Therapeutic applications of enzymes MCQs With Answer are essential for B. Pharm students preparing for pharmacology and clinical pharmacy exams. This concise guide covers enzyme drugs, enzyme replacement therapies, thrombolytics, recombinant enzymes, and enzyme-targeting inhibitors with emphasis on indications, mechanisms, adverse effects, and monitoring. Keywords include enzyme therapy, thrombolytics, recombinant enzymes, enzyme inhibitors, asparaginase, pancrelipase, dornase alfa, imiglucerase, and pharmacokinetics for B. Pharm. The MCQs deepen understanding of clinical use, immunogenicity, dosing adjustments, and formulation strategies such as PEGylation and targeted delivery. Now let’s test your knowledge with 50 MCQs on this topic.
Q1. What is the primary mechanism of action of tissue plasminogen activator (tPA) in thrombolytic therapy?
- Direct cleavage of fibrinogen to fibrin
- Activation of plasminogen to plasmin on fibrin surface
- Inhibition of platelet aggregation
- Blocking thrombin formation
Correct Answer: Activation of plasminogen to plasmin on fibrin surface
Q2. Which enzyme preparation is commonly used as enzyme replacement therapy in Gaucher disease?
- Imiglucerase
- Alglucosidase alfa
- Agalsidase beta
- Idursulfase
Correct Answer: Imiglucerase
Q3. L-asparaginase is used in chemotherapy primarily because it:
- Directly alkylates DNA in leukemic cells
- Depletes circulating asparagine required by some leukemia cells
- Inhibits topoisomerase II
- Stimulates immune-mediated cytotoxicity
Correct Answer: Depletes circulating asparagine required by some leukemia cells
Q4. Dornase alfa (recombinant DNase) is used in cystic fibrosis to:
- Replace deficient pancreatic enzymes
- Hydrolyze extracellular DNA in sputum to reduce viscosity
- Stimulate surfactant production
- Provide systemic anti-inflammatory effects
Correct Answer: Hydrolyze extracellular DNA in sputum to reduce viscosity
Q5. Which of the following is a major concern with therapeutic enzymes derived from non-human sources?
- Excessive half-life
- Immunogenicity and antibody formation
- Overly specific substrate selectivity
- Inability to reach plasma compartment
Correct Answer: Immunogenicity and antibody formation
Q6. Which modification is commonly used to increase circulating half-life and reduce immunogenicity of therapeutic enzymes?
- Micronization
- PEGylation
- Chelation
- Glycolysis
Correct Answer: PEGylation
Q7. Pancrelipase is used therapeutically to treat:
- Lactose intolerance
- Exocrine pancreatic insufficiency
- Urea cycle disorders
- Hypothyroidism
Correct Answer: Exocrine pancreatic insufficiency
Q8. Which thrombolytic agent is non-fibrin specific and works by forming a complex with plasminogen?
- Alteplase (tPA)
- Urokinase
- Streptokinase
- Tenecteplase
Correct Answer: Streptokinase
Q9. Enzyme replacement therapy for Fabry disease uses which agent?
- Imiglucerase
- Agalsidase beta
- Idursulfase
- Alglucosidase alfa
Correct Answer: Agalsidase beta
Q10. Which of the following is an important monitoring parameter during thrombolytic therapy?
- Serum amylase
- Activated partial thromboplastin time (aPTT) alone
- Signs of bleeding and hemodynamic stability
- Serum bilirubin
Correct Answer: Signs of bleeding and hemodynamic stability
Q11. PEG-ADA (pegademase bovine or pegylated adenosine deaminase) is used to treat:
- Type I diabetes
- Severe combined immunodeficiency (SCID) due to ADA deficiency
- Hyperammonemia
- Hemophilia A
Correct Answer: Severe combined immunodeficiency (SCID) due to ADA deficiency
Q12. Which enzyme drug is indicated for acute ischemic stroke within appropriate time window?
- Streptokinase
- Alteplase (tPA)
- Imiglucerase
- Lactase
Correct Answer: Alteplase (tPA)
Q13. Enzyme inhibitors used as drugs typically act by which general mechanism?
- Acting as competitive or noncompetitive inhibitors of target enzymes
- Replacing deficient enzymes in plasma
- Physically breaking down pathological deposits
- Chelating essential cofactors irreversibly
Correct Answer: Acting as competitive or noncompetitive inhibitors of target enzymes
Q14. Which enzyme is the target of ACE inhibitors used in hypertension?
- Angiotensin-converting enzyme
- Renin
- Aldosterone synthase
- ACE2
Correct Answer: Angiotensin-converting enzyme
Q15. Dornase alfa is administered via which route for cystic fibrosis patients?
