Demographic and clinical features of patients with Limbal Stem Cell Deficiency (LSCD) enrolled in HOLOCORE, a European prospective trial

Abstract

Purpose : Efficacy and safety of Autologous Cultivated Limbal Stem Cell Transplantation (ACLSCT; Holoclar®, Chiesi Farmaceutici SpA), for the treatment of unilateral or partial bilateral moderate to severe LSCD due to ocular burns, is under further investigation in the confirmatory HOLOCORE post-approval study. We report demographic and clinical features of the enrolled population

Methods : HOLOCORE is a multinational, multicenter, prospective, open-label, uncontrolled clinical trial. Seventy-eight patients in 8 different European countries were enrolled from October 2015 to May 2018. Key inclusion criteria were a diagnosis of unilateral or partial-bilateral LSCD secondary to physical or chemical ocular burns, superficial neo-vascularization in at least 2 corneal quadrants with evidence of central corneal involvement, time from burn of at least 24 months (12 for minors) as a proxy for disease stability, and severe impairment in best corrected visual acuity (BCVA) defined by a score ≤1/10 on the Snellen chart

Results : Seventy-five adult patients (aged ≥18) and 3 minors were recruited, and most (83%) were male (mean 41.6; SD 16.0). The majority had a history of previous surgical procedures (76%) or concomitant ocular disease (20%). Chemical burns were the most frequent cause of LSCD (82% of the cases) with the majority of them related to alkaline agent injuries (66%). Most patients (69 %) presented with severe LSCD with 4 quadrants (Q) involved and 31.2% were moderate (2-3Q involved). Only 6% of the patients presented with bilateral damage. All subjects reported either photophobia, pain or burning, mainly classified as mild to moderate. Forty-one percent of the patients presented a BCVA corresponding to hand movements, 27% to counting fingers and 6% to light perception. Pannectomy performed in 35 patients at the time of ACLSCT revealed stromal scarring in 32 of them (91.4%) who underwent implantation up to September 2018

Conclusions : Most of the patients enrolled in HOLOCORE had a history of previous surgical procedures and were affected by severe LSCD with significant impairment to vision. Total pannectomy performed at the time of ACLSCT revealed stromal scarring in most of the cases suggesting the need for future corneal transplantation, a procedure that may be considered after complete regeneration of the corneal epithelium with ACLSCT

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.