TAILIEUCHUNG - Chapter 108. Hematopoietic Cell Transplantation (Part 8)

Aplastic Anemia Transplantation from matched siblings after a preparative regimen of highdose cyclophosphamide and antithymocyte globulin can cure up to 90% of patients | Chapter 108. Hematopoietic Cell Transplantation Part 8 Aplastic Anemia Transplantation from matched siblings after a preparative regimen of highdose cyclophosphamide and antithymocyte globulin can cure up to 90 of patients 40 years with severe aplastic anemia. Results in older patients and in recipients of mismatched family member or unrelated marrow are less favorable therefore a trial of immunosuppressive therapy is generally recommended for such patients before considering transplantation. Transplantation is effective in all forms of aplastic anemia including for example the syndromes associated with paroxysmal nocturnal hemoglobinuria and Fanconi s anemia. Patients with Fanconi s anemia are abnormally sensitive to the toxic effects of alkylating agents and so less intensive preparative regimens must be used in their treatment Chap. 102 . Hemoglobinopathies Marrow transplantation from an HLA-identical sibling following a preparative regimen of busulfan and cyclophosphamide can cure 70-90 of patients with thalassemia major. The best outcomes can be expected if patients are transplanted before they develop hepatomegaly or portal fibrosis and if they have been given adequate iron chelation therapy. Among such patients the probabilities of 5-year survival and disease-free survival are 95 and 90 respectively. Although prolonged survival can be achieved with aggressive chelation therapy transplantation is the only curative treatment for thalassemia. Transplantation is being studied as a curative approach to patients with sickle cell anemia. Two-year survival and disease-free survival rates of 90 and 80 respectively have been reported following matched sibling transplantation. Decisions about patient selection and the timing of transplantation remain difficult but transplantation represents a reasonable option for younger patients who suffer repeated crises or other significant complications and who have not responded to other interventions Chap. 99 . Other .

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