Melphalan MCQs With Answer provides B. Pharm students a focused, practical review of melphalan — an alkylating antineoplastic agent used in multiple myeloma and preparative regimens for hematopoietic stem cell transplantation. This resource emphasizes mechanism of action, pharmacokinetics, dosing, routes of administration, toxicity profile (especially myelosuppression and mucositis), drug interactions, and monitoring parameters. Questions explore structural features, clinical uses, high‑dose protocols, renal dosing, and safety precautions for healthcare personnel. Carefully designed to reinforce learning and exam preparation, these MCQs integrate clinical and pharmaceutical concepts to deepen understanding and application. Now let’s test your knowledge with 30 MCQs on this topic.
Q1. What is the primary mechanism of action of melphalan?
- Inhibition of topoisomerase II
- Covalent DNA cross-linking by alkylation
- Inhibition of microtubule polymerization
- Antimetabolite interference with thymidylate synthase
Correct Answer: Covalent DNA cross-linking by alkylation
Q2. Melphalan is chemically classified as which type of alkylating agent?
- Nitrosourea derivative
- Platinum compound
- Nitrogen mustard (phenylalanine derivative)
- Triazene compound
Correct Answer: Nitrogen mustard (phenylalanine derivative)
Q3. Which of the following is a common clinical indication for melphalan?
- Chronic myeloid leukemia
- Multiple myeloma
- Acute lymphoblastic leukemia in children
- Benign prostatic hyperplasia
Correct Answer: Multiple myeloma
Q4. Melphalan is best described as cell cycle:
- Specific for S phase only
- Specific for M phase only
- Non–cell cycle specific
- Specific for G1 phase only
Correct Answer: Non–cell cycle specific
Q5. Which route(s) of administration are commonly used for melphalan?
- Oral and intravenous
- Intramuscular only
- Topical only
- Inhalational only
Correct Answer: Oral and intravenous
Q6. Regarding oral bioavailability of melphalan, which statement is correct?
- It is complete and unaffected by food
- Bioavailability is variable and may be reduced by food
- It is not absorbed orally at all
- Oral form is only used for topical application
Correct Answer: Bioavailability is variable and may be reduced by food
Q7. What is a dose‑limiting toxicity of melphalan?
- Cardiotoxicity
- Severe myelosuppression (bone marrow suppression)
- Cerebral edema
- Peripheral neuropathy
Correct Answer: Severe myelosuppression (bone marrow suppression)
Q8. High‑dose melphalan is commonly used in preparation for:
- Solid organ transplantation
- Autologous hematopoietic stem cell transplantation
- Gene therapy for cystic fibrosis
- Long‑term immunosuppression for rheumatoid arthritis
Correct Answer: Autologous hematopoietic stem cell transplantation
Q9. Which monitoring parameter is most critical during melphalan therapy?
- Liver function tests only
- Complete blood count with differential
- Serum amylase only
- Blood glucose levels only
Correct Answer: Complete blood count with differential
Q10. Melphalan is primarily eliminated by which route?
- Hepatic metabolism and biliary excretion exclusively
- Renal excretion of unchanged drug and metabolites
- Exhalation via lungs
- Secretion in sweat
Correct Answer: Renal excretion of unchanged drug and metabolites
Q11. Dose adjustment of melphalan is most commonly required in patients with:
- Severe renal impairment
- Well‑controlled hypertension
- Mild seasonal allergies
- Hyperthyroidism treated with methimazole
Correct Answer: Severe renal impairment
Q12. Which adverse effect is associated with gonadal toxicity and infertility risk with melphalan?
- Hepatotoxicity
- Ovarian and testicular damage
- Retinal detachment
- Pulmonary fibrosis
Correct Answer: Ovarian and testicular damage
Q13. Melphalan’s transport into cells is facilitated by:
- Glucose transporters (GLUT1)
- Amino acid transporters due to phenylalanine moiety
- ATP‑binding cassette efflux pumps only
- Passive diffusion exclusively
Correct Answer: Amino acid transporters due to phenylalanine moiety
Q14. Compared to cyclophosphamide, melphalan:
- Requires hepatic activation by CYP enzymes
- Does not require bioactivation and acts directly
- Is a platinum analog
- Has identical toxicity profile and activation pathway
Correct Answer: Does not require bioactivation and acts directly
Q15. Which supportive measure reduces risk of severe mucositis during high‑dose melphalan?
- Oral cryotherapy (ice chips) during infusion
- High‑dose vitamin C supplementation
- Prolonged fasting for 48 hours
- Concurrent erythropoietin administration
Correct Answer: Oral cryotherapy (ice chips) during infusion
Q16. A known long‑term risk associated with alkylating agents like melphalan is:
- Secondary malignancies such as therapy‑related AML/MDS
- Development of Alzheimer’s disease
- Endocarditis
- Type 1 diabetes mellitus
Correct Answer: Secondary malignancies such as therapy‑related AML/MDS
Q17. Which precaution is essential for healthcare workers when handling melphalan?
