The European respiratory journal · 2026
Efficacy and safety of cladribine in adult pulmonary Langerhans cell histiocytosis: a phase II study.
Клиническое исследование II фазыСтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Benattia Amira, Lorillon Gwenaël, de Margerie-Mellon Constance, Vercellino Laetitia, Bonniaud Philippe, Marquette Charles-Hugo, Chevret Sylvie, Tazi Abdellatif
Аннотация
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 (FEV1), with an increase of ≥200 mL in the absolute value of FEV1. 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%). The response to cladribine was associated with a median (IQR) decrease of 14.9 (10.5-23.5) points in the St George's Respiratory Questionnaire Total score. At 12 months, five patients were still responders. The median (IQR) duration of infection prophylaxis was 11.8 (11.7-29) months. One patient died before the 12-month visit. The remaining patients were alive at 48 months. No malignancies were detected.
CONCLUSIONS: Cladribine improved lung function in half of the patients at 1 year of follow-up and was well tolerated overall.
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 (FEV1), with an increase of ≥200 mL in the absolute value of FEV1. 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%). The response to cladribine was associated with a median (IQR) decrease of 14.9 (10.5-23.5) points in the St George's Respiratory Questionnaire Total score. At 12 months, five patients were still responders. The median (IQR) duration of infection prophylaxis was 11.8 (11.7-29) months. One patient died before the 12-month visit. The remaining patients were alive at 48 months. No malignancies were detected.
CONCLUSIONS: Cladribine improved lung function in half of the patients at 1 year of follow-up and was well tolerated overall.