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Related Concept Videos

Dosage Regimen: Individualization01:24

Dosage Regimen: Individualization

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Individualization in dosing regimens is the customization of medication doses for individual patients. Its necessity arises from the goal of maximizing therapeutic benefits while minimizing risks. This approach is pivotal because human responses to drugs can vary widely; what is effective for one person may be inadequate or excessive for another. Interpatient (intersubject) variability refers to differences in drug responses between individuals, while intrapatient (intrasubject) variability...
38
Dosage Regimen: Fixed Dose01:01

Dosage Regimen: Fixed Dose

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Fixed-dose regimens are a common approach to administer drugs to achieve and maintain desired levels of the drug in the body. In this dosing strategy, a specific amount of medication is given at regular intervals, often multiple times a day, to ensure a consistent drug concentration in the bloodstream.
Fixed-dose regimens can be used for various routes of administration, including intravenous (IV) injections and oral medications. For IV administration, a predetermined amount of the drug is...
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Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic...
297
Dosage Regimens: Partial Pharmacokinetic Parameters01:01

Dosage Regimens: Partial Pharmacokinetic Parameters

40
It is not uncommon for complete drug pharmacokinetic profiles to remain elusive in pharmacokinetics. This necessitates certain educated assumptions by pharmacokineticists to determine appropriate dosage regimens without comprehensive pharmacokinetic data from animal or human studies. One prevalent assumption is setting the bioavailability factor, denoted as F, to 1 or 100%. This assumption caters to the scenario where a drug doesn't achieve full systemic absorption, resulting in the patient...
40
Dosage Regimens: Designs and Approaches01:28

Dosage Regimens: Designs and Approaches

87
Designing a dosage regimen, which refers to the manner of drug administration, is a complex process involving the selection of drug dose, route, and frequency. This process is underpinned by pharmacokinetic parameters derived from tests and population averages. These parameters are then tailored to patient-specific variables such as diagnosis, demographics, and allergy status. Once therapy commences, therapeutic response monitoring is critical and achieved through clinical and physical...
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Drug Dosing: Infants and Children01:29

Drug Dosing: Infants and Children

68
Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
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Updated: Nov 3, 2025

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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CFTR Modulators: Does One Dose Fit All?

Renske van der Meer1, Erik B Wilms2, Harry G M Heijerman3

  • 1Department of Pulmonology, Haga Teaching Hospital, Els Borst-Eilersplein 275, 2545 AA The Hague, The Netherlands.

Journal of Personalized Medicine
|June 2, 2021
PubMed
Summary

CFTR modulators are key for cystic fibrosis treatment, but individual responses vary. This review explores dosing adjustments based on pharmacokinetics and pharmacodynamics to optimize treatment for people with cystic fibrosis.

Keywords:
CFTR modulatorscystic fibrosisexposure response relationshippharmacodynamicspharmacokinetics

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Area of Science:

  • Pharmacology
  • Medical Genetics
  • Respiratory Medicine

Background:

  • Cystic fibrosis (CF) is a genetic disorder affecting multiple organs.
  • CFTR modulators are a primary treatment for many people with CF (pwCF).
  • Current CFTR modulator therapies demonstrate group-level efficacy but show individual variability.

Purpose of the Study:

  • To review reasons for reconsidering CFTR modulator dosing regimens.
  • To enhance treatment response and minimize side effects in pwCF.
  • To explore pharmacokinetic-pharmacodynamic relationships for optimized dosing.

Main Methods:

  • Discussion of pharmacodynamic properties of CFTR modulators.
  • Analysis of pharmacokinetic properties and their variability in pwCF.
  • Examination of pharmacokinetic-pharmacodynamic relationships to understand dose-response and exposure-response.

Main Results:

  • Individual treatment effects of CFTR modulators are variable.
  • Drug exposure can be influenced by CF disease-related factors.
  • Understanding PK/PD relationships is crucial for personalized dosing.

Conclusions:

  • Specific situations warrant reconsideration of current CFTR modulator dosing regimens.
  • Further research is needed to unravel complex PK/PD interactions.
  • Optimizing dosing strategies can improve therapeutic outcomes for pwCF.