Abstract | 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.
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Authors | Justyna M Starczewska Amelio, Javier Cid Ruzafa, Kamal Desai, Spiros Tzivelekis, Dominic Muston, Javaria Mona Khalid, Philip Ashman, Andrew Maguire |
Journal | BMC cancer
(BMC Cancer)
Vol. 14
Pg. 364
(May 24 2014)
ISSN: 1471-2407 [Electronic] England |
PMID | 24884940
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
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Chemical References |
- Antineoplastic Agents
- Benzamides
- Indoles
- Piperazines
- Protein Kinase Inhibitors
- Pyrimidines
- Pyrroles
- Imatinib Mesylate
- Sunitinib
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Topics |
- Antineoplastic Agents
(therapeutic use)
- Benzamides
(therapeutic use)
- Gastrointestinal Stromal Tumors
(drug therapy, epidemiology, mortality, secondary)
- Humans
- Imatinib Mesylate
- Incidence
- Indoles
(therapeutic use)
- Models, Statistical
- Piperazines
(therapeutic use)
- Prevalence
- Protein Kinase Inhibitors
(therapeutic use)
- Pyrimidines
(therapeutic use)
- Pyrroles
(therapeutic use)
- Rare Diseases
(drug therapy, epidemiology, mortality)
- Registries
- Sunitinib
- Treatment Failure
- United Kingdom
(epidemiology)
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