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Novel Use of Acthar Gel for Treatment of Inflammation Driven Limbal Stem Cell Deficiency

John C Affeldt, MD


John C Affeldt, MD



Loma Linda University Eye Institute

Purpose: To document for the first time the successful use of Acthar Gel in the treatment of inflammation driven partial progressive limbal stem cell deficiency (LSCD).


Methods: Retrospective case series.


Results: Three patients with monocular inflammation driven partial progressive LSCD achieved disease stabilization and control if not regression utilizing standard (80u 2x/week) or high dose (80u 3x/week) Acthar Gel. Underlying etiologies included; Case 1- ocular cicitricial pemphigoid, Case 2- peripherial ulcerative keratitis, Case 3- idiopathic bilaterial ocular inflammation. Time from treatment intervention to observable theraputic benefit was 6.7 weeks, 7.7 weeks and 5.3 weeks repectively. No adverse treatment events were reported. In case #3, the opposite eye suffered from long standing total LSCD and demonstrated no clinical benefit from the Acthar intervention.


Conclusion: Acthar Gel may represent a novel but safe and effective adjunctive or alternative treatment for a spectrum of ocular diseases where inflammation is the common denominator associated with development of partial progressive LSCD. Cinically observable benefit may require months versus weeks of treatment, and may require higher dosages than the current ocular disease recommendations of 80u 2x/week. Finally, Acthar Gel does not appear to offer any clinical benefit in eyes already suffering from total LSCD.


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