Regression to the Mean in Clinical Trials

2014 ◽  
Author(s):  
Clarence E. Davis
Keyword(s):  
Clinical Trials ◽  
Regression To The Mean ◽  
The Mean

Estimating Treatment Effects in Clinical Trials Subject to Regression to the Mean

Biometrics ◽  
1985 ◽  
Vol 41 (2) ◽  
pp. 555 ◽  
Author(s):  
Stephen J. Senn ◽  
Richard A. Brown ◽  
K. E. James
Keyword(s):  
Clinical Trials ◽  
Treatment Effects ◽  
Regression To The Mean ◽  
The Mean

scholarly journals Defining the optimal disease duration of early diffuse systemic sclerosis for clinical trial design

Rheumatology ◽  
2021 ◽  
Author(s):  
Robyn T Domsic ◽  
Shiyao Gao ◽  
Maureen Laffoon ◽  
Steven Wisniewski ◽  
Yuqing Zhang ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Systemic Sclerosis ◽  
Disease Duration ◽  
Disease Onset ◽  
Clinical Trial Design ◽  
Primary Outcome Measure ◽  
Inclusion Criteria ◽  
Regression To The Mean ◽  
The Mean ◽  
Definition Of

Abstract Objectives Clinical trials in early diffuse cutaneous systemic sclerosis (SSc) using the modified Rodnan skin score (mRSS) as the primary outcome measure have most often been negative. We wanted to assess how the definition of disease onset (first SSc manifestation vs first non-Raynaud manifestation), and varying lengths of disease duration at trial entry as an inclusion criteria functioned. Our objective was to optimize trial inclusion criteria Methods We used the prospective, observational University of Pittsburgh Scleroderma Cohort to identify early diffuse SSc patients first evaluated between 1980 and 2015. All had <3 years from first SSc (n = 481) or first non-Raynaud manifestation (n = 514) and 3 or more mRSS scores. We used descriptive, survival and group-based trajectory analyses to compare the different definitions of disease onset and disease duration as inclusion criteria for clinical trials. Results There was no appreciable difference between using first SSc manifestation compared with first non-Raynaud manifestation as the definition of disease onset. Compared with other disease durations, <18 months of disease had >70% of patients fitting into trajectories with worsening cutaneous disease over six months of follow-up. Longer disease durations demonstrated the majority of patients with trajectories showing an improvement in mRSS (regression to the mean) over six months. Conclusions Regardless of whether the first SSc or first non-Raynaud manifestation is used to define disease onset, duration of < 18 months at enrolment is preferable. Longer disease duration criteria more frequently results in regression to the mean of the mRSS score, and likely contributes to negative trial outcomes.

scholarly journals Regression to the Mean: Implications for Clinical Trials of Psychotropic Agents in Dementia

2004 ◽  
Vol 1 (4) ◽  
pp. 323-328 ◽  
Author(s):  
Jeffrey Cummings ◽  
Rochelle Tractenberg ◽  
Anthony Gamst ◽  
Linda Teri ◽  
Donna Masterman ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Regression To The Mean ◽  
Psychotropic Agents ◽  
The Mean

scholarly journals Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Shu Zhang ◽  
◽  
Dongdong Qin ◽  
Liwen Wu ◽  
Man Li ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Hazard Ratio ◽  
Large Cohort ◽  
Muscle Disease ◽  
Clinical Progression ◽  
Large Deletions ◽  
The Mean ◽  
Lower Hazard

