Study sets, textbooks, questions
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Terms in this set (41)
N of 1 trial
One participant is given either a treatment or placebo (or 2 treatments) in a random sequence on multiple occasions
· Randomized multiple crossover study performed in a single individual
· Personalized medicine
· Can balance patient needs with methodological rigor
Cross-Sectional Study Design
a study in which data are collected at only one time point
designs within which random assignment cannot be used
randomized controlled trial
An experimental study in which researchers randomly assign individuals to either an experimental or a control group and expose the experimental group to the manipulated variable of interest.
# of total cases of condition in specific time
-includes both new and existing cases
incidence of death in time period
provides descriptions of groups or population under study in numerical summaries, graphs, or tables
makes inferences and predictions about a population based on data from samples that are from the population.
ratio of the probability of an event occurring in the exposed group versus the probability of the event occurring in the non-exposed group.
is the rate (proportion) of a health outcome (disease or death) in exposed individuals, which can be attributed to the exposure or risk factor.
Relative risk reduction
• Proportion of the relative risk that is attributed to the intervention (exposure) as compared to the control.
Number needed to treat (NNT):
• Number of subjects who must be treated for one person to benefit from the treatment (compared to control).
Number needed to harm
• Number of subjects who must be exposed to a risk factor to cause harm in one subject who otherwise would not have been harmed.
What does 95 CI mean
means that 95% of experiments include the mean
• Two different alleles/mutation are inherited that result a disease phenotype
(red/white/pink flower color) phenotype of a heterozygous organism is a blend between the phenotypes of its homozygous parents
(Blood type) both alleles are simultaneously expressed in the heterozygote
expression of phenotype depends on the sex/sex chromosomes of an individual
• present of 2 genetically or functional distinct cell populations
-->so have presence of 1 wildtype and 1 mutant results in differentt cell populations
is a genotype, an individual is heterozygous for wild type and mutant allele (2 different alleles for 1 gene)
: mechanisms that causes the phenotype
• one copy of a gene is not adequate to produce the needed gene product to preserve normal function.
Not all individuals with a mutant genotype show the mutant phenotype
-• Penetrance can impact disease frequencyà incomplete penetrance reduces disease frequency
genetic mutation (allele)
not all mutations are created equal! Each mutation may have a slightly different phenotype
statistical occurrence of phenotypes in a group of known genotypes"
Lynch Syndrome (Block 5): MLH1 mut - Colorectal cancer: Male - 67% Female - 35%
degree to which trait expression differs among individuals"
• Polydactyly: how many toes?
One gene affecting more than one trait
• Marfan syndrome: phenotype in heart, blood vessels, eyes, and skeleton
the masking of the action of alleles of one gene by allelic combinations of another gene1
Progressively earlier onset of the disease in successive generations with increasing severity within a family8 (Week 3 - Huntington's disease)
What are RCTs best used for?
for studying the effect of an intervention--> showing cause and effect
experimental design where people serve as own control--> decreases variance. You give intervention, then cross-over- now control group.
-not used for treatments with permanent effects
Subjects are randomized to the treatment or control group for the entire study
-randomization and unbiased distribution
-expensive- takes time
-ethically problematic--> since giving med that could be helpful
Cohort is best for?
studying effect of predictive risk factors on outcome
Case-control is best for?
very rare diseases
best to quantify the prevalence of a disease or risk factor and accuracy of a diagnostic test
-->snapshot in time- not good at testing association since no time course
closeness of the measures to the specific value (being studied) - free of error or bias- bullseye, closeness of measurements taking, free from error or bias
producing nearly identical results when repeated under the same conditions aka reproducibility or reliability
Use of clinical case reports and case series
-can help identify recognition patterns for rare clinical conditions
-can alert readers to new reactions to drug
-can highly innovation in medicine
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