This protocol addresses patients with mucocutaneous Langerhans cell histiocytosis whose cutaneous lesions have not responded to a prior oral systemic treatment line. A distinct, locally oriented approach is defined for this refractory situation.
The preceding line involved oral systemic therapy — agents such as methotrexate, prednisone, 6-mercaptopurine, hydroxyurea, thalidomide, or lenalidomide. The treatment goal was any degree of improvement in cutaneous lesions on skin examination. This protocol is indicated when that response was not achieved.
When oral systemic therapy has not achieved cutaneous improvement, this protocol specifies locally directed treatment approaches for refractory cutaneous disease. The specific interventions, their selection, and sequencing are detailed in the full protocol.
Any degree of improvement in cutaneous lesions on skin examination is considered a response.
DOI: 10.1182/blood.2021014343