Int J Stem Cells.  2014 May;7(1):30-32.

Stem Cells and Niemann Pick Disease

Affiliations
  • 1Stamina Foundation, Italy. andolinamarino@gmail.com

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.

Keyword

Niemann Pick; Stem cells; Mesenchymal; Treatment

MeSH Terms

Bone Marrow Transplantation
Humans
Hypersplenism
Niemann-Pick Diseases*
Stem Cells*
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