C044 | CORD BLOOD PLATELET CONCENTRATE FOR THE TREATMENT OF OCULAR CGVHD AND SJÖGREN’S SYNDROME: A PROSPECTIVE PILOT STUDY

INTRODUCTION Ocular cGVHD is a frequent, debilitating complication after Allo-SCT, leading to severe dry eye symptoms and reduced quality of life.Current therapies offer limited, inconsistent benefits.A previous study(Zallio et al, BBMT 2016) using autologous platelet lysate eye drops showed promising results.Building on this, we conducted a single-center prospective study to evaluate the safety and efficacy of allogeneic umbilical cord blood-derived platelet concentrate (CBPC) eye drops. MATERIAL AND METHODS This prospective, single-center interventional study enrolled adults with moderate-to-severe keratopathy due to cGVHD or Sjögren’s syndrome.The study (CPCB19) involved topical use of CBPC eye drops for four months(T0–T4).CBPC was obtained from cord blood units excluded from storage due to low cellularity.Primary endpoints included changes in corneal damage (Oxford score) and tear production (Schirmer test -ST- and Tear Break-Up Time tests- BUT).Secondary endpoints were patient-reported symptoms (pain, photophobia, discomfort) assessed via the Ocular Surface Disease Index (OSDI) and Visual Analogue Scale (VAS). RESULTS From June 2019 to June 2023, 34 patients were enrolled(25 with cGVHD, 9 with Sjögren’s syndrome), median age 52 (21–78), 53% male.Among cGVHD patients: 44% had AML, 12% ALL, 12% B-NHL, 12% MM, 8% BPDCN, and 4% MF. Conditioning regimens were myeloablative in 52% and reduced intensity in 48%. At T4, corneal damage significantly improved, with 83.3% of patients showing resolution of epithelial defects by T4 (median Oxford score at T0 was 2, while at T4 it was 0, with a mean difference of 2, p <0,001).Tear production showed a trend toward improvement, although changes were not statistically significant (ST median differences (T0–T4) were 0.30 mm (LE -2.70 to 2.09; p = 0.28) and 0.88 mm (RE -3.71 to 1.94; p = 0.46). BUT increased by 3.25 s (LE 1.24 to 4.99; p = 0.98) and 3.37 s (RE 1.45 to 5.31; p = 0.99), also without statistical significance.Subjective symptom relief was observed in 90% (VAS) (2,95 - 5,30; p <0,001) and 96.7% (OSDI) (34,14 - 55,17; p <0,001). The NIH ocular score improved in 73.9% of cGVHD patients, with 8.7% reaching a score of 0 (p <0,001).Prednisone use decreased: from five patients at T0 to one at T4 (p <0,001). CONCLUSION CBPC eye drops are a promising treatment for dry eye in cGVHD and Sjögren’s syndrome, especially for relieving symptoms. These encouraging results support further investigation in multicenter trials.