Neurodegeneration in Langerhans cell histiocytosis (ND-LCH) is a major clinical issue requiring urgent resolution. Sveijer et al. showed that plasma neurofilament light protein is a promising biomarker for screening patients with ND-LCH and determining the therapeutic effect of a mitogen-activated protein kinase inhibitor. Therefore, this can be a powerful tool for conducting clinical trials for ND-LCH. Commentary on: Sveijer M, von Bahr Greenwood T, Jädersten M, Kvedaraite E, Zetterberg H, Blennow K, et al. Screening for neurodegeneration in Langerhans cell histiocytosis with neurofilament light in plasma. Br J Haematol. 2022;198:722-729.
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