| 1. |
- Astermark, Jan, et al.
(författare)
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Managing Haemophilia for Life: 4th Haemophilia Global Summit
- 2014
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Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 20, s. 1-20
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Forskningsöversikt (refereegranskat)abstract
- The 4th Haemophilia Global Summit was held in Potsdam, Germany, in September 2013 and brought together an international faculty of haemophilia experts and delegates from multidisciplinary backgrounds. The programme was designed by an independent Scientific Steering Committee of haemophilia experts and explored global perspectives in haemophilia care, discussing practical approaches to the optimal management of haemophilia now and in the future. The topics outlined in this supplement were selected by the Scientific Steering Committee for their relevance and potential to influence haemophilia care globally. In this supplement from the meeting, Jan Astermark reviews current understanding of risk factors for the development of inhibitory antibodies and discusses whether this risk can be modulated and minimized. Factors key to the improvement of joint health in people with haemophilia are explored, with Carlo Martinoli and Victor Jimenez-Yuste discussing the utility of ultrasound for the early detection of haemophilic arthropathy. Other aspects of care necessary for the prevention and management of joint disease in people with haemophilia are outlined by Thomas Hilberg and Sebastian Lobet, who highlight the therapeutic benefits of physiotherapy and sports therapy. Riitta Lassila and Carlo-Federico Perno describe current knowledge surrounding the risk of transmission of infectious agents via clotting factor concentrates. Finally, different types of extended half-life technology are evaluated by Mike Laffan, with a focus on the practicalities and challenges associated with these products.
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| 2. |
- Berntorp, Erik, et al.
(författare)
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Pharmacokinetics, phenotype and product choice in haemophilia B: how to strike a balance?
- 2014
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Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 20, s. 1-11
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Tidskriftsartikel (refereegranskat)abstract
- At the 7th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held in Brussels, Belgium, in February 2014, Pfizer sponsored a satellite symposium entitled: "Pharmacokinetics, phenotype and product choice in haemophilia B: How to strike a balance?" Co-chaired by Cedric Hermans (Cliniques Universitaires Saint Luc, Brussels, Belgium) and Mike Laffan (Imperial College, London, UK), the symposium provided an opportunity to debate whether pharmacokinetic (PK) parameters are good surrogates for clinical efficacy for haemophilia B in clinical practice, consider the perceptions and evidence of disease severity, and examine how these considerations can inform approaches to balancing the potential risks and benefits of the currently available treatment options for haemophilia B. PK parameters are routinely measured in clinical practice and are a requirement of regulatory bodies to demonstrate the clinical efficacy of products; however, the relationship between measured PK parameters and clinical efficacy is yet to be determined, an issue that was debated by Gerry Dolan (University Hospital, Queen's Medical Centre, Nottingham, UK) and Erik Berntorp (Lund University, Malmö Centre for Thrombosis and Haemostasis, Malmö, Sweden). Elena Santagostino (Universita degli Studi di Milano, Milano, Italy) reviewed how differing perceptions on the severity of haemophilia B compared with haemophilia A may have an impact on clinical decision-making. Finally, Andreas Tiede (Hannover Medical School, Hannover, Germany), examined the considerations for balancing the potential risks and benefits of the currently available treatment options for haemophilia B. Although the pathophysiology of haemophilia B has been widely studied and is largely understood, continued investigation and discussion around the optimal management course and appropriate therapeutic choice is warranted.
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| 3. |
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| 4. |
- Matino, D., et al.
(författare)
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Switching clotting factor concentrates: considerations in estimating the risk of immunogenicity
- 2014
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Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 20:2, s. 200-206
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Tidskriftsartikel (refereegranskat)abstract
- The development of neutralizing antibodies to factor VIII (FVIII) is the most serious complication of therapy for haemophilia A. There is now excellent documentation that a large number of both genetic and environmental factors contribute to the risk of FVIII inhibitor incidence. One of the environmental factors that has been proposed as an influence on this complication is the occurrence of FVIII product switching. There are only a small number of clinical studies that have addressed this question, and thus, the amount of objective information available to assess this association is limited. In this review, in addition to summarizing past evidence pertinent to this subject, we present the results of a complementary strategy, a Delphi analysis, to add to the considerations of product switching and FVIII immunogenicity. With the imminent arrival in the clinic of several new FVIII products, the haemophilia community must be prepared to collect prospectively controlled data to better address this important management issue.
