Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

117
In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
117
Acute Kidney Injury IV: Diagnostic Studies and Prevention01:30

Acute Kidney Injury IV: Diagnostic Studies and Prevention

156
Accurate diagnosis and effective prevention are critical in managing Acute Kidney Injury (AKI), which is linked to high mortality rates ranging from 10% to 80%. Timely recognition of at-risk patients and careful monitoring can significantly reduce the likelihood of kidney damage.Diagnostic Assessments:The diagnostic process starts with a comprehensive medical history to identify prerenal, intrarenal, and postrenal causes.Prerenal causes, such as dehydration, hypotension, or blood loss, should...
156
Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

93
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
93
Bioavailability Study Design: Healthy Subjects Versus Patients01:15

Bioavailability Study Design: Healthy Subjects Versus Patients

64
Bioavailability studies are essential for evaluating a drug's therapeutic efficacy and understanding its absorption patterns under various physiological conditions. Conducting such studies on target patient populations provides more relevant data by simulating real-world disease states. However, practical challenges often necessitate the use of young, healthy adult volunteers as study subjects.Patients may exhibit altered drug absorption patterns due to the effects of the disease itself,...
64
Drug Dosing in Renal Diseases: Measurement of Serum Creatinine Concentration and Clearance01:25

Drug Dosing in Renal Diseases: Measurement of Serum Creatinine Concentration and Clearance

79
In healthy individuals, serum creatinine levels remain stable due to a balance between its constant production—primarily from muscle metabolism—and renal excretion. Creatinine is freely filtered by the glomeruli, making it a valuable marker for estimating renal function. When the glomerular filtration rate (GFR) decreases, the kidneys can only eliminate less creatinine, causing serum levels to rise.Serum creatinine concentration is widely used to estimate creatinine clearance...
79
Pharmacokinetics in Pediatric Patients: Drug Distribution01:17

Pharmacokinetics in Pediatric Patients: Drug Distribution

141
Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
141

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Blood proteomics of paediatric bronchiolitis obliterans syndrome after haematopoietic cell transplant.

ERJ open research·2026
Same author

Ten Years of Hydroxyurea for Ugandan Children with Sickle Cell Anemia.

The New England journal of medicine·2026
Same author

Clinical Model-Informed Precision Dosing Consult Service for Accelerating Personalized Medication in Pediatric Patients.

Clinical pharmacology and therapeutics·2026
Same author

Growth and puberty in African children with sickle cell anemia treated with hydroxyurea.

Blood advances·2026
Same author

Liver disease in Shwachman-Diamond syndrome: A comprehensive characterization across the age spectrum.

Journal of pediatric gastroenterology and nutrition·2026
Same author

CPX-351 in High-Risk Relapsed Pediatric Acute Leukemia: Real-World Phase 1 Data Establishing the FDA-Approved Dose.

Pediatric blood & cancer·2026

Related Experiment Video

Updated: Nov 28, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
07:24

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy

1.8K

Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in

Emily R Meier1, Susan E Creary2,3, Matthew M Heeney4,5

  • 1Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA.

Trials
|November 28, 2020
PubMed
Summary
This summary is machine-generated.

This study validates precision dosing for hydroxyurea in children with sickle cell disease (SCD). PK-guided dosing aims to optimize treatment and improve outcomes more rapidly than standard methods.

Keywords:
HydroxyureaPediatricsPharmacokineticsSickle cell anemia

More Related Videos

Endothelialized Microfluidics for Studying Microvascular Interactions in Hematologic Diseases
11:08

Endothelialized Microfluidics for Studying Microvascular Interactions in Hematologic Diseases

Published on: June 22, 2012

16.4K
Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

10.2K

Related Experiment Videos

Last Updated: Nov 28, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
07:24

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy

1.8K
Endothelialized Microfluidics for Studying Microvascular Interactions in Hematologic Diseases
11:08

Endothelialized Microfluidics for Studying Microvascular Interactions in Hematologic Diseases

Published on: June 22, 2012

16.4K
Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

10.2K

Area of Science:

  • Hematology
  • Pharmacology
  • Pediatric Medicine

Background:

  • Sickle cell disease (SCD) affects millions globally, with hydroxyurea as the main therapy.
  • Current hydroxyurea dosing has significant inter-patient variability, often leading to suboptimal exposure.
  • Individualized dosing may improve hydroxyurea's efficacy in SCD patients.

Purpose of the Study:

  • To validate the feasibility and benefits of PK-guided hydroxyurea dosing in a multi-center trial for children with SCD.
  • To compare PK-guided dosing against standard weight-based dosing in pediatric SCD patients.
  • To determine if PK-guided dosing can achieve optimal hydroxyurea doses more rapidly and effectively.

Main Methods:

  • The HOPS trial is a randomized, multicenter study comparing standard vs. PK-guided hydroxyurea dosing.
  • Participants aged 6 months to 21 years with SCD are randomized to either arm.
  • Pharmacokinetic (PK) data is collected via sparse microsampling, with dose adjustments guided by protocol.

Main Results:

  • The primary endpoint is the mean percentage of fetal hemoglobin (%HbF) after 6 months of hydroxyurea therapy.
  • PK-guided dosing aims for more rapid achievement of optimal hydroxyurea doses.
  • This approach is expected to provide superior clinical and laboratory benefits compared to standard dosing.

Conclusions:

  • The HOPS study will assess the clinical feasibility, benefits, and safety of PK-guided hydroxyurea dosing in pediatric SCD.
  • Results have the potential to shift the treatment paradigm for SCD from standard weight-based dosing to precision dosing.
  • Optimizing hydroxyurea therapy through PK-guided dosing may improve laboratory and clinical responses in children with SCD.