ORIGINAL ARTICLE Mesenchymal stem cell therapy unable to rescue the vision from advanced Behcet’s disease retinal vasculitis: report of three patients Fereydoun DAVATCHI, 1 Behrooz NIKBIN, 2 Hormoz SHAMS, 1 Bahar SADEGHI ABDOLLAHI, 1 Mandana MOHYEDDIN 2 and Farhad SHAHRAM 1 1 Rheumatology Research Center, and 2 Molecular Immunology Research Center, Shariati Hospital, Tehran University for Medical Sciences, Tehran, Iran Abstract Objective: Retinal vasculitis (RV) is the most aggressive lesion of ocular manifestations of Behcet’s disease, seen in 32.1% of patients. Although visual acuity (VA) improves with early and aggressive treatment, in the long run it is seen in only 48% of patients. Mesenchymal stem cell (MSC) transplantation (MSCT) can theoretically reverse the RV process. Patients and Methods: Three patients with advanced RV and very low VA were selected. Eyes selected for MSCT were legally blind (no useful vision) with severe retinal damage due to vasculitis, resistant to combinations of monthly pulse-cyclophosphamide (1000 mg) + azathioprine 2–3 mg/kg/day + prednisolone 0.5 mg/kg/day. After patient signed written consent, 30 mL of bone marrow were taken and cultured for MSC growth. After having enough MSCs in culture (4–5 weeks) and taking into consideration all safety measures, cells were injected in one eye of each patient (approximately 1.8 million MSCs). VA was measured. Disease Activity Index (DAI) was calculated for anterior uveitis (AU), posterior uveitis (PU) and RV. Results: Visual acuity was light perception (LP) for two patients and finger count (FC) for the third. Follow-up at 1, 6 and 12 months were respectively LP/LP/FC at 0.5 m, no-light perception (NLP)/LP/LP, NLP/LP/NLP. Discussion: Results showed a total failure of the procedure, essentially due to the late and advanced state of vasculitis. However, the autoimmune/inflammatory reaction was greatly controlled by the procedure. Conclusion: Earlier cases have to be selected for further trials. Key words: Behcet’s disease retinal vasculitis, mesenchymal stem cell, stem cell transplantation, treatment. INTRODUCTION Behcet’s disease (BD) is a rare disease classified among the vasculitides. It is mainly seen along the Silk Road 1 Considering its rarity, the prevalence is rather high in Iran, 2 around 80 for 100 000 inhabitants. 3 Ocular manifestations are frequent, evident in 56.8% of patients. Retinal vasculitis (RV) is the most aggressive ocular lesion, seen in 32.1% of patients. 4 Although visual acuity (VA) improves with early and aggressive treatment (cytotoxics + corticosteroids), in the long run it effects 47.1% of men and 48.8% of women. In males, 5.6% of eyes lose their useful vision (legal blindness) and 8.6% become blind: a total of 14.2% of eyes. In females, 5.7% lose their useful vision and 6.2% become blind. RV is the most treatment-resistant ocular lesion. The inflammatory index of RV improved in 62% of males and 64.4% of females, by combination of cyto- toxic agents and corticosteroids. 5 The remaining, 1/3 of Correspondence: Professor Fereydoun Davatchi, Rheumatology Research Center, Tehran University of Medical Sciences, Shariati Hospital, Kargar Avenue, Tehran 14114, Iran. Email: fddh@davatchi.net © 2013 The Authors International Journal of Rheumatic Diseases © 2013 Asia Pacific League of Associations for Rheumatology and Wiley Publishing Asia Pty Ltd International Journal of Rheumatic Diseases 2013; 16: 139–147