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Principles of Clinical Pharmacology

Chapter 71 | Part 3: Pharmacology · Part 3 – Pharmacology · Chapter 71


Key Clinical Points

  1. Adherence is preferred over 'compliance' to remove the implication that patients are at fault.
  2. Pharmacokinetics (PK) describes drug concentration vs. time; Pharmacodynamics (PD) describes drug concentration vs. effect.
  3. Therapeutic index (ratio/window) defines the margin between effective doses and toxic doses.
  4. First-pass elimination reduces bioavailability of oral drugs via intestinal and hepatic metabolism.
  5. P-glycoprotein (P-gp) acts as an efflux pump limiting systemic availability and brain penetration.
  6. Type A ADRs are dose-dependent; Type B ADRs are dose-independent and often unexpected.
  7. Steady state is achieved after approximately 5 elimination half-lives.
  8. Loading doses are used to reach therapeutic concentrations more rapidly in urgent cases.
  9. Renal and liver disease can significantly alter drug levels due to changes in excretion or metabolism.
  10. Heart failure can lead to redistribution of cardiac output, increasing CNS or cardiac effects of drugs.
  11. Drug interactions can be pharmacokinetic (changes in concentration) or pharmacodynamic (changes in effect without concentration change).

1. DEFINITION & OVERVIEW

Core Concepts: Drugs are the cornerstone of modern therapeutics; outcomes vary based on individual factors (genetics, disease, interactions).

Goals of Clinical Pharmacology: 1. Provide a description of conditions under which drug actions vary among human subjects. 2. Determine mechanisms underlying this variability to improve therapy with available drugs.

Evolution of Field: Shift from empirical descriptions of sensitivity/disease influence to understanding molecular mechanisms (pharmacogenomics) and systems biology. Systems biology involves complex computational models, gene editing, and multi-omic measurements (transcriptomics, metabolomics, proteomics).

Adherence vs. Compliance: The term 'adherence' is preferred over 'compliance' because it removes the idea that the patient is at fault.

Therapeutic Index: Defined as the margin between doses required for effect and those producing toxicity. Also known as therapeutic ratio or therapeutic window. Monitoring is useful when a clear relationship exists between plasma concentration and effect (e.g., anticonvulsants, antirejection agents, antiarrhythmics).


2. EPIDEMIOLOGY

Adverse Drug Reactions (ADRs): Can be mistaken for signs of underlying disease because they involve every organ system.

Failure of Efficacy: The most common 'adverse' drug effect; occurs in up to half of patients with conditions like psychiatric disease or hypertension.

Statistics (UK): 6.5% of hospital admissions are due to ADRs → 2.3% of these result in death.

Common Culprit Drugs: Aspirin, nonsteroidal anti-inflammatory drugs (NSAIDs), diuretics, warfarin, ACE inhibitors, antidepressants, opiates, digoxin, steroids, and clopidogrel.

US Data: ADRs were responsible for >100,000 in-hospital deaths (4th to 6th most common cause).


3. ETIOLOGY & PATHOPHYYSOLOGY

Pharmacokinetics (PK): Relationship between drug concentration and time (delivery/removal).

Bioavailability: Fraction of drug available to the systemic circulation. - <100% due to: 1. Incomplete absorption. 2. Metabolism or elimination prior to entering the systemic circulation (presystemic/first-pass effect).

First-Pass Effect: Occurs in intestinal enterocytes and the liver before reaching systemic circulation. - Enterocyte barrier: Drug may be metabolized, excreted into the lumen, or transported via P-glycoprotein. - Hepatic barrier: Drug may be metabolized or excreted into the bile. - Clinical Examples: - Nitroglycerin: 0% bioavailability orally due to complete extraction; used sublingually, transdermally, or intravenously. - Verapamil: High first-pass metabolism requires much higher oral doses (40–120 mg) compared to IV (1–5 mg).

Drug Transport: Combination of passive diffusion and active transport. - P-glycoprotein (ABCB1/MDR1): - Function: Efflux pump. - Locations: Apical enterocyte, canicular hepatocyte, cerebral capillaries. - Effect: Limits drug availability to systemic circulation and brain penetration; forms part of the blood-brain barrier.

Drug Metabolism: Converts drugs into more polar compounds for easier excretion. - Phase I: Chemical modification (mostly oxidation via Cytochrome P450/CYP). - Phase II: Conjugation of endogenous compounds (glucuronyl-, acetyl-, sulfo-, and methyltransferases).

