Cyclosporine A for the Prevention of Ocular Graft versus Host Disease in Allogeneic Hematopoietic Stem Cell Transplant Recipients Is Safe and Feasible

Abstract

To evaluate the safety and efficacy of ocular cyclosporine in the prevention of the development of ocular graft versus host disease (oGVHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (AHSCT) in comparison with historic data. Design: We developed a longitudinal, observational, prospective nonrandomized study. We evaluated the feasibility of prophylactic use of topical cyclosporine A (CsA) to prevent or decrease the incidence of oGVHD and compared this with historic data. Methods: Patients undergoing AHSCT were treated with prophylactic topical CsA for 12 months after engraftment, followed by serial ophthalmic evaluations, including the Schirmer test. Results: Twenty patients were included. No serious adverse effects were reported. Poor adherence was documented in 15% of patients. In spite of observing extra-ocular GVHD (acute and chronic GVHD incidence of 50 and 45%, respectively), only 1 in 20 patients developed oGVHD over the 20-month follow-up for the entire cohort. No statistically significant difference was observed in the incidence of oGVHD when compared to a historical cohort. Conclusions: Topical CsA as a prophylactic measure for oGVHD, administered over a period of 1 year after grafting, is safe and feasible and may decrease the incidence of ophthalmic manifestations of GVHD. These findings must be confirmed in a randomized trial.

Long-term clinical outcomes and predictive factors in patients with chronic ocular graft-versus-host disease

“…Several case studies have reported successful treatment using oral cyclosporine for ocular surface inflammatory disorders including limbal stem cell deficiency (LSCD), necrotizing scleritis, severe keratoconjunctivitis, and corneal graft rejection 37 – 41 . Several studies have reported that the initiation of topical cyclosporine in the pre-transplantation phase could reduce the inflammatory response in the lacrimal glands that may be responsible for the development of keratitis sicca 35 , 42 . They proposed that topical cyclosporine treatment in the early phase could achieve greater tissue levels of CsA and possibly forestall inflammatory damage to the lacrimal acini in GVHD 35 .…”

Ocular graft-versus-host disease: An update on immune pathogenesis and therapies

“…oGVHD can be prevented with the use of topical cyclosporine. Cantú-Rodríguez et al 21 reported a decrease in oGVHD cases due to the immunosuppressive properties of cyclosporine, demonstrating its effectiveness as prophylaxis for oGVHD to prevent ocular damage.…”

“…In addition, it was directly effective in improving aqueous tear production, reducing GVHD-ocular surface inflammation, and increasing cell density 27 . Moreover, CsA has been demonstrated to be a feasible prophylactic measure for oGVHD 21 . Dietrich-Ntoukas et al 16 reported the effectiveness of topical CsA when steroids did not work.…”

Involvement of ocular surface in graft‐versus‐host disease: An update from immunopathogenesis to treatment

“…(Dietrich‐Ntoukas et al, 2012) A recent paper showed that topical CsA treatment after HSCT was effective in preventing the onset of ocular complications of GVHD, during a period of 1 year of follow‐up. (Cantu‐Rodriguez et al, 2020) Topical CsA showed also to be effective in controlling ocular inflammation management when steroid does not work (Dietrich‐Ntoukas et al, 2012). Specifically, few case series and two CRT showed the improvement of tear function and ocular signs in moderate to severe dry eye related to cGVHD (Lelli et al, 2006; Sall et al, 2000; Wang et al, 2008).…”