Number needed to treat relation US Medical PG Practice Questions and MCQs
Practice US Medical PG questions for Number needed to treat relation. These multiple choice questions (MCQs) cover important concepts and help you prepare for your exams.
Number needed to treat relation US Medical PG Question 1: You are conducting a study comparing the efficacy of two different statin medications. Two groups are placed on different statin medications, statin A and statin B. Baseline LDL levels are drawn for each group and are subsequently measured every 3 months for 1 year. Average baseline LDL levels for each group were identical. The group receiving statin A exhibited an 11 mg/dL greater reduction in LDL in comparison to the statin B group. Your statistical analysis reports a p-value of 0.052. Which of the following best describes the meaning of this p-value?
- A. There is a 95% chance that the difference in reduction of LDL observed reflects a real difference between the two groups
- B. Though A is more effective than B, there is a 5% chance the difference in reduction of LDL between the two groups is due to chance
- C. If 100 permutations of this experiment were conducted, 5 of them would show similar results to those described above
- D. This is a statistically significant result
- E. There is a 5.2% chance of observing a difference in reduction of LDL of 11 mg/dL or greater even if the two medications have identical effects (Correct Answer)
Number needed to treat relation Explanation: **There is a 5.2% chance of observing a difference in reduction of LDL of 11 mg/dL or greater even if the two medications have identical effects**
- The **p-value** represents the probability of observing results as extreme as, or more extreme than, the observed data, assuming the **null hypothesis** is true (i.e., there is no true difference between the groups).
- A p-value of 0.052 means there's approximately a **5.2% chance** that the observed 11 mg/dL difference (or a more substantial difference) occurred due to **random variation**, even if both statins were equally effective.
*There is a 95% chance that the difference in reduction of LDL observed reflects a real difference between the two groups*
- This statement is an incorrect interpretation of the p-value; it confuses the p-value with the **probability that the alternative hypothesis is true**.
- A p-value does not directly tell us the probability that the observed difference is "real" or due to the intervention being studied.
*Though A is more effective than B, there is a 5% chance the difference in reduction of LDL between the two groups is due to chance*
- This statement implies that Statin A is more effective, which cannot be concluded with a p-value of 0.052 if the significance level (alpha) was set at 0.05.
- While it's true there's a chance the difference is due to chance, claiming A is "more effective" based on this p-value before statistical significance is usually declared is misleading.
*If 100 permutations of this experiment were conducted, 5 of them would show similar results to those described above*
- This is an incorrect interpretation. The p-value does not predict the outcome of repeated experiments in this manner.
- It refers to the **probability under the null hypothesis in a single experiment**, not the frequency of results across multiple hypothetical repetitions.
*This is a statistically significant result*
- A p-value of 0.052 is generally considered **not statistically significant** if the conventional alpha level (significance level) is set at 0.05 (or 5%).
- For a result to be statistically significant at alpha = 0.05, the p-value must be **less than 0.05**.
Number needed to treat relation US Medical PG Question 2: A medical research study is evaluating an investigational novel drug (medication 1) as compared with standard therapy (medication 2) in patients presenting to the emergency department with myocardial infarction (MI). The study enrolled a total of 3,000 subjects, 1,500 in each study arm. Follow-up was conducted at 45 days post-MI. The following are the results of the trial:
Endpoints Medication 1 Medication 2 P-Value
Primary: death from cardiac causes 134 210 0.03
Secondary: hyperkalemia 57 70 0.4
What is the relative risk of death from a cardiac cause, expressed as a percentage? (Round to the nearest whole number.)
- A. 64% (Correct Answer)
- B. 42%
- C. 72%
- D. 36%
- E. 57%
Number needed to treat relation Explanation: ***64%***
- The **relative risk (RR)** is calculated as the event rate in the exposed group divided by the event rate in the unexposed (control) group.
- For cardiac death, the event rate for Medication 1 is 134/1500 = 0.0893, and for Medication 2 is 210/1500 = 0.14. Therefore, RR = 0.0893 / 0.14 = 0.6378.
- Expressing as a percentage: 0.6378 × 100 = 63.78%, which rounds to **64%**.
- This indicates that Medication 1 has 64% of the risk of cardiac death compared to Medication 2, representing a **36% relative risk reduction**.
