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Long‐term outcomes of children with extracutaneous juvenile xanthogranulomas in Japan
Author(s) -
Maeda Miho,
Morimoto Akira,
Shioda Yoko,
Asano Takeshi,
Koga Yuhki,
Nakazawa Yozo,
Kanegane Hirokazu,
Kudo Kazuko,
Ohga Shouichi,
Ishii Eiichi
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28381
Subject(s) - medicine , diabetes insipidus , juvenile xanthogranuloma , histiocytosis , pediatrics , disease , surgery , langerhans cell histiocytosis , pathology , histiocyte
Background Juvenile xanthogranuloma (JXG) is the most common non‐Langerhans cell histiocytosis in children. The mortality and morbidity of JXG with extracutaneous lesions remain unclear. Methods Data of patients aged < 18 years who were diagnosed with JXG between 2001 and 2010 were retrospectively collected through a nationwide survey. Results Twenty patients (11 male and nine female) had extracutaneous lesions. The median observation time was 10 years (range, 0‐17). Six patients presented with symptoms at birth. The median age at diagnosis was 8.5 months (range, 0 month‐13 years). Fifteen patients underwent treatment for JXG, including chemotherapy ( n = 11), and five did not receive treatment. All patients except one survived; 17 were disease‐free and two survived with disease. One newborn‐onset patient with liver, spleen, and bone marrow involvement died of the disease. Permanent sequelae included central diabetes insipidus, growth hormone deficiency, and panhypopituitarism detected at diagnosis in three, one, and two patients, respectively. Four patients had visual impairment (optic nerve compression and intraocular invasion in two each), three had epilepsy, one had mental retardation, and one had a skin scar. Eight patients who had intracranial lesions were older at diagnosis, and had a higher frequency of disease‐related comorbidities and permanent sequelae than those without intracranial involvement. Conclusions Patients with extracutaneous JXG had good outcomes, although those with intracranial lesions had serious permanent sequelae. Effective and safe treatment regimens for patients with intracranial JXG need to be developed.