Validation of Microsimulation Models against Alternative Model Predictions and Long-Term Colorectal Cancer Incidence and Mortality Outcomes of Randomized Controlled Trials

2020 ◽  
Vol 40 (6) ◽  
pp. 815-829
Author(s):  
Jie-Bin Lew ◽  
Marjolein J. E. Greuter ◽  
Michael Caruana ◽  
Emily He ◽  
Joachim Worthington ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Randomized Controlled Trials ◽  
Fecal Occult Blood ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Incidence And Mortality ◽  
Long Term Follow Up ◽  
Microsimulation Models

Background. This study aimed to assess the validity of 2 microsimulation models of colorectal cancer (CRC), Policy1-Bowel and ASCCA. Methods. The model-estimated CRC risk in population subgroups with different health statuses, “dwell time” (time from incident precancerous polyp to symptomatically detected CRC), and reduction in symptomatically detected CRC incidence after a one-time complete removal of polyps and/or undetected CRC were compared with published findings from 3 well-established models ( MISCAN, CRC-SPIN, and SimCRC). Furthermore, 6 randomized controlled trials (RCTs) that provided screening using a guaiac fecal occult blood test (Funen trial, Burgundy trial, and Minnesota Colon Cancer Control Study [MCCCS]) or flexible sigmoidoscopy (NORCCAP, SCORE, and UKFSST) with long-term follow-up were simulated. Model-estimated long-term relative reductions of CRC incidence (RR inc) and mortality (RR mort) were compared with the RCTs’ findings. Results. The Policy1-Bowel and ASCCA estimates showed more similarities to CRC-SPIN and SimCRC. For example, overall dwell times estimated by Policy1-Bowel (24.0 years) and ASCCA (25.3) were comparable to CRC-SPIN (25.8) and SimCRC (25.2) but higher than MISCAN (10.6). In addition, ∼86% of Policy1-Bowel’s and ∼74% of ASCCA’s estimated RR inc and RR mort were consistent with the RCTs’ long-term follow-up findings. For example, at 17 to 18 years of follow-up, the MCCCS reported RR mort of 0.67 (95% confidence interval [CI], 0.51–0.83) and 0.79 (95% CI, 0.62–0.97) for the annual and biennial screening arm, respectively, and the UKFSST reported RR mort of 0.70 (95% CI, 0.62–0.79) for CRC at all sites and 0.54 (95% CI, 0.46–0.65) for distal CRC. The corresponding model estimates were 0.65, 0.74, 0.81, and 0.61, respectively, for Policy1-Bowel and 0.65, 0.70, 0.75, and 0.58, respectively, for ASCCA. Conclusion. Policy1-Bowel and ASCCA’s estimates are largely consistent with the data included for comparisons, which indicates good model validity.

Quality of Randomized Controlled Trials Published By Plastic Surgeons: Long-Term Follow-Up

2019 ◽  
Vol 43 (3) ◽  
pp. 866-873
Author(s):  
Thiago Bezerra de Morais ◽  
Daniela Francescato Veiga ◽  
Joel Veiga-Filho ◽  
Andréia Cristina Feitosa do Carmo ◽  
Rosely de Fátima Pellizzon ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Long Term Follow Up

scholarly journals Minimally Invasive Surgery in Patients With Intracerebral Hemorrhage: A Meta-Analysis of Randomized Controlled Trials

2022 ◽  
Vol 12 ◽  
Author(s):  
Duanlu Hou ◽  
Ying Lu ◽  
Danhong Wu ◽  
Yuping Tang ◽  
Qiang Dong
Keyword(s):  
Minimally Invasive Surgery ◽  
Minimally Invasive ◽  
Randomized Controlled Trials ◽  
Conventional Treatment ◽  
Meta Analysis ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Long Term Follow Up

Background: Minimally invasive surgery for intracerebral hemorrhage (ICH) has been evaluated in clinical trials. Although meta-analyses on this topic have been performed in the past, recent trials have added important information to the results of the comparison. However, little work has been done to compare the effect of MIS and conventional treatment on patient prognosis, especially mortality.Methods: PubMed, EMBASE, Web of Science, Ovid, China National Knowledge Infrastructure, and ClinicalTrials.gov were searched on May 1, 2021, for randomized controlled trials of MIS for spontaneous ICH. The primary outcome was defined as death at follow-up, while the secondary outcome was defined as death in different comparisons between MIS and craniotomy (CT) or medication (Me).Results: The initial search yielded 12 high-quality randomized controlled trials involving 2,100 patients. We analyzed the odds ratios (ORs) for MIS compared with conventional treatment, including Me and conventional CT. The OR and confidence intervals (CIs) of the primary and secondary outcomes were 0.62 (0.45–0.85) for MIS vs. conventional treatment. We also conducted subgroup analyses and found that the ORs and CIs for MIS compared with that of conventional treatment in the short-term follow-up were 0.58 (0.42–0.80), and, in the long-term follow-up, was 0.67 (0.46–0.98); and found that ORs were 0.68 (0.48–0.98) for MIS vs. CT and 0.57 (0.41–0.79) for MIS vs. Me.Conclusions: This meta-analysis demonstrates that certain patients with ICH benefit in short- and long-term follow-up from MIS over other treatments, including open surgery and conventional Me.Systematic Review Registration:https://www.crd.york.ac.uk/PROSPERO/.

Clinical trials of multiple sclerosis therapies: improvements to demonstrate long-term patient benefit

2009 ◽  
Vol 15 (8) ◽  
pp. 951-958 ◽  
Author(s):  
WM Carroll
Keyword(s):  
Multiple Sclerosis ◽  
Randomized Controlled Trials ◽  
Controlled Trials ◽  
Patient Benefit ◽  
Short Term ◽  
Randomized Controlled ◽  
Long Term Follow Up ◽  
Term Benefit

Background The therapeutic goal for multiple sclerosis (MS) is to achieve a better long-term outcome. However, since available data come from short-term studies, it is important to review the evidence that current therapies provide long-term benefit. Method and results Long-term data from both registry studies and long-term follow-up studies, and efficacy treatment data were reviewed. Registry data show that the course of MS is predictable after a certain level of disability is reached, indicating that short-term efficacy data from randomized, controlled trials provide evidence of long-term benefit. Long-term studies of patients originally enrolled in pivotal randomized, controlled trials consistently show that delayed or discontinued treatment provides less benefit than continuous therapy. The 16-Year Long-Term Follow-Up Study of interferon beta-1b (IFNβ-1b; Betaferon®/Betaseron®) therapy had the highest ascertainment of long-term follow-up efforts of the pivotal trials, which led to the currently approved therapies. Disability scores at the start of treatment were predictive of their current disability scores. In addition, this 16-year study showed an excellent safety profile with no unexpected side effects to IFNβ-1b and a lower mortality rate after 16 years compared with those receiving placebo treatment during the pivotal study (6 deaths vs 20 deaths). Conclusion This article reviews the key data and provides recommendations for optimizing clinical studies in MS to demonstrate long-term patient benefit.

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