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51.
Phillips PP Gillespie SH Boeree M Heinrich N Aarnoutse R McHugh T Pletschette M Lienhardt C Hafner R Mgone C Zumla A Nunn AJ Hoelscher M 《The Journal of infectious diseases》2012,205(Z2):S250-S257
A growing number of new drugs for the treatment of tuberculosis are in clinical development. Confirmatory phase 3 trials are expensive and time-consuming and the question of whether one particular drug combination can be used to treat tuberculosis is less important from a public health perspective than the question of which are the shortest, simplest, most effective, and safest regimens. While preclinical and phase 1 studies provide some guidance in the selection of combinations for clinical evaluation, a large number of combinations will require phase 2 testing to ensure that only the best regimens advance to phase 3. The multi-arm multi-stage trial design is an example of a treatment selection-adaptive design where multiple experimental arms are each simultaneously compared with a common control and interim analyses allow for poor performing arms to be dropped early. Such designs, if designed and implemented correctly, require fewer patients, can be completed in a shorter time frame, and answer more relevant questions without any loss in statistical validity or scientific integrity. There are, however, practical issues that must be considered in applying this in tuberculosis treatment trials. More innovative trials designs should be considered to speed drug and regimen development for the treatment of tuberculosis. 相似文献
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Gossec L Smolen JS Gaujoux-Viala C Ash Z Marzo-Ortega H van der Heijde D FitzGerald O Aletaha D Balint P Boumpas D Braun J Breedveld FC Burmester G Cañete JD de Wit M Dagfinrud H de Vlam K Dougados M Helliwell P Kavanaugh A Kvien TK Landewé R Luger T Maccarone M McGonagle D McHugh N McInnes IB Ritchlin C Sieper J Tak PP Valesini G Vencovsky J Winthrop KL Zink A Emery P;European League Against Rheumatism 《Annals of the rheumatic diseases》2012,71(1):4-12
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Pulmonary arterial hypertension (PAH) is one of the leading causes of mortality in systemic sclerosis (SSc). We audited adherence
with available recommendations regarding cardiopulmonary screening for PAH in SSc and explored potential factors influencing
clinical practice. A retrospective case note review of 108 patients with SSc who had attended outpatient clinic over the previous
year was undertaken. Records were scrutinised for evidence of previous assessment with trans-thoracic echocardiography (TTE)
and pulmonary function tests (PFT), along with information regarding clinical phenotype and serological subset. The proportion
of patients for whom screening had been undertaken within the previous 12 months was low, with significantly fewer having
TTE compared with PFT assessment (34.7% vs. 53.1%, p = 0.014). The majority of patients had undergone TTE and PFT assessment within the previous 2 years, but a lower proportion
had undergone TTE compared with PFT (69.4% vs. 82.7%, p = 0.044). There were strong trends for more frequent PFT assessment in younger patients, limited cutaneous SSc and worse
previous PFT results. In contrast, the frequency of TTE assessment was not associated with previous investigation results
or disease subtype. Serological profile did not influence the frequency of either TTE or PFT assessments. Disparity between
available published guidelines may influence both the frequency and preference of PAH screening in SSc in clinical practice.
The higher frequency of PFT assessment might reflect a perceived superiority amongst clinicians of PFT over TTE in the early
identification of SSc-PAH. SSc-specific guidelines, possibly incorporating additional independent risk factors, may improve
the cost-effectiveness and clinical efficacy of screening recommendations designed to ensure the early identification of PAH
in patients with SSc. 相似文献
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Background: Although aphasia affects quality of life (QoL), the impact within specific domains (e.g., psychosocial, communication) is poorly understood. Moreover, the complex and multidimensional nature of QoL renders it difficult to measure accurately using a single global scale. Aims: Using two recently developed QoL scales, the Stroke and Aphasia Quality of Life Scale‐39, (SAQOL; Hilari, Byng, Lamping, & Smith, 2003a) and the American Speech Language Hearing Association's Quality of Communication Life Scale (QCL; Paul et al., 2004), this study aimed to document the domains of QoL that were most affected for participants with aphasia compared to control participants, as well as to determine the relationship between the two scales, their sub‐domains, and linguistic variables in aphasia. Methods & Procedures: The two scales were administered to a group of 19 participants with aphasia (14 male, 5 female), ages ranging from 27 to 79 years, and 19 age‐ and gender‐matched control participants. Various types and severity of aphasia were represented in the aphasia group. The performances of aphasia and control groups were compared, and correlation analyses examined the relationship between the two scales and their sub‐domains in the aphasia group only. Outcomes & Results: Compared to control participants, QoL was lower in participants with aphasia, with the communication sub‐domain of SAQOL and socialisation/activities sub‐domain of QCL being the most affected areas of functioning. Between the two scales, the communication sub‐domain of SAQOL correlated with the socialisation/activities sub‐domain and the QCL mean. Moreover, linguistic variables correlated strongly with psychosocial, communication and socialisation/activities sub‐domains of QoL. Conclusions: Measuring QoL using the SAQOL and the QCL captures different but equally important aspects of experiences of living with aphasia. When interpreted together, they provide a holistic picture of functioning in aphasia that includes broad overviews of QoL from the SAQOL and a finer‐grained analysis of communication impairments on QoL from the QCL. 相似文献
57.
Matthew M. Carper R. Kathryn McHugh David H. Barlow 《Administration and policy in mental health》2013,40(2):87-95
Computerized cognitive behavioral therapy is an efficacious treatment for anxiety and depression with the potential to improve access to evidence-based care. However, its adoption in clinical practice in the US has been low and thus there is a need for identification of barriers to its use. We examined treatment-seeking patient (n = 55) and clinician (n = 26) perceptions of computer-based psychological treatment (CBPT) using Diffusion of Innovations theory as a conceptual framework. Diffusion of Innovations theory emphasizes potential adopter perceptions as being key to understanding adoption decisions, thus making it an ideal framework for evaluating barriers to use. Overall, treatment-seeking patients held slightly negative perceptions of CBPT, while clinicians’ perceptions were more neutral. In both groups, perceptions of observability (seeing or hearing about the treatment in use) were rated lowest. Implications for dissemination efforts and suggestions for future research are discussed. 相似文献
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Fibroblast growth factor family aberrations as a putative driver of head and neck squamous cell carcinoma in an epidemiologically low‐risk patient as defined by targeted sequencing
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Brittny N. Tillman MD Megan Yanik BS Andrew C. Birkeland MD Chia‐Jen Liu MS Daniel H. Hovelson MS Andi K. Cani MS Nallasivam Palanisamy PhD Shannon Carskadon MS Thomas E. Carey PhD Carol R. Bradford MD Scott A. Tomlins MD PhD Jonathan B. McHugh MD Matthew E. Spector MD J. Chad Brenner PhD 《Head & neck》2016,38(Z1):E1646-E1652