Allogeneic mesenchymal stem cell transplantation for lupus nephritis patients refractory to conventional therapy

Clin Rheumatol. 2014 Nov;33(11):1611-9. doi: 10.1007/s10067-014-2754-4. Epub 2014 Aug 14.


Allogeneic mesenchymal stem cell transplantation (MSCT) has been shown to be clinically efficacious in the treatment of various autoimmune diseases. Here, we analyzed the role of allogeneic MSCT to induce renal remission in patients with active and refractory lupus nephritis (LN). This is an open-label and single-center clinical trial conducted from 2007 to 2010 in which 81 Chinese patients with active and refractory LN were enrolled. Allogeneic bone marrow- or umbilical cord-derived mesenchymal stem cells (MSCs) were administered intravenously at the dose of 1 million cells per kilogram of bodyweight. All patients were then monitored over the course of 12 months with periodic follow-up visits to evaluate renal remission, as well as possible adverse events. The primary outcome was complete renal remission (CR) and partial remission (PR) at each follow-up, as well as renal flares. The secondary outcome included renal activity score, total disease activity score, renal function, and serologic index. During the 12-month follow-up, the overall rate of survival was 95 % (77/81). Totally, 60.5 % (49/81) patients achieved renal remission during 12-month visit by MSCT. Eleven of 49 (22.4 %) patients experienced renal flare by the end of 12 months after a previous remission. Renal activity evaluated by British Isles Lupus Assessment Group (BILAG) scores significantly declined after MSCT (mean ± SD, from 4.48 ± 2.60 at baseline to 1.09 ± 0.83 at 12 months), in parallel with the obvious amelioration of renal function. Glomerular filtration rate (GFR) improved significantly 12 months after MSCT (mean ± SD, from 58.55 ± 19.16 to 69.51 ± 27.93 mL/min). Total disease activity evaluated by Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) scores also decreased after treatment (mean ± SD, from 13.11 ± 4.20 at baseline to 5.48 ± 2.77 at 12 months). Additionally, the doses of concomitant prednisone and immunosuppressive drugs were tapered. No transplantation-related adverse event was observed. Allogeneic MSCT resulted in renal remission for active LN patients within 12-month visit, confirming its use as a potential therapy for refractory LN.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adrenal Cortex Hormones / therapeutic use
  • Adult
  • Child
  • Cyclophosphamide / therapeutic use
  • Drug Therapy, Combination
  • Female
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Lupus Nephritis / drug therapy
  • Lupus Nephritis / therapy*
  • Male
  • Mesenchymal Stem Cell Transplantation / methods*
  • Middle Aged
  • Mycophenolic Acid / analogs & derivatives
  • Mycophenolic Acid / therapeutic use
  • Remission Induction
  • Retreatment
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult


  • Adrenal Cortex Hormones
  • Immunosuppressive Agents
  • Cyclophosphamide
  • Mycophenolic Acid