, 12 males and 6 females. Nine participants were each enrolled into Groups 1 (A/S) and 2 (S/A). The average age of Group 1 was 45 years old and the average age for Group 2 was 53 years old. Twelve participants (6 in Group 1 (A/S) and 6 in Group 2 (S/A) completed the first 3-month period before the study was prematurely halted due to shutting down of the compounding Pharmacy that was manufacturing the ASED drops. Of the 12, only one participant achieved the primary outcome. This resulted in a success rate of 16.7% for those in Group 1 (A/S) compared with 0% for those in Group 2 (S/A), with a p-value of 1.00 using a 2-sided Fisher’s Exact test.

Conclusions : The study failed to identify a significant difference between Group 1 (A/S) and Group 2 (S/A). Of the 18 enrolled in the study, the ASEDs were well-tolerated by study participants and were not associated with any serious adverse effects. The limitations of this study were the small sample size and the premature shut down of the manufacturing Pharmacy; therefore, larger randomized trials are needed to better assess safety and potential efficacy of ASEDs.

This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.

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