Dramatic response to trametinib in a male child with neurofibromatosis type 1 and refractory astrocytoma

Pediatr Blood Cancer. 2019 Jan;66(1):e27474. doi: 10.1002/pbc.27474. Epub 2018 Sep 24.

Authors

Tristan Knight^{1

2}, Margaret Shatara^{1

2}, Lindsey Carvalho³, Deniz Altinok^{2

4}, Janet Poulik^{2

5}, Zhihong Joanne Wang¹

Affiliations

¹ Division of Pediatric Hematology and Oncology, Department of Pediatrics, Children's Hospital of Michigan, Detroit, Michigan.
² Wayne State University School of Medicine, Detroit, Michigan.
³ John A. Burns School of Medicine, University of Hawaii, Honolulu, Hawaii.
⁴ Department of Radiology, Children's Hospital of Michigan, Detroit, Michigan.
⁵ Department of Pathology, Children's Hospital of Michigan, Detroit, Michigan.

PMID: 30251337
DOI: 10.1002/pbc.27474

No abstract available

Keywords: MEK inhibitor; low-grade glioma; neurofibromatosis type 1; trametinib.

Publication types

Case Reports
Letter

MeSH terms

Antineoplastic Agents / therapeutic use*
Astrocytoma / complications
Astrocytoma / drug therapy*
Astrocytoma / pathology
Drug Resistance, Neoplasm / drug effects*
Humans
Infant
Male
Neurofibromatosis 1 / complications
Neurofibromatosis 1 / drug therapy*
Neurofibromatosis 1 / pathology
Prognosis
Pyridones / therapeutic use*
Pyrimidinones / therapeutic use*
Remission Induction
Salvage Therapy*

Substances

Antineoplastic Agents
Pyridones
Pyrimidinones
trametinib