Stem Cells and Niemann Pick Disease.
- Author:
Marino ANDOLINA
1
Author Information
1. Stamina Foundation, Italy. andolinamarino@gmail.com
- Publication Type:Retracted Publication ; Original Article
- Keywords:
Niemann Pick;
Stem cells;
Mesenchymal;
Treatment
- MeSH:
Bone Marrow Transplantation;
Humans;
Hypersplenism;
Niemann-Pick Diseases*;
Stem Cells*
- From:International Journal of Stem Cells
2014;7(1):30-32
- CountryRepublic of Korea
- Language:English
-
Abstract:
BACKGROUND AND OBJECTIVES: Niemann Pick A disease causes a progressive accumulation of sphyngomyelin in several organs and the survival of the patients is usually limited to three years. We describe the outcome of a patient suffering from Niemann Pick A disease, who first underwent an haploidentical bone marrow transplantation, and then intrathecal and I.V injections of mesenchymal cells. METHODS AND RESULTS: While the outcome of bone marrow transplantation was a complete failure, one month after the treatment with the mesenchymal cells the patient improved from the psychomotor and the parenchymal storage perspective. When hypersplenism was solved platelets rose quickly from 20,000 to 120,000/microliter. CONCLUSIONS: Therefore cellular therapy should be considered as a possible choice of treatment of NPA disease.
