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BACKGROUND: This study aims to evaluate the clinical efficacy and prognosis of currently popular treatments for patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA). METHODS: Medical records of patients with TD-NSAA were collected from three hospitals and divided into four groups based on their treatment. Treatment efficacy was evaluated at 6 months after treatment, with subgroup analysis based on patients' disease courses. Adverse events and 3-year survival status were also reviewed. RESULTS: A total of 175 patients' records were collected in this study. The analysis of treatment efficacy indicated that the most effective treatment was allo-hematopoietic stem cell transplant (HSCT) with the response rate of 84.62%, which was followed by Anti thymocyte globulin and ciclosporin A (ATG + CsA) (68.52%), CsA + androgen (26.92%), and androgen (0%), respectively. The subgroup analysis showed that for patients with less than 6-month disease courses, ATG + CsA treatment could achieve similar response rate as allo-HSCT (84.00%). Besides, ATG + CsA showed better efficacy than CsA + androgen in all stratified arms (P CONCLUSIONS: Therefore, we concluded that ATG+ CsA could achieve better efficacy to treat TS-NSAA than did CsA+ androgen. Disclosures No relevant conflicts of interest to declare.

The induction of donor T cell anergy to recipient cells for reducing GVHD could be one way of expanding donor candidates for HLA-mismatched hematopoietic stem cell transplantation. The present study was designed to clarify whether recipient cell-specific T cell anergy could be induced by priming donor lymphocytes with recipient monocyte-derived dendritic cells (mo-DCs) irradiated with UV-C. By irradiation of mo-DCs with 100 J/m2 or more UV-C, the expression of DC-associated surface phenotypes such as CD1a, CD54, CD40, CD80, CD83 and CD86 was reduced in one day after irradiation and the effects of UV-C irradiation continued for at least 7 days. By irradiation of mo-DCs with 100 J/m2 or more UV-C, the antigen-presenting ability of both immature and mature mo-DCs, which was examined by 3H-thymidine incorporation assay, was clearly decreased at UV-C dose-dependent manner. Proliferation of CSFE-labeled lymphocytes by the stimulation with immature or mature Mo-DCs was suppressed by 300 J/m2 UV-C irradiation to immature or mature mo-DCs. The response of normal donor 1 lymphocytes, which had been co-cultured with 300–3,000 J/m2 UV-C-irradiated donor 2 immature mo-DCs for 7 days, against mature donor 2 mo-DCs in mixed leukocyte culture (MLC) for 7 days was markedly reduced, compared with the response of the donor 1 lymphocytes co-cultured with non-irradiated donor 2 mo-DCs or UV-C-irradiated mo-DCs derived from a different individual donor 3. CFSE-labeling analysis of donor 1 lymphocytes, which were co-cultured with 300 J/m2 UV-C irradiated donor 2 mo-DCs in the first MLC and then stimulated with donor 2 mature mo-DCs in the second MLC, showed that by stimulation with mature mo-DCs in the second MLC, the proliferation of donor 1 lymphocytes co-cultured with UV-C irradiated donor 2 mo-DCs in the first MLC was less than that of the lymphocytes co-cultured with non-irradiated mature mo-DCs. Flow cytometry analysis of the lymphocytes co-cultured with 300 J/m2 UV-C irradiated mo-DCs using surface CD4/CD25 and cytoplasmic Foxp3 monoclonal antibodies revealed that there was no increase of regulatory T cell population in the lymphocytes co-cultured with UV-C-irradiated immature mo-DCs, compared with the lymphocytes co-cultured with non-irradiated immature mo-DCs. Cell proliferation in allogeneic MLC consisting of lymphocytes as responder cells and mature mo-DCs as stimulator cells was not suppressed by the addition of the lymphocytes co-cultured with UV-C-irradiated immature mo-DCs. The present study demonstrated that recipient cell-specific T cell anergy could be induced by priming donor lymphocytes with UV-C-irradiated immature mo-DCs derived from a recipient and the T cell anergy was not associated with regulatory T cells. These data suggest the applicability of donor graft cells, which have been pre-stimulated with UV-C-irradiated recipient immature mo-DCs for expanding donor candidates for HLA-mismatched hematopoietic stem cell transplantation.

Objective: To explore the feasibility of Chinese herbal medicine in haploidentical related allogeneic bone marrow transplantation (Allo-BMT) without T-cell depletion for the treatment of leukemia. Methods: From 2003 to 2005, four patients with CML and four patients with AML received allo-BMT with grafts from 1 or 3 HLA antigen mismatched related donors. All patients were preconditioned with high dose Ara-c, cyclophosphamide (CY)and total body irradiation (TBI) or Busulfan (Bu). Donors were given G-CSF 250μg/d for seven days prior to marrow harvest. GVHD prevention regimen consisted of CSA, MTX, ATG, MMF and CD25-mab. Chinese herbal medicine was given orally to all patients after transplantation according to the theory (syndrome differentiation) of Traditional Chinese Medicine (TCM). One or 2 weeks after transplantation, TCM were given with the purpose to increase Qi and cultivate Yin, strengthen the function of Spleen and Stomach. At the time of bone marrow convalescence, about 1 month post transplantation, different TCM was prescribed for increasing Qi, cultivating Blood, strengthening the function of Kidney, and fasting distillation. Results: All patients were successfully engrafted. The median days of granulocyte >0.5×109/L and platelet >20×109/L were 12 (10–14) and 20(18–25) days, respectively. Seven patients developed grade I-IVGVHD, with two of them advanced to grade IV gut and liver GVHD and died of concurrent infection eventually. Two patients developed hemorrhagic cystitis. Six patients were alive in a disease-free situation after median following-up of 20 (6–36) months. Conclusion: The major histoincompatibility barriers in the haploidentical related donor/recipient allo-BMT could be successfully carried out with G-CSF stimulated donor bone marrow combined with GVHD preservation and Chinese Herbal Medicine. Our patients were treated with TCM to regulate immunity immediately after clearing out of their bong marrow; With this remedy method, we improved the curative effect of bone marrow transplant and this deserves further study with a well designed clinical trial.