scholarly journals Using short-term response information to facilitate adaptive randomization for survival clinical trials

2009 ◽  
Vol 28 (12) ◽  
pp. 1680-1689 ◽  
Author(s):  
Xuelin Huang ◽  
Jing Ning ◽  
Yisheng Li ◽  
Elihu Estey ◽  
Jean-Pierre Issa ◽  
...  
2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Peter Witters ◽  
Andrew C. Edmondson ◽  
Christina Lam ◽  
Christin Johnsen ◽  
Marc C. Patterson ◽  
...  

AbstractA recent report on long-term dietary mannose supplementation in phosphomannomutase 2 deficiency (PMM2-CDG) claimed improved glycosylation and called for double-blind randomized study of the dietary supplement in PMM2-CDG patients. A lack of efficacy of short-term mannose supplementation in multiple prior reports challenge this study’s conclusions. Additionally, some CDG types have previously been reported to demonstrate spontaneous improvement in glycosylated biomarkers, including transferrin. We have likewise observed improvements in transferrin glycosylation without mannose supplementation. This observation questions the reliability of transferrin as a therapeutic outcome measure in clinical trials for PMM2-CDG. We are concerned that renewed focus on mannose therapy in PMM2-CDG will detract from clinical trials of more promising therapies. Approaches to increase efficiency of clinical trials and ultimately improve patients’ lives requires prospective natural history studies and identification of reliable biomarkers linked to clinical outcomes in CDG. Collaborations with patients and families are essential to identifying meaningful study outcomes.


Author(s):  
Carlota Rigotti ◽  
Júlia Zomignani Barboza

Abstract The return of foreign fighters and their families to the European Union has mostly been considered a security threat by member States, which consequently adopt repressive measures aimed at providing an immediate, short-term response to this perceived threat. In addition to this strong-arm approach, reintegration strategies have also been used to prevent returnees from falling back into terrorism and to break down barriers of hostility between citizens in the long term. Amidst these different strategies, this paper seeks to identify which methods are most desirable for handling returnees.


2016 ◽  
Vol 25 (1) ◽  
pp. 119-128
Author(s):  
Chao Gong ◽  
Changchun Song ◽  
Xinhou Zhang ◽  
Wenwen Tan ◽  
Tianhua Qiao

2016 ◽  
Vol 233 ◽  
pp. 308-317 ◽  
Author(s):  
Chris Bamminger ◽  
Christian Poll ◽  
Christina Sixt ◽  
Petra Högy ◽  
Dominik Wüst ◽  
...  

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