Multiple bone lesions in combination with skin, lymph node, or diabetes insipidus (low-risk multisystem LCH)

Chemotherapy (vinblastine and prednisone in combination). Based on the results of the randomized HISTSOC-LCH-III (NCT00276757) trial, the same chemotherapy regimen of vinblastine and prednisone, as described above, is used for 12 months. Patients without risk-organ involvement who were randomly assigned to receive 12 months of treatment with vinblastine/prednisone had a lower 5-year reactivation rate (37%) than did patients who received only 6 months of treatment (54%; P = .03) and patients treated with historical 6-month schedules (52% [HISTSOC-LCH-I] and 48% [HISTSOC-LCH-II]; P < .001). Most disease reactivations were in bone, skin, or other non-risk locations.[29]Patients with low-risk multisystem LCH have a survival rate of almost 100%, but reactivations were shown to be major risk factors for significant late effects on the DAL and Histiocyte Society trials.[29,76]

Chemotherapy (other regimens). Other chemotherapy regimens have also been effective, including the following:

Chemotherapy (other regimens). Other chemotherapy regimens have also been effective, including the following:

Vincristine, cytarabine, and prednisone in combination.[108][Level of evidence C2] This combination has proven effectiveness as frontline or salvage therapy. However, prednisone is now given for a much shorter time than was originally published (52 weeks): 4 weeks at 40 mg/m2 then tapered to 20 mg/m2 by week 6 during the induction phase, and for 5 days every 3 weeks at 20 mg/m2 with a single dose of vincristine and 5 days of cytarabine during maintenance.

Chemotherapy (other regimens). Other chemotherapy regimens have also been effective, including the following:

Cladribine. Cladribine given at 5 mg/m2 per day for 5 days every 3 weeks for two to six cycles can be an effective salvage therapy for recurrent bone or low-risk multisystem disease.[109][Level of evidence C2] More than six cycles is not recommended because of the risk of cumulative cytopenias.

Bisphosphonate therapy. Bisphosphonate therapy can also be effective for treating LCH bone lesions.[110,111][Level of evidence C2] A nationwide survey from Japan described 16 children treated with bisphosphonates for bone LCH. None had risk-organ disease. Most patients received six cycles of pamidronate at 1 mg/kg per course given at 4-week intervals. In 12 of 16 patients, all active lesions including skin (n = 3) and soft tissues (n = 3) resolved. Eight patients remained disease free at a median of 3.3 years.[112] Other bisphosphonates such as zoledronate have been used to successfully treat bone LCH.[113]Although bisphosphonates are used for bone LCH, some publications report response in other organs, such as skin.[111,112]