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Published in 2021 at "Pediatrics"
DOI: 10.1542/peds.2020-0784
Abstract: A toddler presents with chronic transfusion-dependent hemolytic anemia, hepatosplenomegaly, failure to thrive, and developmental delay with dramatic improvement after anakinra administration. PSTPIP1-associated myeloid-related proteinemia inflammatory (PAMI) syndrome is a rare early-onset autoinflammatory disease associated with…
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Keywords:
hemolysis neurologic;
pami syndrome;
neurologic impairment;
disease ... See more keywords