Trial reportJournal of clinical pharmacology2016
Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia.
Trial report in Journal of clinical pharmacology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
15 citing papers in PubMed, 1 synthesis or guideline pooled it, 30 citations in OpenAlex.
- Hydroxyurea (hydroxycarbamide) for sickle cell disease.The Cochrane database of systematic reviews · 2022Pooled it
- Hydroxycarbamide in children with sickle cell anaemia after first-dose vs. chronic therapy: pharmacokinetics and predictive models for drug exposure.British journal of clinical pharmacology · 2018Trial
- Physiologically Based Pharmacokinetic Modelling of Hydroxyurea in Patients with Sickle Cell Disease: A Special Focus on Lactating Women and Breastfed Infants to Inform Safe Dosing and Breastfeeding Strategies.Pharmaceuticals (Basel, Switzerland) · 2026Article
- Hydroxyurea pharmacokinetics in children with sickle cell anemia across different global populations.Blood advances · 2026Article
- Assessing multilevel barriers to hydroxyurea adherence in youth with sickle cell disease using pharmacy-based refill records.Pediatric blood & cancer · 2024Article
- Intensive hydroxyurea dosing in very young children with sickle cell anemia.Blood advances · 2023Article
- Efficacy, safety, and pharmacokinetics of a new, ready-to-use, liquid hydroxyurea in children with sickle cell anemia.Blood advances · 2023Article
- Effectiveness of Pharmacokinetic-Guided Hydroxyurea Dose Individualization in Patients with Sickle Cell Anemia: A Mini-Review.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Mathematical Modeling of Hydroxyurea Therapy in Individuals with Sickle Cell Disease.Pharmaceutics · 2022Article
- Compounded Nonsterile Preparations and FDA-Approved Commercially Available Liquid Products for Children: A North American Update.Pharmaceutics · 2022Article
- Article
- Tolerability and age-dependent toxicokinetics following perinatal hydroxyurea treatment in Sprague Dawley rats.Journal of applied toxicology : JAT · 2021Article
- Model-based dosing with concentration feedback as an integral part of personalized hydroxycarbamide management.British journal of clinical pharmacology · 2018Article
- A clinically meaningful fetal hemoglobin threshold for children with sickle cell anemia during hydroxyurea therapy.American journal of hematology · 2017Article
- Minireview: Prognostic factors and the response to hydroxurea treatment in sickle cell disease.Experimental biology and medicine (Maywood, N.J.) · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors at 14 institutions in 2 countries.
Funding
Abstract
Hydroxyurea (HU) is a crucial therapy for children with sickle cell anemia, but its off-label use is a barrier to widespread acceptance. We found HU exposure is not significantly altered by liquid vs capsule formulation, and weight-based dosing schemes provide consistent exposure. HU is recommended for all children starting as young as 9 months of age with sickle cell anemia (SCA; HbSS and HbSβspan(0) thalassemia); however; a paucity of pediatric data exists regarding the pharmacokinetics (PK) or the exposure-response relationship of HU. This trial aimed to characterize the PK of HU in children and to evaluate and compare the bioavailability of a liquid vs capsule formulation. This multicenter; prospective; open-label trial enrolled 39 children with SCA who provided 682 plasma samples for PK analysis following administration of HU. Noncompartmental and population PK models are described. We report that liquid and capsule formulations of HU are bioequivalent; weight-based dosing schemes provide consistent drug exposure; and age-based dosing schemes are unnecessary. These data support the use of liquid HU in children unable to swallow capsules and in those whose weight precludes the use of fixed capsule formulations. Taken with existing safety and efficacy literature; these findings should encourage the use of HU across the spectrum of age and weight in children with SCA; and they should facilitate the expanded use of HU as recommended in the National Heart; Lung; and Blood Institute guidelines for individuals with SCA.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.