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Safety and efficacy of human amniotic epithelial stem cell eye drops for ocular cGvHD

By Ella Dixon

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Apr 28, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in chronic GvHD.


 

Ocular chronic graft-versus-host disease (ocGvHD) is a severe complication that occurs in 40–60% of patients following allogeneic hematopoietic stem cell transplant.1 Current treatments for ocGvHD can cause side effects including blurred vision, infections, and localized burning, highlighting the need for novel treatments in these patients.2

A study (ChiCTR2200057857) was initiated to assess the safety and efficacy of eye drops containing human amniotic epithelial stem cells (hAESCs), which are characterized by low immunogenicity and a promising safety profile. In total, 20 patients received 1 × 106/mL hAESCs for 2 drops four-times daily in each eye for 2–6 weeks. The primary outcome was safety, as measured by incidence of adverse events. Results were presented by Wang at the 51st Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT).2 

 

Key learnings2

No serious adverse events were reported. No patients experienced hypersensitivity or application site pain; there was one case of mild ocular itching that was managed by discontinuation of the treatment for 1 day. There were no significant changes to intraocular pressure after treatment.
After treatment with hAESCs, the symptoms of dry eye – including stinging, burning, crustiness, and/or redness – were significantly relieved, which was reflected by reduced OSDI and NIH scores. In addition, fluorescein staining showed a reduction in corneal epithelial damage after treatment.
In a mouse model used to assess the mechanism of hAESCs in ocGvHD, similar results were observed. Single cell sequencing revealed that treatment with hAESCs expanded LSC cluster 3 and reduced LSC cluster 2 in the cornea, demonstrating the potential of hAESCs in restoring self-renewal and differentiation of LSCs. 
This study demonstrated that hAESCs are a safe, effective, and non-invasive treatment for ocGvHD. Additional studies are warranted to further investigate the ocGvHD inflammatory microenvironment, particularly the mechanism of fibroblast suppression.

Abbreviations: hAESC, human amniotic epithelial stem cell; LSC, limbal stem cell; NIH, National Institute of Health; ocGvHD, ocular graft-versus-host disease; OSDI, Ocular Surface Disease Index.

References

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