Abstract Background Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease in human. We aimed to describe the genotype distribution in a large cohort of Chinese DMD patients and their delayed loss of ambulation by glucocorticoid (GC) treatments. This is to facilitate protocol designs and outcome measures for the emerging DMD clinical trials. Results A total of 1163 patients with DMD were recruited and genotyped. Genotype variations were categorized as large deletions, large duplications, and small mutations. Large deletions were further analyzed for those amenable to exon-skipping therapies. Participants aged 5 years or older were grouped into GC-treated and GC-naïve groups. Clinical progression among different genotypes and their responses to GC treatments were measured by age at loss of ambulation (LOA). Among the mutation genotypes, large deletions, large duplications, and small mutations accounted for 68.79%, 7.14%, and 24.07%, respectively. The mean age at diagnosis was 4.59 years; the median ages at LOA for the GC-naïve, prednisone/prednisolone-treated, and deflazacort-treated groups were 10.23, 12.02, and 13.95 years, respectively. The “deletion amenable to skipping exon 44” subgroup and the nonsense-mutation subgroup had older ages at LOA than the “other deletions” subgroup. Subgroups were further analyzed by both genotypes and GC status. All genotypes showed significant beneficial responses to GC treatment. Deletions amenable to skipping exon 44 showed a lower hazard ratio (0.155). The mean age at death was 18.57 years in this DMD group. Conclusion Genotype variation influences clinical progression in certain DMD groups. Beneficial responses to GC treatment were observed among all DMD genotypes. Compared with other genotypes, deletions amenable to skipping exon 44 had a lower hazard ratio, which may indicate a stronger protective effect of GC treatments on this subgroup. These data are valuable for designing future clinical trials, as clinical outcomes may be influenced by the genotypes.

scholarly journals Impact on actual clinical practice of the latest evidence for percutaneous closure of patent foramen ovale associated with stroke. A single center experience

2021 ◽  
Vol 22 (Supplement_1) ◽  
Author(s):  
L Carnero Montoro ◽  
M Ruiz Ortiz ◽  
N Paredes Hurtado ◽  
M Delgado Ortega ◽  
A Rodriguez Almodovar ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Patent Foramen Ovale ◽  
Cryptogenic Stroke ◽  
Percutaneous Closure ◽  
Publication Date ◽  
Foramen Ovale ◽  
Complex Anatomy ◽  
The Mean ◽  
The Impact

Abstract Funding Acknowledgements Type of funding sources: None. Background and aims Since september 14th, 2017. Three large clinical trials demonstrated that, in selected patients, percutaneous closure of patent foramen ovale (PFO) was associated with lower recurrence in patients with cryptogenic stroke (CS). Our aim was to determine the impact of these findings on routine  clinical practice in a tertiary hospital. Methods Patients with percutaneous closure of PFO due to CS (January 2001-January 2020) were included. The clinical characteristics were analyzed individually and grouped in the RoPE score, before and after the publication date. Complex anatomy (CA) defined as interatrial septum aneurysm or basal wide bubble passage was evaluated in both periods. Results 293 patients were included. The mean age was 49 ± 11 years, 15% were older than 60 years, 60% men, 26% hypertensive, 28% smokers and 7%diabetics. The median RoPEscore was 6 [p25-75, 5-7] and 75% met CA criteria. Since september 14th, 2017, the frequency of CA and the mean age of the patients were significantly higher (89% vs. 69% p <0.0005 and 51 ± 11 vs. 48 ± 11 years, p = 0.02, respectively), and RoPEscore, significantly lower (5 [5-7] vs. 6 [5-7], p = 0.02). Conclusion The publication of clinical trials wich demonstrated the benefit of percutaneous closure of PFO in CS had a significant impact on the daily clinical practice of our institution, with an increase in indications for CA, despite a clinical profile suggestive of lower causal probability of PFO.

Mines in the End Zone: Are there Downsides to Team Performance?

2020 ◽  
Vol 23 ◽  
Author(s):  
Troy V. Mumford ◽  
M. Travis Maynard
Keyword(s):  
Team Performance ◽  
Role Overload ◽  
Future Research ◽  
Negative Consequences ◽  
Regression To The Mean ◽  
Occupational Psychology ◽  
Team Success ◽  
The Mean ◽  
Overconfidence Bias

Abstract Research on teams in organizations tends to focus on understanding the causes of team performance with a focus on how to enjoy the benefits of team success and avoid the negative consequences of team failure. This paper instead asks the question, ‘what are some of the negative consequences of team success?’ A review of the literature on teams is augmented with research from cognitive science, sociology, occupational psychology, and psychology to explore the potential negative long-term consequences of teamwork success. The general topics of groupthink, overconfidence bias, regression to the mean, role overload, and strategy calcification are reviewed while discussing the implications for future research streams and practical team management.

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