Which drugs have been proposed for use in the treatment of Langerhans cell histiocytosis (LCH)?

Updated: Jun 12, 2020
  • Author: Christopher R Shea, MD; Chief Editor: William D James, MD  more...
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Other potential treatments include monoclonal antibody targeting with indium–labeled anti-CD1a, cytokine inhibitors, alemtuzumab (anti-CD52), low-dose oral cyclophosphamide, and all-trans retinoic acid. [88, 89, 90] Imatinib has been proposed as a potential therapy for LCH, owing to its ability to inhibit signaling through the platelet-derived growth factor (PDGF), macrophage-colony stimulating factor (MCSF), and c-kit receptors, which are present on the surface of histiocytes. However, clinical application of this therapy has shown mixed efficacy. [91, 92] Finally, the discovery of oncogenic BRAF V600E mutation in more than half of LCH specimens [82] suggests that B-RAF inhibitors such as vemurafenib could be applied to treatment of those cases shown to bear that mutation.

Finally, the MEK1/2 inhibitor cobimetinib has been found to be efficacious in treating a variety of multisystem or refractory histiocytic neoplasms, including LCH. A phase II study of 18 patients had an 89% response rate, with 95% progression-free at 1 year. [87]

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