Concerns regarding clinical trials in haemophilia-Augmenting Iorio's “Research and policy implications of a recently published controlled study in previously untreated haemophilia patients at high risk of inhibitor development” Haemophilia (2017), 1-3 htt

Haemophilia ◽  
2017 ◽  
Vol 23 (5) ◽  
pp. e475-e476
Author(s):  
A. Farrugia ◽  
U. Schlenkrich
2021 ◽  
Vol 10 (3) ◽  
pp. 421
Author(s):  
Niccolo’ Bolli ◽  
Nicola Sgherza ◽  
Paola Curci ◽  
Rita Rizzi ◽  
Vanda Strafella ◽  
...  

Smoldering multiple myeloma (SMM), an asymptomatic plasma cell neoplasm, is currently diagnosed according to the updated IMWG criteria, which reflect an intermediate tumor mass between monoclonal gammopathy of undetermined significance (MGUS) and active MM. However, SMM is a heterogeneous entity and individual case may go from an “MGUS-like” behavior to “early MM” with rapid transformation into symptomatic disease. This wide range of clinical outcomes poses challenges for prognostication and management of individual patients. However, initial studies showed a benefit in terms of progression or even survival for early treatment of high-risk SMM patients. While outside of clinical trials the conventional approach to SMM generally remains that of close observation, these studies raised the question of whether early treatment should be offered in high-risk patients, prompting evaluation of several different therapeutic approaches with different goals. While delay of progression to MM with a non-toxic treatment is clearly achievable by early treatment, a convincing survival benefit still needs to be proven by independent studies. Furthermore, if SMM is to be considered less biologically complex than MM, early treatment may offer the chance of cure that is currently not within reach of any active MM treatment. In this paper, we present updated results of completed or ongoing clinical trials in SMM treatment, highlighting areas of uncertainty and critical issues that will need to be addressed in the near future before the “watch and wait” paradigm in SMM is abandoned in favor of early treatment.


2021 ◽  
Vol 164 (1-2) ◽  
Author(s):  
Chad S. Boda ◽  
Turaj Faran ◽  
Murray Scown ◽  
Kelly Dorkenoo ◽  
Brian C. Chaffin ◽  
...  

AbstractLoss and damage from climate change, recognized as a unique research and policy domain through the Warsaw International Mechanism (WIM) in 2013, has drawn increasing attention among climate scientists and policy makers. Labelled by some as the “third pillar” of the international climate regime—along with mitigation and adaptation—it has been suggested that loss and damage has the potential to catalyze important synergies with other international agendas, particularly sustainable development. However, the specific approaches to sustainable development that inform loss and damage research and how these approaches influence research outcomes and policy recommendations remain largely unexplored. We offer a systematic analysis of the assumptions of sustainable development that underpins loss and damage scholarship through a comprehensive review of peer-reviewed research on loss and damage. We demonstrate that the use of specific metrics, decision criteria, and policy prescriptions by loss and damage researchers and practitioners implies an unwitting adherence to different underlying theories of sustainable development, which in turn impact how loss and damage is conceptualized and applied. In addition to research and policy implications, our review suggests that assumptions about the aims of sustainable development determine how loss and damage is conceptualized, measured, and governed, and the human development approach currently represents the most advanced perspective on sustainable development and thus loss and damage. This review supports sustainable development as a coherent, comprehensive, and integrative framework for guiding further conceptual and empirical development of loss and damage scholarship.


2018 ◽  
Vol 199 (1) ◽  
pp. 43-52 ◽  
Author(s):  
Andrew T. Lenis ◽  
Nicholas M. Donin ◽  
David C. Johnson ◽  
Izak Faiena ◽  
Amirali Salmasi ◽  
...  

2021 ◽  
Vol 80 ◽  
pp. 102226
Author(s):  
Magnus C. Abraham-Dukuma ◽  
Michael O. Dioha ◽  
Okechukwu C. Aholu ◽  
Nnaemeka Vincent Emodi ◽  
Chitzi Ogbumgbada ◽  
...  

