Clinical analysis of pediatric systemic juvenile xanthogranulomas: A
retrospective single-center study
Abstract
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.