Key result
ALK inhibitors yield robust responses in adult ALK-positive histiocytosis but fail in pediatric CNS disease.
Why the study?
The full clinicopathological features and molecular lineage of ALK-positive histiocytosis had not been fully elucidated.
Case Report (n=4)
No
ALK inhibitors may provide robust responses in adult patients with multisystem ALK-positive histiocytosis, but efficacy in young children with CNS involvement remains poor.
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Hypothesis-generating for ALK inhibitors in adult multisystem disease; leaves open pediatric CNS efficacy.
Liu et al. (2023) conducted a case report in Multisystem ALK-positive histiocytosis (n=4). ALK inhibitors (alectinib, lorlatinib, crizotinib) was evaluated on Clinical response. ALK inhibitors presented robust and durable responses in adult patients with multisystem ALK-positive histiocytosis, but a poor response in a young child with central nervous system involvement.
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