*42%*
- This option is incorrect as it does not reflect the accurate calculation of **relative risk** using the provided event rates.
- A calculation error or conceptual misunderstanding of the relative risk formula would lead to this value.
*72%*
- This percentage is higher than the calculated relative risk, suggesting an incorrect application of the formula or a misinterpretation of the event rates.
- It does not represent the ratio of risk between the two medication groups for cardiac death.
*36%*
- This value represents the **relative risk reduction** (100% - 64% = 36%), not the relative risk itself.
- This is a common error where students confuse relative risk with relative risk reduction.
*57%*
- While closer to the correct answer, this value is not the precise result when rounding to the nearest whole number.
- Small calculation discrepancies or rounding at intermediate steps could lead to this slightly different percentage.
Number needed to treat relation US Medical PG Question 3: A researcher is trying to determine whether a newly discovered substance X can be useful in promoting wound healing after surgery. She conducts this study by enrolling the next 100 patients that will be undergoing this surgery and separating them into 2 groups. She decides which patient will be in which group by using a random number generator. Subsequently, she prepares 1 set of syringes with the novel substance X and 1 set of syringes with a saline control. Both of these sets of syringes are unlabeled and the substances inside cannot be distinguished. She gives the surgeon performing the surgery 1 of the syringes and does not inform him nor the patient which syringe was used. After the study is complete, she analyzes all the data that was collected and performs statistical analysis. This study most likely provides which level of evidence for use of substance X?
- A. Level 3
- B. Level 1 (Correct Answer)
- C. Level 4
- D. Level 5
- E. Level 2
Number needed to treat relation Explanation: ***Level 1***
- The study design described is a **randomized controlled trial (RCT)**, which is considered the **highest level of evidence (Level 1)** in the hierarchy of medical evidence.
- Key features like **randomization**, **control group**, and **blinding (double-blind)** help minimize bias and strengthen the validity of the findings.
*Level 2*
- Level 2 evidence typically comprises **well-designed controlled trials without randomization** (non-randomized controlled trials) or **high-quality cohort studies**.
- While strong, they do not possess the same level of internal validity as randomized controlled trials.
*Level 3*
- Level 3 evidence typically includes **case-control studies** or **cohort studies**, which are observational designs and carry a higher risk of bias compared to RCTs.
- These studies generally do not involve randomization or intervention assignment by the researchers.
*Level 4*
- Level 4 evidence is usually derived from **case series** or **poor quality cohort and case-control studies**.
- These studies provide descriptive information or investigate associations without strong control for confounding factors.
*Level 5*
- Level 5 evidence is the **lowest level of evidence**, consisting of **expert opinion** or **animal research/bench research**.
- This level lacks human clinical data or systematic investigative rigor needed for higher evidence levels.
Number needed to treat relation US Medical PG Question 4: A researcher is designing an experiment to examine the toxicity of a new chemotherapeutic agent in mice. She splits the mice into 2 groups, one of which she exposes to daily injections of the drug for 1 week. The other group is not exposed to any intervention. Both groups are otherwise raised in the same conditions with the same diet. One month later, she sacrifices the mice to check for dilated cardiomyopathy. In total, 52 mice were exposed to the drug, and 50 were not exposed. Out of the exposed group, 13 were found to have dilated cardiomyopathy on necropsy. In the unexposed group, 1 mouse was found to have dilated cardiomyopathy. Which of the following is the relative risk of developing cardiomyopathy with this drug?
