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Sorafenib Plus Valproic Acid for Infant Spinal Glioblastoma

Rokes, Christopher Alan MD*; Remke, Mark MD; Guha-Thakurta, Nandita MD; Witt, Olaf MD§; Korshunov, Andrey MD; Pfister, Stephan MD† §; Wolff, Johannes E. MD, PhD*

Journal of Pediatric Hematology/Oncology: August 2010 - Volume 32 - Issue 6 - p 511-514
doi: 10.1097/MPH.0b013e3181d74702
Clinical and Laboratory Observations

Spinal glioblastoma multiforme (GBM) is rare in children. New therapeutic options should be explored given the poor outcomes reported. We describe the case of an infant with spinal GBM whose condition worsened despite radiotherapy and chemotherapy. Immunohistochemical analysis of the tumor sample showed activation of the Raf-MEK-ERK pathway. Targeted pharmacologic therapy with sorafenib plus valproic acid led to decrease in the size of the tumor and improvement of symptoms. We conclude that regulation of the mitogen-activated protein kinase pathway using sorafenib plus valproic acid warrants further investigation for the management of childhood GBM.

*Children's Cancer Hospital

Neuro-Radiology Department at The University of Texas M.D. Anderson Cancer Center, Houstan, TX

The Molecular Genetics Division, German Cancer Research Center-DKFZ

§Department of Pediatric Hematology, Oncology, and Immunology, University of Heidelberg

Department of Neuropathology, University Hospital Heidelberg, Heidelberg, Germany

Reprints: Johannes E. Wolff, MD, PhD, The University of Texas M.D. Anderson Cancer Center, Department of Pediatrics, Unit 87, 1515 Holcombe Boulevard, Houston, Texas 77030 (e-mail:

Received for publication October 27, 2009; accepted February 1, 2010

© 2010 Lippincott Williams & Wilkins, Inc.