Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria.

Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired clonal disorder of the haemopoietic stem cells for which the only curative treatment is bone marrow transplantation. There are few reports on the use of allogeneic transplantation for PNH, and nearly all of them include only a few patients. Between September 1978 and December 1997, 16 patients underwent marrow transplantation for PNH at the Hospital Saint Louis. The 5-year survival rate for the 16 patients was 58 +/- 13%. Two factors, an absolute neutrophil count >1.0 x 109/l and haemoglobin level >9 g/dl at transplant, were found to be statistically associated with a better outcome.