Umbilical cord-derived mesenchymal stem cell therapy for acute Stevens Johnson syndrome/Toxic Epidermal Necrolysis with severe ocular involvement

Acute Stevens–Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) frequently result in severe ocular surface inflammation and long-term vision-threatening sequelae, largely driven by early aqueous deficiency and epithelial disruption. In this preliminary clinical experience, we explored the safety and potential therapeutic benefit of intralacrimal administration of umbilical cord–derived mesenchymal stem cells (UC-MSCs) in three patients (six eyes) with acute-phase SJS/TEN and significant ocular involvement. Each patient received a single bilateral subconjunctival injection of 1×10⁶ UC-MSCs (0.2 mL/eye), with cells prepared under GMP/GTP regulatory standards and fully characterized for phenotypic markers, sterility, viability, and genomic stability. All patients tolerated the procedure without ocular or systemic adverse events. Over one-year follow-up, we observed a meaningful improvement in tear secretion as measured by Schirmer’s test (p = 0.02, ANOVA) and a significant reduction in fluorescein punctate keratopathy (NEI grading, p = 0.002, ANOVA). Best-corrected visual acuity was preserved in all treated eyes. These clinical outcomes were corroborated by anterior segment imaging, revealing improved epithelial integrity and decreased corneal surface staining. These findings support the concept that UC-MSC therapy may modulate ocular surface inflammation and—importantly—enhance lacrimal function, thereby addressing aqueous deficiency early in the disease course. As a complementary approach to conventional management such as amniotic membrane application and topical anti-inflammatory therapy, MSC-based regenerative treatment may help reduce the progression toward chronic dry eye and structural ocular damage. While preliminary, our experience provides encouraging evidence to justify further controlled studies to better define optimal dosage, timing, and patient selection for UC-MSC therapy in SJS/TEN.