期刊论文详细信息
BMC Cancer
Prevalence of gastrointestinal stromal tumour (GIST) in the United Kingdom at different therapeutic lines: an epidemiologic model
Andrew Maguire1  Philip Ashman2  Javaria Mona Khalid1  Dominic Muston2  Spiros Tzivelekis3  Kamal Desai1  Javier Cid Ruzafa1  Justyna M Starczewska Amelio1 
[1]Health Economics and Epidemiology, Evidera, Metro Building, 6th floor, No.1 Butterwick, London W6 8DL, UK
[2]Health Economics, Bayer plc, Strawberry Hill, Newbury RG14 1JA, UK
[3]Global Market Access, Bayer Pharma AG, Berlin S157, 03, 305, Germany
关键词: Ultra-orphan disease;    Model;    GIST;    Gastrointestinal stromal tumour;    Prevalence;    Epidemiology;   
Others  :  858794
DOI  :  10.1186/1471-2407-14-364
 received in 2013-01-30, accepted in 2014-05-12,  发布年份 2014
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【 摘 要 】

Background

The prevalence of patients with gastrointestinal stromal tumourgst (GIST) who fail currently available treatments imatinib and sunitinib (third-line treatment-eligible GIST) is unknown, but is expected to be below an ultra-orphan disease threshold of 2/100,000 population used in England and Wales. Our study was designed to estimate the prevalence and absolute number of UK patients with unresectable/metastatic GIST at first-, second- and eventually third-line treatment.

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. Model parameters for incidence, proportion of unresectable/metastatic disease and survival estimates for GIST patients were obtained from a targeted literature review and a UK cancer register. The robustness of the results was checked through differing scenarios taking extreme values of the input parameters.

Results

The base-case scenario estimated a prevalence of third-line treatment-eligible GIST of 1/100,000 and a prevalence count of 598 with a 99.9% likelihood of being below the ultra-orphan disease threshold. The extreme scenarios, one-way and probabilistic sensitivity analyses and threshold analysis confirmed the robustness of these results.

Conclusions

The prevalence of third-line treatment-eligible GIST is very low and highly likely below the ultra-orphan disease threshold.

【 授权许可】

   
2014 Starczewska Amelio et al.; licensee BioMed Central Ltd.

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