P1275: safety and efficacy of human amniotic epithelial stem cells eye drop in ocular chronic graft-versus-host disease

Topic: 22. Stem cell transplantation - Clinical Background: Around 50% of patients suffer from ocular chronic GVHD (ocGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT), which causes serious dryness and visual impairment. Nevertheless, the current clinical therapies for ocGVHD including artificial tears, immunosuppression therapy, or punctual occlusion usually provide limited benefits. Amniotic membrane transplantation (AMT) can significantly alleviate ocular symptoms in ocGVHD patients, but AMT is an invasive surgery and is not applicable to all patients with ocGVHD. Therefore, we designed a clinical trial to demonstrate the safety and efficacy of human amniotic epithelial stem cells (hAESC) eye drops in ocGVHD. Aims: To investigate the safety and efficacy of hAESC therapy for ocGVHD in patients undergoing allo-HSCT. Methods: Register Number: ChiCTR2200057857. The enrolled eyes received 1*106 cells/mL of hAESC eye drops four times a day for a period of one to three cycles of treatment (A cycle was defined as two weeks of continuous administration and two weeks without hAESC eye drops). Additionally, the side effects or adverse events relevant to the topical application of the hAESC eye drops would be recorded during the follow-up. The primary endpoint is the ocular surface disease index (OSDI) score after treatment with hAESC eye drops. Results: A total of 17 post-allo-HSCT ocGVHD patients (39±11y; range 21-56y) involving 34 eyes were enrolled. After treatment with hAESC eye drops, 82.35% of ocGVHD patients demonstrated a significant reduction in OSDI score (P=0.0009). There was also a statistical improvement in Schirmer basal secretion score (P=0.0353), corneal fluorescein staining score (P<0.0001), and fluorescein tear breakup time (P=0.0268). No adverse events associated with hAESC were observed. The results revealed that hAESC eye drops ameliorated ocular symptoms, increased tear secretion, and improved corneal epithelial recovery of ocGVHD. Conclusion: The study suggested that hAESC eye drop is a safe and efficient therapy for ocGVHD patients. Keywords: Chronic graft-versus-host