Autologous Serum Containing Platelet Derived Growth Factors (PDGFs) as Topical Treatment for “Sicca Syndrome” Associated with Chronic GvHD.

Introduction: Severe “sicca syndrome” after haemopoietic stem cell transplantation impairs the quality of life of transplanted patients and no valid therapeutic options are available. Autologous serum enriched with PDGFs is a new therapeutic option because of its high content in different growth factors and fibronectin able to restore the epithelial integrity of cornea and conjunctive. Herein we present our experience on 16 allotransplanted patients (pts) undergoing extracorporeal photochemotherapy treatment for drug resistant chronic GvHD associated with severe eye dryness. All pts were treated with autologous serum eye drops prepared in our laboratory under gmp conditions. Material and Methods: A peripheral blood sample of 30 ml was obtained from each pt in sterile conditions and centrifuged at 1500 rpm per 15 min. Serum was diluted with sterile saline to obtain a final concentration of 30%. No antibiotic was added. The solution was aliquoted in 1 ml plastic tubes (with the purpose of single use) and freezed at −20°C. The maximum storage time allowed was 30 days. At time of preparation PDGFs (PDGF, TGF b1, VEGF, EGF, IGF) content was assessed by ELISA technique and bacterial and fungal contamination was tested. 16 pts (11 M, 5 F), median age 41 yrs (range: 14–61), median plt count 170x103/mL (range: 110–530), median Hct% 36 (32–46) were enrolled and instructed to instill two autologous serum eye drops per eye four times/day. A written informed consent was always obtained. Schirmer’s test was performed immediately before treatment and bi-monthly. The median follow up was 12,5 months (4–16). Results: 14/16 pts experienced a significative dry eye improvement that was mantained during the course of treatment. The need of topical therapy diminished progressively till two drops per eye once a day. The mean Schirmer’s test value improved from 1.5 to 4.6 mm/5 mins during the follow up. Neither microbial contamination on the samples tested nor oftalmic infectious episodes were documented. Conclusions: Autologous serum eye drops demonstrated to be a valid, safe and low cost new therapeutic option for treatment of “sicca syndrome” associated with chronic GVHD. Preparation under gmp conditions, storage in single use vials and detailed instructions to the patient demonstrated to be an effective approach to avoid the addition of antibiotic and microbial contamination of the solution.