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Langerhans Cell Histiocytosis with BRAF p.N486_P490del or MAP2K1 p.K57_G61del Treated by the MEK Inhibitor Trametinib
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  • Yoav Messinger,
  • Bruce Bostrom,
  • Damon Olson,
  • Nathan Gossai,
  • Lane Miller,
  • Michael Richards
Yoav Messinger
Children's Hospitals and Clinics of Minnesota
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Bruce Bostrom
Children's Hospitals and Clinics of Minnesota
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Damon Olson
Children's Hospitals and Clinics of Minnesota
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Nathan Gossai
Children's Hospitals and Clinics of Minnesota
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Lane Miller
Children's Hospitals and Clinics of Minnesota
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Michael Richards
Children's Hospitals and Clinics of Minnesota
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Abstract

Langerhans Cell Histiocytosis (LCH) is characterized by activating variants of the MAPK pathway. Inhibition of the MAPK pathway with trametinib (MEK inhibitor) has been shown to induce responses in LCH patients. Two adolescent males with LCH driven by BRAF p.N486_P490del have received trametinib for >1 year with no reactivation in one and partial response in another (including stable lung disease). A third male with neonatal LCH and MAP2K1 p.K57_G61del had a complete response to trametinib with no active disease after 22 months. All patients continue on trametinib monotherapy with tolerable skin and CPK toxicity.

Peer review status:ACCEPTED

27 Jul 2020Submitted to Pediatric Blood & Cancer
27 Jul 2020Submission Checks Completed
27 Jul 2020Assigned to Editor
28 Jul 2020Reviewer(s) Assigned
06 Aug 2020Review(s) Completed, Editorial Evaluation Pending
06 Aug 2020Editorial Decision: Revise Major
15 Aug 2020Submission Checks Completed
15 Aug 2020Assigned to Editor
15 Aug 20201st Revision Received
17 Aug 2020Reviewer(s) Assigned
03 Sep 2020Review(s) Completed, Editorial Evaluation Pending
03 Sep 2020Editorial Decision: Accept