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Tumor Discovery Expert consensus of NUT carcinoma

rates compared to conventional treatments, offering a non-continuous data. The 199 reviewed papers did not
new promising therapeutic avenue. Considering the uniformly or comprehensively study and report all relevant
poor prognosis of NUT carcinoma, where most patients indicators, resulting in potential discrepancies in IHC and
have short survival times and are unsuitable for repeat fusion gene results. These findings must be interpreted
surgery, post-operative recurrent or metastatic patients within the clinical context. Future large-scale, multi-center
should prioritize refining high-throughput sequencing to studies are required for a more precise investigation.
identify sensitive drugs. Given that there are currently no
approved targeted therapeutic agents specifically for NUT 6. Conclusion
carcinoma, it is recommended that patients either directly Based on the existing literature and insights from
participate in clinical trials involving BET inhibitors, multidisciplinary experts, we have developed an expert
HDAC inhibitors, and other novel therapies, or on disease consensus on the diagnosis and treatment of NUT
progression following conventional anti-tumor treatments, carcinoma. This consensus provides comprehensive and
patients are encouraged to actively participate in clinical practical recommendations for healthcare providers
trials involving BET inhibitors, HDAC inhibitors, and managing NUT carcinoma throughout patient care.
other novel therapies.
Given the poor prognosis and high invasiveness of
(a) Recommendation 8 NUT carcinoma as a rare tumor, its non-specific features
For patients with post-operative recurrence or distant complicate diagnosis, and the overall effectiveness
metastasis who are unsuitable for further surgery, apart of existing conventional treatment methods remains
from optimizing high-throughput sequencing to select insufficient. Although the prospects for targeted agents
sensitive drugs. It is recommended that patients either such as BET and HDAC inhibitors are promising,
directly participate in clinical trials involving BET overcoming drug resistance to these agents and selecting
inhibitors, HDAC inhibitors, and other novel therapies, or the most appropriate treatment combinations still
on disease progression following conventional anti-tumor require further exploration. Therefore, focused research
treatments, patients are encouraged to actively participate on NUT carcinoma patients is essential to enhance our
in clinical trials involving BET inhibitors, HDAC understanding of the disease and improve diagnostic and
inhibitors, and other novel therapies (evidence level: treatment strategies in the future. Specialized committees
grade 3; recommendation level: strongly recommended). have recently been established in various countries. 87-89
In cases of treatment progression, high-throughput For example, the International NUT Carcinoma Registry
sequencing should be refined, suitable drugs should be was established in the United States to record the clinical,
selected based on the results, and participation in relevant genetic, and pathological characteristics and outcomes
clinical trials should be actively considered (evidence level: of NUT carcinoma patients diagnosed since 1993.
18
grade 5; recommendation level: strongly recommended). However, the patients included are predominantly from
the American and European populations.
5. Limitations
According to the most recent data on population
This study has several limitations. First, high-quality and vital statistics from the United Nations (April
data were not available for some of the topics discussed. 2024), the United States and Europe account for
90
Observational studies inherently carry a high risk of 4.16% (331,449,281/7,975,105,000) and 9.32%
incomplete outcome data and selective outcome reporting. (743,556,000/7,975,105,000) of the global population,
Publication bias may also influence researchers to prioritize respectively. In contrast, China and the other Asian
reporting positive results over negative ones. In case series, countries account for 17.8% (1,419,873,864/7,975,105,000)
selective reporting may lead to an emphasis on successful and 59.2% (4,722,635,000/7,975,105,000) of the total
surgical outcomes. Other potential sources of bias include population, respectively. These demographic data indicate
baseline imbalances, such as population selection in a significant potential number of NUT carcinoma cases
observational studies. in China and Asia. In response to these statistics, we
In addition, some relevant articles may have been established the first specialized center for NUT carcinoma
overlooked. Second, due to the extreme rarity and poor in China in January 2023. In June 2023, the NUT Carcinoma
prognosis of NUT carcinoma, physicians often lack Gene Diagnosis Working Group was established under
sufficient awareness of the disease. Consequently, we aimed the Tumor Gene Diagnosis Special Committee of the
to include all available data on NUT carcinoma, but large- Chinese Anti-Cancer Association. We initiated a real-
scale studies remain scarce. Much of the data analyzed in world cohort study (NCT06073938) investigating the
this review derived from case reports, leading to gaps and efficacy of the BET inhibitor (NHWD 870 HCl) for solid

Volume 3 Issue 4 (2024) 22 doi: 10.36922/td.4904