- A. 12.5 (Correct Answer)
- B. 25.0
- C. 13.7
- D. 16.3
- E. 23.0
Number needed to treat relation Explanation: ***Correct Option: 12.5***
- The **relative risk (RR)** is calculated as the **risk in the exposed group divided by the risk in the unexposed group**: RR = [a/(a+b)] / [c/(c+d)]
- **Risk in exposed group** = 13/52 = 0.25 (25%)
- **Risk in unexposed group** = 1/50 = 0.02 (2%)
- **RR = 0.25 / 0.02 = 12.5**
- This indicates that mice exposed to the chemotherapeutic agent are **12.5 times more likely** to develop dilated cardiomyopathy compared to unexposed mice
- An **RR > 1** indicates increased risk with exposure, supporting the drug's cardiotoxicity
*Incorrect Option: 25.0*
- This value results from **miscalculating the unexposed group risk** (e.g., using 0.01 instead of 0.02 as the denominator)
- If the unexposed risk was halved incorrectly: 0.25 / 0.01 = 25.0
- This overestimates the relative risk by a factor of 2
*Incorrect Option: 13.7*
- This value does not result from the correct **relative risk formula**
- May arise from an **arithmetic error** or confusion with other epidemiological measures
- The correct calculation of 13/52 ÷ 1/50 does not yield this result
*Incorrect Option: 16.3*
- This might result from **miscounting the number of subjects** in either group or confusing **relative risk with odds ratio**
- The **odds ratio** would be calculated as (13/39) / (1/49) = 16.3
- Remember: **Relative risk uses total exposed/unexposed as denominators**, while odds ratio uses non-diseased counts
*Incorrect Option: 23.0*
- This value suggests a **fundamental error** in applying the relative risk formula
- Could result from using incorrect numerators or denominators (e.g., 13/1 instead of proper risk calculation)
- Significantly overestimates the true relative risk of 12.5
Number needed to treat relation US Medical PG Question 5: A physician attempts to study cirrhosis in his state. Using a registry of admitted patients over the last 10 years at the local hospital, he isolates all patients who have been diagnosed with cirrhosis. Subsequently, he contacts this group of patients, asking them to complete a survey assessing their prior exposure to alcohol use, intravenous drug abuse, blood transfusions, personal history of cancer, and other medical comorbidities. An identical survey is given to an equal number of patients in the registry who do not carry a prior diagnosis of cirrhosis. Which of the following is the study design utilized by this physician?
- A. Randomized controlled trial
- B. Case-control study (Correct Answer)
- C. Cross-sectional study
- D. Cohort study
- E. Meta-analysis
Number needed to treat relation Explanation: ***Case-control study***
- This study design **identifies subjects based on their outcome (cases with cirrhosis, controls without cirrhosis)** and then retrospectively investigates their past exposures.
- The physician selected patients with cirrhosis (cases) and patients without cirrhosis (controls), then assessed their prior exposures to risk factors like alcohol use and intravenous drug abuse.
*Randomized controlled trial*
- This design involves randomly assigning participants to an **intervention group** or a **control group** to assess the effect of an intervention.
- There is no intervention being tested or randomization occurring in this study; it is observational.
*Cross-sectional study*
- A cross-sectional study measures the **prevalence of disease and exposure at a single point in time** in a defined population.
- This study collects retrospective exposure data and compares two distinct groups (cases and controls), rather than assessing prevalence at one time point.
*Cohort study*
- A cohort study **follows a group of individuals over time** to see if their exposure to a risk factor is associated with the development of a disease.
- This study starts with the outcome (cirrhosis) and looks backward at exposures, which is the opposite direction of a cohort study.
*Meta-analysis*
- A meta-analysis is a statistical method that **combines the results of multiple independent studies** to produce a single, more powerful estimate of treatment effect or association.
- This is an original research study collecting new data, not a systematic review or synthesis of existing studies.
Number needed to treat relation US Medical PG Question 6: An epidemiologist is evaluating the efficacy of Noxbinle in preventing HCC deaths at the population level. A clinical trial shows that over 5 years, the mortality rate from HCC was 25% in the control group and 15% in patients treated with Noxbinle 100 mg daily. Based on this data, how many patients need to be treated with Noxbinle 100 mg to prevent, on average, one death from HCC?
- A. 20
- B. 73
- C. 10 (Correct Answer)
- D. 50
- E. 100
Number needed to treat relation Explanation: ***10***
- The **number needed to treat (NNT)** is calculated by first finding the **absolute risk reduction (ARR)**.
- **ARR** = Risk in control group - Risk in treatment group = 25% - 15% = **10%** (or 0.10).
- **NNT = 1 / ARR** = 1 / 0.10 = **10 patients**.
- This means that **10 patients must be treated with Noxbinle to prevent one death from HCC** over 5 years.
*20*
- This would result from an ARR of 5% (1/0.05 = 20), which is not supported by the data.
- May arise from miscalculating the risk difference or incorrectly halving the actual ARR.
*73*
- This value does not correspond to any standard calculation of NNT from the given mortality rates.
