calcimycin and Anemia--Aplastic

Aplastic anemia (AA) is a rare but potentially life-threatening disease. There is a need for the development of new, more effective and less toxic therapies for treating AA. The safety and efficacy of an immune cell-based therapy for AA was examined.. Thirty-one patients with idiopathic AA received intravenous infusions of ex vivo-activated autologous and allogeneic immune cells at least once a week. Response to therapy was assessed by symptoms, transfusion dependency, blood counts, bone marrow biopsy and survival.. Of the 31 patients, 25 (81%) had either complete (11, 35%) or partial (14, 45%) responses, while six (19%) showed no response to the therapy. The overall survival rates at 3 years were 90%.. The therapy described appears to be safe and effective. The data from this pilot study suggest that a larger, controlled study is warranted.

Topics: Adolescent; Adult; Anemia, Aplastic; Benzene; Calcimycin; Cell Count; Child; Disease Progression; Female; Follow-Up Studies; Hematopoiesis; Humans; Immunotherapy, Adoptive; Interleukin-2; Leukocytes, Mononuclear; Lymphocyte Activation; Male; Middle Aged; Pilot Projects; Treatment Outcome

Aplastic anemia is a bone marrow failure disorder characterized by pancytopenia and a hypocellular marrow. Benzene is one of the etiologic agents capable of inducing the disease. With modest to severe aplastic anemia, one previously untreated patient and 13 patients who had failed immunosuppressive therapy were studied. Peripheral blood mononuclear cells from patients were expanded in vitro with a combination of cytokines and a calcium-mobilizing agents for 2 days, and the activated cells were infused intravenously once a week. In some cases, we used allogenic leukocytes instead of autologous cultured lymphocytes. After 6-35 weeks of the treatment, all patients had multilineage responses to this therapy and achieved complete disease remission, defined as normal blood count, independence from transfusion, and normal bone marrow histology. The therapy was safe and well tolerated with minimal side effects. The cultured cells produced interleukin-1 and induced immune responses in vivo. Serum interleukin-2 and interferon- gamma were detected following cell infusion. Finally, patients had sustained responses to the therapy and no relapse was found up to 18 months after cellular therapy.

Topics: Adult; Anemia, Aplastic; Benzene; Blood Cell Count; Blood Transfusion; Bone Marrow; Calcimycin; CD4-CD8 Ratio; Enzyme-Linked Immunosorbent Assay; Female; Granulocyte-Macrophage Colony-Stimulating Factor; Hemoglobins; Humans; Immunotherapy; Interferon-gamma; Interleukin-1; Interleukin-2; Leukocytes, Mononuclear; Male; Occupational Exposure; Recurrence; Remission Induction; Stem Cell Transplantation