Prevalence of gastrointestinal stromal tumour (GIST) in the United Kingdom at different therapeutic lines: an epidemiologic model.
Prevalence of gastrointestinal stromal tumour (GIST) in the United Kingdom at different therapeutic lines: an epidemiologic model.
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DOI：
10.1186/1471-2407-14-364
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发表时间：
2014-05-24
期刊：
影响因子：
3.8
通讯作者：
Maguire A
中科院分区：
文献类型：
--
作者：
Starczewska Amelio JM;Cid Ruzafa J;Desai K;Tzivelekis S;Muston D;Khalid JM;Ashman P;Maguire A
目前可用的伊马替尼和舒尼替尼（三线治疗合格的胃肠道间质瘤）治疗失败的胃肠道间质瘤（GIST）患者的患病率未知，但预计低于英格兰和威尔士使用的2/100，000人群的超孤儿疾病阈值。我们的研究旨在估计一线、二线和最终三线治疗时不可切除/转移性GIST的英国患者的患病率和绝对数量。我们的开放人群模型估计了英国三线治疗合格的GIST患者的患病率将保持在超孤儿疾病阈值以下的概率。GIST患者的发病率、不可切除/转移性疾病的比例和生存估计的模型参数来自有针对性的文献综述和英国癌症登记。通过采用输入参数极值的不同情景检查结果的稳健性。基础病例情景估计符合三线治疗条件的GIST的患病率为1/100，000，患病率计数为598，低于超孤儿疾病阈值的可能性为99.9%。极端情景、单向和概率敏感性分析以及阈值分析证实了这些结果的稳健性。符合三线治疗条件的GIST的患病率非常低，极有可能低于超孤儿疾病阈值。
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. 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. 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. The prevalence of third-line treatment-eligible GIST is very low and highly likely below the ultra-orphan disease threshold.
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