Hongyun Lian1, Lei Cui2, Ying Yang1, Ang Wei1, Hua Cheng3, Na Li2, Li Zhang1, Honghao Ma1, Xiaoxi Zhao1, Tianyou Wang1, Zhigang Li4, Rui Zhang5. 1. Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Key Laboratory of Major Diseases in Children, Ministry of Education, Hematology Oncology Center, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, 100045, China. 2. Laboratory of Hematologic Diseases, Beijing Pediatric Research Institute, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, 100045, China. 3. Radiology Department, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, 100045, China. 4. Laboratory of Hematologic Diseases, Beijing Pediatric Research Institute, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University, Beijing, 100045, China. ericlzg70@hotmail.com. 5. Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Key Laboratory of Major Diseases in Children, Ministry of Education, Hematology Oncology Center, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, 100045, China. ruizh1973@126.com.
Abstract
PURPOSE: Langerhans cell histiocytosis (LCH) can affect any organ. Central nervous system (CNS) involvement is rare, and its management is poorly understood. This study aimed to analyze the clinical response and prognosis of pediatric LCH with central diabetes insipidus (CDI) treated with second-line therapy with cytarabine (Ara-c), cladribine (2-cdA), dexamethasone, and vindesine. METHODS: This retrospective case series study included pediatric LCH with CDI treated at Beijing Children's Hospital affiliated with Capital Medical University (11/2012-01/2018). After the first-line 2009-LCH regimen, patients with active disease/worse response, relapse, or no significant improvement in risk organs, pituitary, or lung were given the second-line therapy. Baseline characteristics, clinical response and adverse reactions were observed. RESULTS: Twenty-six children with CDI and disappearance of hyperintensity in the posterior pituitary were included. They received "Regimen A" Ara-c + dexamethasone + vindesine (n = 7) or "Regimen B" Ara-c + dexamethasone + vindesine + 2-cdA (n = 19) as second-line therapy. There were 14 patients with CDI but without pituitary stalk thickening (PST) and 12 with CDI and PST. In patients with CDI alone, 4/4 patients receiving Regimen A and 3/10 receiving Regimen B improved. All patients with CDI and PST showed improvement for PST. The reappearance of hyperintensity at the posterior pituitary was observed in 10 patients with CDI. All 26 children were alive after a median follow-up of 40.5 months. There were no chemotherapy-related deaths. CONCLUSION: A combined therapy with Ara-c, 2-cdA, dexamethasone, and vindesine could partially alleviate pituitary disease conditions in pediatric LCH with CNS involvement, with good tolerance.
PURPOSE: Langerhans cell histiocytosis (LCH) can affect any organ. Central nervous system (CNS) involvement is rare, and its management is poorly understood. This study aimed to analyze the clinical response and prognosis of pediatric LCH with central diabetes insipidus (CDI) treated with second-line therapy with cytarabine (Ara-c), cladribine (2-cdA), dexamethasone, and vindesine. METHODS: This retrospective case series study included pediatric LCH with CDI treated at Beijing Children's Hospital affiliated with Capital Medical University (11/2012-01/2018). After the first-line 2009-LCH regimen, patients with active disease/worse response, relapse, or no significant improvement in risk organs, pituitary, or lung were given the second-line therapy. Baseline characteristics, clinical response and adverse reactions were observed. RESULTS: Twenty-six children with CDI and disappearance of hyperintensity in the posterior pituitary were included. They received "Regimen A" Ara-c + dexamethasone + vindesine (n = 7) or "Regimen B" Ara-c + dexamethasone + vindesine + 2-cdA (n = 19) as second-line therapy. There were 14 patients with CDI but without pituitary stalk thickening (PST) and 12 with CDI and PST. In patients with CDI alone, 4/4 patients receiving Regimen A and 3/10 receiving Regimen B improved. All patients with CDI and PST showed improvement for PST. The reappearance of hyperintensity at the posterior pituitary was observed in 10 patients with CDI. All 26 children were alive after a median follow-up of 40.5 months. There were no chemotherapy-related deaths. CONCLUSION: A combined therapy with Ara-c, 2-cdA, dexamethasone, and vindesine could partially alleviate pituitary disease conditions in pediatric LCH with CNS involvement, with good tolerance.
Authors: G A Kaltsas; T B Powles; J Evanson; P N Plowman; J E Drinkwater; P J Jenkins; J P Monson; G M Besser; A B Grossman Journal: J Clin Endocrinol Metab Date: 2000-04 Impact factor: 5.958
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Authors: H Gadner; N Grois; M Arico; V Broadbent; A Ceci; A Jakobson; D Komp; J Michaelis; S Nicholson; U Pötschger; J Pritchard; S Ladisch Journal: J Pediatr Date: 2001-05 Impact factor: 4.406
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