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ORIGINAL RESEARCH article

Front. Immunol.
Sec. Multiple Sclerosis and Neuroimmunology
Volume 15 - 2024 | doi: 10.3389/fimmu.2024.1422802
This article is part of the Research Topic Central nervous system autoimmunity and autoinflammation: insights from inborn errors of immunity View all articles

A MULTIDISCIPLINARY NON-INVASIVE APPROACH TO MONITOR RESPONSE TO INTRAVENOUS IMMUNOGLOBULIN TREATMENT IN NEURODEGENERATIVE LANGERHANS CELL HISTIOCYTOSIS: A REAL-WORLD STUDY

Provisionally accepted
Irene Trambusti Irene Trambusti 1Carmen Barba Carmen Barba 1Marzia Mortilla Marzia Mortilla 1Susanna Rizzi Susanna Rizzi 1Katiuscia Romano Katiuscia Romano 1Maria Luisa Coniglio Maria Luisa Coniglio 1Ersilia Lucenteforte Ersilia Lucenteforte 2ANNALISA TONDO ANNALISA TONDO 1Renzo Guerrini Renzo Guerrini 1,3Elena Sieni Elena Sieni 1*
  • 1 Azienda Ospedaliera Universitaria Meyer IRCCS - Firenze, Florence, Italy
  • 2 University of Pisa, Pisa, Tuscany, Italy
  • 3 University of Florence, Florence, Tuscany, Italy

The final, formatted version of the article will be published soon.

    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.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.

    Keywords: neurodegenerative Langerhans cell histiocytosis, Evoked Potentials, immunoglobulin, SEPs, ND-LCH

    Received: 25 Apr 2024; Accepted: 29 Jul 2024.

    Copyright: © 2024 Trambusti, Barba, Mortilla, Rizzi, Romano, Coniglio, Lucenteforte, TONDO, Guerrini and Sieni. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Elena Sieni, Azienda Ospedaliera Universitaria Meyer IRCCS - Firenze, Florence, Italy

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