Biologic medicines for blood disorders MCQs With Answer

Biologic medicines for blood disorders MCQs With Answer is a focused quiz resource for M.Pharm students studying Biological Evaluation of Drug Therapy. This set of 20 multiple-choice questions explores therapeutic biologics used in hematology — including recombinant growth factors, clotting factors, monoclonal antibodies, antibody–drug conjugates, Fc‑fusion proteins and gene therapy approaches. Questions emphasize mechanisms of action, clinical indications, adverse effects, immunogenicity, production platforms and regulatory concepts relevant to biologics. Each MCQ tests both conceptual understanding and practical knowledge required for evaluation and safe use of these agents in clinical and research settings. Carefully review explanations and references in your coursework to complement this practice set.

Q1. Which biologic agent is a recombinant erythropoiesis-stimulating protein primarily used to treat anemia in chronic kidney disease?

• Epoetin alfa
• Filgrastim
• Romiplostim
• Etanercept

Correct Answer: Epoetin alfa

Q2. Filgrastim is a colony‑stimulating factor used to reduce neutropenia. What is its primary receptor-mediated mechanism?

• Activation of the G-CSF receptor on neutrophil precursors to stimulate proliferation and differentiation
• Blockade of TNF-alpha signaling to reduce marrow suppression
• Agonism of thrombopoietin receptor (c-Mpl) to increase platelet production
• Inhibition of complement C5 to prevent hemolysis

Correct Answer: Activation of the G-CSF receptor on neutrophil precursors to stimulate proliferation and differentiation

Q3. Which agent is a thrombopoietin receptor (TPO-R) agonist used for chronic immune thrombocytopenia (ITP)?

• Romiplostim
• Rituximab
• Desmopressin
• Eculizumab

Correct Answer: Romiplostim

Q4. Recombinant factor VIII products are used to treat hemophilia A. Which statement correctly describes a key challenge with these biologics?

• Development of neutralizing anti‑factor VIII inhibitors that reduce efficacy
• Universal oral bioavailability making subcutaneous dosing unnecessary
• Complete lack of post‑translational modifications simplifies production
• They are routinely metabolized by cytochrome P450 enzymes

Correct Answer: Development of neutralizing anti‑factor VIII inhibitors that reduce efficacy

Q5. Rituximab targets which antigen and is used in certain hematologic conditions such as autoimmune cytopenias and B‑cell malignancies?

• CD20
• CD3
• CD52
• Complement C5

Correct Answer: CD20

Q6. Alemtuzumab is a monoclonal antibody used in some hematologic malignancies; which cell surface protein does it target?

• CD52
• CD19
• CD33
• CD117

Correct Answer: CD52

Q7. Gemtuzumab ozogamicin is an antibody–drug conjugate used in acute myeloid leukemia. What is the targeted antigen?

• CD33
• CD22
• CD20
• CD19

Correct Answer: CD33

Q8. Which serious adverse immunologic phenomenon can occur with repeated erythropoiesis‑stimulating agent therapy and leads to severe anemia?

• Pure red cell aplasia due to neutralizing anti‑erythropoietin antibodies
• Immune thrombocytopenia due to anti‑platelet factor antibodies
• Aplastic anemia mediated by T‑cell activation against megakaryocytes
• Hemolytic anemia from complement activation against RBCs

Correct Answer: Pure red cell aplasia due to neutralizing anti‑erythropoietin antibodies

Q9. PEGylation of therapeutic proteins is commonly performed to achieve which pharmacokinetic benefit?

• Increase circulating half‑life by reducing renal clearance and proteolysis
• Enhance receptor internalization for faster onset of action
• Improve binding affinity to Fc receptors to increase ADCC
• Promote enhanced cellular uptake via endocytosis

Correct Answer: Increase circulating half‑life by reducing renal clearance and proteolysis

Q10. For a biosimilar to gain approval, regulatory agencies generally require which core demonstration?

