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Langerhans cell histiocytosis (LCH) is a rare hematologic neoplasm characterized by a clonal proliferation of Langerhans-like cells. LCH affects all ages and in children with a frequency ranging from 4.1 to 8.9/million/year with an increased incidence in children <1 year of age. Infantile LCH refractory to frontline therapy is difficult to treat but targeted therapies in patients with BRAF mutations are promising. We treated an infant of refractory multisystem LCH with risk organ involvement with MAP kinase inhibitor (trametinib) with excellent outcomes.
Khandelwal et al. (Sat,) studied this question.
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