Study designs

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Study designs

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PED STEP2

Eating disorders: Pathology review
Psychiatric emergencies: Pathology review
Attributable risk (AR)
Bias in interpreting results of clinical studies
Bias in performing clinical studies
Clinical trials
Confounding
DALY and QALY
Direct standardization
Disease causality
Incidence and prevalence
Indirect standardization
Interaction
Mortality rates and case-fatality
Odds ratio
Positive and negative predictive value
Prevention
Relative and absolute risk
Selection bias
Sensitivity and specificity
Study designs
Test precision and accuracy
Acyanotic congenital heart defects: Pathology review
Adrenal masses: Pathology review
Bacterial and viral skin infections: Pathology review
Bone tumors: Pathology review
Coagulation disorders: Pathology review
Congenital neurological disorders: Pathology review
Cyanotic congenital heart defects: Pathology review
Extrinsic hemolytic normocytic anemia: Pathology review
Eye conditions: Inflammation, infections and trauma: Pathology review
Eye conditions: Refractive errors, lens disorders and glaucoma: Pathology review
Headaches: Pathology review
Intrinsic hemolytic normocytic anemia: Pathology review
Leukemias: Pathology review
Lymphomas: Pathology review
Macrocytic anemia: Pathology review
Microcytic anemia: Pathology review
Mixed platelet and coagulation disorders: Pathology review
Nasal, oral and pharyngeal diseases: Pathology review
Nephritic syndromes: Pathology review
Nephrotic syndromes: Pathology review
Non-hemolytic normocytic anemia: Pathology review
Pediatric brain tumors: Pathology review
Pediatric musculoskeletal disorders: Pathology review
Platelet disorders: Pathology review
Renal and urinary tract masses: Pathology review
Seizures: Pathology review
Viral exanthems of childhood: Pathology review
Pharmacodynamics: Agonist, partial agonist and antagonist
Pharmacodynamics: Desensitization and tolerance
Pharmacodynamics: Drug-receptor interactions
Pharmacokinetics: Drug absorption and distribution
Pharmacokinetics: Drug elimination and clearance
Pharmacokinetics: Drug metabolism
Cystic fibrosis: Pathology review
Diabetes mellitus: Pathology review
HIV and AIDS: Pathology review
Obstructive lung diseases: Pathology review
Papulosquamous and inflammatory skin disorders: Pathology review
Antidiuretic hormone
Body fluid compartments
Movement of water between body compartments
Sodium homeostasis
Acid-base disturbances: Pathology review
Diabetes insipidus and SIADH: Pathology review
Electrolyte disturbances: Pathology review
Renal failure: Pathology review
Growth hormone and somatostatin
Childhood and early-onset psychological disorders: Pathology review
Breastfeeding
Central nervous system infections: Pathology review
Congenital TORCH infections: Pathology review
Jaundice: Pathology review
Respiratory distress syndrome: Pathology review
Ectoderm
Endoderm
Human development days 1-4
Human development days 4-7
Human development week 2
Human development week 3
Mesoderm
Cell cycle
DNA damage and repair
DNA mutations
DNA replication
DNA structure
Epigenetics
Gene regulation
Mitosis and meiosis
Nuclear structure
Transcription of DNA
Translation of mRNA
Hardy-Weinberg equilibrium
Independent assortment of genes and linkage
Inheritance patterns
