This protocol covers unifocal Langerhans cell histiocytosis confined to the pituitary or hypothalamus in an adult, where the lesion has led to central diabetes insipidus and anterior pituitary dysfunction.
The patient presents with unifocal LCH of the pituitary/hypothalamus accompanied by central diabetes insipidus and anterior pituitary dysfunction. Hormone replacement for these endocrine deficits is an important part of management alongside any disease-directed therapy.
When symptoms are recent-onset or a radiologic lesion is present, systemic chemotherapy — applied in an approach broadly similar to multisystem disease — may be considered. The full regimen, agent selection, and treatment schedule are detailed in the structured protocol.
DOI: 10.1182/blood.2021014343
For unifocal LCH of pituitary/hypothalamus resulting in DI and anterior pituitary dysfunction, hormone replacement should be undertaken.
The role of systemic therapy is unclear and is recommended in cases with symptoms that are recent-onset or when a radiologic lesion is present.
Among systemic treatment options for LCH, chemotherapy using either cladribine or cytarabine is preferred because of relatively high overall response rates and the potential for long-term remissions with limited cycles of treatment.
View source ↗