Dyskeratosis congenita (DC) is a severe inherited disease characterized by a triad of clinical manifestations including abnormal skin pigmentation, nail dystrophy, and mucosal leukoplakia. 1 Bone marrow failure is th...Dyskeratosis congenita (DC) is a severe inherited disease characterized by a triad of clinical manifestations including abnormal skin pigmentation, nail dystrophy, and mucosal leukoplakia. 1 Bone marrow failure is the principal cause of early mortality, together with an increased predisposition to malignancy and fatal pulmonary complications. According to the dyskeratosis congenita registry, a peripheral blood cytopenia of one or more lineages is reported in 93% of patients, with 51% developing pancytopenia before the age of 10 years. 2 In patients with DC, bone marrow failure or bone marrow failure treatment-associated complications account for 67% of total mortality. 3 Therefore, management of bone marrow failure syndrome is crucial in patients with DC.展开更多
There are two arms in the management of aplastic anemia,one allogeneic bone marrow transplantation(BMT) and the other immunomodulation therapy with antilymphocyte globulin(ALG).
Background Immunological sensitization remains a major problem following renal transplantation. There is no consensus for the management of sensitized renal allograft recipients. The patients become tethered to dialys...Background Immunological sensitization remains a major problem following renal transplantation. There is no consensus for the management of sensitized renal allograft recipients. The patients become tethered to dialysis while waiting for compatible donors. This study was designed to evaluate the efficacy and safety of preoperative single- bolus high-dose antithymocyte globulin （ATG） as induction therapy in sensitized renal transplant recipients.Methods A total of 56 patients were divided into two groups according to the level of panel reactive antibody （PRA）： non-sensitized group （PRA〈10%, n=30） and sensitized group （PRA≥ 10%, n=26）. The characteristics of the recipients and donors were comparable between the two groups. Mycophenolate mofetil （MMF, 1 g） or ATG （iv. 9 mg/kg） were given preoperatively in the two groups as induction therapy. After the transplantation, the patients were treated with standard triple therapy regimen consisting of tacrolimus （FK-506） or cyclosporine A, MMF, and prednisolone. Acute rejection （AR） and infection episodes were recorded and renal function was monitored during a 12-month follow-up. Х^2 test and t test were used to analyze the data. Results During the follow-up, 6 patients （20.0%） suffered AR episodes in the non-sensitized group and 4 （15.4%） in the sensitized group （P=0.737）; 8 patients （26.7%） experienced 11 infection episodes （average, 1.4 episodes per infected patient） in the non-sensitized group, and 6 （23.1%） experienced 10 infection episodes （average, 1.7 episodes per infected patient） in the sensitized group （P=0.757, 0.890）. The safety of the drugs, which was assessed by the occurrence of side effects, was comparable between the two groups. The hospital stay was 13-25 days （mean, 16.7±3.3） in the non-sensitized group and 14-29 days （mean, 16.2±3.1） in the sensitized group, respectively （P=0.563）. No delayed graft function （DGF） was observed in all the patients. Both the 12-month actuarial patient and graft survival rates were 100% in the two groups. Conclusion Preoperative single-bolus high-dose ATG is an effective and safe induction therapy yielding acceptable acute rejection rate in sensitized renal transplant recipients.展开更多
文摘Dyskeratosis congenita (DC) is a severe inherited disease characterized by a triad of clinical manifestations including abnormal skin pigmentation, nail dystrophy, and mucosal leukoplakia. 1 Bone marrow failure is the principal cause of early mortality, together with an increased predisposition to malignancy and fatal pulmonary complications. According to the dyskeratosis congenita registry, a peripheral blood cytopenia of one or more lineages is reported in 93% of patients, with 51% developing pancytopenia before the age of 10 years. 2 In patients with DC, bone marrow failure or bone marrow failure treatment-associated complications account for 67% of total mortality. 3 Therefore, management of bone marrow failure syndrome is crucial in patients with DC.
文摘There are two arms in the management of aplastic anemia,one allogeneic bone marrow transplantation(BMT) and the other immunomodulation therapy with antilymphocyte globulin(ALG).
文摘Background Immunological sensitization remains a major problem following renal transplantation. There is no consensus for the management of sensitized renal allograft recipients. The patients become tethered to dialysis while waiting for compatible donors. This study was designed to evaluate the efficacy and safety of preoperative single- bolus high-dose antithymocyte globulin （ATG） as induction therapy in sensitized renal transplant recipients.Methods A total of 56 patients were divided into two groups according to the level of panel reactive antibody （PRA）： non-sensitized group （PRA〈10%, n=30） and sensitized group （PRA≥ 10%, n=26）. The characteristics of the recipients and donors were comparable between the two groups. Mycophenolate mofetil （MMF, 1 g） or ATG （iv. 9 mg/kg） were given preoperatively in the two groups as induction therapy. After the transplantation, the patients were treated with standard triple therapy regimen consisting of tacrolimus （FK-506） or cyclosporine A, MMF, and prednisolone. Acute rejection （AR） and infection episodes were recorded and renal function was monitored during a 12-month follow-up. Х^2 test and t test were used to analyze the data. Results During the follow-up, 6 patients （20.0%） suffered AR episodes in the non-sensitized group and 4 （15.4%） in the sensitized group （P=0.737）; 8 patients （26.7%） experienced 11 infection episodes （average, 1.4 episodes per infected patient） in the non-sensitized group, and 6 （23.1%） experienced 10 infection episodes （average, 1.7 episodes per infected patient） in the sensitized group （P=0.757, 0.890）. The safety of the drugs, which was assessed by the occurrence of side effects, was comparable between the two groups. The hospital stay was 13-25 days （mean, 16.7±3.3） in the non-sensitized group and 14-29 days （mean, 16.2±3.1） in the sensitized group, respectively （P=0.563）. No delayed graft function （DGF） was observed in all the patients. Both the 12-month actuarial patient and graft survival rates were 100% in the two groups. Conclusion Preoperative single-bolus high-dose ATG is an effective and safe induction therapy yielding acceptable acute rejection rate in sensitized renal transplant recipients.
