Papers of note in Science Translational Medicine

2016 ◽  
Vol 9 (411) ◽  
pp. ec13-ec13
Author(s):  
Nancy R. Gough

ImmunodeficiencySAPping immunopathologyDGKα inhibition restores apoptosis in SAP-deficient lymphocytes, decreasing immunopathology in XLP-1 patients.E. Ruffo, V. Malacarne, S. E. Larsen, R. Das, L. Patrussi, C. Wülfing, C. Biskup, S. M. Kapnick, K. Verbist, P. Tedrick, P. L. Schwartzberg, C. T. Baldari, I. Rubio, K. E. Nichols, A. L. Snow, G. Baldanzi, A. Graziani, Inhibition of diacylglycerol kinase α restores restimulation-induced cell death and reduces immunopathology in XLP-1. Sci. Transl. Med.8, 321ra7 (2016). [Abstract]AllergyFighting food allergyInfants who develop food allergy display hyperresponsive innate immunity at birth that promotes nonclassical TH2 differentiation.Y. Zhang, F. Collier, G. Naselli, R. Saffery, M. L. K. Tang, K. J. Allan, A.-L. Ponsonby, L. C. Harrison, P. Vuillermin, on behalf of the BIS Investigator Group, Cord blood monocyte–derived inflammatory cytokines suppress IL-2 and induce nonclassic “TH2-type” immunity associated with development of food allergy. Sci. Transl. Med.8, 321ra8 (2016). [Abstract]Fragile X SyndromeThe mGluR theory of fragile X, put to the testIn contrast to previous studies, targeting the mGluR pathway in fragile X syndrome patients did not improve behavior independent of FMR1 methylation.E. Berry-Kravis, V. Des Portes, R. Hagerman, S. Jacquemont, P. Charles, J. Visootsak, M. Brinkman, K. Rerat, B. Koumaras, L. Zhu, G. M. Barth, T. Jaecklin, G. Apostol, F. von Raison, Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci. Transl. Med.8, 321ra5 (2016). [Abstract]S. S. Jeste, D. H. Geschwind, Clinical trials for neurodevelopmental disorders: At a therapeutic frontier. Sci. Transl. Med.8, 321fs1 (2016). [Abstract]

2016 ◽  
Vol 8 (321) ◽  
pp. 321ra5-321ra5 ◽  
Author(s):  
Elizabeth Berry-Kravis ◽  
Vincent Des Portes ◽  
Randi Hagerman ◽  
Sébastien Jacquemont ◽  
Perrine Charles ◽  
...  

Cancers ◽  
2020 ◽  
Vol 12 (11) ◽  
pp. 3251
Author(s):  
Jennifer G. Le-Rademacher ◽  
Shauna Hillman ◽  
Elizabeth Storrick ◽  
Michelle R. Mahoney ◽  
Peter F. Thall ◽  
...  

This article introduces the adverse event (AE) burden score. The AE burden by treatment cycle is a weighted sum of all grades and AEs that the patient experienced in a cycle. The overall AE burden score is the total AE burden the patient experienced across all treatment cycles. AE data from two completed Alliance multi-center randomized double-blind placebo-controlled trials, with different AE profiles (NCCTG 97-24-51: 176 patients, and A091105: 83 patients), were utilized for illustration. Results of the AE burden score analyses corroborated the trials’ primary results. In 97-24-51, the overall AE burden for patients on the treatment arm was 2.2 points higher than those on the placebo arm, with a higher AE burden for patients who went off treatment early due to AE. Similarly, in A091105, the overall AE burden was 1.6 points higher on the treatment arm. On the placebo arms, the AE burden in 97-24-51 remained constant over time; and increased in later cycles in A091105, likely attributable to the increase in disease morbidity. The AE burden score enables statistical comparisons analogous to other quantitative endpoints in clinical trials, and can readily accommodate different trial settings, diseases, and treatments, with diverse AE profiles.


2016 ◽  
Vol 37 (8) ◽  
pp. 619-628 ◽  
Author(s):  
Laura Greiss Hess ◽  
Sarah E. Fitzpatrick ◽  
Danh V. Nguyen ◽  
Yanjun Chen ◽  
Kimberly N. Gaul ◽  
...  

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