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Cancer Health Center

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


Skeletal involvement

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

  • Curettage only is the recommended therapy, when possible for isolated bone lesions; curettage plus injection of methylprednisolone may be used. Low-dose radiation therapy is effective but its use is limited in pediatric patients to lesions that threaten organ function.[10,11]; [12][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 isolated skull lesions in the mastoid, temporal, or orbital bones is to decrease the chance of developing diabetes insipidus and other long-term problems.[13] Comparison of diabetes insipidus incidence with no systemic therapy (40%) versus 6 months of vinblastine/prednisone (20%) strongly supports treatment of the central nervous system (CNS)-risk bones even when it occurs in a single site.[14] However, the efficacy of therapy and the optimal length of therapy have yet to be proven in a prospective trial.

  • Twelve months of vinblastine and prednisone as per the LCH-III study results: 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.[2,13]
  • There is some controversy about whether systemic therapy is required for the first presentation with unifocal bone LCH even in the CNS risk bones. Ear, nose, and throat surgeons have reported a series of patients with orbital or mastoid lesions who received only surgical curettage.[15] 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 (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 chemotherapy treatment to prevent diabetes insipidus in patients with LCH of the mastoid, temporal, or orbital bones.[16,17] It should be noted, however, that the DAL HX studies used more drugs and treated patients for a duration of 12 months.
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