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Phase II for Allogeneic BM-MSC Treatment of Aplastic Anemia!

Review of “Allogeneic Bone Marrow-Derived Mesenchymal Stromal Cells Expanded In Vitro for Treatment of Aplastic Anemia: A Multicenter Phase II Trial” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Aplastic anemia is a bone marrow failure syndrome characterized by the exhaustion of hematopoietic precursors, which, in turn, leads to a reduction in the number of red blood cells, white blood cells, and platelets. Current treatment strategies include immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT).

Researchers from the laboratory of Yang Xiao (Hainan Cancer Hospital, Haikou, Hainan, PR China) knew that bone marrow-derived mesenchymal stromal cells (BM-MSCs) could suppress the immune system and promote hematopoiesis; so, do BM-MSCs represent an attractive treatment therapy for aplastic anemia patients? 

A previous phase I/II trial discovered that around one-third of patients who did not respond to IST achieved complete response (CR) or partial response (PR) to ex vivo expanded allogeneic BM-MSCs treatment [1]. In their new STEM CELLS Translational Medicine article, Pang et al. now report back on their a phase II, non-comparative, open-label, multicenter study. So just what did the team find [2]?

The trial enrolled 74 patients at seven centers to receive BM-MSC infusions over a 4-week period, with responses recorded up to a year after the first cell infusion. Results demonstrated an overall response rate of 28.4% and median times of leukocytic, erythrocytic, and megakaryocytic lineage responses of 19, 17, and 31 days, respectively. Furthermore, the authors noted an overall survival of 87.8% (at 17 months), no serious adverse effects, and the utility of anti-thymocyte globulin and no activated infection as predictors for response. 

MSCs represent a cheaper and safer option for aplastic anemia treatment and, given the similar response rate of MSCs with other methods, the authors suggest that their strategy may be successful. Moving forward, the team now hope to explore additional means to improve this strategy, including refining the in vitro culture of MSCs and altering MSC dosage to enhance response rates.

Another encouraging trial result; stay tuned to the Stem Cells Portal to keep up to date with this interesting new treatments strategy for this bone marrow failure disorder.

Furthermore, see the Press Release associated with this study!


  1. Xiao Y, Jiang ZJ, Pang Y, et al. Efficacy and safety of mesenchymal stromal cell treatment from related donors for patients with refractory aplastic anemia. Cytotherapy 2013;15:760-766.
  2. Pang Y, Xiao H-W, Zhang H, et al. Allogeneic Bone Marrow-Derived Mesenchymal Stromal Cells Expanded In Vitro for Treatment of Aplastic Anemia: A Multicenter Phase II Trial. STEM CELLS Translational Medicine 2017;6:1569-1575.