Efficacy of MAPK inhibitors in children with neurodegenerative Langerhans cell histiocytosis: Results from the European Consortium for Histiocytosis
Francesco Pegoraro, Francois Chalard, Dmitry Evseev, Mohamed Barkaoui, Solenne Le Louet, Julian Thalhammer, Nathalie Aladjidi, Alexandra Salmon, Perrine Marec‐Berard, Stefania Gaspari, Carmela De Fusco, Irene Trambusti, Antonio Verrico, Anna Raciborska, Karel Svojgr, Zdenka Křenová, Olga Slater, Ger · 2026
Read Efficacy of MAPK inhibitors in children with neurodegenerative Langerhans cell histiocytosis: Results from the European Consortium for Histiocytosis by Francesco Pegoraro, Francois Chalard, Dmitry Evseev, Mohamed Barkaoui, Solenne Le Louet, Julian Thalhammer, Nathalie Aladjidi, Alexandra Salmon, Perrine Marec‐Berard, Stefania Gaspari, Carmela De Fusco, Irene Trambusti, Antonio Verrico, Anna Raciborska, Karel Svojgr, Zdenka Křenová, Olga Slater, Ger (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13242968.txt.
Description
Francesco Pegoraro and colleagues pooled the largest experience yet of MAPK inhibitors for a devastating brain complication of Langerhans cell histiocytosis. Their study appeared in HemaSphere in 2026. The neurodegenerative form of the disease damages the brain years after the initial illness and has had no effective treatment. Sixty children treated between 2013 and 2025 were assessed with clinical and scan scoring systems, some newly developed for this work.
Just over half of symptomatic children improved clinically, and most of the rest stabilized rather than worsening. Brain scans improved in about three-quarters of those followed, a highly significant change. None of the nine children treated before symptoms began developed any. Treatment started a median of about two years after the brain diagnosis. The authors read the results as the first real evidence that these targeted drugs can bend the course of this complication. Longer follow-up will show whether gains endure.
Catalog record
| CREATOR | Francesco Pegoraro, Francois Chalard, Dmitry Evseev, Mohamed Barkaoui, Solenne Le Louet, Julian Thalhammer, Nathalie Aladjidi, Alexandra Salmon, Perrine Marec‐Berard, Stefania Gaspari, Carmela De Fusco, Irene Trambusti, Antonio Verrico, Anna Raciborska, Karel Svojgr, Zdenka Křenová, Olga Slater, Ger |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 37 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |