Bayer is conducting research on GIST therapy. Appendix I. Appendix II. Model Specifications. Appendix III. Key Data Sources for Model Parameters.
Appendix IV. Literature Search Strategy [ 54 - 58 ]. Each of the authors was funded by his or her employer at the time of study conduct and manuscript preparation. This research and this manuscript were commissioned and sponsored by Bayer.
Study design, data collection, analysis and interpretation, and writing of the manuscript were conducted by Evidera authors. Bayer authors reviewed and provided feedback.
Submission of the resulting manuscript to a scientific journal was included in the initial project scope. National Center for Biotechnology Information , U. BMC Cancer. Published online May Author information Article notes Copyright and License information Disclaimer. Corresponding author. Justyna M Starczewska Amelio: moc. Received Jan 30; Accepted May This article has been cited by other articles in PMC. Methods Our open population model estimates the probability that the prevalence of UK third-line treatment-eligible GIST patients will remain under the ultra-orphan disease threshold.
Conclusions The prevalence of third-line treatment-eligible GIST is very low and highly likely below the ultra-orphan disease threshold. Background Gastrointestinal stromal tumours GIST are relatively rare soft tissue mesenchymal tumours occurring in the gastrointestinal tract, originating in the interstitial cells of Cajal involved in the regulation of the digestive system [ 1 , 2 ].
Table 1 Model parameters. Open in a separate window. Model overview We constructed a model for calculating the prevalence of GIST in the UK population Figure 1 that represents the flow of patients with GIST from first diagnosis to death via two possible treatment pathways and four treatment states. Figure 1. Model parameters, analysis and sensitivity analysis The model base-case scenario was defined by base-case values for each model parameter.
Model outcomes Model outcomes for each scenario included absolute number and prevalence per , population of all patients with GIST, patients in first-line receiving imatinib and second-line receiving sunitinib treatment and third-line treatment-eligible GIST patients who experienced imatinib and sunitinib treatment failures. Results Scenario analysis The base-case and alternative scenarios 1, 2 and 3 are presented in Table 2. Univariate sensitivity analysis Univariate sensitivity analysis performed on the base-case and alternative scenarios demonstrate that the initial incidence of GIST and third-line treatment-eligible GIST survival were most influential on the prevalence of third-line treatment-eligible GIST, as illustrated in the tornado diagram Figure 2.
Figure 2. Threshold analysis To better quantify the risk associated with the two most influential parameters, we determined the threshold incidence and third-line treatment-eligible GIST survival that would cause the third-line treatment-eligible GIST prevalence to exceed 2.
Conclusion Despite the study limitations, there is a very high probability of third-line treatment-eligible GIST prevalence being below 2. Bayer sponsored this research. Click here for file K, docx. Gastrointestinal stromal tumors GIST : a single center experience. Acta Chir Belg. Gastrointestinal stromal tumors GISTs : a review. Aberdeen HTA Group. Gastrointestinal stromal tumors: the incidence, prevalence, clinical course, and prognostication in the preimatinib mesylate era—a population-based study in western Sweden.
Gastrointestinal stromal tumors in a cohort of Chinese patients in Hong Kong. World J Gastroenterol. Prognostic factors after surgery of primary resectable gastrointestinal stromal tumours. Eur J Surg Oncol. A whole population study of gastrointestinal stromal tumors in the Czech Republic and Slovakia. Ann Oncol. Population-based study of the diagnosis and treatment of gastrointestinal stromal tumours.
Br J Surg. In the West, between and , the incidence was initially decreasing APC Subsequently, between and , the incidence started to decrease again with an APC of Our study, to the best of our knowledge, is the first to evaluate the incidence of GIST between and using the USCS database, which covers all 50 states. In our analysis, we found the overall incidence of GIST to be 0. There was an overall 1. However, in contrast to prior studies, we found that the incidence, between and , increased at a greater rate in females compared to males.
Similar to prior studies, we found that blacks had a greater incidence of GIST compared to other races [ 11 ]. However, we also found that the APC in blacks increased at a faster rate than other races. In the study by Ma et al.
We found that the overall incidence between APIs and whites is similar, however, contrary to prior findings, the incidence in whites increased at more than double the rate than in APIs. When comparing incidence by stage at diagnosis, we found that the overall incidence was greater in localized disease compared to regional and distant disease, which had similar overall incidences.
However, we also found that the incidence of localized disease increased at over three times the rate as distant disease. Moreover, the incidence of regional disease has decreased. The incidence of GISTs, when stratified by primary location within the GI tract, is considerably greater in the stomach than other locations such as the small intestine or colorectum, which was also found by other studies [ 3 ]. We also found that the incidence in both the stomach and the small intestine is increasing between and , whereas the incidence in the colorectum is decreasing.
We found that the overall incidence, although similar, was slightly greater in the Northeast compared to other regions. Moreover, when we analyzed how the incidence changed over the years, we found that the incidence initially increased at an expeditious rate only in the Northeast. However, the incidence also increased at a substantial rate in the South and Midwest.
Between and , the West had a fluctuating incidence of GISTs, and after the incidence started to decrease again. There were several strengths and limitations to our study. As the first study to use the USCS data to evaluate the incidence of GISTs in all 50 states, we were able to more accurately identify at-risk populations compared to prior studies.
Other studies have used the SEER database which has data on a limited percentage of the US population and can underrepresent incidence patterns for cancers, unlike the USCS dataset [ 6 ]. Moreover, our study was done after the period of misdiagnosis of GISTs and thus represents a more accurate representation of incidence patterns compared to prior studies.
A limitation of our study was the inability to stratify data by specific risk factors that may be contributing to the incidence patterns we found for the subpopulations we analyzed. Ultimately, further studies are needed to help determine correlations between specific risk factors and the results we found.
In our study, we investigated the incidence of GIST in all 50 states. We found that the overall incidence was greatest in males, blacks, localized disease, the stomach, and those in the Northeast. The APC in these groups also increased at a rapid rate between and The reasons for these trends are still unclear and require further studies that look at specific risk factors and how they influence the incidence of GIST in specific stratifications.
Our findings will undoubtedly aid in the development of surveillance guidelines that can help reduce the morbidity and mortality of this cancer. Cureus is not responsible for the scientific accuracy or reliability of data or conclusions published herein. All content published within Cureus is intended only for educational, research and reference purposes.
Additionally, articles published within Cureus should not be deemed a suitable substitute for the advice of a qualified health care professional. GIST may be curable if there is one early stage tumor that can be surgically removed without damaging vital organs. This can sometimes be done with minimally invasive laparoscopic surgery. Some people make a full recovery without needing further treatment. Cases of GIST with tumors that are smaller than 2 centimeters cm are most likely to be cured with surgery alone.
Tumors that are larger than 2 cm are more likely to recur. Tumors that are 10 cm in size or larger are very likely to recur. The cancer is still treatable. These therapies can help shrink tumors, slow the spread of cancer, and relieve symptoms. The outlook for people with GIST varies.
Due to evolving therapies, people diagnosed within the last several years have more treatment options than ever. TKIs are paving the way for a more personalized approach to treatment. These drugs target specific genetic mutations that are responsible for GIST. But your general health may benefit from behaviors such as:.
Some people may need to take TKIs long term. GIST is a type of tumor that starts in the gastrointestinal tract. So the estimate may not show the results of better diagnosis or treatment available for less than 5 years. Talk with your doctor if you have any questions about this information. Learn more about understanding statistics. The next section in this guide is Risk Factors. It explains what factors may increase the chance of developing a GIST.
Use the menu to choose a different section to read in this guide. Types of Cancer.
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