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Ferrata Storti Foundation, Haematologica, 7(99), p. 1168-1175

DOI: 10.3324/haematol.2014.103580

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Bone marrow mesenchymal stem cells from patients with aplastic anemia maintain functional and immune properties and do not contribute to the pathogenesis of the disease

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

Aplastic anemia is a life-threatening bone marrow failure disorder characterized by peripheral pancitopenia and marrow hipoplasia. The majority of aplastic anemia cases remain idiopathic, and hematopoietic stem cell deficiency and impaired immune responses are hallmark underlying mechanisms causative for the bone marrow failure in aplastic anemia. Mesenchymal stem/stromal cells constitute an essential component of the bone marrow hematopoietic microenvironment because of their immunomodulatory properties and their ability to support hematopoiesis, and they have been involved in the pathogenesis of several hematological malignances. We have addressed whether bone marrow mesenchymal stem cells contribute, directly or indirectly, to the aplastic anemia pathogenesis. Here, we report that mesenchymal stem cell cultures can be established from the bone marrow of aplastic anemia patients and display the same phenotype and differentiation potential as their counterparts from normal bone marrow. Mesenchymal stem cells from aplastic anemia patients support the in vitro homeostasis and the in vivo repopulating function of CD34+ cells, and preserve their immunosuppressive and anti-inflammatory properties. These data demonstrates that bone marrow mesenchymal stem cells from aplastic anemia do not exert impaired functional and immunological properties, suggesting that they do not seem to contribute to the pathogenesis of the disease.