Related Experiment Video

Updated: Jan 3, 2026

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

1.9K

Effect of High-dose Vitamin A Supplementation in Children With Sickle Cell Disease: A Randomized, Double-blind,

Jefferson N Brownell1,2, Joan I Schall1, Carolyn R Mcanlis1

  • 1Division of Gastroenterology, Hepatology, and Nutrition.

Journal of Pediatric Hematology/Oncology
|November 26, 2019
PubMed

Insights

High-dose vitamin A supplementation did not improve vitamin A status in children with hemoglobin-SS disease (HbSS). However, it led to improvements in fetal hemoglobin and erythrocyte indices.

Area of Science:

  • Pediatric Hematology
  • Nutritional Science
  • Clinical Research

Background:

  • Suboptimal vitamin A status is linked to adverse outcomes in children with hemoglobin-SS disease (HbSS).
  • Standard Recommended Daily Allowance (RDA) of retinol is ineffective for improving vitamin A status in this population.

Purpose of the Study:

  • To compare vitamin A and nutritional status in children with HbSS versus healthy children.
  • To explore the impact of high-dose vitamin A supplementation on serum vitamin A status and other health parameters in children with HbSS.

Main Methods:

  • A single-center, randomized, blinded, dose-finding pilot study.
  • 22 children with HbSS were randomized to receive 3000 or 6000 IU/d of oral retinol for 8 weeks.
  • Mixed-effects linear regression was used to analyze associations between vitamin A dose, serum retinol, and outcomes.

Main Results:

  • Children with HbSS exhibited suboptimal serum retinol, growth, and nutritional status at baseline.
  • Vitamin A supplementation did not alter serum vitamin A levels.
  • Significant improvements were observed in fetal hemoglobin, mean corpuscular volume, mean corpuscular hemoglobin, and mean corpuscular hemoglobin concentration.

Conclusions:

  • High-dose vitamin A supplementation does not improve vitamin A status in children with HbSS.
  • Supplementation showed positive effects on hematologic parameters, including fetal hemoglobin and erythrocyte indices.
  • Mild improvements in growth and muscle function were noted, independent of hydroxyurea treatment.

More Related Videos

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload05:23

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

20.2K
Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

10.3K

Related Concept Videos

Bioavailability Study Design: Healthy Subjects Versus Patients01:15

Bioavailability Study Design: Healthy Subjects Versus Patients

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,...
115
Bioavailability Study Design: Single Versus Multiple Dose Studies01:11

Bioavailability Study Design: Single Versus Multiple Dose Studies

Bioavailability studies are essential for understanding how a drug is absorbed, distributed, metabolized, and excreted in the body. These studies assess the extent and rate at which the active pharmaceutical agent becomes available at the site of action. The design of bioavailability studies can involve single-dose or multiple-dose regimens, each with distinct advantages and limitations.Single-dose studies are the preferred approach due to their simplicity and reduced drug exposure for...
164