Nelson Textbook of Pediatrics 22nd Edition | Shwachman-Diamond syndrome: inherited bone marrow failure syndrome with exocrine pancreatic insufficiency, neutropenia, and metaphyseal dysplasia. Most common gene mutation: SBDS (80-90%). High risk of MDS/AML (monosomy 7). Treatment: pancreatic enzyme replacement, G-CSF for neutropenia, HSCT for marrow failure/MDS.
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