Hematopoietic stem cell transplantation in Niemann–Pick disease type B monitored by chitotriosidase activity

Here, we report a patient with Niemann–Pick disease type B, with early severe onset of disease and pulmonary involvement, treated with hematopoietic stem cell transplant (HSCT) from a bone marrow matched unrelated donor. We confirm that HSCT is feasible and potentially beneficial for patients with s...

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Bibliographic Details
Published inPediatric blood & cancer Vol. 65; no. 2
Main Authors Quarello, Paola, Spada, Marco, Porta, Francesco, Vassallo, Elena, Timeus, Fabio, Fagioli, Franca
Format Journal Article
LanguageEnglish
Published United States Wiley Subscription Services, Inc 01.02.2018
Subjects
Bone marrow
Bone marrow transplantation
Brain diseases
chitotriosidase activity
Genetic disorders
Hematology
hematopoietic stem cell transplantation
Hematopoietic stem cells
Lung diseases
Metabolic disorders
Niemann-Pick disease
Niemann–Pick disease type B
Oncology
Pediatrics
Stem cell transplantation
Stem cells
Transplantation
Transplants & implants
hematopoietic stem cell transplantation
Niemann-Pick disease type B
chitotriosidase activity
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Summary:Here, we report a patient with Niemann–Pick disease type B, with early severe onset of disease and pulmonary involvement, treated with hematopoietic stem cell transplant (HSCT) from a bone marrow matched unrelated donor. We confirm that HSCT is feasible and potentially beneficial for patients with severe phenotype. Noteworthy, we discussed the potential usefulness of the activity of peripheral chitotriosidase for the longitudinal evaluation of HSCT success and effectiveness.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
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ISSN:1545-5009
1545-5017
DOI:10.1002/pbc.26811