Open-Label & Single-Blind Studies - Levien

What are the 5 Purposes of Case reports and case series? 1. Di... 2. Ad... 3. Ne.. 4. Ed... 5. Qu...

1. Disease identification 2. Adverse drug reaction reporting 3. New treatment approach 4. Educational 5. Quality assurance

4 advantages to Open-label techniques 1. Easier to... 2. Less... 3. Easier to get... 4. Has better what?

1. Easier to conduct no palcebo or dummy needed 2. Less costly 3. Easier to get patients enrolled in higher numbers 4. Has better real world external validity

3 Advantages to Single-Blind techniques 1. Easier to... 2. May be... 3. May...

1. Easier to conduct/monitor, lessens need for sham treatments 2. May be less costly 3. May reduce bias

6 Disadvantages to Open-label techniques 1. Subject to what? 2. Issue with selection bias? 3. Issue about it being easier to get more patients? 4. Psychological effects of what?

1. Subject to bias on assessments and safety 2. Patients more fragile because its easier to comply with study treatment 3. More patients will drop out 4. Effect on outcome because they know what treatment they are on

Disadvantages to Single-Blind techniques A) If Investigators are blinded what 5 things are less likely to occur? (Tr, Add, Adj, With, Enc)

1. Transfer attitude to patient 2. Add intervention 3. Adjust dose 4. Withdraw participants 5. Encourage or discourage study participation

6 Disadvantages to Open-label techniques 5. Risk of what bias? 6. Affect what type of care?

5. Risk of reporting bias from patient/investigators being more likely to report/under report effects 6. Could affect additional care if they know they are getting substandard treatment

Differentiating Study designs A) What is a double blind study? B) What is a double-dummy design?

A) 2 parties are blinded typically investigators and patients B) Technique for matching different dosage form, route, frequency with a placebo

Score interpretation of DIPS A) What is highly probably? B) What is Probable? C) What is Possible? D) What is doubtful?

A) > 8 B) 5-8 C) 2-4 D) < 2

Score interpretation of Naranjo Algorithim A) What is a definite? B) What is Probable? C) What is possible? D) What is doubtful?

A) > 9 B) 5-8 C) 1-4 D) 0 or less

Differentiating Study designs A) What is an open-Label? B) What is a single-blind? C) When are sing-blind typically used?

A) All participants aware of study assignment B) Either investigator or patient is aware of the study C) When it's important for one party to be aware of the risk

Weakness of Case reports/Case series A) A... B) Ge... C) Po...

A) Anecdotal evidence B) Generalizability is low C) Potential for reporting bias

Validity A) How is this an attempt to reduce bias? B) What does the source population give?

A) By being Internally valid or unbiased giving an estimate of a true effect on exposure aside from errors. B) An Externally valid estimate of the study to the outside population

Selection Bias A) Which is this more common in? B) What is this an error in? C) What does it cause differences in?

A) Case-control studies B) Estimated effect of an exposure on outcome. C) Probability of choosing or assigning individuals

Case-Control Studies A) How does it compare exposures? B) How are subjects selected? C) What is compared in result? D) Example?

A) Cases with outcome and Controls without outcome B) Basis of the outcome C) Exposures in cases VS controls D) Alzheimers with and without benzo use

Confounding A) What are Covariates? B) How is confounding introduced? C) How is Bias introduced?

A) Confounding variables measured and accounted for in statistical analysis B) Non-systematically C) Systematically

Disadvantages to Single-Blind techniques A) How are they subject to bias? B) If patients are blinding what 3 things will less likely occur? (Ps,Seek,Drop)

A) Conscious or subconsciously affect perception of response & Attitudes transfer to participants affecting care offered B) - Psychological/physical response - Seek additional treatment - Drop out

Cohort Advantages A) Does what directly? B) Study rare what? C) Study what outcomes? D) Less prone to what? E) More opportunity for what if prospective? F) Less risk of what?

A) Directly estimates risks B) Study rare exposures C) Study multiple outcomes D) Less prone to recall biases E) More opportunity to validate study measures F) less risk of selection bias & Temporal ambiguity

Observational Studies A) What are the studies driven on? B) What should comparison groups have? C) What can results speak for only? D) What is it useful in?

A) Exposure driven B) Same background risk for outcome C) Only associations not casualty D) Rare outcomes like cancer

Prospective Cohort Studies A) What do they study? B) How is study tailored? C) How is data captured? D) What can they be designed?

A) Exposures and disease outcomes as they occur B) To measure research question C) Purposefully on important risk factors D) Use reliable and validated techniques

Minimizing Selection Bias A) How to do this for cases and controls? B) What limits this?

A) Get subjects from same identifiable base population B) Randomization

Disadvantages to Single-Blind techniques A) What is less likely to occur of the Assessor is blinded? B) What are the 2 Risks of reporting Bias? C) What has a more complete unbiased report?

A) Have biases that affect outcome assessment especially with subjective outcomes B) - Patients/investigator reporting bias - Underreport known effects and perceived as normal C) Double-blind

Minimizing Confounding A) What is a big way to do this? B) How can you do this in the analysis? C) What models can you use?