1984 ◽  
Vol 4 (4) ◽  
pp. 311-341 ◽  
Author(s):  
Snehendu B. Kar

This article presents a multidimensional model of psychosocial determinants of health behavior for health promotion research and policy analysis. Frequently, health promotion focuses almost exclusively on intrapsychic determinants and on individual level behavior. Based upon Field Theory and attitude theories, this proposed model holds that in populations with comparable sociodemographic and biological status (exogenous variables) a health behavior is a function of direct and interaction effects of five key intrapsychic and external variables. These are: behavioral intentions, social support, accessibility of means for action, personal autonomy, and action situation. Empirical tests with cross-cultural studies in Venezuela, Kenya, and the Philippines provide substantial support for the model. The findings suggest that while health promotion strategies should deal with intrapsychic determinants of behavior, key extrapsychic factors (such as social support, quality and accessibility of health care measures, and situational factors) all have direct and independent effects on health behavior as well. Health promotion research and interventions which aim exclusively at intrapsychic determinants would thus have rather limited overall value. The article discusses key research and policy implications of the model presented.


Author(s):  
Fenton McCarthy ◽  
Katherine M McDermott ◽  
Vinay Kini ◽  
Dale Kobrin ◽  
Nimesh D Desai ◽  
...  

Background: Transcatheter Aortic Valve Replacement (TAVR) demonstrated excellent outcomes in clinical trials of inoperable/high-risk patients. Subsequent approval by the Food and Drug Administration and National Coverage Determination by the Centers for Medicare and Medicaid Services established unique volume requirements for institutions and physicians to perform TAVR. Diffusion of prior cardiovascular interventions has involved less stringent policies and exhibited significant institutional variation in clinical outcomes. Our objective is to compare risk-standardized procedural outcomes across US hospitals performing TAVR to identify hospitals with outlying post-procedure mortality rates. Methods: All Medicare fee-for-service beneficiaries who underwent TAVR between January 1, 2011 and November 30, 2012 were identified. Thirty-day risk-standardized mortality rates (RSMR) were calculated using the Hospital Compare statistical method, a well-validated hierarchical generalized linear model. Results: Claims were examined from 5044 patients undergoing TAVR at 199 hospitals, with a crude 30-day mortality rate of 5.97%. RSMRs modeled using patient-level predictors varied from 4.5 % to 9.0 % (Figure 1). One hospital had a RSMR statistically lower than the national mean (4.5%, P<0.05), and two hospitals had RSMRs statistically higher than the national mean (8.5% and 6.9%, P<0.05). Conclusions: Clinical outcomes among TAVR hospitals in high-risk/inoperable patients demonstrated very little variability, few outliers, and excellent outcomes comparable to pre-approval clinical trials. This may be the result of the unique policy and regulatory environment governing the CMS coverage determination for TAVR institutions. As TAVR disseminates to additional hospitals and other new cardiovascular interventions are inevitably introduced, risk-standardized outcome comparisons across hospitals may facilitate ongoing surveillance to ensure high quality outcomes at all active centers.


2018 ◽  
Vol 25 (5) ◽  
Author(s):  
T. Sharma ◽  
C. Tajzler ◽  
A. Kapoor

BackgroundAlthough surgical resection remains the standard of care for localized kidney cancers, a significant proportion of patients experience systemic recurrence after surgery and hence might benefit from effective adjuvant therapy. So far, several treatment options have been evaluated in adjuvant clinical trials, but only a few have provided promising results. Nevertheless, with the recent development of targeted therapy and immunomodulatory therapy, a series of clinical trials are in progress to evaluate the potential of those novel agents in the adjuvant setting. In this paper, we provide a narrative review of the progress in this field, and we summarize the results from recent adjuvant trials that have been completed.MethodsA literature search was conducted. The primary search strategy at the medline, Cochrane reviews, and http://ClinicalTrials.gov/ databases included the keywords “adjuvant therapy,” “renal cell carcinoma,” and “targeted therapy or/and immunotherapy.”ConclusionsData from the s-trac study indicated that, in the “highest risk for recurrence” patient population, disease-free survival was increased with the use of adjuvant sunitinib compared with placebo. The assure trial showed no benefit for adjuvant sunitinib or sorafenib in the “intermediate- to high-risk” patient population. The ariser (adjuvant girentuximab) and protect (adjuvant pazopanib) trials indicated no survival benefit, but subgroup analyses in both trials recommended further investigation. The inconsistency in some of the current results can be attributed to a variety of factors pertaining to the lack of standardization across the trials. Nevertheless, patients in the “high risk of recurrence” category after surgery for their disease would benefit from a discussion about the potential benefits of adjuvant treatment and enrolment in ongoing adjuvant trials.


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