A Mortal Complication in a Case with Mucopolysaccharidosis type I Following Hematopoietic Stem Cell Transplantation: Pulmonary Haemorrhage
- 1 January 2021
- journal article
- research article
- Published by Logos Medical Publication (Logos Yayincilik Tic. A.S.) in Journal of Dr Behcet Uz Children s Hospital
- Vol. 11 (2), 198-201
- https://doi.org/10.5222/buchd.2021.26539
Abstract
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease due to mutations within the gene IDUA encoding the "alpha-L-iduronidase". The clinical manifestations concern multisystemic involvement. There are two disease modifying therapies, enzyme replacement therapy and haematopoietic stem cell transplantation (HSCT). Pulmonary haemorrhage (PH) is a rare complication of HSCT and the case was presented with the reason that the related reports were few in MPS I.Keywords
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