Cladribine treatment of patients with pulmonary Langerhans cell histiocytosis

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Cladribine treatment in patients with PLCH 1 Patients and methods From 2010 to 2021, 20 patients with histologically confirmed LCH were hospitalized in our department.All those who required chemotherapy (20 out of a total of 62 patients) received 2 -CdA.Evaluation of the patients was performed according to the recommendations of the Histiocyte Society.2,3 2 -CdA was administered intravenously at a dose of 0.15 or 0.12 mg/kg per day for 5 consecutive days at monthly intervals.Data on patients' demographics, concomitant diseases, smoking status, clinical symptoms, organ involvement, pulmonary function tests (PFTs), and echocardiography were gathered.Lung and bone computed tomography (CT) scans, positron emission tomography, and magnetic resonance imaging (MRI) scans of the brain, pituitary gland, and abdomen were interpreted by experienced radiologists (KB, MJ, and JW).Pulmonary function improvement or deterioration were assessed in terms of changes in the forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1) exceeding 200 ml, or transfer factor of the lung for carbon monoxide (T LCO ) above 10% from baseline.PLCH stratification, evaluation of the disease status, and response to treatment, were based on the Histiocyte Society criteria.2,3 The patients were assessed at the beginning of the treatment, before every course, at 3, 6, and 12 months after the treatment concluded, and annually thereafter.Further methodological details and information on statistical analysis of the results can be found in Supplementary material.The study adhered to all relevant tenets of the Helsinki Declaration.All patients provided their written informed consent for the use of offlabel 2 -CdA prior to the treatment, and approval for inclusion of each patient was given by Introduction Langerhans cell histiocytosis (LCH) is a rare disease of unknown etiology caused by clonal proliferation and infiltration of various organs by bone marrow -derived dendritic cells in the lymph nodes, bones, skin, lungs, pituitary gland, spleen, liver, and central nervous system (CNS).1-3 The lungs may be involved as an isolated organ or as a part of a multisystem disease.Pulmonary LCH (PLCH) can occur in patients of all ages but it is usually a disease of young, adult smokers, and no sex predilection is apparent.1-3 In the lungs, Langerhans cells proliferate to form bronchiolocentric nodules, which may cavitate to form thick -walled confluent cysts with relative sparing of the costophrenic angles.PLCH progression may trigger fibrocystic lung destruction.Mutations in the BRAF (V600E), MAP2K1, ARAF, NRAS, and KRAS genes have been identified in more than two -thirds of PLCH cases, likely contributing to Langerhans cells proliferation. 1-3The course of the disease is unpredictable.In some patients, smoking cessation induces regression, but in others the course may be aggressive. 2-5Treatment strategies of adult -onset LCH are based on extrapolation of pediatric data, and on small retrospective studies.1-10 At present, no standard treatment for adult PLCH is available.2,3 Growing evidence suggests that cladribine (2 -CdA) effectively cures PLCH, and a prospective clinical trial (www.clinicaltrials.gov,NCT01477397) is being conducted to verify this.8-20 2 -CdA is a purine nucleoside analogue, which accumulates in the cells containing active deoxycytidine kinase, such as lymphocytes and dendritic cells.The principal indication for 2 -CdA treatment is hairy cell leukemia.1,2 This work describes the results of a single--center observational study on 2 -CdA treatment in adults with progressive PLCH.

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