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. 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.
Incidence and Mortality
Estimated new cases and deaths from soft tissue sarcoma in the United States in 2013:
New cases: 11,410.
Soft tissue sarcomas are malignant tumors that arise in any of the mesodermal tissues of the extremities (50%), trunk and retroperitoneum (40%), or head and neck (10%). The reported international incidence rates range from 1.8 to 5 per 100,000 per year.
Risk Factors and Genetic Factors
The risk of sporadic soft tissue sarcomas...
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
Topical steroids, although topical steroid creams are rarely effective.
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).
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]; [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.
Skull lesions in the mastoid, temporal, or orbital bones
The purpose of treating patients with skull lesions in the mastoid, temporal, or orbital bones is to decrease the chance of developing diabetes insipidus and other long-term problems, although the efficacy of this, and the optimal length of therapy, have yet to be proven in a prospective trial.
Twelve months of vinblastine and prednisone (based upon comparative results noted above regarding DAL-HX trials versus the LCH-I and LCH-II studies): Weekly vinblastine (6 mg/m2) for 7 weeks then every 3 weeks for good response. Daily prednisone (40 mg/m2) for 4 weeks then tapered over 2 weeks. Afterward prednisone is given for 5 days at 40 mg/m2 every 3 weeks with the vinblastine injections.
There is some controversy about whether systemic therapy is required for the first presentation with unifocal bone even in the central nervous system (CNS) risk bones. Ear, nose, and throat surgeons have reported a series of patients with orbital or mastoid lesions who received only surgical curettage. None of these patients developed diabetes insipidus. However, when comparing the incidence rates of diabetes insipidus in patients who received little or no chemotherapy (20%–50% incidence of diabetes insipidus) versus diabetes insipidus incidence rates reported by the German-Austrian-Dutch (Deutsche Arbeits-gemeinschaft für Leukaemieforschung und-therapie im Kindesalter [DAL]) Group HX-83 trial (10% incidence of diabetes insipidus in patients treated for LCH), it appears that the weight of evidence from the DAL HX-83 trial supports treatment to prevent diabetes insipidus in patients with LCH of the mastoid, temporal, or orbital bones.[13,14] It should be noted, however, that the DAL HX studies used more drugs and treated for a duration of 12 months. Nonetheless, the CNS study group of the Histiocyte Society believes prevention of the potentially devastating consequences of CNS and endocrine disease with relatively low-toxicity chemotherapy is worthwhile; acknowledging that the overall level of evidence is low (Level of evidence: 3iii) and that prospective trials are needed.