
Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies.
Publication
, Journal Article
Wagner, LM; Myseros, JS; Lukins, DE; Willen, CM; Packer, RJ
Published in: Pediatr Blood Cancer
May 2018
Young children with emaciation caused by a hypothalamic glioma are considered to have diencephalic syndrome (DS), which is often poorly controlled with conventional treatment. We describe an infant with DS whose tumor progressed following chemotherapy. Biopsy was performed for molecular testing and demonstrated a BRAF fusion. Treatment with the MEK inhibitor trametinib for 18 months resulted in reduction of tumor size, normalization of his weight curve, and marked neurodevelopmental improvement. Our results build on earlier reports of using targeted agents for low-grade glioma, and we review the evolving management strategy for such patients in the era of precision medicine.
Duke Scholars
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Published In
Pediatr Blood Cancer
DOI
EISSN
1545-5017
Publication Date
May 2018
Volume
65
Issue
5
Start / End Page
e26917
Location
United States
Related Subject Headings
- Pyrimidinones
- Pyridones
- Proto-Oncogene Proteins c-met
- Protein Kinase Inhibitors
- Prognosis
- Oncology & Carcinogenesis
- Molecular Targeted Therapy
- Male
- Infant
- Hypothalamic Diseases
Citation
APA
Chicago
ICMJE
MLA
NLM
Wagner, L. M., Myseros, J. S., Lukins, D. E., Willen, C. M., & Packer, R. J. (2018). Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies. Pediatr Blood Cancer, 65(5), e26917. https://doi.org/10.1002/pbc.26917
Wagner, Lars M., John S. Myseros, Douglas E. Lukins, Christi M. Willen, and Roger J. Packer. “Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies.” Pediatr Blood Cancer 65, no. 5 (May 2018): e26917. https://doi.org/10.1002/pbc.26917.
Wagner LM, Myseros JS, Lukins DE, Willen CM, Packer RJ. Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies. Pediatr Blood Cancer. 2018 May;65(5):e26917.
Wagner, Lars M., et al. “Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies.” Pediatr Blood Cancer, vol. 65, no. 5, May 2018, p. e26917. Pubmed, doi:10.1002/pbc.26917.
Wagner LM, Myseros JS, Lukins DE, Willen CM, Packer RJ. Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies. Pediatr Blood Cancer. 2018 May;65(5):e26917.

Published In
Pediatr Blood Cancer
DOI
EISSN
1545-5017
Publication Date
May 2018
Volume
65
Issue
5
Start / End Page
e26917
Location
United States
Related Subject Headings
- Pyrimidinones
- Pyridones
- Proto-Oncogene Proteins c-met
- Protein Kinase Inhibitors
- Prognosis
- Oncology & Carcinogenesis
- Molecular Targeted Therapy
- Male
- Infant
- Hypothalamic Diseases