Table 1 Summary: - CYP3A: Substrates include CCBs, Antiarrhythmics (lidocaine, quinidine, mexiletine), HMG-CoA reductase inhibitors (statins), Cyclosporine, tacrolimus, Indinavir, saquinavir, ritonavir, Timolol, metoprolor, carvedilol, Propafenone, flecainide, Tricyclic antidepressants, Fluoxetine, paroxetine. Inhibitors: Amiodarone, Ketoconazole, Itraconazole, Erythromycin, Clarithromycin, Ritonavir, Gemfibrozil/fibrates. - CYP2C9: Substrates include Warfarin, Phenytoin, Glipizide, Losartan, Omeprazole, Mephenytoin, Clopidogrel. Inhibitors: Amiodarone, Fluconazole, Phenytoin. - CYP2B6: Substrates include Efavirenz, 6-Mercaptopurine, azathioprine. Inhibitor: Ticlopidine. - N-acetyltransferase: Isoniazid, Procainamide, Hydralazine, Sulfonamides, Irinotecan. - Pseudocholinesterase: Succinylcholine. - P-glycoprotein: Substrates include Digoxin, HIV protease inhibitors, many CYP3A substrates. Inhibitors: Quinidine, Amiodarone, Verapamil, Cyclosporine, Itraconazole, Erythromycin.

Drug Elimination: Most processes are first-order (rate depends on amount present). - Half-life: Time for 50% of a first-order process to be completed. - Steady State: Achieved after approximately 5 elimination half-lives. - Distribution vs. Elimination: Initial rapid drop in concentration = distribution into tissues; Slower decline = elimination from system.

Pharmacodynamics (PD): Relationship between drug concentration and effect (action). The linear framework of PK/PD is a first approximation for understanding drug effects.


4. CLINICAL FEATURES

Adverse Drug Reactions (ADRs): Categorized by mechanism and predictability. - Type A: - Mechanism: Exaggeration of intended pharmacologic action. - Characteristics: Dose-dependent, predictable (e.g., bleeding with anticoagulants). - Type B: - Mechanism: Toxic effects unrelated to intended actions. - Characteristics: Dose-independent, often unanticipated; may result from toxic effects unrelated to intended actions.


5. DIFFERENTIAL DIAGNOSIS

Drug Interactions: Must be considered in any unusual response during system-wide drug therapy. - Pharmacokinetic (PK) Interactions: - Result from changes in drug disposition (absorption, distribution, metabolism, excretion). - Pharmacodynamic (PD) Interactions: - Result from changes in drug response without changes in plasma concentration.

Table 2 Summary: - Decreased Effect (PK): - Due to gut binding: Antacids/tetracyclines, Cholestyramine/digoxin. - Due to induction of metabolism/transport: Rifampin, Carbamazepine, Phenytoin, St. John's wort, Glutethimide, smoking, etc. - Increased Effect (PK): - Due to inhibited transport: Amiodarone (inhibits many CYPs and P-glycoprotein). - Due to reduced renal clearance: Probenecid, probenecid, salicylates (increases methotrexate toxicity). - Antagonistic (PD): - Example: Loss of antihypertensive drug effects with NSAIDs.


6. INVESTIGATIONS & DIAGNOSIS

Monitoring Parameters: Identify drugs where plasma concentration correlates with effect to guide dosing. - Anticonvulsants. - Antirejection agents. - Antiarrhythmics.


7. MANAGEMENT & TREATMENT

Dosing Strategies: 1. Loading Doses: - Used for urgent indications to reach therapeutic levels faster. - Note: Does not alter the final steady state, only the time required to achieve it. 2. Steady State Adjustments: - For first-order elimination: Doubling the dose results in a doubling of the steady-state concentration. - Timeframe: Steady state is reached after ~5 half-lives. 3. Route Selection based on Bioavailability: - Drugs with high first-pass metabolism (e.g., Nitroglycerin) → use sublingual, transdermal, or IV routes to bypass presystemic metabolism. - Drugs with high first-pass but usable oral doses (e.g., Verapamil) → require significantly higher oral doses than IV equivalents.


