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Long‐term outcome, clinical course and treatment approaches of paediatric langerhans cell histiocytosis: A greek reference centre report
Author(s) -
Tzotzola Vasiliki,
Petrikkos Loizos,
Papadakis Vassilios,
Mitropoulou Georgia,
Kelaidi Charikleia,
Dimitriadis Efthymios,
Polychronopoulou Sophia
Publication year - 2021
Publication title -
acta paediatrica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.772
H-Index - 115
eISSN - 1651-2227
pISSN - 0803-5253
DOI - 10.1111/apa.15743
Subject(s) - medicine , langerhans cell histiocytosis , cohort , histiocytosis , diabetes insipidus , pediatrics , disease , retrospective cohort study , cohort study , surgery
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.