Clinicopathologic Features and Long-term Outcomes of NUT Midline Carcinoma

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Clinicopathologic Features and Long-term Outcomes of NUT Midline Carcinoma

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Title: Clinicopathologic Features and Long-term Outcomes of NUT Midline Carcinoma
Author: Bauer, Daniel Evan; Mitchell, C. M.; Strait, K. M.; Lathan, Christopher Scott; Stelow, E. B.; Luer, S. C.; Muhammed, S.; Evans, A. G.; Sholl, Lynette Marie; Rosai, J.; Giraldi, E.; Oakley, R. P.; Rodriguez-Galindo, C; London, Wendy B; Sallan, Stephen Earl; Bradner, James Elliott; French, Christopher Alexander

Note: Order does not necessarily reflect citation order of authors.

Citation: Bauer, D. E., C. M. Mitchell, K. M. Strait, C. S. Lathan, E. B. Stelow, S. C. Luer, S. Muhammed, et al. 2012. “Clinicopathologic Features and Long-Term Outcomes of NUT Midline Carcinoma.” Clinical Cancer Research 18 (20) (August 15): 5773–5779. doi:10.1158/1078-0432.ccr-12-1153.
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Abstract: Purpose

NUT midline carcinoma (NMC) is a poorly differentiated squamous cancer characterized by rearrangement of the NUT gene. Research advances have provided opportunities for targeted therapy in NMC, yet the clinical features of this rare disease have not been systematically characterized. We report on a large population of such patients to identify the disease characteristics and treatments, correlate them with outcome, and to consider clinical recommendations.

Experimental Design

A clinical database was established using retrospective demographic and outcomes data available on all known cases of NMC. Questionnaires were completed by treating physicians. Pathologic, demographic, and clinical variables were assessed for 63 patients, the largest cohort of NMC patients studied to date. Outcome data from 54 patients were available for survival analyses.


The diagnosis of NMC has increased annually since 2007. Since 2009, there has been an observed increase in the age at diagnosis (p<0.05). Geographic distribution of NMC patients has been concentrated in the United States (n=41, 65%). The median overall survival for patients with NMC was 6.7 months. The 2-year progression-free survival (PFS) was 9% with a 95% CI of 1%–17% (1-year PFS 15% (5%–24%)) and 2-year overall survival (OS) was 19% with a 95% CI of 7%–31% (1-year OS: 30% (27%–34%). Multivariate analysis suggested that extent of surgical resection and initial radiotherapy were independent predictors of PFS and OS. Notably, no chemotherapeutic regimen was associated with improved outcome.


NMC portends a poor prognosis among all squamous cell neoplasms and appears to be frequently unrecognized. The finding that conventional chemotherapy has been inadequate indicates a pressing need for the development of targeted therapeutics. Intensive local therapies such as gross total resection and radiotherapy might be associated with enhanced survival.
Published Version: doi:10.1158/1078-0432.CCR-12-1153
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