Clinicopathologic Features and Long-Term Outcomes of NUT Midline Carcinoma: An Index Report of the International NMC Registry
Daniel Bauer, Chelsey Mitchell, Kelly Straight, Christopher Lathan, Edward Stelow, Sonja Luer, Somala Muhammed, Andrew Evans, Lynette Sholl, Juan Rosai, Eugenia Giraldi, Richard Oakley, Carlos Rodriguez-Galindo, Wendy London, Stephen Sallan, James Bradner, Christopher French. Dana-Farber Cancer Institute, and Division of Hematology/Oncology, Boston, MA; Brigham & Women's Hospital, Harvard Medical School, Boston; Dana-Farber Harvard Cancer Care and Children's Hospital, Boston; Dana-Farber Cancer Institute, Boston; University of Virginia, Charlottesville; University Children's Hospital of Bern, Inselspital, Bern, Switzerland; Centro Diagnostico Italiano International Center for Oncologic Pathology Consultations, Milan, Italy; Ospedali Riuniti, Bergamo, Italy
Background: 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. An International NMC Registry has been created to identify patient characteristics and treatments correlating with outcome, and to start providing first clinical recommendations.
Design: A clinical database was established first 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. Outcome data from 54 patients were available for survival analyses.
Results: The diagnosis of NMC is increasing annually since 2007. A significant increase in age at diagnosis has been observed since 2009 (p < 0.05). Geographic distribution of NMC registry patients is heavily 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 % +/- 4 % and 2-year overall survival (OS) was 19 % +/- 6 %. Extent of surgical resection and initial radiotherapy were independently predictors of PFS and OS in a multivariate analysis. Notably, no chemotherapeutic regimen was associated with improved outcome.
Conclusions: NMC portends a particularly poor prognosis among all squamous cell neoplasms. The inadequacy of conventional chemotherapy establishes a pressing need for the development of targeted therapeutics. Intensive local therapies such as gross total resection and radiotherapy are associated with enhanced survival.
Wednesday, March 21, 2012 9:30 AM
Poster Session V # 292, Wednesday Morning