Long‐term outcome, clinical course and treatment approaches of paediatric langerhans cell histiocytosis: A greek reference centre report. (10th January 2021)

• Record Type: Journal Article
• Authors: Tzotzola, Vasiliki; Petrikkos, Loizos; Papadakis, Vassilios; Mitropoulou, Georgia; Kelaidi, Charikleia; Dimitriadis, Efthymios; Polychronopoulou, Sophia
• Is Part Of: Acta pædiatrica. Volume 110:Number 6(2021)
• Page Start: 1944
• Page End: 1951
• Publication Date: 2021-01-10
• Subjects: langerhans cell histiocytosis -- neurodegeneration -- reactivation -- survival; Pediatrics -- Periodicals; Pediatrics; 618.92
• DOI: 10.1111/apa.15743
• Languages: English
• ISSNs: 0803-5253

Abstract

Abstract: Aim: Langerhans cell histiocytosis (LCH) is an inflammatory myeloid neoplasia with diverse clinical behaviour. In this article, we studied the clinical course, management and long‐term outcomes of a paediatric cohort treated by our reference centre. Methods: We retrospectively studied 66 children with LCH, consecutively diagnosed by a Greek reference centre from 1974 to 2020. Results: The patients had a median age of 3.9 (range 0.0–15.9) years, 39 and 6 patients were diagnosed with unifocal or multifocal single system disease and 14 and 7 had multisystem disease with or without risk organ involvement. No late occurrence of clinical neurodegenerative disease or diabetes insipidus were observed at a median follow‐up period of 4.1 (range 0.5–27.7) years. The 10‐year event‐free survival and overall survival were 65.0% and 90.3% and improved significantly over a 45‐year period. Survival was superior in single system than multisystem cases. BRAF V600E mutation was found in 8/14 tested patients. Reactivation occurred in 12/66 patients (18.2%); 11 achieved remission and one patient died after a second relapse. Conclusion: LCH survival rates significantly increased in our cohort over time. Reactivation occurred in 18.2% patients, but no late neurodegeneration was found. The prognostic value of single system disease status vs. multisystem LCH was confirmed.