Author:
Starczewska Amelio Justyna M,Cid Ruzafa Javier,Desai Kamal,Tzivelekis Spiros,Muston Dominic,Khalid Javaria Mona,Ashman Philip,Maguire Andrew
Abstract
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.
Publisher
Springer Science and Business Media LLC
Subject
Cancer Research,Genetics,Oncology
Reference58 articles.
1. Kostka R, Gurlich R, Koldova L: Gastrointestinal stromal tumors (GIST): a single center experience. Acta Chir Belg. 2012, 112: 33-39.
2. Steigen SE, Eide TJ: Gastrointestinal stromal tumors (GISTs): a review. APMIS. 2009, 117: 73-86. 10.1111/j.1600-0463.2008.00020.x.
3. Ahmed I, Welch NT, Parsons SL: Gastrointestinal stromal tumours (GIST) - 17 years experience from mid trent region (United Kingdom). Eur J Surg Oncol. 2008, 34: 445-449. 10.1016/j.ejso.2007.01.006.
4. Hislop J, Quayyum Z, Elders A, Fraser C, Jenkinson D, Mowatt G, Sharma P, Vale L, Petty R: Systematic Review of the Clinical and Cost-Effectiveness of Imatinib at Escalated Doses of 600 mg/day or 800 mg/day for the Treatment of Unresectable and/or Metastatic Gastrointestinal Stromal Tumours Which Have Progressed on Treatment at a Dose of 400 mg/day. Aberdeen HTA Group. 2010, Aberdeen, UK: Institute of Applied Health Sciences, University of Aberdeen
5. Nilsson B, Bumming P, Meis-Kindblom JM, Oden A, Dortok A, Gustavsson B, Sablinska K, Kindblom LG: Gastrointestinal stromal tumors: the incidence, prevalence, clinical course, and prognostication in the preimatinib mesylate era–a population-based study in western Sweden. Cancer. 2005, 103: 821-829. 10.1002/cncr.20862.
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