A Single-Center Pilot Prospective Study of Topical Application of Platelet-Derived Eye Drops for Patients with Ocular Chronic Graft-versus-Host Disease

Abstract

a b s t r a c tOcular involvement of chronic graft-versus-host disease (cGVHD) is a complication that occurs in up to 60% of patients after allogeneic hematopoietic stem cell transplantation. Conventional therapeutic options include medical and surgical procedures that are administered depending on the severity of the condition, but most of them have provided unsatisfactory results and, to date, there is no consensus about treatment. We considered that topical application of a platelet lysate, administered as eye drops, might be considered an alternative worthwhile of investigation to treat ocular surface disorders in patients suffering from cGVHD. Therefore, we conducted a single-center prospective pilot study to assess the efficacy and safety of using eye drops made from reconstituted lysed platelet concentrate. Twenty-six patients with ocular cGVHD were eligible for the study; all but 2 completed their scheduled 1-year treatment and complied with the hematologic and ophthalmic regimen. At their first assessment interviews, after 30 days of treatment, 91% of patients reported an improvement in their symptoms and for 32%, substantive objective differences were measured. Remission of corneal damage was seen for 86% of our cohort, and improved National Institutes of Health scores for 73%, of whom 8% achieved the best score of 0 (ie, nonedry eye). Similar results were seen at later time points. Comparing outcomes for our patient cohort to those determined retrospectively for patients in our institutional database revealed a 5-year overall survival (OS) of 65%. This OS is comparable to patients with limited cGVHD (75%) and is superior to that of patients with nonocular extensive cGVHD or without cGVHD (30% and 59%, respectively) (P ¼ .013). Our results suggest that platelet-derived eye drops are a safe, practical, and well-tolerated therapeutic option that offers substantial benefits for most patients affected by ocular cGVHD at onset. The favorable OS of our patient cohort suggests that this topical therapy, rather than systemic immunosuppression, may be the treatment of choice. Ó

<p>Human Platelets and Derived Products in Treating Ocular Surface Diseases – A Systematic Review</p>

“… 31 Zallio et al similarly used autologous PL eye drops over an extended period (12 months) in patients suffering from chronic GVHD and found that 86% experienced remission of objective corneal signs and 73% had improved subjective national standardized health scores including 8% of patients who described complete resolution of dry eye symptoms. 32 …”

“… Sanchez-Avila et al 2017 35 Retrospective, non-comparative, consecutive study (Level IV) Sjogren syndrome 26 No Immunosafe PRGF: PRGF-Endoret eye drops 4/day 12 months Significant improvement in OSDI and CDVA Adverse events (n = 2) Pezzotta et al 2012 64 Prospective, non-randomized, intervention (Phase II clinical trial) (Level IV) GVHD 23 No Plasma rich in PDGF eye drops (PRGD) 4/day 6 months 73.9% patients showed improvement of symptoms. Zallio et al 2016 32 Single-center, prospective pilot study (Level IV) GVHD 26 No Autologous PL 6/day 12 months 91% patients reported improvement. Remission of clinical signs in 86% eyes.…”

“…31 Zallio et al similarly used autologous PL eye drops over an extended period (12 months) in patients suffering from chronic GVHD and found that 86% experienced remission of objective corneal signs and 73% had improved subjective national standardized health scores including 8% of patients who described complete resolution of dry eye symptoms. 32 Laser in situ keratomileusis (LASIK) surgery is the most commonly performed refractive surgical procedure. 33 Post-LASIK ocular surface syndrome (OSS) is used to describe a spectrum of DED following surgery.…”

Ocular Graft-versus-Host Disease after Hematopoietic Cell Transplantation: Expert Review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation

“…Autologous serum eye drops must be prepared according to local regulations for blood products, are contraindicated for patients with active infections, and can be difficult to prepare in patients with anemia. In a prospective pilot study of 26 patients, platelet-derived eye drops improved ocular symptoms in 91% of patients and improved objective findings in 32% [77].…”

Blood-Based Treatments for Severe Dry Eye Disease: The Need of a Consensus

“…A sample for microbiological investigations is taken at the time of preparation. Next, the final preparation is frozen at −20 °C and stored in the patients’ own freezer for a maximum of 45 days [103,104].…”