Table 2.
Clinical trials using regenerative medicine for rheumatic diseases.
| Intervention | Comparison | Disease | Study phase | Patient number | Treatment effect | Treatment toxicity | References |
|---|---|---|---|---|---|---|---|
| Autologous PBSCT/BMT | - | SSc | I/II | 57 | Improvement in skin sclerosis at 6–36 months | Treatment-related mortality 8.7% | (22) |
| Autologous PBSCT | IVCY | SSc | II | 19 (10 vs. 9) | Improvement in skin sclerosis and pulmonary function at 12 months (vs. IVCY) | No deaths | (23) |
| Autologous PBSCT | IVCY | SSc | III | 156 (79 vs. 77) | Improvement in skin sclerosis and pulmonary function at 2 years (vs. IVCY), Improvement in event-free survival at 1–10 years (vs. IVCY) | Treatment-related mortality 10.1% at 1 year | (24) |
| Autologous PBSCT | IVCY | SSc | II | 75 (36 vs. 39) | Improvement in the global rank composite score and event-free survival at 54 months (vs. IVCY) | Treatment-related mortality 3% at 54 months and 6% at 72 months | (25) |
| Autologous PBSCT | - | SLE | I/II | 7 | Achievement of clinical remission in all cases, Disappearance of serum anti-dsDNA antibody in all cases within 1 month | One death due to invasive central nervous system aspergillosis at 3 months | (32) |
| Autologous PBSCT | - | SLE | II | 50 | Decrease in disease activity at 6 months to 5 years, Improvement in pulmonary function at 12–60 months | Treatment-related mortality 2% | (33) |
| Autologous PBSCT | - | SLE | II | 32 | Decrease in disease activity at 6 months to 5 years | No treatment-related deaths | (35) |
| Autologous PBSCT (Unmanipulated cells) | Autologous PBSCT (CD34-selected cells) | RA | II | 33 (15 vs. 18) | Decrease in disease activity over a median follow-up of 167 (range 45–374) days (Similar outcomes in both groups) | No deaths | (37) |
| Autologous PBSCT | - | RA | I/II | 14 | Decrease in disease activity at 3–12 months | No deaths | (38) |
| Allogeneic UC-MSCT | - | SLE | I/IIa | 16 | Decrease in disease activity at 1–24 months | No deaths | (70) |
| Allogeneic BM-/UC-MSCT | - | SLE | I/II | 81 | Improvement in renal function and decrease in disease activity at 12 months | No deaths | (71) |
| Autologous SVF transplantation | - | SSc | I | 12 | Improvement in hand disability, pain, Raynaud's phenomenon, finger edema and quality of life at 6 months | No deaths | (76) |
PBSCT, peripheral blood stem cell transplantation; BMT, bone marrow transplantation; SSc, systemic sclerosis; IVCY, intravenous cyclophosphamide; SLE, systemic lupus erythematosus; RA, rheumatoid arthritis; UC-MSCT, umbilical cord-derived mesenchymal stem cell transplantation; BM-MSCT, bone marrow-derived mesenchymal stem cell transplantation; SVF, stromal-vascular fraction.