8. PROGNOSIS & COMPLICATIONS

Clinical Implications of Drug Distribution: - Heart Failure: Decreased tissue perfusion redistributes cardiac output, increasing CNS or cardiac effects of drugs. - P-glycoprotein (P-gp) Dysfunction: - Reduced P-gp function (e.g., due to drug interactions) can increase penetration of substrate drugs into the brain even when plasma concentrations are unchanged.


9. SPECIAL CONSIDERATIONS

Renal and Liver Disease: - Metabolism/Excretion: - Drugs with predominant renal excretion (e.g., digoxin, dofetilide) require dose reduction in renal disease. - In liver disease, oral bioavailability of high first-pass drugs (e.g., morphine, nifedipine) is almost doubled.

Drug Interaction Risks: - Amiodarone: Potent inhibitor of many CYPs and P-glycoprotein; can significantly increase levels of warfarin, digoxin, and dabigatran.


10. KEY PEARLS & CLINICAL TRAPS

P-glycoprotein (P-gp): Acts as a critical gatekeeper at the blood-brain barrier; its inhibition can lead to CNS toxicity even if plasma levels are stable. • First-Pass Effect: Crucial for determining oral vs. parenteral dosing requirements; determines bioavailability of drugs like Nitroglycerin and Verapamil. • Type A vs. Type B: Distinguishing between dose-dependent (A) and dose-independent/unexpected (B) reactions is vital for safety monitoring. • Steady State Rule: Always remember the 5 half-lives rule for reaching steady state after any change in dosing or initiation of therapy.


Reference Tables

TABLE 71-1 Molecular Pathways Mediating Drug Disposition

Harrison's 22e, p.485

ENZYME SUBSTRATESa INHIBITORSa
CYP3A Calcium channel blockers Amiodarone
Antiarrhythmics (lidocaine,
quinidine, mexiletine)
Ketoconazole,
itraconazole
HMG-CoA reductase
inhibitors (“statins”; see
text)
Erythromycin,
clarithromycin
Cyclosporine, tacrolimus Ritonavir
Indinavir, saquinavir,
ritonavir
Gemfibrozil and other
fibrates
Timolol, metoprolol,
carvedilol
Propafenone, flecainide
Tricyclic antidepressants
Fluoxetine, paroxetine
CYP2C9b Warfarin Amiodarone
Phenytoin Fluconazole
Glipizide Phenytoin
Losartan
Omeprazole
Mephenytoin
Clopidogrel
CYP2B6b Efavirenz Ticlopidine
6-Mercaptopurine,
azathioprine
N-acetyltransferaseb Isoniazid
Procainamide
Hydralazine
Some sulfonamides
Irinotecan
Pseudocholinesteraseb Succinylcholine
TRANSPORTER SUBSTRATESa INHIBITORSa
P-glycoprotein Digoxin Quinidine
HIV protease inhibitors Amiodarone
Many CYP3A substrates Verapamil
Cyclosporine
Itraconazole
Erythromycin
Simvastatin and some
other statins

TABLE 71-2 Drug Interactions MECHANISM Pharmacokinetic Interactions Causing Decreased Drug Effect Decreased absorption…

Harrison's 22e, p.489

MECHANISM EXAMPLE
Pharmacokinetic Interactions Causing Decreased Drug Effect
Decreased absorption due to drug
binding in the gut
Antacids or bile acid sequestrants
decrease the absorption of many drugs:
Antacids/tetracyclines
Cholestyramine/digoxin
Induction of drug metabolism and/
or drug transport:
Rifampin
Carbamazepine
Phenytoin
St. John’s wort
Glutethimide
(also smoking, exposure to
chlorinated insecticides, and
chronic alcohol ingestion)
Decreased concentrations and effects of:
Warfarin
Quinidine
Cyclosporine
Losartan
Oral contraceptives
Methadone
Dabigatran
Reduced delivery of drug to active
sites of action
Tricyclics prevent clonidine uptake
into adrenergic neurons, preventing
antihypertensive effects
Pharmacokinetic Interactions Causing Increased Drug Effect
Inhibited drug transport Amiodarone (inhibits many CYPs and
P-glycoprotein):
Warfarin
Digoxin
Dabigatran
Decreased elimination due to
altered renal function
Inhibitors of renal tubular transport
(phenylbutazone, probenecid, salicylates)
increase methotrexate toxicity
Pharmacodynamic Drug Interactions
Antagonistic effects on the same
biologic process
Loss of antihypertensive drug effects with
NSAIDs