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| 5. |
- Perry, D., et al.
(författare)
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FEIBA prophylaxis in haemophilia patients: a clinical update and treatment recommendations
- 2010
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 16:1, s. 80-89
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Tidskriftsartikel (refereegranskat)abstract
- In patients with severe haemophilia and inhibitors, regular factor VIII inhibitor bypassing activity (FEIBA) prophylaxis has been shown to reduce the frequency of bleeding by up to 85% and to improve patient quality of life. FEIBA is well tolerated; the incidence of thrombotic events and of allergic reactions is extremely low. The concept of prophylaxis in haemophilia patients with inhibitors is relatively new and some clinicians may be unsure of how to use FEIBA in this context. These treatment recommendations, based on published evidence plus the collective experience of a group of haematologists (with practical knowledge of managing inhibitor patients with FEIBA prophylaxis), are intended to provide guidance to clinicians considering initiating and maintaining patients on FEIBA prophylaxis with specific focus on practical aspects of patient selection, dosing, monitoring and stop criteria.
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| 6. |
- Sinn, T., et al.
(författare)
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Lessons learned from three university experiments onboard the REXUS/BEXUS sounding rockets and stratospheric balloons
- 2013
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Ingår i: 64th International Astronautical Congress 2013. - : International Astronautical Federation. - 9781629939094 ; , s. 7965-7976
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Konferensbidrag (refereegranskat)abstract
- Over the last three years the authors have been involved in three experiments that were or will be launched on sounding rockets and high altitude balloons with the REXUS/BEXUS program (Rocket-borne / Balloon-borne Experiments for University Students). The first experiment, called Suaineadh was launched from Esrange (Kiruna, Sweden) onboard REXUS 12 in March 2012. Suaineadh had the purpose of deploying a web in space by using centrifugal forces. The payload was lost during re-entry but was recovered 18 month later in early September 2013. StrathSat-R is the second experiment, which had the purpose of deploying two cube satellites with inflatable structures from the REXUS 13 sounding rocket, was launched first in May 2013 and will be launched a second time in spring 2014. The last experiment is the iSEDE experiment which has the goal of deploying an inflatable structure with disaggregated electronics from the high altitude balloon BEXUS15/16 in October 2013. All these experiments have been designed, built and flown in a timeframe of one and a half to two years. This paper will present the lessons learned in project management, outreach, experiment design, fabrication and manufacturing, software design and implementation, testing and validation as well as launch, flight and post-flight. Furthermore, the lessons learned during the recovery mission of Suaineadh will be discussed as well. All these experiments were designed, built and tested by a large group of university students of various disciplines and different nationalities. StrathSat-R and iSEDE were built completely at Strathclyde but the Suaineadh experiment was a joint project between Glasgow and Stockholm which was especially tricky during integration while approaching the experiment delivery deadline. This paper should help students and professionals across various disciplines to build and organise these kinds of projects more efficiently without making the same, sometimes expensive, mistakes all over again.
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| 7. |
- Teitel, J, et al.
(författare)
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A consensus statement on clinical trials of bypassing agent prophylaxis in inhibitor patients.
- 2011
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Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 17, s. 516-521
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Tidskriftsartikel (refereegranskat)abstract
- Summary. The haemophilia literature increasingly contains reports describing the use of bypassing agent prophylaxis (BAP) in patients with severe haemophilia A and inhibitors. However, it is difficult to interpret and compare the results and draw conclusions about treatment efficacy because of small patient numbers and a lack of standardization among BAP studies. This article presents consensus recommendations for standardizing future BAP clinical trials developed by an international panel of haemophilia opinion leaders.
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