Allogenic mesenchymal stem cells transplantation in refractory systemic lupus erythematosus: a pilot clinical study

Ann Rheum Dis 2010;69:1423–1429. doi:10.1136/ard.2009.123463 1423 ABSTRACT Objective To determine the safety and effi cacy of allogeneic mesenchymal stem cell transplantation (MSCT) in refractory systemic lupus erythematosus (SLE). Methods A total of 15 patients with persistently active SLE underwent MSCT. Outcome was evaluated by changes in the SLE disease activity index (SLEDAI), serological features (anti-nuclear antibodies and antidouble-stranded DNA (anti-dsDNA)), renal function and percentage of peripheral blood regulatory T cells. Results From 11 March 2007 to 4 November 2008, 15 patients with persistently active SLE were enrolled and underwent MSCT. The mean follow-up period was 17.2±9.5 months. A total of 13 patients have been followed for more than 12 months. All patients clinically improved following treatment with mesenchymal stem cells with a marked decrease in the SLEDAI score and 24 h proteinuria. At 12-month follow-up, SLEDAI scores decreased from 12.2±3.3 to 3.2±2.8 and proteinuria decreased from 2505.0±1323.9 to 858.0±800.7 mg/24 h (all p<0.05, by paired t test, n=12). At 1-year follow-up in 13 patients, 2 had a relapse of proteinuria, while the other 11 continue to have decreased disease activity on minimal treatment. Anti-dsDNA levels decreased. Improvement in glomerular fi ltration rate was noted in two patients in which formal testing was performed. Non-renal-related manifestations also improved signifi cantly. No serious adverse events were reported. Conclusion Allogeneic MSCT in patients with refractory lupus resulted in amelioration of disease activity, improvement in serological markers and stabilisation of renal function. MSCT appears benefi cial in treatment of patients with SLE refractory to conventional treatment options.