MEK inhibition achieves robust and durable responses in Erdheim–Chester disease - Takeaways - MDSpire

MEK Inhibitors Produce Strong, Sustained Responses in Erdheim–Chester Disease

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This international study included 170 patients with Erdheim–Chester disease treated with MEK inhibitor monotherapy across eight countries.
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The cohort was predominantly male (71%), and 69% had multisystem disease involving more than four sites.
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Skeletal involvement was most common (74%), followed by perirenal (66%), large-vessel (55%), cardiac (39%), and central nervous system disease (38%).
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Treatment response was assessed using a composite of clinical, radiologic, and metabolic criteria, and adverse events were graded using CTCAE criteria.
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The study evaluated long-term efficacy, tolerability, response timing, treatment retention, survival, and baseline features associated with MEK inhibitor response.

Original Source(s)

Hemasphere

MEK inhibition achieves robust and durable responses in Erdheim–Chester disease

by Francesco Pegoraro, Félicien Triboulet, Matthias Papo, Francesco Peyronel, Anita Argentieri, Jerome Razanamahery, Ahmed Idbaih, Polyzois Makras, Ofer Shpilberg, Oshrat Hershkovitz-Rokah, Michael Girschikofsky, Matthew Collin, Kristian Bowles, Satyen H. Gohil, Rodothea Amerikanou, Emmanuel Ledoult, Tanguy Le Scornet, Mathilde de Menthon, Etienne Riviere, Xavier Boulu, Henry Dupuy, Achille Aouba, Stanislas Faguer, Xavier Solanich, Elena Sieni, Stéphane Barete, Chiara Bellino, Alessandro Tomelleri, Corrado Campochiaro, Lorenzo Dagna, Zahir Amoura, Jean-François Emile, Fleur Cohen-Aubart, Augusto Vaglio, Julien Haroche
October 6, 2026

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