- May result from confusion with other epidemiological measures or calculation error.
*50*
- This would correspond to an ARR of 2% (1/0.02 = 50), which significantly underestimates the actual risk reduction.
- Could result from incorrectly calculating the difference as a proportion rather than absolute percentage points.
*100*
- This would correspond to an ARR of 1% (1/0.01 = 100), grossly underestimating the treatment benefit.
- May result from confusing ARR with relative risk reduction or other calculation errors.
Number needed to treat relation US Medical PG Question 7: You submit a paper to a prestigious journal about the effects of coffee consumption on mesothelioma risk. The first reviewer lauds your clinical and scientific acumen, but expresses concern that your study does not have adequate statistical power. Statistical power refers to which of the following?
- A. The probability of detecting an association when no association exists.
- B. The probability of not detecting an association when an association does exist.
- C. The probability of detecting an association when an association does exist. (Correct Answer)
- D. The first derivative of work.
- E. The square root of the variance.
Number needed to treat relation Explanation: ***The probability of detecting an association when an association does exist.***
- **Statistical power** is defined as the probability that a study will correctly reject a false null hypothesis, meaning it will detect a true effect or association if one exists.
- A study with **adequate statistical power** is less likely to miss a real effect.
*The probability of detecting an association when no association exists.*
- This describes a **Type I error** or **false positive**, often represented by **alpha (α)**.
- It is the probability of incorrectly concluding an effect or association exists when, in reality, there is none.
*The probability of not detecting an association when an association does exist.*
- This refers to a **Type II error** or **false negative**, represented by **beta (β)**.
- **Statistical power** is calculated as **1 - β**, so this option describes the complement of power.
*The first derivative of work.*
- The first derivative of work with respect to time represents **power** in physics, which is the rate at which work is done.
- This option is a **distractor** from physics and is unrelated to statistical power in research.
*The square root of the variance.*
- The **square root of the variance** is the **standard deviation**, a measure of the dispersion or spread of data.
- This is a statistical concept but is not the definition of statistical power.
Number needed to treat relation US Medical PG Question 8: You are currently employed as a clinical researcher working on clinical trials of a new drug to be used for the treatment of Parkinson's disease. Currently, you have already determined the safe clinical dose of the drug in a healthy patient. You are in the phase of drug development where the drug is studied in patients with the target disease to determine its efficacy. Which of the following phases is this new drug currently in?
- A. Phase 4
- B. Phase 1
- C. Phase 2 (Correct Answer)
- D. Phase 0
- E. Phase 3
Number needed to treat relation Explanation: ***Phase 2***
- **Phase 2 trials** involve studying the drug in patients with the target disease to assess its **efficacy** and further evaluate safety, typically involving a few hundred patients.
- The question describes a stage after safe dosing in healthy patients (Phase 1) and before large-scale efficacy confirmation (Phase 3), focusing on efficacy in the target population.
*Phase 4*
- **Phase 4 trials** occur **after a drug has been approved** and marketed, monitoring long-term effects, optimal use, and rare side effects in a diverse patient population.
- This phase is conducted post-market approval, whereas the question describes a drug still in development prior to approval.
*Phase 1*
- **Phase 1 trials** primarily focus on determining the **safety and dosage** of a new drug in a **small group of healthy volunteers** (or sometimes patients with advanced disease if the drug is highly toxic).
- The question states that the safe clinical dose in a healthy patient has already been determined, indicating that Phase 1 has been completed.
*Phase 0*
- **Phase 0 trials** are exploratory, very early-stage studies designed to confirm that the drug reaches the target and acts as intended, typically involving a very small number of doses and participants.
- These trials are conducted much earlier in the development process, preceding the determination of safe clinical doses and large-scale efficacy studies.
*Phase 3*
- **Phase 3 trials** are large-scale studies involving hundreds to thousands of patients to confirm **efficacy**, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug to be used safely.
- While Phase 3 does assess efficacy, it follows Phase 2 and is typically conducted on a much larger scale before submitting for regulatory approval.