Mendelian genetics and punnett squares
Autosomal trisomies: Pathology review
Disorders of sex chromosomes: Pathology review
Miscellaneous genetic disorders: Pathology review
Baroreceptors
Cardiac preload
Chemoreceptors
Renin-angiotensin-aldosterone system
Adrenal insufficiency: Pathology review
Congenital gastrointestinal disorders: Pathology review
Environmental and chemical toxicities: Pathology review
Gastrointestinal bleeding: Pathology review
GERD, peptic ulcers, gastritis, and stomach cancer: Pathology review
Inflammatory bowel disease: Pathology review
Medication overdoses and toxicities: Pathology review
Pneumonia: Pathology review
Shock: Pathology review
Supraventricular arrhythmias: Pathology review
Traumatic brain injury: Pathology review
Ventricular arrhythmias: Pathology review
Introduction to pharmacology
Androgens and antiandrogens
Estrogens and antiestrogens
Miscellaneous cell wall synthesis inhibitors
Protein synthesis inhibitors: Tetracyclines
Cell wall synthesis inhibitors: Penicillins
Antihistamines for allergies
Acetaminophen (Paracetamol)
Non-steroidal anti-inflammatory drugs
Antimetabolites: Sulfonamides and trimethoprim
Antituberculosis medications
Cell wall synthesis inhibitors: Cephalosporins
DNA synthesis inhibitors: Fluoroquinolones
DNA synthesis inhibitors: Metronidazole
Miscellaneous protein synthesis inhibitors
Protein synthesis inhibitors: Aminoglycosides
Bronchodilators: Beta 2-agonists and muscarinic antagonists
Bronchodilators: Leukotriene antagonists and methylxanthines
Pulmonary corticosteroids and mast cell inhibitors
Glucocorticoids
Azoles
Anticonvulsants and anxiolytics: Barbiturates
Anticonvulsants and anxiolytics: Benzodiazepines
Nonbenzodiazepine anticonvulsants
Developmental milestones: Clinical
Disruptive, impulse-control and conduct disorders: Clinical
Eating disorders: Clinical
Elimination disorders: Clinical
Neurodevelopmental disorders: Clinical
Child abuse: Clinical
BRUE, ALTE, and SIDS: Clinical
Congenital heart defects: Clinical
Fever of unknown origin: Clinical
Kawasaki disease: Clinical
Pediatric bone and joint infections: Clinical
Pediatric constipation: Clinical
Pediatric ear, nose, and throat conditions: Clinical
Pediatric gastrointestinal bleeding: Clinical
Pediatric infectious rashes: Clinical
Pediatric lower airway conditions: Clinical
Pediatric ophthalmological conditions: Clinical
Pediatric orthopedic conditions: Clinical
Pediatric upper airway conditions: Clinical
Pediatric urological conditions: Clinical
Pediatric vomiting: Clinical
Adrenal masses and tumors: Clinical
Asthma: Clinical
Cystic fibrosis: Clinical
Diabetes mellitus: Clinical
Leukemia: Clinical
Lymphoma: Clinical
Pediatric allergies: Clinical
Pediatric bone tumors: Clinical
Seizures: Clinical
Sickle cell disease: Clinical
Chronic kidney disease: Clinical
Heart failure: Clinical
Hyperkalemia: Clinical
Hypernatremia: Clinical
Hypokalemia: Clinical
Hyponatremia: Clinical
Metabolic and respiratory acidosis: Clinical
Shock: Clinical
Mood disorders: Clinical
Congenital disorders: Clinical
Neonatal ICU conditions: Clinical
Neonatal jaundice: Clinical
Newborn management: Clinical
Perinatal infections: Clinical
Bleeding disorders: Clinical
Immunodeficiencies: Clinical
Brain tumors: Clinical
Meningitis, encephalitis and brain abscesses: Clinical
Toxidromes: Clinical
Vaccinations: Clinical

