Intended for healthcare professionals

Research Article

Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation.

Br Med J (Clin Res Ed) 1987; 295 doi: (Published 28 November 1987) Cite this as: Br Med J (Clin Res Ed) 1987;295:1375
  1. A Vellodi,
  2. J R Hobbs,
  3. N M O'Donnell,
  4. B S Coulter,
  5. K Hugh-Jones
  1. Bone Marrow Transplant Unit, Westminster Children's Hospital, London.


    Allogenic bone marrow transplantation was carried out on a 3 year old girl with Niemann-Pick disease type B. Successful engraftment was achieved, and nine months after the procedure there was definite clearing of the sphingomyelin from the liver and pronounced clearing from the bone marrow. Any patient with Niemann-Pick disease type B complicated by early or severe hepatic impairment should be considered for bone marrow transplantation.