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

Abstract

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.

• Authors: A Vellodi, J R Hobbs, N M O'Donnell, B S Coulter, K Hugh-Jones
• Journal: British medical journal (Clinical research ed.) (Br Med J (Clin Res Ed)) Vol. 295 Issue 6610 Pg. 1375-6 (Nov 28 1987) ISSN: 0267-0623 [Print] England
• PMID: 3121020 (Publication Type: Case Reports, Journal Article)

Topics

• Bone Marrow Transplantation
• Child, Preschool
• Female
• Humans
• Niemann-Pick Diseases (therapy)
• Time Factors
• Transplantation, Homologous