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A researcher is attempting to better understand correlational risk factors for hyperlipidemia. He obtains data on approximately 30,000 patients in a small city in the United States and finds a higher prevalence of hyperlipidemia in patients with a high meat intake when compared to those who identify as vegetarians. Which of the following best describes this type of study design?  

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There are six basic types of epidemiological study designs, and they can each be distinguished using certain criteria.

The first criterion for deciding which study design to use is whether you have individual or group data.

For example, let’s say we want to know how many people out of 100 people had migraines in the past year.

Now, with individual data, we have information about each person, so we can tell whether or not each of the 100 people had a migraine.

So, let’s say that 9 people had migraines. If we have individual data, we can look at the individual characteristics for each of the 9 people that had migraines, like their sex, age, race, or past history of migraines, and we can compare them to the people that didn’t have migraines.

On the other hand, if we have group data, we don’t actually know which specific individuals out of the 100 people had migraines.

So even though we know that 9 people had them, we don’t know which 9 people they were or any of their individual characteristics.

Now, ecological studies are a type of study design that uses group data to figure out if there is a potential association between two variables.

For example, let’s say you want to figure out if people who sleep less are more likely to get migraines.

And perhaps you have information about average sleep duration for populations in ten different cities.

You could plot this information on a graph with average sleep duration on the x-axis and the prevalence of migraines—which is the number of people that suffer from migraines, per 100,000 people—on the y-axis.

Generally, we can see that the less sleep a city gets, the higher the prevalence of migraines is for that city.

The thing is, we can’t actually say that getting less sleep causes migraines, since we don’t have information about each individual in each city.

All we can say is that there’s an association between sleep duration and prevalence of migraines.

Ecological studies are helpful for making hypotheses, though, that can later be tested using individual-level studies.

And, in general, individual-level studies are considered stronger than ecological studies, because knowing individual characteristics can help us determine what risk factors are associated with certain diseases.

So now let’s talk about studies that use individual data.

The next criterion we use to decide on a study design is whether or not there’s an intervention, and an intervention is basically just an exposure that the researcher controls.

Studies with interventions are also called experimental studies, randomized controlled trials, or RCTs for short.

So, for example, let’s say we want to find out if a newly discovered drug, we’ll call it Drug A, can prevent migraines for up to a year. In this example, Drug A is the intervention and having a migraine is the outcome.

In the most basic RCT, the sample population might be randomly split into two treatment groups, an intervention group that receives Drug A, and a control group that receives a placebo.

The placebo looks and tastes like Drug A but is completely harmless and ineffective - like a tiny capsule filled with water.

After both groups get their treatments, researchers would compare the incidence of migraines in each group—which is the number of individuals in each group who got migraines over the next year.

Now, RCTs are considered to be the gold standard study design because they’re able to determine causality.

In other words, we can determine if taking Drug A causes people to have fewer migraines compared to taking the placebo.

Determining causality is possible because the intervention group and the control group are randomly selected from the larger target population, so there’s a good chance that people in each group are similar and that the only difference between the two groups is whether or not they were exposed to Drug A.

There are some downsides to RCTs though, mainly that they can sometimes be really expensive, time consuming, and in some cases unethical, depending on the intervention.

Next, let’s talk about studies that don’t have an intervention, and these are called observational studies, because you simply observe what happens to individuals without controlling their exposure.

There are a few different types of observational studies, and the main criterion used to distinguish them is when you measure the exposure.

In other words, whether you measure the exposure before the outcome, after the outcome, or at the same time.

The first observational study is cohort or longitudinal studies, and cohort studies measure the exposure before the outcome.

Now, a cohort is simply a group of people who share a common characteristic.

So, cohort studies are a type of study design that look at individuals in a cohort who have a certain exposure, as well as individuals in a cohort who have not had that exposure, and then follow both groups over time and compare the incidence of a certain outcome.

For example, let’s say we follow a group of 100 individuals that smoke cigarettes, the exposed group, and 100 people that don’t smoke cigarettes, the unexposed group, and compare the incidence of migraines during the next five years.

Cohort studies are useful when you want to show the timing or temporality of the relationship between the exposure and the outcome.

For example, out of 200 people, 100 that smoke and 100 that don’t smoke, none of them have migraines at the beginning of the study.

But after five years, more individuals that smoke have migraines compared to individuals that don’t smoke, so it’s pretty clear that smoking happened first and migraines happened second.

Cohort studies are also good for looking at rare exposures, like if a certain uncommon medication causes an increased risk of migraines.

It makes more sense to recruit 100 people who were all already using the uncommon medication rather than start with 100 people with migraines and try to figure out if any of them had been exposed to the medication in the past; because since it’s such a rare exposure, there might be only 1 or 2 people out of the 100 individuals with migraines who were exposed to the medication.

Summary