Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br J Haematol 1999 Feb;104(2):392-6 PMID: 10050724

Pubmed ID



Paroxysmal nocturnal haemoglobinuria (PNH) is a rare clonal haematological disorder characterized by intravascular haemolysis and increased risk of thrombosis. PNH is associated with bone marrow failure syndromes including aplastic anaemia, myelodysplasia and leukaemia. Bone marrow transplants are sometimes used to treat PNH, but small series and reporting biases make assessment of transplant outcome difficult. The outcome of 57 consecutive allogeneic bone marrow transplants for PNH reported to the International Bone Marrow Transplant Registry (IBMTR) between 1978 and 1995 was analysed. The 2-year probability of survival in 48 recipients of HLA-identical sibling transplants was 56% (95% confidence interval 49-63%). Two recipients of identical twin transplants remain alive 8 and 12 years after treatment. One of seven recipients of alternative donor allogeneic transplants is alive 5 years after transplant. The most common causes of treatment failure were graft failure and infections. Our results indicate that bone marrow transplantation can restore normal bone marrow function in about 50% of PNH patients.

Author List

Saso R, Marsh J, Cevreska L, Szer J, Gale RP, Rowlings PA, Passweg JR, Nugent ML, Luzzatto L, Horowitz MM, Gordon-Smith EC


Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-0032936695   95 Citations

MESH terms used to index this publication - Major topics in bold

Bone Marrow Transplantation
Hemoglobinuria, Paroxysmal
Middle Aged
Survival Analysis
Transplantation, Homologous
Treatment Outcome
Twins, Monozygotic
jenkins-FCD Prod-321 98992d628744e349846c2f62ac68f241d7e1ea70