- Intravenous infusion
- Oral tablet
- Inhalation (nebulization)
- Subcutaneous injection
Correct Answer: Inhalation (nebulization)
Q16. Which adverse effect is commonly associated with L-asparaginase therapy?
- Hyperglycemia and pancreatitis
- Severe hypotension only
- Renal calculi
- Arrhythmias
Correct Answer: Hyperglycemia and pancreatitis
Q17. Enzyme replacement therapy for Pompe disease uses which recombinant enzyme?
- Imiglucerase
- Alglucosidase alfa
- Agalsidase alfa
- Idursulfase
Correct Answer: Alglucosidase alfa
Q18. Which property of enzymes determines affinity for substrate and can be altered by competitive inhibitors?
- Vmax
- Kcat
- Km
- Isoelectric point
Correct Answer: Km
Q19. Which enzyme therapy is used to treat cystic fibrosis by reducing sputum viscosity?
- Lactase
- Dornase alfa
- Pancrelipase
- Reteplase
Correct Answer: Dornase alfa
Q20. Enzyme prodrug activation is important in pharmacology. Which enzyme family commonly activates many prodrugs in the liver?
- CYP450 enzymes
- Proteases
- DNases
- Glycosidases
Correct Answer: CYP450 enzymes
Q21. Which of the following enzymes is used therapeutically to digest necrotic tissue and promote wound debridement?
- Streptokinase
- Collagenase
- Lactase
- Alteplase
Correct Answer: Collagenase
Q22. Which lysosomal storage disorder is treated with idursulfase?
- Gaucher disease
- Fabry disease
- Hunter syndrome (MPS II)
- Pompe disease
Correct Answer: Hunter syndrome (MPS II)
Q23. What is a clinical limitation of systemic administration of many therapeutic enzymes?
- Unlimited tissue penetration
- Rapid renal clearance and proteolytic degradation
- Excessively long half-life making dosing difficult
- No immunologic response
Correct Answer: Rapid renal clearance and proteolytic degradation
Q24. Which therapeutic enzyme degrades uric acid and is used in refractory hyperuricemia or tumor lysis syndrome?
- Uricase (rasburicase)
- Xanthine oxidase
- Allopurinol
- Guanase
Correct Answer: Uricase (rasburicase)
Q25. Which strategy improves targeted delivery of enzymes to lysosomes for enzyme replacement therapy?
- Conjugation with mannose-6-phosphate residues
- PEGylation only
- Reducing molecular weight below 5 kDa
- Administering with proton pump inhibitors
Correct Answer: Conjugation with mannose-6-phosphate residues
Q26. Which of the following is a recombinant enzyme used to treat Gaucher disease that requires macrophage uptake?
- Alglucosidase alfa
- Imiglucerase
- Reteplase
- Dornase alfa
Correct Answer: Imiglucerase
Q27. Tenecteplase differs from alteplase primarily by having:
- Shorter half-life and less fibrin specificity
- Greater fibrin specificity and longer half-life allowing bolus dosing
- Higher immunogenicity
- Direct thrombin inhibition
Correct Answer: Greater fibrin specificity and longer half-life allowing bolus dosing
Q28. Which enzyme is inhibited by statins leading to cholesterol-lowering effects?
- HMG-CoA reductase
- Acetyl-CoA carboxylase
- Cholesterol esterase
- Lipase
Correct Answer: HMG-CoA reductase
Q29. Which therapeutic enzyme is used orally to treat lactose intolerance?
- Pancrelipase
- Lactase
- Uricase
- Sucrase-isomaltase
Correct Answer: Lactase
Q30. The formation of neutralizing antibodies against therapeutic enzymes most directly affects:
- Route of administration
- Oral bioavailability only
- Clinical efficacy and safety through inactivation and hypersensitivity
- Molecular weight of the enzyme
Correct Answer: Clinical efficacy and safety through inactivation and hypersensitivity
Q31. Which enzyme drug is indicated for acute pulmonary embolism and can be given as intravenous bolus or infusion?
- Alteplase (tPA)
- Imiglucerase
- Rasburicase
- Lactase
Correct Answer: Alteplase (tPA)
Q32. In enzyme kinetics, a noncompetitive inhibitor typically changes which parameter?
- Km only
- Vmax only
- Km and Vmax identically
- Isoelectric point
Correct Answer: Vmax only
Q33. Which enzyme therapy is administered to patients with chronic pancreatitis to assist digestion?
- Pancrelipase
- Alteplase
- Agalsidase beta
- Dornase alfa
Correct Answer: Pancrelipase
Q34. Which of the following enzyme-targeting drugs is used in heart failure by inhibiting neprilysin?
- ACE inhibitors
- ARNI (sacubitril combined with valsartan)
- Statins
- Beta-blockers
Correct Answer: ARNI (sacubitril combined with valsartan)
Q35. Which therapeutic enzyme is contraindicated in patients with active internal bleeding or recent intracranial hemorrhage?