A) Inclusion & Exclusion criteria B) Stratify the analysis (make separate risk for kids attending day care) C) Use multivariate statistical models with confounding variables (daycare attendance)

Minimizing Confounding A) How to minimize in cohort study? B) How to minimize in case-control? C) How does matching minimize this?

A) Match patients based on similar propensity to have the disease B) Match patients based on similar baseline characteristics C) Study subjects will be similar or balanced in regards to confounders

Information Bias A) What 4 errors occur after enrollment? B) What is the result of differences in measurement or collection between study groups?

A) Measurement, Analysis, Interpretation, Reporting B) Biases on estimated effect of an exposure outcome

Information Bias A) What is bias by interpretation? B) What is recall bias? C) What is temporal ambiguity?

A) Misclassification B) When subjects in one group remember events more inaccurately than another group C) Confusion about Exposure affect disease or disease affect exposure

Observational studies A) Cohort focus on what? B) Case-control focus on what?

A) Natural disease progression and risk factors B) Relationship between disease and exposure

Retrospective Cohort Studies A) What Limitations? B) How will errors exist?

A) Only study exposures that have had time to produce an outcome B) Errors in measuring factors from retrospective data sources

What are characteristics of a good case report/Case series? A) Detailed description of what what? B) What 4 things should be included? (T,L,M,I) C) What can be generated?

A) Patients clinical course B) Timeline, Lab values, Medications, Interventions C) Hypothesis

Retrospective Cohort Studies A) How are they done? B) What 3 ways are they measured? C) What is an advantage?

A) Previous exposure and disease outcomes. B) Subject recall, stored biological samples, and existing records C) Existing data quickly produces results

Cohort Studies A) How can they be performed? B) What is the focus? C) How are patients categorized? D) What endpoint are they followed for?

A) Propsective OR Retrospective, Open OR Closed B) Exposure first then outcome C) By exposure (smoker vs non-smoker) D) If endpoint occurs at a greater frequency in one group

Case-Control Advantages A) Typically what 2 things? B) Study rare what? C) Study what outcomes? D) Study what exposures? E) Require fewer what?

A) Quicker and less expensive B) Rare outcomes C) Outcomes with long latency/induction periods D) Multiple exposures E) Fewer subjects

Limitations to Randomized controlled trials A) When are they not ethical? B) When are they not feasible C) How is efficacy assessed? D) How can they not be powered?

A) Randomizing subjects to exposures known to be harmful B) Randomize subjects to exposure like obesity or heart disease C) Effects in ideal settings D) Estimate effects of treatment on rare endpoints or clinical endpoints that take a long time to develop

Disadvantages of studies A) What is a disadvantages of Cohort studies? B) What is a disadvantage of Case-Control studies?

A) Rare outcomes require large studies followed for many years B) More susceptible to biases (recall, Selection, Temporal ambiguity)

Concealed Allocation A) What is it? B) Do open-label studies have this?

A) Related to randomization when the study assignment group is concealed from individual recruiting subjects B) They can and should if it is a randomized study

Tools used to assess probability of AE and Drug interactions. A) What does the Naranjo Algorithm Grade? B) What does the DIPS Grade?

A) Relationship between medication and adverse reactions B) Relationship between Drug-Drug Interaction and adverse reaction

Case-control Studies A) How are they performed? B) What do they focus on? C) How are patients categorized? D) How are results compared?

A) Retrospective population based B) Outcome first then exposure C) Having disease or experienced event D) Previous exposures to see if they occurred at a greater freqeuncy

Statistical Measures A) What is used in a Cohort Study? B) What is used in a Case-control study?

A) Risk Ratio or Relative Risk - risk of outcome in exposed group relative to unexposed B) Odds ratio - odds of exposure in cases over controls

What will a Cohort study diagram look like? A) First split? B) Second split C) Two end results?

A) Source population --> study sample B) Exposed and Unexposed C) No outcome or Outcome in Exposed and Unexposed

Prospective Cohort Studies A) How does this compare to other types of observational designs? B) What are 2 disadvantages?

A) Strongest type of observational study B) Expensive to recruit subjects & Takes a long time to complete.

Purpose of Case report/Case series A) What dose Disease identification focus on? B) What does ADR reporting focus on? C) What does New treatment approach focus on? D) What does education include? E) What does Quality assurance focus on?

A) Uncommon/unknown disease/condition B) Rare & Serious reactions in special populations C) Novel therapy indication & Caution in result interpreting D) Common or unique in clinical situations E) Medication/practice errors

Confounding A) What is this? B) What is an example?

A) When another factor (confounder) distorts or causes error in interpretation. B) Children going to daycare when your looking at flu vaccination efficacy

Minimizing Information Bias A) How to decrease misclassification bias? B) How to decrease recall bias? C) How to decrease temporal ambiguity?

A) using same method to measure disease/exposure in both groups B) Use same methods to measure exposure data in case-control C) Designing a nested case-control study

What is a Case series?

Description of events in two or more similar patients

What is a Case report?

Retrospective description of an event or treatment approach occurring in a single patient