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- Beeton, K, et al.
(author)
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Recent developments in clinimetric instruments
- 2006
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In: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 12:Suppl. 3, s. 102-107
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Journal article (peer-reviewed)abstract
- Assessment of impairment and function is essential in order to monitor joint status and evaluate therapeutic interventions in patients with haemophilia. The improvements in the treatment of haemophilia have required the development of more sensitive tools to detect the more minor dysfunctions that may now be apparent. This paper outlines some of the recent developments in this field. The Haemophilia Joint Health Score (HJHS) provides a systematic and robust measure of joint impairment. The MRI Scoring System has been designed to provide a comprehensive scoring system combining both progressive and additive scales. The Functional Independence Score for Haemophilia (FISH) has been developed to assess performance of functional activities and can be used in conjunction with the Haemophilia Activities List (HAL) which provides a self report measure of function. It is recommended that both measures are evaluated as these tools measure different constructs. Further refinement and testing of the psychometric properties of all of these tools is in progress. More widespread use of these tools will enable the sharing of data across the world so promoting best practice and ultimately enhancing patient care.
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- Feldman, E. J., et al.
(author)
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On the use of lonafarnib in myelodysplastic syndrome and chronic myelomonocytic leukemia
- 2008
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In: Leukemia. - : Springer Science and Business Media LLC. - 0887-6924 .- 1476-5551. ; 22:9, s. 1707-1711
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Journal article (peer-reviewed)abstract
- Lonafarnib is an orally bio-available farnesyltransferase inhibitor that prevents farnesylation of specific target proteins including Ras. In a multicenter study, 67 patients with advanced myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) were treated with a continuous oral dose of 200-300 mg of lonafarnib and were evaluated for hematologic, pathologic and pharmacodynamic response. The median age of patients was 70 years (range 44-86). There were 32 patients with MDS (RAEB-20 and RAEB-t-12) and 35 with CMML. Overall 16 (24%) of the patients responded with two patients achieving a complete remission and one a partial response. Responses were seen in 6/32 and 10/35 patients with MDS and CMML, respectively. Of the 19 patients who were platelet transfusion-dependent prior to treatment, 5 (26%) became transfusion-free for a median duration of 185 days. A decrease in the farnesylation of the HDJ-2 protein measured in patient-derived cells was observed in the majority of patients during treatment with lonafarnib, but no clear correlation between changes in farnesylation and clinical effect could be made. Gastrointestinal toxicity was significant with 19% of patients discontinuing therapy due to diarrhea, nausea and/or anorexia. Lonafarnib has demonstrable activity in patients with advanced MDS and CMML.
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3. |
- Bonaca, Marc P, et al.
(author)
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Antithrombotics in acute coronary syndromes
- 2009
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In: Journal of the American College of Cardiology. - : Elsevier BV. - 0735-1097 .- 1558-3597. ; 54:11, s. 969-984
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Journal article (peer-reviewed)abstract
- Antithrombotic agents are an integral component of the medical regimens and interventional strategies currently recommended to reduce thrombotic complications in patients with acute coronary syndromes (ACS). Despite great advances with these therapies, associated high risks for thrombosis and hemorrhage remain as the result of complex interactions involving patient comorbidities, drug combinations, multifaceted dosing adjustments, and the intricacies of the care environment. As such, the optimal combinations of antithrombotic therapies, their timing, and appropriate targeted subgroups remain the focus of intense research. During the last several years a number of new antithrombotic treatments have been introduced, and new data regarding established therapies have come to light. Although treatment guidelines include the most current available data, subsequent findings can be challenging to integrate. This challenge is compounded by the complexity associated with different efficacy and safety measures and the variability in study populations, presenting syndromes, physician, and patient preferences. In this work we review recent data regarding clinically available antiplatelet and anticoagulation agents used in the treatment of patients with ACS. We address issues including relative efficacy, safety, and timing of therapies with respect to conservative and invasive treatment strategies. In specific cases we will highlight remaining questions and controversies and ongoing trials, which will hopefully shed light in these areas. In addition to reviewing existing agents, we take a look forward at the most promising new antithrombotics currently in late-stage clinical development and their potential role in the context of ACS management.
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5. |
- Gauthier, S, et al.
(author)
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Mild cognitive impairment
- 2006
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In: Lancet (London, England). - 1474-547X. ; 367:9518, s. 1262-1270
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Journal article (peer-reviewed)
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7. |
- Braunerhjelm, Pontus, et al.
(author)
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Cluster Genesis : Technology-Based Industrial Development
- 2007
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Book (other academic/artistic)abstract
- This book examines the origins and emergence of technology-based industrial clusters - regional concentrations of related firms and organizations - in order to understand the forces that promoted economic development. Many places attempt to emulate the world’s most famous industrial cluster Silicon Valley, with its rich institutional landscape of engaged and leveraged research universities, high-flying local venture capitalists, world class supporting business and legal consultants, and rich collaborative networks. While mature clusters may look similar, what really matters is the process by which clusters come into existence. But there is little understanding of such processes, and little guidance provided on the role of policies in promoting cluster emergence. The book attempts to bridge this gap in the literature by focusing on the early origins of high-technology cluster in Europe, the United States, and China, and the ensuing policy implications. The book is organized around three main themes: Creation Myths Revisited, Considering the Development Cluster Context, and Crafting Cluster and Economic Development Policy. The empirical analyses suggest that clusters that grow rapidly as compared to the less successful ones are distinguished by vigorous entrepreneurial activity and the active building of institutions aided by the forces of agglomeration economies.
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