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Langerhans Cell Histiocytosis Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Childhood LCH

Depending on the site and extent of disease, treatment of Langerhans cell histiocytosis (LCH) may include surgery, radiation therapy, or oral, topical, and intravenous medication. The recommended duration of therapy is 6 months for patients who require chemotherapy for bone, skin, or lymph node involvement. For patients with liver, spleen, bone marrow, or lung involvement, treatment is based upon data from the German-Austrian-Dutch (Deutsche Arbeits-gemeinschaft für Leukaemieforschung und-therapie im Kindesalter [DAL]) Group trials, which treated patients for 1 year and had fewer relapses (29%) than the LCH-I and LCH-II trials, in which patients received 6 months of treatment and had a 50% chance of relapse.[1] Future trials will assess whether even longer duration of therapy will reduce the incidence of reactivations and late effects.

It is preferable that LCH patients be enrolled in a clinical trial whenever possible so that advances in therapy can be achieved more quickly, utilizing evidence-based recommendations and to ensure optimal care. Information about clinical trials for LCH in children is available from the Histiocyte Society Web site.

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Standard Treatment Options by Organ, Site or System Involvement

The standard treatment of LCH is best chosen based on data from international trials with large numbers of patients. However, some patients may have LCH involving only the skin, mouth, pituitary gland, or other sites not studied in these international trials. In such cases therapy recommendations are based upon case series which lack the evidence-based strength of the trials.

Treatment of low-risk disease (single-system or multisystem)

Isolated skin involvement

  • Observation.
  • Topical steroids,[2] although topical steroid creams are rarely effective.
  • Oral methotrexate (20 mg/m2) weekly for 6 months.[3]
  • Oral thalidomide 50 mg to 200 mg nightly.[4]
  • Topical application of nitrogen mustard is effective for cutaneous LCH that is resistant to oral therapies, but not for disease involving large areas of skin.[5,6]
  • Psoralen and long-wave ultraviolet radiation (PUVA).[7]

Single skull lesions of the frontal, parietal, or occipital regions, or single lesions of any other bone

  • Curettage only, curettage plus injection of methylprednisolone, or radiation therapy may be used.[8,9]; [10][Level of evidence: 3iiiA] LCH bone lesions may not need complete excision, as this only increases healing time and the risk of long-term complications.
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