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Bone Marrow Transplantation and Mesenchymal Stem Cells in Niemann Pick A Disease

Marino Andolina

A patient suffering from Niemann Pick A Disease, underwent an haploidentical bone marrow transplantation, which was then followed by intrathecal and I.V injections of mesenchymal cells.
Results: While the outcome of the bone marrow transplantation was a complete failure, after one month after from the treatment with the mesenchymal cells the patient improved from the psychomotor and from the parenchymal storage perspective. Hypersplenism was solved and platelets rose quickly from 20,000 to 120,000/ microliter. After the discontinuation of the therapy the disease slowly relapsed; however the patient is alive at the age of 8 years, while the survival of this disease does not exceed 3 years.
Conclusions: A bone marrow transplantation resulted ineffective while cellular therapy should be considered as a possible choice for of treatment of for Niemann Pick disease.