Number needed to treat relation US Medical PG Question 9: A researcher is investigating the effects of a new antihypertensive medication on renal physiology. She gives a subject a dose of the new medication, and she then collects plasma and urine samples. She finds the following: Hematocrit: 40%; Serum creatinine: 0.0125 mg/mL; Urine creatinine: 1.25 mg/mL. Urinary output is 1 mL/min. Renal blood flow is 1 L/min. Based on the above information and approximating that the creatinine clearance is equal to the GFR, what answer best approximates filtration fraction in this case?
- A. 10%
- B. 17% (Correct Answer)
- C. 33%
- D. 50%
- E. 25%
Number needed to treat relation Explanation: ***17%***
- First, calculate **GFR** using the creatinine clearance formula: GFR = (Urine creatinine × Urinary output) / Serum creatinine = (1.25 mg/mL × 1 mL/min) / 0.0125 mg/mL = **100 mL/min**.
- Next, calculate **Renal Plasma Flow (RPF)** from Renal Blood Flow (RBF) and Hematocrit: RPF = RBF × (1 - Hematocrit) = 1000 mL/min × (1 - 0.40) = **600 mL/min**.
- Finally, calculate **Filtration Fraction (FF)** = GFR / RPF = 100 mL/min / 600 mL/min = 0.1667 = **16.7%, which approximates to 17%**.
- This is the correct answer based on the physiological calculations and represents a normal filtration fraction.
*10%*
- This would correspond to a filtration fraction of 0.10, which would require either a GFR of 60 mL/min (lower than calculated) or an RPF of 1000 mL/min (higher than calculated).
- This value is too low given the provided parameters and doesn't match the calculation from the given data.
*25%*
- This value would suggest FF = 0.25, requiring a GFR of 150 mL/min with the calculated RPF of 600 mL/min.
- This is higher than the calculated GFR of 100 mL/min and doesn't match the given creatinine values.
*33%*
- This would imply FF = 0.33, requiring a GFR of approximately 200 mL/min with RPF of 600 mL/min.
- This is significantly higher than the calculated GFR and would represent an abnormally elevated filtration fraction.
*50%*
- A filtration fraction of 50% is unphysiologically high and would indicate severe pathology.
- This would require a GFR of 300 mL/min with the calculated RPF, which is impossible given the provided creatinine clearance data.
Number needed to treat relation US Medical PG Question 10: You are reviewing raw data from a research study performed at your medical center examining the effectiveness of a novel AIDS screening examination. The study enrolled 250 patients with confirmed AIDS, and 240 of these patients demonstrated a positive screening examination. The control arm of the study enrolled 250 patients who do not have AIDS, and only 5 of these patients tested positive on the novel screening examination. What is the NPV of this novel test?
- A. 240 / (240 + 15)
- B. 240 / (240 + 5)
- C. 240 / (240 + 10)
- D. 245 / (245 + 10) (Correct Answer)
- E. 245 / (245 + 5)
Number needed to treat relation Explanation: ***245 / (245 + 10)***
- The **negative predictive value (NPV)** is calculated as **true negatives (TN)** divided by the sum of **true negatives (TN)** and **false negatives (FN)**.
- In this study, there are 250 patients with AIDS; 240 tested positive (true positives, TP), meaning 10 tested negative (false negatives, FN = 250 - 240). There are 250 patients without AIDS; 5 tested positive (false positives, FP), meaning 245 tested negative (true negatives, TN = 250 - 5). Therefore, NPV = 245 / (245 + 10).
*240 / (240 + 15)*
- This calculation incorrectly uses the number of **true positives** (240) in the numerator and denominator, which is relevant for **positive predictive value (PPV)**, not NPV.
- The denominator `(240 + 15)` does not correspond to a valid sum for calculating NPV from the given data.
*240 / (240 + 5)*
- This calculation incorrectly uses **true positives** (240) in the numerator, which is not part of the NPV formula.
- The denominator `(240 + 5)` mixes true positives and false positives, which is incorrect for NPV.
*240 / (240 + 10)*
- This incorrectly places **true positives** (240) in the numerator instead of **true negatives**.
- The denominator `(240+10)` represents **true positives + false negatives**, which is related to sensitivity, not NPV.
*245 / (245 + 5)*
- This calculation correctly identifies **true negatives** (245) in the numerator but incorrectly uses **false positives** (5) in the denominator instead of **false negatives**.
- The denominator for NPV should be **true negatives + false negatives**, which is 245 + 10.
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