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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 12 months for patients who require chemotherapy for single-system bone, skin, or lymph node involvement. For patients with both high-risk and low-risk multisystem disease, the reactivation rate following 6 months of therapy was as high as 50% on the LCH-I and LCH-II trials.[1]. 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%),[1] the LCH-III trial was designed to give 12 months of chemotherapy for all high-risk multisystem patients and to randomly assign low-risk multisystem patients to either 6 months or 12 months of therapy. In patients with low-risk or high-risk disease who received 12 months of therapy, the reactivation rate was significantly reduced to approximately 30%.[2] The LCH-IV trial will assess whether extending the duration of therapy further will reduce the incidence of reactivations and late effects. Although the LCH-IV study is open in several European centers, it is not open in the United States.

It is preferable that patients with LCH 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 for Pediatric Patients

The standard treatment for 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 that lack the evidence-based strength of the trials.

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

Isolated skin involvement

  • Observation. Observation is recommended for all pediatric patients with skin-only LCH. Therapy is suggested only for symptomatic disease such as extensive rashes, pain, ulceration, or bleeding.
  • Topical steroids. Medium to high potency steroids are effective but the effect is usually not long lasting.[3]
  • Oral methotrexate (20 mg/m2) weekly for 6 months.[4]
  • Oral thalidomide 50 mg to 200 mg nightly.[5] Oral thalidomide may be effective for both pediatric and adult patients.
  • Topical application of nitrogen mustard can be effective for cutaneous LCH that is resistant to oral therapies, but not for disease involving large areas of skin.[6,7]
  • Psoralen and long-wave ultraviolet A radiation (PUVA) and UVB can be effective in skin LCH but its use is limited by the potential for late skin cancers, especially in patients with light skin tones.[8,9]
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