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Background: Severe acquired aplastic anemia (SAA) is an uncommon disease of childhood. Patients with SAA receive supportive care with transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be allogenic stem cell transplantation (SCT) from a matched sibling or immunosupressive therapy (IT). Aim: To report the experience in the management of SAA. Patients and methods: Twenty five children with acquired SAA were treated from July 1992 to September 2005. Patients with full matched sibling donors received allogenic SCT after conditioning with a cyclophosphamide containing regimen. The other patients received immune suppression with cyclosporine plus methylprednisolone (n= 18) plus ATG (n=17). All received supportive care until recovery of hematopoietic function. Those who had severe opportunistic infections at diagnosis or did not respond to two cycles of ATG were evaluated for unrelated donor SCT. Results: Seven patients received sibling donor SCT and 18 IT, which was repeated in six. Three patients received mismatched related (1) or unrelated (2) SCT. Nineteen patients survived with a median follow up time of 4 years, 14 with full hematologic recovery. Six patients died: four due to infections after IT or SCT, one due to intracranial hemorrhage and one with secondary myelodysplasia 12 years after IT. Conclusions: Most children with SAA can be treated successfully with sibling donor SCT or IT. Patients without a histocompatible sibling who fail to respond to IS have a worse prognosis

Background: Severe acquired aplastic anemia (SAA) is an uncommon disease of childhood. Patients with SAA receive supportive care with transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be allogenic stem cell transplantation (SCT) from a matched sibling or immunosupressive therapy (IT). Aim: To report the experience in the management of SAA. Patients and methods: Twenty five children with acquired SAA were treated from July 1992 to September 2005. Patients with full matched sibling donors received allogenic SCT after conditioning with a cyclophosphamide containing regimen. The other patients received immune suppression with cyclosporine plus methylprednisolone (n= 18) plus ATG (n=17). All received supportive care until recovery of hematopoietic function. Those who had severe opportunistic infections at diagnosis or did not respond to two cycles of ATG were evaluated for unrelated donor SCT. Results: Seven patients received sibling donor SCT and 18 IT, which was repeated in six. Three patients received mismatched related (1) or unrelated (2) SCT. Nineteen patients survived with a median follow up time of 4 years, 14 with full hematologic recovery. Six patients died: four due to infections after IT or SCT, one due to intracranial hemorrhage and one with secondary myelodysplasia 12 years after IT. Conclusions: Most children with SAA can be treated successfully with sibling donor SCT or IT. Patients without a histocompatible sibling who fail to respond to IS have a worse prognosis

A 28 years old male on chronic hemodialysis for 40 months due to a IgA crescentic glomerulonephritis developed pancytopenia (hematocrit 16%, white blood cell count 3.800 mm³ and platelets 11.000 mm³. The bone marrow aspirate showed erythropoietic hyperplasia. Hemolytic anemia, folate or vitamin B12 deficiency and paroxysmal nocturnal hemoglobinuria were ruled out. Steroids were given with a transient elevation of red cells and platelets, which lasted only for some weeks. Afterwards, intravenous immunoglobulin was given without benefit. Two months after, a bone marrow biopsy and a bone marrow magnetic resonance imaging showed severe aplasia. Cyclosporine was started with a rapid increase in blood cells count. Eight months later, he received a renal transplant from a cadaveric donor. Immunosupression was achieved with cyclosporine, prednisone and mycofenolate mofetil. The patient required hemodialysis for the first three weeks and a mild acute cellular rejection was treated with methylprednisolone. At discharge, 6 weeks later, serum creatinine was 2.4 mg/dl and creatinine clearance 37.6 ml/min. During the first months after transplant, platelet count and hemoglobin decreased and a bone marrow biopsy showed only mild hypoplasia. Four months after renal transplant the hematocrit was 43%, white blood cell count 6.600 mm³ and platelets, 150.000 mm³ and did not change during the first year of follow up (Rev Méd Chile 2004; 132: 989-94

A 28 years old male on chronic hemodialysis for 40 months due to a IgA crescentic glomerulonephritis developed pancytopenia (hematocrit 16%, white blood cell count 3.800 mm³ and platelets 11.000 mm³. The bone marrow aspirate showed erythropoietic hyperplasia. Hemolytic anemia, folate or vitamin B12 deficiency and paroxysmal nocturnal hemoglobinuria were ruled out. Steroids were given with a transient elevation of red cells and platelets, which lasted only for some weeks. Afterwards, intravenous immunoglobulin was given without benefit. Two months after, a bone marrow biopsy and a bone marrow magnetic resonance imaging showed severe aplasia. Cyclosporine was started with a rapid increase in blood cells count. Eight months later, he received a renal transplant from a cadaveric donor. Immunosupression was achieved with cyclosporine, prednisone and mycofenolate mofetil. The patient required hemodialysis for the first three weeks and a mild acute cellular rejection was treated with methylprednisolone. At discharge, 6 weeks later, serum creatinine was 2.4 mg/dl and creatinine clearance 37.6 ml/min. During the first months after transplant, platelet count and hemoglobin decreased and a bone marrow biopsy showed only mild hypoplasia. Four months after renal transplant the hematocrit was 43%, white blood cell count 6.600 mm³ and platelets, 150.000 mm³ and did not change during the first year of follow up (Rev Méd Chile 2004; 132: 989-94