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| Autori principali: | , , , , , , |
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| Natura: | Artículo Open Access |
| Pubblicazione: |
Wiley
2025
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| Accesso online: | https://onlinelibrary.wiley.com/doi/10.1002/pbc.32016 |
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- Targeted Therapy in Pediatric Langerhans Cell Histiocytosis: Describing a Novel Strategy to Minimize Long‐Term Exposure While Maintaining Efficacy Natalia Wojciechowska Alexandra E. Richards Aly Anthony Maranda Diaz Kelly Vallance Chelsee Greer Anish Ray Pediatric Blood & Cancer ABSTRACTBackgroundLangerhans cell histiocytosis (LCH) is a rare malignancy driven by MAPK pathway activation and often involves BRAF V600E mutations. Targeted therapy with trametinib, a MEK inhibitor, is a promising alternative to conventional chemotherapy.ProcedureWe retrospectively analyzed the records of patients treated with trametinib either at relapse or as front‐line therapy between 2020 and 2024.ResultsFifteen pediatric patients received trametinib either at diagnosis (n = 6), relapse (n = 7), or due to chemotherapy intolerance (n = 2). Molecular testing identified MAPK pathway mutations in 11 patients. The median age at treatment initiation was 5 years (range: 0.1–16.5). Notably, five of these patients started trametinib before the age of 1. The median treatment duration was 2.2 years (range: 0.1–4.7 years). All 15 patients achieved favorable responses without concerns regarding growth and development. The adverse effects included rash (40%) and diarrhea (13%), which were all mild and were managed with temporary dose adjustments. A self‐weaning dosing strategy minimized long‐term exposure while maintaining disease control.ConclusionOur data suggest that trametinib is a safe and effective therapy for pediatric LCH with broad efficacy and tolerability. However, prospective studies are needed to confirm these findings and refine targeted treatment protocols. 10.1002/pbc.32016 http://onlinelibrary.wiley.com/termsAndConditions#vor