1. A multidisciplinary non-invasive approach to monitor response to intravenous immunoglobulin treatment in neurodegenerative Langerhans cell histiocytosis: a real-world study.
- Author
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Trambusti I, Barba C, Mortilla M, Rizzi S, Romano K, Coniglio ML, Lucenteforte E, Tondo A, Guerrini R, and Sieni E
- Subjects
- Humans, Male, Female, Evoked Potentials, Somatosensory, Treatment Outcome, Child, Preschool, Child, Adolescent, Neurodegenerative Diseases drug therapy, Neurodegenerative Diseases diagnosis, Infant, Adult, Evoked Potentials, Auditory, Brain Stem, Immunoglobulins, Intravenous therapeutic use, Immunoglobulins, Intravenous administration & dosage, Histiocytosis, Langerhans-Cell drug therapy, Histiocytosis, Langerhans-Cell diagnosis, Magnetic Resonance Imaging
- Abstract
Aims: Early detection and treatment of neurodegenerative Langerhans cell histiocytosis (ND-LCH) have been suggested to prevent neurodegenerative progression. The aim of the study is to validate a standardized multidisciplinary diagnostic work-up to monitor the intravenous immunoglobulins (IVIG) treatment response and the natural course of the disease in untreated patients., Methods: Patients with abnormal somatosensory evoked potentials (SEPs) received monthly 0.5 g/kg IVIG. The diagnostic protocol included structural 3T MRI, neurological examination, brainstem auditory evoked potentials (BAEPs) and SEPs., Results: Twenty-two patients were followed for 5.2 years (median) from the first MRI evidence of ND-LCH. Eleven patients received IVIG for 1.7 years (median). At treatment start neurological examination was abnormal in 10 patients, of whom two had severe clinical impairment and four had abnormal BAEPs. At last follow-up, 1/11 remained stable and 7/11 improved, while worsening of neurological or neurophysiological findings, or both, occurred in 3/11. Risk factors for worsening were a severe clinical or MRI ND-LCH at treatment initiation and prolonged exposure to LCH. Of the 11 untreated patients, none improved and three worsened., Conclusions: Using a standardized diagnostic protocol, we demonstrated that IVIG treatment can lead to clinical stabilization or improvement in all pauci-symptomatic patients with an MRI grading of less than 4., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2024 Trambusti, Barba, Mortilla, Rizzi, Romano, Coniglio, Lucenteforte, Tondo, Guerrini and Sieni.)
- Published
- 2024
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