Abstract
In this thesis, various studies are presented related to gout and hyperuricemia, divided among three parts. The first part focused on the clinical management of gout and hyperuricemia. The main study of this section revealed that the interleukin-1 inhibitor anakinra is an efficacious treatment for managing acute gout flares by showing non-inferiority to conventional treatments in reducing pain in patients with an acute gout flare, further supporting the validity of the role of interleukin-1 in gout. Furthermore, studies in this section showed that the long-term management of gout and hyperuricemia is suboptimal in terms of the one-year medication adherence of urate-lowering therapy (ULT) in the Netherlands, and the high frequency of gout flare recurrences within three months after initiating ULT in gout patients. Lastly, this section revealed that the concept of treating to a serum urate target with ULT needs further substantiation in gout, but may be of added value in lowering serum urate levels.
Studies included in the second part focused on measuring the effects that gout may have on patients using patient-reported outcome measures (PROMs). It was shown that more evidence on the psychometric properties of commonly used PROMs in gout clinical research should become available, based on modern psychometric techniques and methodological sound studies. Moreover, the Gout Attack Intensity Score (GAIS) was introduced as a score for use in acute gout trials for determining the severity of gout flare symptom severity, considering the levels of patient-reported pain, swelling and tenderness. The GAIS was shown to be a reliable and responsive PROM.
The third part examined the societal burden of gout and hyperuricemia in terms of the cost-effectiveness of the treatment options for these diseases. It revealed that the ULT allopurinol and febuxostat were both cost-effective compared to no ULT, and that at higher willingness-to-pay thresholds febuxostat was the favored treatment. For managing acute gout flares, conventional therapies offered similar health economic implications, although naproxen was overall most favored. Compared to conventional therapies anakinra was not cost-effective, suggesting from a health economic perspective, it would most likely be suited as a second-line therapy for treating gout flares.
Studies included in the second part focused on measuring the effects that gout may have on patients using patient-reported outcome measures (PROMs). It was shown that more evidence on the psychometric properties of commonly used PROMs in gout clinical research should become available, based on modern psychometric techniques and methodological sound studies. Moreover, the Gout Attack Intensity Score (GAIS) was introduced as a score for use in acute gout trials for determining the severity of gout flare symptom severity, considering the levels of patient-reported pain, swelling and tenderness. The GAIS was shown to be a reliable and responsive PROM.
The third part examined the societal burden of gout and hyperuricemia in terms of the cost-effectiveness of the treatment options for these diseases. It revealed that the ULT allopurinol and febuxostat were both cost-effective compared to no ULT, and that at higher willingness-to-pay thresholds febuxostat was the favored treatment. For managing acute gout flares, conventional therapies offered similar health economic implications, although naproxen was overall most favored. Compared to conventional therapies anakinra was not cost-effective, suggesting from a health economic perspective, it would most likely be suited as a second-line therapy for treating gout flares.
| Original language | English |
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| Qualification | Doctor of Philosophy |
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| Award date | 11 Jul 2019 |
| Place of Publication | Enschede |
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| Print ISBNs | 978-90-365-4811-3 |
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| Publication status | Published - 11 Jul 2019 |