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Hongyun Lian

and 11 more

Objective To investigate the clinical characteristics, treatment, and prognosis of children with systemic juvenile xanthogranuloma (JXG). Methods Children with JXG from January 2012 to December 2019 were retrospectively analyzed. Data relating to the clinical manifestations, laboratory values, treatment, and prognosis of the children were extracted from medical records. Patients underwent vindesine +prednisone as the first-line treatment and cytarabine + vindesine + dexamethasone +/- cladribine as the second-line treatment. Results Ten patients, including 8 males and 2 females, with an onset age of 1.95 (0.80-7.30) years, exhibited multi-system dysfunction. The median age of diagnosis was 2.45 (1.30-12.10) years. The most common location of extracutaneous lesions was the central nervous system (6 cases), followed by the lung (5 cases) and bone (4 cases). Nine patients underwent first-line chemotherapy, and 6 patients underwent second-line chemotherapy, including 5 patients with poorly controlled disease after first-line treatment. The median observation time was 20 (3-106) months. Nine patients survived, whereas one patient died of respiratory failure caused by pulmonary infection. By the end of follow-up, 7 patients were in an active disease (AD) state but better (AD-better), and 2 patients were in an AD-stable state. Three patients had permanent sequelae, mainly, central diabetes insipidus. The first-line treatment response rate was 40.0%, and the second-line treatment response rate was 66.7%. Conclusion The chemotherapy protocol for Langerhans cell histiocytosis (LCH) was effective for patients with systemic JXG, which also resulted in good outcomes. Central nervous system involvement did not impact overall survival, but serious permanent sequelae remained.