Objectives: The experience with bone marrow transplantation (BMT) from matched unrelated donors (MUD) for paroxysmal nocturnal hemoglobinuria (PNH) is limited and optimal preparative regimen has been not established.
Methods: We report first two MUD BMTs for patients with PNH in Poland. Preparative regimen consisted of Treosulfan, Fludarabine and Thymoglobulin. We also present the review of published reports on allogeneic transplantations for PNH and discuss important transplant-related issues.
Results: Both patients are alive and are doing well over 12 and over 4 months following BMT. Regeneration is complete with full 100% donor chimerism and the eradication of PNH clone.
Conclusions: MUD BMT is an effective treatment for PNH. Treosulfan, Fludarabine and Thymoglobulin treatment can be safely and effectively used for conditioning in PNH.