Background The efficacy of cladribine in treating pulmonary Langerhans cell histiocytosis (PLCH) has been suggested in select case reports. Treatment-related malignancies remain a concern.
Methods In this phase II trial, the efficacy and safety of cladribine was evaluated in symptomatic PLCH patients with airflow obstruction and/or a decrease in lung function within the previous year. Cladribine was administered for 4 monthly cycles combined with infectious prophylaxis.
Patients were followed up every 3 months during the first year to assess efficacy and then until 48 months. The primary end-point was the cumulative incidence of response to treatment at 6 months, defined as ≥10% improvement in forced vital capacity (FVC) and/or forced expiratory volume in 1 s (FEV 1 ), with an increase of ≥200 mL in the absolute value of FEV 1 .
The study was registered with ClinicalTrials.gov (NCT01473797). Results 10 patients (six men; median (interquartile range (IQR)) age 37 (33 - 47.5) years; six current smokers) were included.
The cumulative incidence of response to treatment at 6 months was 70% (95% CI 28.4 - 90.4%).
European Respiratory Journal published a clinical update in Critical Care on 21 May 2026.
The item focuses on Efficacy and safety of cladribine in adult pulmonary Langerhans cell histiocytosis: a phase II study.
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