Treatment of bone LCH

Curettage followed by observation, with or without intralesional corticosteroids. As in children, adults with single-bone lesions should undergo curettage of the lesion followed by observation, with or without intralesional corticosteroids.[50] Extensive or radical surgery leading to loss of function and disfigurement is contraindicated at any site, including the teeth or jaw bones.

Systemic chemotherapy. Systemic chemotherapy causes bone lesions to regress. A variety of chemotherapy regimens, including cytarabine and cladribine, have been used in the treatment of a relatively limited number of patients.[51,52] For more information, see the Chemotherapy and radiation therapy for the treatment of other single-system disease and multisystem disease section.

Low-dose radiation therapy. For patients who do not respond to chemotherapy, low-dose radiation therapy may be indicated and should be attempted before any radical surgery. Radiation therapy is also indicated for impending neurological deficits from vertebral body lesions or visual problems from orbital lesions. Two series and a study have reported the following:

Low-dose radiation therapy. For patients who do not respond to chemotherapy, low-dose radiation therapy may be indicated and should be attempted before any radical surgery. Radiation therapy is also indicated for impending neurological deficits from vertebral body lesions or visual problems from orbital lesions. Two series and a study have reported the following:

A German cooperative radiation therapy group reported on a series of 98 adult patients with LCH. Most of the patients (60 of 98) had only bone lesions and 24 had multisystem disease including bone, who were treated with radiation therapy.[53][Level of evidence C3] Of 89 evaluable patients, 77% achieved a complete remission, 9% developed an infield recurrence, and 15.7% (14 of 89) experienced a progression outside the radiation field(s).

Low-dose radiation therapy. For patients who do not respond to chemotherapy, low-dose radiation therapy may be indicated and should be attempted before any radical surgery. Radiation therapy is also indicated for impending neurological deficits from vertebral body lesions or visual problems from orbital lesions. Two series and a study have reported the following:

A retrospective analysis of 80 patients treated with radiation therapy alone reported a complete remission rate of 77% and a partial remission rate of 12.5%. The long-term control rate was 80% in adults. No adverse late effects were reported.[54][Level of evidence C3]

Low-dose radiation therapy. For patients who do not respond to chemotherapy, low-dose radiation therapy may be indicated and should be attempted before any radical surgery. Radiation therapy is also indicated for impending neurological deficits from vertebral body lesions or visual problems from orbital lesions. Two series and a study have reported the following:

A single-institution study included 39 patients with LCH (age range, 1.5–67 years; 24 patients aged >18 years) who received radiation therapy to 46 lesions. The study reported no local recurrences in the 31 bony sites. In comparison, the 3-year freedom from local failure rate was 63% in the 15 nonbone lesions (95% confidence interval, 32%–83%; P = .0008).[55]

Bisphosphonate therapy. Case reports and case series have described the successful use of bisphosphonates, both intravenous pamidronate and oral zoledronate, in controlling severe bone pain in patients with multiple osteolytic LCH bone lesions.[56-58] A multi-institutional review of bisphosphonate therapy in children and adults with LCH found that most adult patients were given oral zoledronic acid, and most pediatric patients were given pamidronate.[59][Level of evidence C3] Because of the increased toxicity of chemotherapy in adults, bisphosphonate therapy could be used before chemotherapy in multifocal bone disease. Response of other organs, such as skin and soft tissue, to bisphosphonate therapy has been reported.[60]

Anti-inflammatory agents with trofosfamide. Another approach using anti-inflammatory agents (pioglitazone and rofecoxib) coupled with trofosfamide in a specific timed sequence was successful in two patients who had disease resistant to standard chemotherapy treatment.[61][Level of evidence C3]