Results were encouraging, but not excellent. Improvement of the technique may improve the results.

Aim: Osteoarthritis is a degenerative joint disease characterized by the destruction of joint cartilage. Mesenchymal stem cells (MSCs) are found in low numbers in normal cartilage, mainly in the superficial layer, acting as repairing agents. In OA, MSCs are seen in larger numbers, but act chaotic and are unable to repair the cartilage. The synovial membrane becomes inflamed and interacts with the cartilage. Transplanted MSC have the ability to normalize them, redirecting them to their normal function. In a preliminary study, we showed that MSC could improve knee OA in four patients at 6 months. This report shows their long-term follow-up at 5 years.Methods: One patient was lost to follow-up at 2 years and three were followed for 5 years. They were aged 55, 57, 65 and 54 years, and had moderate to severe knee osteoarthritis. The worse knee of each patient was injected with 8-9 9 10 6 MSC.Results: As previously reported, all parameters improved in transplant knees at 6 months (walking time, stair climbing, gelling pain, patella crepitus, flection contracture and the visual analogue score on pain). Then, they started gradually to deteriorate, but at 5 years they were still better than at baseline. PGA (Patient Global Assessment) improved from baseline to 5 years. The better knee at baseline (no MSC), continued its progression toward aggravation and at 5 years became the worse knee. Conclusion:Transplant knees were all in a rather advanced stage of OA. Earlier transplantation may give better results in long-term follow-up. This is what future studies have to demonstrate.

Summary:This study concerns the effects of several pre-transplant features on outcome for patients with beta thalassemia major who underwent bone marrow transplantation (BMT). Seventy patients with beta thalassemia major underwent bone marrow transplantation during the period 1991-1997 in Shariati Hospital in Tehran, Iran. The survival and rejection curves levelled off at 8 and 18 months after transplantation at 82.6% and 11.4%, respectively. Pre-transplant clinical features (age, serum ferritin, portal fibrosis, hepatomegaly and quality of chelation therapy) were examined for their effects on survival and recurrence of thalassemia in this group of patients who were less than 16 years old. Increasing age, presence of portal fibrosis and increasing serum ferritin were significantly associated with reduced probability of survival (P = 0.0047, P = 0.016 and P = 0.024, respectively). Hepatomegaly and inadequate pretransplant chelation therapy which were documented as poor prognostic factors in previous studies, were not evaluable in this study. We also showed the benefits of transplanting more than 5.5 ؋ 10 8 /kg cells in this group of patients with no increase in complications. Keywords: marrow transplantation; thalassemia; prognostic factors Homozygous ␤-thalassemia is a congenital hemolytic anemia due to a genetic defect in globulin chain synthesis. Thalassemia is probably the most common single gene disorder causing a major public health problem in the world. In the absence of specific gene therapy, allogeneic bone marrow transplantation is the only rational therapeutic modality for eradication of beta thalassemia major.2 The effects of pre-transplant prognostic factors on BMT outcome were reviewed for the first time by Lucarelli et al in a large series of patients who had been transplanted in Pesaro, Italy.3-5 They concluded that for patients under 16 years of age, transplantation of bone marrow offers a high probability of complication-free survival, particularly in the absence of hepatomegaly, portal fibrosis and history of poor chelation therapy. MethodsBetween March 1991 and September 1997, 70 patients between the ages of 2.5 and 16 years underwent BMT in Shariati Hospital in Tehran, Iran. In 67 cases, donor and recipient were genotypically HLA identical siblings and in three cases they were phenotypically identical. Thirty-eight patients were male and 32 female. In the presence of major ABO incompatibility, red cells were removed from donor marrow by sedimentation with hydroxy ethyl starch. The mean number of cells transplanted was 5.5 × 10 8 /kg (s.d. = 1.9). For all patients regardless of age, busulfan was administered orally at a dose of 3.5 mg per kg per day in three divided doses for 4 days (total dose:14 mg per kg). This treatment was followed by 50 mg of cyclophosphamide per kg intravenously on each of 4 successive days. All patients received cyclosporin A and methotrexate (10 mg/m 2 on days 1, 3, 6 and 11) to prevent acute graftversus-host disease. Cyclosporin A was administered intravenously at a...

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.

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