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

• Published in: Haematologica (Roma) Vol. 99; no. 7; pp. 1168 - 1175
• Main Authors: Bueno, Clara, Roldan, Mar, Anguita, Eduardo, Romero-Moya, Damia, Martín-Antonio, Beatriz, Rosu-Myles, Michael, del Cañizo, Consuelo, Campos, Francisco, García, Regina, Gómez-Casares, Maite, Fuster, Jose Luis, Jurado, Manuel, Delgado, Mario, Menendez, Pablo
• Format: Journal Article
• Language: English
• Published: Italy Ferrata Storti Foundation 01.07.2014
• Subjects: Adolescent; Adult; Aged; Anemia, Aplastic - diagnosis; Anemia, Aplastic - etiology; Anemia, Aplastic - immunology; Anemia, Aplastic - metabolism; Antigens, CD34 - metabolism; Case-Control Studies; Cell Differentiation; Cells, Cultured; Child; Coculture Techniques; Female; Fetal Blood - cytology; Graft Survival; Hematopoietic Stem Cell Transplantation; Hematopoietic Stem Cells - cytology; Hematopoietic Stem Cells - metabolism; Humans; Immunomodulation; Immunophenotyping; Male; Mesenchymal Stromal Cells - cytology; Mesenchymal Stromal Cells - immunology; Mesenchymal Stromal Cells - metabolism; Middle Aged; Phenotype; Young Adult
• DOI: 10.3324/haematol.2014.103580
• ISSN: 0390-6078

Aplastic anemia is a life-threatening bone marrow failure disorder characterized by peripheral pancytopenia and marrow hypoplasia. The majority of cases of aplastic anemia remain idiopathic, although hematopoietic stem cell deficiency and impaired immune responses are hallmarks underlying the bone marrow failure in this condition. 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 hematologic malignancies. We investigated whether bone marrow mesenchymal stem cells contribute, directly or indirectly, to the pathogenesis of aplastic anemia. We found 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 maintain their immunosuppressive and anti-inflammatory properties. These data demonstrate that bone marrow mesenchymal stem cells from patients with aplastic anemia do not have impaired functional and immunological properties, suggesting that they do not contribute to the pathogenesis of the disease.