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Table 1 Overview of open-label phase I/II studies to evaluate mesenchymal stem cells (1 × 10 6 per kg) in treatment-refractory systemic lupus erythematosus

From: Safety and feasibility of umbilical cord mesenchymal stem cells in treatment-refractory systemic lupus erythematosus nephritis: time for a double-blind placebo-controlled trial to determine efficacy

Authors (date) ClinicalTrials.gov protocol number Study design/duration of follow-up Number of patients MSC type/regimen Conditioning Safety: deaths/serious infection PD markera Efficacy
Sun et al. [2] NR Single-arm/ median of 8.25 months (range of 3 to 28 months) 16 (15 SLEN) UC, single infusion CYC 0.8 to 1.8 mg/kg intravenously, 2 to 4 days 0/0 Percentage of Treg cells increased at 3 months (P = 0.03) ‘Decreasing SLEDAI and proteinuriab in all patients’
Liang et al. [3] NCT 00698191 Single-arm/17.2 ± 9.5 months 15 SLEN BM, single infusion Included in protocol, but NR 0/0 Percentage of Treg cells increased at 1 week and 3 and 6 months (P <0.05) ‘Decreasing SLEDAI and proteinuriab in all patients’
Wang et al. [4] NCT 00698191 Unblinded-randomized, 2-arm/12 months 58 (~88% SLEN) BM, UC, single versus 2× (7 days apart) CYC 10 mg/kg per day, day 4, 3, and 2 1/NR ND CR single: 16/30 (53%); double: 8/27 (29%)
Wang et al. [5] NR Single-arm/mean of 27 months 87 (84% SLEN) BM, UC, single infusion, 18 patients retreated at relapse CYC 10 mg/ kg/day, day 4, 3, and 2 5/NR ND CR in 23/83, relapse 10/83
Wang et al. [1] cNCT 01741857 Single-arm 40 (38 SLEN) UC, 2× infusion, 7 days apart) No 3/4 ND MCR 13/PCR 11, 7 relapse
  1. aPharmacodynamic (PD) markers = increased peripheral blood regulatory T (Treg) cells, balanced T helper 1 (Th1)/Th2 cytokines; b[2] baseline (BL) proteinuria 3.1 (±1.2) g/day versus 3 months, 1.3 (±0.9) g/day (P <0.001, n = 15); [3] BL proteinuria 2.7 (±1.2) g/day versus 6 months, 0.9 (±0.8) g/day (P <0.01, n = 12); cprotocol described at ClinicalTrials.gov consistent with this study, but not explicitly noted in report. BM, bone marrow; CR, complete remission; CYC, cyclophosphamide; MCR, major clinical response; MSC, mesenchymal stem cell; ND, not done; NR, not reported; PCR, partial clinical response; SLEDAI, Systemic Lupus Erythematosus Disease Activity Index; SLEN, systemic lupus erythematosus nephritis; UC, umbilical cord.