- No special precautions are needed
- Use of personal protective equipment and safe handling to avoid exposure
- Only handwashing after handling is sufficient
- Melphalan can be handled without gloves if diluted
Correct Answer: Use of personal protective equipment and safe handling to avoid exposure
Q18. In combination regimens for myeloma, melphalan is often paired with:
- Prednisone
- Insulin
- Metformin
- Levothyroxine
Correct Answer: Prednisone
Q19. Which laboratory change is typically seen after melphalan therapy?
- Marked leukocytosis within 48 hours
- Progressive neutropenia and thrombocytopenia reaching nadir in 7–21 days
- Immediate rise in platelet count
- Isolated hyperkalemia without blood count changes
Correct Answer: Progressive neutropenia and thrombocytopenia reaching nadir in 7–21 days
Q20. Melphalan is contraindicated or used with extreme caution during:
- Pregnancy due to teratogenicity
- Uncomplicated eczema
- Mild seasonal rhinitis
- Controlled hyperlipidemia
Correct Answer: Pregnancy due to teratogenicity
Q21. Which interaction could increase melphalan toxicity?
- Concurrent use of granulocyte colony‑stimulating factor (G-CSF)
- Co‑administration with other myelosuppressive chemotherapy agents
- Vitamin D supplementation
- Topical antifungal use
Correct Answer: Co‑administration with other myelosuppressive chemotherapy agents
Q22. The amino acid analog property of melphalan contributes to:
- Its rapid hepatic metabolism by CYP3A4
- Cell uptake via amino acid transporters and some tumor selectivity
- Exclusive renal tubular secretion without filtration
- Resistance to DNA alkylation
Correct Answer: Cell uptake via amino acid transporters and some tumor selectivity
Q23. For outpatient oral melphalan dosing, which counseling point is appropriate?
- Take with a high‑fat meal to increase absorption
- Take on an empty stomach or consistently with regard to meals because food can reduce and vary absorption
- Crush and mix tablets into any beverage to improve taste
- Missed doses should be doubled the next day
Correct Answer: Take on an empty stomach or consistently with regard to meals because food can reduce and vary absorption
Q24. Which adverse effect requires immediate medical attention during melphalan therapy?
- Mild hair thinning
- Fever or signs of infection suggesting neutropenic sepsis
- Transient mild nausea easily controlled with antiemetics
- Temporary taste changes
Correct Answer: Fever or signs of infection suggesting neutropenic sepsis
Q25. Melphalan’s stability and preparation considerations include:
- It is stable indefinitely in solution at room temperature
- Follow institutional guidelines for reconstitution, use promptly, and dispose of cytotoxic waste safely
- It can be stored in unsealed containers on the ward for later use
- No special disposal required
Correct Answer: Follow institutional guidelines for reconstitution, use promptly, and dispose of cytotoxic waste safely
Q26. Which pharmacokinetic property is true for melphalan?
- Extensive enterohepatic recirculation
- Moderate plasma protein binding and variable distribution
- Exclusively metabolized to inactive glucuronides
- Undergoes significant fecal elimination only
Correct Answer: Moderate plasma protein binding and variable distribution
Q27. Melphalan-induced hemorrhagic cystitis risk is:
- High and requires routine MESNA prophylaxis
- Low compared with cyclophosphamide/ifosfamide but hydration and monitoring are prudent
- Nonexistent; no precautions needed
- Only relevant with topical use
Correct Answer: Low compared with cyclophosphamide/ifosfamide but hydration and monitoring are prudent
Q28. Which statement about resistance to melphalan is correct?
- Resistance may develop via enhanced DNA repair and increased glutathione‑mediated detoxification
- Melphalan resistance is impossible because it binds irreversibly
- Resistance occurs only through decreased renal excretion
- Resistance is always due to decreased drug absorption in the gut
Correct Answer: Resistance may develop via enhanced DNA repair and increased glutathione‑mediated detoxification
Q29. In pediatric oncology, melphalan dosing considerations primarily require attention to:
- Standard adult fixed dosing without adjustment
- BSA‑based dosing and careful organ function assessment
- Avoiding use in all pediatric patients regardless of indication
- Only using intramuscular administration
Correct Answer: BSA‑based dosing and careful organ function assessment
Q30. Which statement best describes melphalan’s role in modern oncology therapy?
- Obsolete and no longer used in any regimen
- Remains valuable for specific indications like myeloma, in combination therapies, and as high‑dose conditioning prior to stem cell transplant
- Used exclusively for infectious diseases
- Preferred first‑line agent for all solid tumors
Correct Answer: Remains valuable for specific indications like myeloma, in combination therapies, and as high‑dose conditioning prior to stem cell transplant

I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
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