Bone marrow transplantation and immunosuppressive treatment

At the moment, the only form of treatment that can provide a cure for PNH is allogeneic bone marrow transplantation (BMT); when an HLA-identical sibling is available, BMT should be offered to any young patient with PNH, especially if there is severe pancytopenia. Results similar to those for AA can be expected, with long-term disease-free survival ranging from 60% to 100% in the few series that have been published. Recently, BMT from unrelated donors in PNH has been also successful. The majority of patients will not have a potential sibling donor, and some of those who do may not wish to undergo BMT. Given the common pathogenesis of PNH and

Figure 11.4 Successful thrombolytic therapy in PNH. (a) Magnetic resonance venography in a 29-year-old woman who presented with acute Budd-Chiari syndrome, and who also had a thrombus partially occluding the inferior vena cava. (b) Repeat study after a 48-h infusion of tissue plasminogen activator (from R Thertulien et al., unpublished).

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