- Lactase
- Thrombolytics such as alteplase
- Imiglucerase
- Collagenase
Correct Answer: Thrombolytics such as alteplase
Q36. Which of the following describes a common delivery challenge for oral enzyme therapies?
- High permeability across intestinal epithelium
- Degradation by gastric acid and proteases
- Excessive systemic absorption leading to toxicity
- No interaction with food components
Correct Answer: Degradation by gastric acid and proteases
Q37. Which enzyme replacement is used for mucopolysaccharidosis type I (Hurler syndrome)?
- Idursulfase
- Laronidase
- Imiglucerase
- Alglucosidase alfa
Correct Answer: Laronidase
Q38. Rasburicase works by converting uric acid into:
- Xanthine
- Allantoin
- Hypoxanthine
- Guanine
Correct Answer: Allantoin
Q39. Which factor increases the risk of hypersensitivity reactions to bacterial-derived therapeutic enzymes?
- Human origin of enzyme
- Prior exposure and antibody formation to bacterial proteins
- Low molecular weight
- Oral route of administration only
Correct Answer: Prior exposure and antibody formation to bacterial proteins
Q40. Which enzyme class is a common drug target for anti-inflammatory PDE inhibitors used in respiratory and cardiac indications?
- Proteases
- Phosphodiesterases (PDEs)
- Kinases only
- Glycosidases
Correct Answer: Phosphodiesterases (PDEs)
Q41. Which of the following is a recombinant enzyme therapy used in Pompe disease to replace deficient acid alpha-glucosidase?
- Alglucosidase alfa
- Imiglucerase
- Agalsidase beta
- Rasburicase
Correct Answer: Alglucosidase alfa
Q42. Which enzyme inhibitor class is the mainstay therapy for peptic ulcer disease by inhibiting gastric acid secretion?
- Protease inhibitors
- Proton pump inhibitors (PPIs) targeting H+/K+ ATPase
- Acetylcholinesterase inhibitors
- Lipase inhibitors
Correct Answer: Proton pump inhibitors (PPIs) targeting H+/K+ ATPase
Q43. Which enzyme therapy is indicated for lysosomal glucocerebrosidase deficiency?
- Imiglucerase
- Alglucosidase alfa
- Idursulfase
- Rasburicase
Correct Answer: Imiglucerase
Q44. Which biochemical parameter is most directly affected by thrombolytic-induced fibrinolysis?
- Serum creatinine
- Plasma fibrinogen levels and fibrin degradation products
- Serum glucose
- Liver transaminases only
Correct Answer: Plasma fibrinogen levels and fibrin degradation products
Q45. Which of the following is a therapeutic use of collagenase topical preparations?
- Oral enzyme replacement in cystic fibrosis
- Debridement of chronic wounds and removal of necrotic tissue
- Treatment of thrombosis
- Systemic enzyme replacement for lysosomal disorders
Correct Answer: Debridement of chronic wounds and removal of necrotic tissue
Q46. Which approach reduces immunogenicity of therapeutic enzymes and may improve tissue penetration?
- Increasing dosing frequency without modification
- PEGylation and human sequence engineering
- Using higher molecular weight animal-derived enzymes
- Co-administering with antibiotics only
Correct Answer: PEGylation and human sequence engineering
Q47. Which enzyme-based drug is commonly used in acute management of myocardial infarction when percutaneous coronary intervention is unavailable?
- Pancrelipase
- Thrombolytics such as alteplase or tenecteplase
- Imiglucerase
- Rasburicase
Correct Answer: Thrombolytics such as alteplase or tenecteplase
Q48. In designing enzyme therapeutics, which factor is crucial for ensuring activity at target site?
- Maintaining tertiary structure and catalytic site integrity
- Maximizing degradation in plasma
- Ensuring no glycosylation at all
- Making enzyme highly hydrophobic only
Correct Answer: Maintaining tertiary structure and catalytic site integrity
Q49. Which lab finding suggests successful enzymatic thrombolysis?
- Decreased D-dimer levels
- Increased fibrinogen and clot stability
- Reduction in thrombus size on imaging and improved perfusion
- Marked leukocytosis only
Correct Answer: Reduction in thrombus size on imaging and improved perfusion
Q50. Which of the following best summarizes the therapeutic role of enzymes in modern medicine?
- They are rarely used due to instability and have no clinical relevance
- They serve as replacement therapies, thrombolytics, mucolytics, digestive aids, and targets for inhibitors, with formulation and immunogenicity challenges
- They are only used in laboratory diagnostics
- They function exclusively as antibiotics
Correct Answer: They serve as replacement therapies, thrombolytics, mucolytics, digestive aids, and targets for inhibitors, with formulation and immunogenicity challenges
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