• Analytical similarity plus targeted nonclinical and clinical comparability studies
• Only a full clinical efficacy trial identical to the originator product
• No data if the manufacturing host cell line is different
• Only pharmacovigilance data after marketing without pre‑approval studies

Correct Answer: Analytical similarity plus targeted nonclinical and clinical comparability studies

Q11. Which expression system is most commonly used for complex glycosylated recombinant hematology biologics such as clotting factors and monoclonal antibodies?

• Chinese hamster ovary (CHO) cells
• Escherichia coli
• Yeast (Saccharomyces cerevisiae)
• Plant-based transient expression systems

Correct Answer: Chinese hamster ovary (CHO) cells

Q12. Adeno-associated virus (AAV) vectors are commonly used in gene therapy for hemophilia because they primarily provide which advantage?

• Stable, long‑term expression in non‑dividing hepatocytes with low pathogenicity
• Integration into the host genome leading to high insertional mutagenesis risk
• Ability to package very large transgenes over 100 kb
• High innate immune activation that enhances transgene expression

Correct Answer: Stable, long‑term expression in non‑dividing hepatocytes with low pathogenicity

Q13. Eculizumab is indicated for paroxysmal nocturnal hemoglobinuria (PNH). What is its molecular target?

• Complement protein C5
• Factor VIII
• Thrombopoietin receptor
• Interleukin‑6 receptor

Correct Answer: Complement protein C5

Q14. Which clinical risk is notably increased when erythropoiesis‑stimulating agents are used to target hemoglobin levels above recommended thresholds?

• Thromboembolic events due to increased hematocrit
• Severe neutropenia from marrow suppression
• Hypoglycemia from insulin mimetic effects
• Renal stone formation from hypercalciuria

Correct Answer: Thromboembolic events due to increased hematocrit

Q15. Fc‑fusion proteins exploit the Fc domain primarily to achieve which pharmacological benefit?

• Extended plasma half‑life via neonatal Fc receptor (FcRn) mediated recycling
• Direct complement activation to enhance cytotoxicity
• Increased renal filtration for rapid clearance
• Conversion to small peptide fragments for oral absorption

Correct Answer: Extended plasma half‑life via neonatal Fc receptor (FcRn) mediated recycling

Q16. Emicizumab (Hemlibra) is a bispecific antibody used in hemophilia A. What is its primary mechanism?

• Bridging activated factor IX and factor X to mimic factor VIII cofactor activity
• Directly replacing factor VIII in circulation
• Neutralizing anti‑factor VIII inhibitors by Fc‑mediated clearance
• Inhibiting thrombin generation to prevent bleeding

Correct Answer: Bridging activated factor IX and factor X to mimic factor VIII cofactor activity

Q17. Inotuzumab ozogamicin is an antibody–drug conjugate used in B‑cell acute lymphoblastic leukemia. Which antigen does it target?

• CD22
• CD33
• CD20
• CD52

Correct Answer: CD22

Q18. A common adverse effect of granulocyte colony‑stimulating factor (G‑CSF) therapy is bone pain. What is the primary cause of this symptom?

• Rapid marrow expansion and stimulation of myeloid progenitors increasing intraosseous pressure
• Direct infection of bone by endotoxin contamination in the product
• Autoimmune attack on osteoblasts mediated by anti‑G‑CSF antibodies
• Calcium mobilization caused by Fc receptor binding in bone tissue

Correct Answer: Rapid marrow expansion and stimulation of myeloid progenitors increasing intraosseous pressure

Q19. Proper storage of most biologic hematology agents requires which condition to preserve stability and activity?

• Cold chain storage at 2–8°C avoiding freeze–thaw cycles
• Room temperature exposure to light for activation
• Repeated warming and cooling to maintain potency
• Storage at –80°C for immediate bedside use

Correct Answer: Cold chain storage at 2–8°C avoiding freeze–thaw cycles

Q20. Which factor increases the immunogenicity risk of therapeutic proteins and the likelihood of anti‑drug antibody formation?

• Presence of non‑human sequences, protein aggregates and subcutaneous administration
• Strictly human sequence and monomeric formulation given intravenously
• Use of glycosylation patterns identical to endogenous human proteins
• Lower protein dose and single bolus intravenous dosing only

Correct Answer: Presence of non‑human sequences, protein aggregates and subcutaneous administration

Download