Initial chemotherapy for locally advanced and metastatic NUT carcinoma Journal Article
| Authors: | Luo, J.; Sanchez, M.; Lee, E.; Hertzler, H.; Luong, N.; Mazzola, E.; Finstein, B.; Tamen, R.; Brisbane, G.; Nguyen, T.; Paik, P. K.; Chaft, J. E.; Cheng, M. L.; Khalil, H.; Piha-Paul, S. A.; Sholl, L. M.; Nishino, M.; Jänne, P. A.; DuBois, S. G.; Hanna, G. J.; Shapiro, G. I.; French, C. A. |
| Article Title: | Initial chemotherapy for locally advanced and metastatic NUT carcinoma |
| Abstract: | Introduction: NUT carcinoma (NC) is an underdiagnosed and aggressive poorly differentiated or squamous cell cancer. A subset of NC is sensitive to chemotherapy, but the optimal regimen is unknown. Experts have recommended platinum- and ifosfamide-based therapy based on case reports. Methods: Patients with pathologically confirmed NC with known survival outcomes after chemotherapy and consented to participate in a worldwide registry were studied. Results were summarized using descriptive methods. Results: The study included 118 patients with NC. Median age was 34 (range: 1–82) years, 39% were women, and 61% harbored a BRD4::NUTM1 fusion. Patients received platinum (74%) or ifosfamide (26%, including regimens with both, 13%). Of 62 patients with nonmetastatic disease, 40% had a thoracic primary. Compared with platinum-based chemotherapy, patients who received ifosfamide-based chemotherapy had nominally higher progression-free survival (12 mo: 59% [95% CI: 32–87] versus 37% [95% CI: 22–52], hazard ratio = 0.68 [0.32, 1.42], p = 0.3) but not overall survival (OS). Among the 56 patients with metastatic disease, 80% had a thoracic primary. Ifosfamide had an objective response rate (ORR) of 75% (six of eight) and platinum had an ORR of 31% (11 of 36). Nevertheless, there was no difference in progression-free survival or OS. The 3-year OS of the entire cohort was 19% (95% CI: 10%–28%). Of the 11 patients alive greater than 3 years, all presented with nonmetastatic and operable or resectable disease. Conclusion: There is a numerically higher ORR for ifosfamide-based therapy compared with platinum-based therapy, with limited durability. OS at 3 years is only 19%, and development of effective therapies is an urgent unmet need for this patient population. © 2023 International Association for the Study of Lung Cancer |
| Keywords: | adolescent; adult; cancer chemotherapy; cancer survival; child; treatment outcome; treatment response; aged; major clinical study; overall survival; squamous cell carcinoma; cisplatin; doxorubicin; advanced cancer; cancer patient; chemotherapy; metastasis; progression free survival; etoposide; cohort analysis; vincristine; ifosfamide; register; fusion gene; carcinoma; platinum derivative; taxane derivative; human; male; female; article; poorly differentiated carcinoma; nut carcinoma; long term responders |
| Journal Title: | Journal of Thoracic Oncology |
| Volume: | 19 |
| Issue: | 5 |
| ISSN: | 1556-0864 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2024-05-01 |
| Start Page: | 829 |
| End Page: | 838 |
| Language: | English |
| DOI: | 10.1016/j.jtho.2023.12.022 |
| PUBMED: | 38154515 |
| PROVIDER: | scopus |
| PMCID: | PMC11081848 |
| DOI/URL: | |
| Notes: | Article -- Source: Scopus |
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