Sotorasib for Vascular Malformations Associated with KRAS G12C Mutation.
N Engl J Med
; 391(4): 334-342, 2024 Jul 25.
Article
in En
| MEDLINE
| ID: mdl-39018528
ABSTRACT
KRAS gain-of-function mutations are frequently observed in sporadic arteriovenous malformations. The mechanisms underlying the progression of such KRAS-driven malformations are still incompletely understood, and no treatments for the condition are approved. Here, we show the effectiveness of sotorasib, a specific KRAS G12C inhibitor, in reducing the volume of vascular malformations and improving survival in two mouse models carrying a mosaic Kras G12C mutation. We then administered sotorasib to two adult patients with severe KRAS G12C-related arteriovenous malformations. Both patients had rapid reductions in symptoms and arteriovenous malformation size. Targeting KRAS G12C appears to be a promising therapeutic approach for patients with KRAS G12C-related vascular malformations. (Funded by the European Research Council and others.).
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Arteriovenous Malformations
/
Proto-Oncogene Proteins p21(ras)
Limits:
Adult
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Animals
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Female
/
Humans
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Male
/
Middle aged
Language:
En
Journal:
N Engl J Med
Year:
2024
Type:
Article