Myelodysplasia and acute myeloid leukemia fifteen years after high-dose cyclophosphamide in a child with severe aplastic anemia
نویسندگان
چکیده
Aplastic anemia (AA) is a rare disorder characterized by suppression of bone marrow function. It can develop as the result of congenital marrow disease and chemical exposure; however, most cases are idiopathic.1 Treatment with immunosuppressive therapy (IST) for patients who do not have an human leukocyte antigen (HLA)-compatible donor relies on the evidence that a deregulated immune system drives T lymphocytes to cytokine-mediated destruction of their own hematopoietic stem cells.1 The majority of these patients respond well to up-front administration of IST, including antithymocyte globulin (ATG) and cyclosporine (CsA), which is successful in around 80%.2 Unfortunately, ATG and CsA can lead to clonal disorders, in particular myelodysplastic syndrome (MDS) and paroxysmal nocturnal hemoglobinuria.3 On the other hand, high doses of cyclophosphamide (HDCY) have been administered as a sole immunosuppressive agent in severe aplastic anemia (SAA), principally in adults, with no
منابع مشابه
Pilot trial of risk-adapted cyclophosphamide intensity based conditioning and HLA matched sibling and unrelated cord blood stem cell transplantation in newly diagnosed pediatric and adolescent recipients with acquired severe aplastic anemia.
BACKGROUND Cyclophosphamide-based conditioning regimens and allogeneic hematopoietic stem cell transplantation (AlloHSCT) from matched related donors (MRD) has resulted in the highest survival rates in children and adolescents with acquired severe aplastic anemia (SAA). Time to transplant has consistently been associated with decreased overall survival. Reduced toxicity conditioning and AlloHSC...
متن کاملHigh-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder that can be treated with bone marrow transplantation, immunosuppressive therapy, and high-dose cyclophosphamide. Here, we report long-term follow-up on 67 SAA patients (44 treatment-naive and 23 refractory) treated with high-dose cyclophosphamide. At 10 years, the overall actuarial survival was 88%, the response rat...
متن کاملAcute myeloid leukemia after intensive immunosuppressive therapy in aplastic anemia.
A 10-year-old boy was admitted with complaints of fever, pallor, fatigue and skin bleeds of 10 days duration and diagnosed as very severe aplastic anemia. He was given intensive immunosuppressive therapy but showed no response to therapy. He later evolved into acute myeloid leukemia. The occurrence of AML is reviewed and possible pathogenesis is discussed.
متن کاملCLINICAL TRIALS AND OBSERVATIONS High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up
Severe aplastic anemia (SAA) is a lifethreatening bone marrow failure disorder that can be treated with bone marrow transplantation, immunosuppressive therapy,and high-dose cyclophosphamide. Here, we report long-term follow-up on 67 SAA patients (44 treatment-naiveand23refractory) treated with high-dose cyclophosphamide. At 10 years, the overall actuarial survival was 88%, the response rate was...
متن کاملALLOGENIC BONE MARROW TRANSPLANTATION IN APLASTIC ANEMIA
Eighteen patients, twelve men and six women, with aplastic anemia underwent allogenic bone marrow transplantation (BMT) from HLA-matched siblings during the period of 1990 to 1996. The conditioning regimen was cyclophosphamide with or without busulfan, depending on the cause of aplasia. Antilymphocyte globulin (ALG) and cyclosporine were used for rejection and acute GVHD prophylaxis, respe...
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