Abstract:
:We conducted a phase II, noncomparative, multicenter study to assess the efficacy and safety of allogeneic bone marrow-derived mesenchymal stromal cells (BM-MSCs) expanded in vitro for patients with aplastic anemia (AA) refractory to immunosuppressive therapy. Seventy-four patients from seven centers received allogeneic BM-MSCs at a dose of 1-2 × 106 cells/kg per week for 4 weeks. Responses were assessed at 0.5, 1, 2, 3, 6, 9, and 12 months after the first cells infusion. Patients with response at 1 month continued to receive four infusions. All patients were evaluable. The overall response rate was 28.4% (95% confidence interval, 19%-40%), with 6.8% complete response and 21.6% partial response. The median times to response of leukocytic, erythrocytic, and megakaryocytic linages were 19 (range, 11-29), 17 (range, 12-25), and 31 (range, 26-84) days, respectively. After median follow-up of 17 months, overall survival was 87.8%. Seven patients developed transitory and mild headache and fever, but no other adverse events were observed. Antithymocyte globulin used in previous treatment and no activated infection throughout treatment were predictors for response. Allogeneic BM-MSCs infusion is a feasible and effective treatment option for refractory AA. The trial was registered at www.clinicaltrials.gov as NCT00195624. Stem Cells Translational Medicine 2017;6:1569-1575.
journal_name
Stem Cells Transl Medjournal_title
Stem cells translational medicineauthors
Pang Y,Xiao HW,Zhang H,Liu ZH,Li L,Gao Y,Li HB,Jiang ZJ,Tan H,Lin JR,Du X,Weng JY,Nie DN,Lin DJ,Zhang XZ,Liu QF,Xu DR,Chen HJ,Ge XH,Wang XY,Xiao Ydoi
10.1002/sctm.16-0227subject
Has Abstractpub_date
2017-07-01 00:00:00pages
1569-1575issue
7eissn
2157-6564issn
2157-6580journal_volume
6pub_type
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