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Langerhans cell histiocytosis: diagnosis, natural history, management, and outcome
D M Howarth1, G S Gilchrist, B P Mullan
1Department of Nuclear Medicine, Mayo Clinic Rochester, Minnesota, USA.
Insights
Langerhans cell histiocytosis (LCH) patients with isolated bone lesions show the best prognosis. Multisystem LCH has a higher risk of progressive disease, underscoring the need for prognostic indicators and surveillance.
Area of Science:
- Oncology
- Pediatric Hematology
- Pathology
Background:
- Langerhans cell histiocytosis (LCH) is a rare clonal proliferative disorder of myeloid lineage cells.
- Understanding the natural history and outcomes of LCH is crucial for effective management.
Purpose of the Study:
- To analyze the natural history, management, and outcomes of a large cohort of Langerhans cell histiocytosis (LCH) patients.
- To compare outcomes between patients with single-system versus multisystem LCH.
Main Methods:
- A retrospective descriptive analysis of 314 histologically confirmed LCH patients at Mayo Clinic.
- Patients were categorized by disease extent (single-system vs. multisystem).
- Data on clinical features, treatments (chemotherapy, radiotherapy, surgery), and outcomes (disease-free survival, active disease, death) were collected.
Main Results:
- Isolated bone LCH had a 97% disease-free survival rate. Multisystem LCH affected 96 patients, with 25 experiencing active disease post-treatment.
- Pulmonary LCH (87 patients) had a high mortality rate (10 deaths), particularly in smokers.
- Pituitary-thalamic axis involvement (44 patients) often required long-term hormone replacement.
Conclusions:
- Isolated bone LCH offers the best prognosis.
- Multisystem LCH carries a significant risk of progressive disease despite treatment.
- Identifying prognostic indicators is essential for tailored treatment and long-term follow-up.
Background:
The objective of this descriptive analysis of a large cohort of patients with Langerhans cell histiocytosis (LCH) was to add to the understanding of the natural history, management, and outcome of this disease.
Methods:
Three hundred fourteen Mayo Clinic patients with histologically proven LCH were categorized into those patients with multisystem disease and those patients with single system disease. Clinical features, treatment, and outcome were determined from the case history notes and tumor registry correspondence. Treatment included chemotherapy, radiotherapy, and surgical excision. The end points were disease free survival, active disease, or death. The median time of follow-up was 4 years (range, 1 month to 47.5 years).
Results:
The age of the patients ranged from 2 months to 83 years. Of the 314 patients, there were 28 deaths. Multisystemic LCH was found in 96 patients, 25 of whom had continuing active disease after treatment. Isolated bone LCH lesions were observed in 114 of the 314 patients, 111 of whom (97%) achieved disease free survival after treatment. The most common sites of osseous LCH were the skull and proximal femur. Of the 87 patients with isolated pulmonary involvement, only 3 were nonsmokers. After treatment with corticosteroids (+/- cyclophosphamide or busulphan), 74 patients achieved disease free survival, but 10 patients died. Pituitary-thalamic axis LCH, characterized by diabetes insipidus, was found in 44 patients. After treatment, 30 of these patients had disease free survival, but all required long term hormone replacement with desmopressin acetate. Lymph node involvement was found in 21 patients, and mucocutaneous involvement was found in 77 patients.
Conclusions:
Patients with isolated bone LCH lesions have the best prognosis compared with patients with LCH involvement of other systems. By contrast, 20% of patients with multisystem involvement have a progressive disease course despite treatment. The identification of prognostic indicators to facilitate appropriate treatment and long term follow-up surveillance is recommended.
