Allogeneic bone marrow transplantation for Pearson's syndrome

Abstract

Pearson’s syndrome (PS) is a generalized mitochondropathy that occurs in infancy owing to a deletion or duplication of mitochondrial DNA (mtDNA). PS is characterized by hypoplastic macrocytic anemia alone or associated with thrombocytopenia or granulocytopenia, proximal tubular insufficiency, exocrine and endocrine pancreas insufficiency, failure to thrive… (More)
DOI: 10.1038/sj.bmt.1705638

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