Abstract
Purpose :
Stage II (visual axis invaded) limbal stem cell deficiency (LSCD) is one of the most serious of the 6 clinical stages of LSCD. Disease control and reversal are extremely difficult, and the available therapeutic options are all surgical with problematic outcomes. There is currently no recognized pharmacologic treatment option for LSCD. We report for the first-time successful pharmacologic control and reversal of progressive Stage II LSCD with the novel usage of Acthar Gel, a repository corticotropin immunomodulator.
Methods :
Retrospective observational case series
Results :
Four patients with monocular progressive Stage II-A or II-B LSCD were identified. All suffered different underlying etiologies including rosacea keratoconjunctivitis, ocular cicatricial pemphigoid, peripheral ulcerative keratitis, and idiopathic ocular inflammation. All however shared the single common pathologic denominator of poorly controlled ocular inflammation. All had failed traditional supportive and topical and systemic immunosuppressive therapy and were therefore treated adjunctively with either standard (80u 2x/week) or high dose (80u 3x/week) Agthar Gel. All 4 patients responded with LSCD regression to Stage I with a clearing of their visual axis. The mean treatment time to observable therapeutic effect was 5.5 weeks, while the final mean visual improvement was 5 lines.
Conclusions :
Acthar gel appears to represent a novel yet an effective nonsurgical pharmacologic adjunctive option for potential control and reversal of inflammation driven progressive Stage II LSCD. Higher than standard Acthar dosages of 80u 2x/week may be required, and therapeutic benefit may require weeks to months of treatment. These cases may also demonstrate the criticality of inflammation control in an LSCD setting, whether that inflammation is the primary LSCD trigger, secondary and inherent to the LSCD itself, or a self-promoting synergistically destructive combination of both.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.