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- Vihinen, M, et al.
(författare)
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BTKbase: XLA-mutation registry
- 1996
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Ingår i: Immunology today. - 0167-5699. ; 17:11, s. 502-506
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Tidskriftsartikel (refereegranskat)
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| 5. |
- Cicardi, M., et al.
(författare)
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Icatibant, a New Bradykinin-Receptor Antagonist, in Hereditary Angioedema
- 2010
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Ingår i: New England Journal of Medicine. - 0028-4793. ; 363:6, s. 532-541
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Tidskriftsartikel (refereegranskat)abstract
- BACKGROUND Hereditary angioedema is characterized by recurrent attacks of angioedema of the skin, larynx, and gastrointestinal tract. Bradykinin is the key mediator of symptoms. Icatibant is a selective bradykinin B2 receptor antagonist. METHODS In two double-blind, randomized, multicenter trials, we evaluated the effect of icatibant in patients with hereditary angioedema presenting with cutaneous or abdominal attacks. In the For Angioedema Subcutaneous Treatment (FAST) 1 trial, patients received either icatibant or placebo; in FAST-2, patients received either icatibant or oral tranexamic acid, at a dose of 3 g daily for 2 days. Icatibant was given once, subcutaneously, at a dose of 30 mg. The primary end point was the median time to clinically significant relief of symptoms. RESULTS A total of 56 and 74 patients underwent randomization in the FAST-1 and FAST-2 trials, respectively. The primary end point was reached in 2.5 hours with icatibant versus 4.6 hours with placebo in the FAST-1 trial (P=0.14) and in 2.0 hours with icatibant versus 12.0 hours with tranexamic acid in the FAST-2 trial (P<0.001). In the FAST-1 study, 3 recipients of icatibant and 13 recipients of placebo needed treatment with rescue medication. The median time to first improvement of symptoms, as assessed by patients and by investigators, was significantly shorter with icatibant in both trials. No icatibant-related serious adverse events were reported. CONCLUSIONS In patients with hereditary angioedema having acute attacks, we found a significant benefit of icatibant as compared with tranexamic acid in one trial and a nonsignificant benefit of icatibant as compared with placebo in the other trial with regard to the primary end point. The early use of rescue medication may have obscured the benefit of icatibant in the placebo trial. (Funded by Jerini; ClinicalTrials.gov numbers, NCT00097695 and NCT00500656.)
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- Drees, Holger, et al.
(författare)
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On a Minimum Distance Procedure for Threshold Selection in Tail Analysis
- 2020
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Ingår i: SIAM Journal on Mathematics of Data Science. - : Society for Industrial & Applied Mathematics (SIAM). - 2577-0187. ; 2:1, s. 75-102
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Tidskriftsartikel (refereegranskat)abstract
- Power-law distributions have been widely observed in different areas of scientific research. Practical estimation issues include selecting a threshold above which observations follow a power-law distribution and then estimating the power-law tail index. A minimum distance selection procedure (MDSP) proposed by Clauset, Shalizi, and Newman [SIAM Rev., 51 (2009), pp. 661-703] has been widely adopted in practice for the analyses of social networks. However, theoretical justifications for this selection procedure remain scant. In this paper, we study the asymptotic behavior of the selected threshold and the corresponding power-law index given by the MDSP. For independent and identically distributed (iid) observations with Pareto-like tails, we derive the limiting distribution of the chosen threshold and the power-law index estimator, where the latter estimator is not asymptotically normal. We deduce that in this iid setting MDSP tends to choose too high a threshold level and show with asymptotic analysis and simulations how the variance increases compared to Hill estimators based on a nonrandom threshold. We also provide simulation results for dependent preferential attachment network data and find that the performance of the MDSP procedure is highly dependent on the chosen model parameters.
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- Lindskog, Filip, et al.
(författare)
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Regularly varying measures on metric spaces : Hidden regular variation and hidden jumps
- 2014
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Ingår i: Probability Surveys. - : Institute of Mathematical Statistics. - 1549-5787. ; 11:2014, s. 270-314
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Tidskriftsartikel (refereegranskat)abstract
- We develop a framework for regularly varying measures on complete separable metric spaces S with a closed cone C removed, extending material in [15, 24]. Our framework provides a flexible way to consider hidden regular variation and allows simultaneous regular-variation properties to exist at different scales and provides potential for more accurate estimation of probabilities of risk regions. We apply our framework to iid random variables in ℝ∞+ with marginal distributions having regularly varying tails and to càdlàg Lévy processes whose Lévy measures have regularly varying tails. In both cases, an infinite number of regular-variation properties coexist distinguished by different scaling functions and state spaces.
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