This protocol addresses adults presenting with bone-only Langerhans cell histiocytosis characterised by multifocal lytic osseous lesions. Disease is confined to the skeleton at multiple sites, without soft-tissue or visceral involvement.
Current evidence supports systemic bone-targeted therapy for this presentation — bisphosphonate agents are among the recognised options — along with consideration of localised approaches depending on lesion number and suitability. The complete regimen is available in the full protocol.
Treatment aims for resolution of bone pain and radiographic ossification and normalisation of bone lesions. Response is assessed with FDG-PET/CT at 2–3 months.
DOI: 10.1182/blood.2021014343
For multifocal osseous LCH, recommended treatments are radiation therapy (<3 lesions safely amenable to radiation), bisphosphonates, or systemic chemotherapy.
In a combined adult and pediatric series of patients with skeletal disease, the use of bisphosphonate therapy (alendronate, pamidronate, or zoledronate) was associated with durable resolution of bone pains as well as radiographic ossification and normalization of bone lesions in the majority of the cases (92%).
Radiation therapy is an effective option for symptomatic bone-only disease with a limited number of lesions (<3).
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