Rapamycin is highly effective in murine models of immune-mediated bone marrow failure

• Published in: Haematologica (Roma) Vol. 102; no. 10; pp. 1691 - 1703
• Main Authors: Feng, Xingmin, Lin, Zenghua, Sun, Wanling, Hollinger, Maile K., Desierto, Marie J., Keyvanfar, Keyvan, Malide, Daniela, Muranski, Pawel, Chen, Jichun, Young, Neal S.
• Format: Journal Article
• Language: English
• Published: Italy Ferrata Storti Foundation 01.10.2017
• Subjects: Anemia, Aplastic - drug therapy; Anemia, Aplastic - immunology; Anemia, Aplastic - metabolism; Anemia, Aplastic - mortality; Anemia, Aplastic - pathology; Animals; Bone Marrow - drug effects; Bone Marrow - immunology; Bone Marrow - pathology; Bone Marrow Diseases - drug therapy; Bone Marrow Diseases - immunology; Bone Marrow Diseases - mortality; Bone Marrow Diseases - pathology; Disease Models, Animal; Epitopes, T-Lymphocyte - immunology; Hemoglobinuria, Paroxysmal - drug therapy; Hemoglobinuria, Paroxysmal - immunology; Hemoglobinuria, Paroxysmal - mortality; Hemoglobinuria, Paroxysmal - pathology; Immunologic Memory; Immunosuppressive Agents - pharmacology; Mice; Pancytopenia - immunology; Pancytopenia - pathology; Signal Transduction; Sirolimus - pharmacology; T-Lymphocyte Subsets - drug effects; T-Lymphocyte Subsets - immunology; T-Lymphocyte Subsets - metabolism; T-Lymphocytes, Regulatory - drug effects; T-Lymphocytes, Regulatory - immunology; T-Lymphocytes, Regulatory - metabolism; Treatment Outcome

Acquired aplastic anemia, the prototypical bone marrow failure disease, is characterized by pancytopenia and marrow hypoplasia. Most aplastic anemia patients respond to immunosuppressive therapy, usually with anti-thymocyte globulin and cyclosporine, but some relapse on cyclosporine withdrawal or re...