What is the role of medications in the treatment of Langerhans cell histiocytosis (LCH)?

Updated: Sep 16, 2020
  • Author: Cameron K Tebbi, MD; Chief Editor: Vikramjit S Kanwar, MBBS, MBA, MRCP(UK), FAAP  more...
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The aim of therapy in histiocytosis is to relieve clinical symptoms and prevent complications of the disease. For single-system disease (eg, of the skin or bone), no therapy or only local therapy may be necessary, although further treatment may be needed in certain circumstances. [46, 62, 282, 283, 12]

Localized skin lesions, especially in infants, can spontaneously regress. If treatment is required, topical corticosteroids may be tried. Use of extemporaneously prepared topical 0.02% nitrogen mustard has also been advocated, but concerns of its mutagenic activity, especially in children, should be considered. [283, 74, 284] This agent, initially used systemically, appears to provide rapid response within 10 days [285] with minimal adverse effects, such as contact allergy. Scarring at the site of the lesion is thought to be due to the disease and not therapy. [284] In one study, skin lesions promptly healed in 14 of 22 children, and two had partial responses. [285] Low-dose radiation therapy to the local lesions is often effective but is rarely needed. For unresponsive skin lesions, low-dose mild systemic therapy can be used.

Chemotherapy for multisystemic disease with local or constitutional symptoms is used. [283] Single agents or adjuvant use of several chemotherapeutic agents and/or biologic-response modifiers may be effective. Published therapies include corticosteroids, vinca alkaloids, antimetabolites-nucleoside analogs, immune modulators such as cyclosporine, [286] antithymocyte globulin, [286] biologic-response modifiers such as interleukin (IL)-2 and interferon alpha (INF-alpha), [287] cellular treatment, and exchange transfusion. [288] Most reports of treatment modalities lack controls, with most authors citing the rarity of the disease as justification for this deficiency. [289, 77]

Phase I and II investigational therapies have included the use of single agents such as dabrafenib, GSK2110183, cladribine, [290] imatinib mesylate, [291] vemurafenib, [192] clofarabine, [292] and alemtuzumab [293] or combinations of agents such as alemtuzumab plus cyclophosphamide and busulfan plus fludarabine plus melphalan. Longer duration of therapy appear to improve the outcome in multisystem LCH. [294]

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