Estimating Causal Treatment Effects from Longitudinal HIV Natural History Studies Using Marginal Structural Models

Biometrics ◽  
2003 ◽  
Vol 59 (1) ◽  
pp. 152-162 ◽  
Author(s):  
Hyejin Ko ◽  
Joseph W. Hogan ◽  
Kenneth H. Mayer
2012 ◽  
Vol 12 (1) ◽  
Author(s):  
Tobias Gerhard ◽  
Joseph AC Delaney ◽  
Rhonda M Cooper-DeHoff ◽  
Jonathan Shuster ◽  
Babette A Brumback ◽  
...  

Author(s):  
Lorena Lúcia Costa Ladeira ◽  
Sarah Pereira Martins ◽  
Cayara Mattos Costa ◽  
Elizabeth Lima Costa ◽  
Rubenice Amaral da Silva ◽  
...  

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.


2012 ◽  
Vol 27 (1_suppl) ◽  
pp. 143-148 ◽  
Author(s):  
C W K P Arnoldussen ◽  
C H A Wittens

In this article we want to discuss the potential of lower extremity deep vein thrombosis (DVT) imaging and propose a systematic approach to DVT management based on a DVT classification of the lower extremity; the LET classification. Identifying and reporting DVT more systematically allows for accurate stratification for initial patient care, future clinical trials and appropriate descriptions for natural history studies.


Biometrics ◽  
2015 ◽  
Vol 71 (2) ◽  
pp. 299-301 ◽  
Author(s):  
Olli Saarela ◽  
David A. Stephens ◽  
Erica E. M. Moodie ◽  
Marina B. Klein

Biostatistics ◽  
2018 ◽  
Vol 21 (1) ◽  
pp. 172-185 ◽  
Author(s):  
Pål Christie Ryalen ◽  
Mats Julius Stensrud ◽  
Sophie Fosså ◽  
Kjetil Røysland

Abstract In marginal structural models (MSMs), time is traditionally treated as a discrete parameter. In survival analysis on the other hand, we study processes that develop in continuous time. Therefore, Røysland (2011. A martingale approach to continuous-time marginal structural models. Bernoulli 17, 895–915) developed the continuous-time MSMs, along with continuous-time weights. The continuous-time weights are conceptually similar to the inverse probability weights that are used in discrete time MSMs. Here, we demonstrate that continuous-time MSMs may be used in practice. First, we briefly describe the causal model assumptions using counting process notation, and we suggest how causal effect estimates can be derived by calculating continuous-time weights. Then, we describe how additive hazard models can be used to find such effect estimates. Finally, we apply this strategy to compare medium to long-term differences between the two prostate cancer treatments radical prostatectomy and radiation therapy, using data from the Norwegian Cancer Registry. In contrast to the results of a naive analysis, we find that the marginal cumulative incidence of treatment failure is similar between the strategies, accounting for the competing risk of other death.


Sign in / Sign up

Export Citation Format

Share Document