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

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Vertebral or femoral bone lesions at risk for collapse

  • A single vertebral body lesion without soft tissue extension to the extradural space may be observed only.
  • Low-dose radiation therapy may be used to try to promote resolution in an isolated vertebral body lesion or a large femoral neck lesion at risk for fracture, where chemotherapy is not usually indicated (single bone lesion). Despite the low dose required (700-1,000 cGy), radiation therapy should be used with caution in the area of the thyroid gland, brain, or any growth plates.[18]
  • Patients with soft tissue extension from vertebral lesions are often treated successfully with chemotherapy,[19][Level of evidence: 3iiDiii] but prolonged therapy does not appear to be needed beyond the period required to reduce the mass and any risk to the spinal cord. The risk of reactivation of a single bone lesion was only 9% in one large retrospective series.[20]
  • When instability of the cervical vertebrae and/or neurologic symptoms are present, bracing, or rarely, spinal fusion may be needed.[21] Patients with soft tissue extension from the vertebral lesions are often treated successfully with chemotherapy.[19][Level of evidence: 3iiDiii]

Multiple bone lesions (single-system multifocal bone)

  • The most commonly used systemic chemotherapy regimen is the combination of vinblastine and prednisone. Based on the results of the HISTSOC-LCH-III trial, 12 months of treatment with weekly vinblastine (6 mg/m2) for 7 weeks then every 3 weeks is used for good responders.[2] Prednisone (40 mg/m2) is given daily for 4 weeks then tapered over 2 weeks. Afterwards prednisone is given for 5 days at 40 mg/m2 every 3 weeks with the vinblastine injections. A short (<6 months) treatment course with only a single agent (e.g., prednisone) is not sufficient, and the number of relapses is higher. A reactivation rate of 18% was reported with a multidrug treatment regimen that was used for 6 months versus a historical reactivation rate of 50% to 80% with surgery alone or with a single-drug treatment regimen.[22]

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

  • Vinblastine and prednisone in combination. Based on the results of the randomized HISTSOC-LCH-III 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 12 months of 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% [LCH-I] and 48% [LCH-II]; P < .001). Most disease reactivations were in bone, skin, or other nonrisk locations.[2]
  • Other chemotherapy regimens have also been effective, including the following:
    • Vincristine, cytosine arabinoside, and prednisone in combination.[23] This combination has been proven to be an effective frontline or salvage therapy. However, prednisone is given for a much shorter duration than was originally published; currently, prednisone is given for 4 to 6 weeks during the induction phase and then for 5 days every 3 weeks with a single dose of vincristine and 5 days of cytosine arabinoside during maintenance.
    • Cladribine. Cladribine given at 5 mg/m2 /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. More than six cycles is not recommended because of the risk of cumulative cytopenias.
    • Pamidronate can also be effective for treating LCH bone lesions.[24] A nationwide survey from Japan described 16 children treated with bisphosphonates for bone LCH. All had bone disease; none had risk-organ disease. Most patients received six cycles of pamidronate at 1 mg/kg/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 remained disease free at a median of 3.3 years.[25] Other bisphosphonates, such as zoledronate and oral alendronate, have been used to successfully treat bone LCH.
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