Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children
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STUDY PROTOCOL
Open Access
Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia Emily R. Meier1, Susan E. Creary2,3, Matthew M. Heeney4,5, Min Dong6,7, Abena O. Appiah-Kubi8, Stephen C. Nelson9, Omar Niss7,10, Connie Piccone11, Maa-Ohui Quarmyne12, Charles T. Quinn7,10, Kay L. Saving13, John P. Scott14, Ravi Talati15, Teresa S. Latham10, Amanda Pfeiffer10, Lisa M. Shook7,10, Alexander A. Vinks6,7, Adam Lane7,10 and Patrick T. McGann7,10*
Abstract Background: Sickle cell disease (SCD) is a severe and devastating hematological disorder that affects over 100,000 persons in the USA and millions worldwide. Hydroxyurea is the primary disease-modifying therapy for the SCD, with proven benefits to reduce both short-term and long-term complications. Despite the well-described inter-patient variability in pharmacokinetics (PK), pharmacodynamics, and optimal dose, hydroxyurea is traditionally initiated at a weight-based dose with a subsequent conservative dose escalation strategy to avoid myelosuppression. Because the dose escalation process is time consuming and requires frequent laboratory checks, many providers default to a fixed dose, resulting in inadequate hydroxyurea exposure and suboptimal benefits for many patients. Results from a singlecenter trial of individualized, PK-guided dosing of hydroxyurea for children with SCD suggest that individualized dosing achieves the optimal dose more rapidly and provides superior clinical and laboratory benefits than traditional dosing strategies. However, it is not clear whether these results were due to individualized dosing, the young age that hydroxyurea treatment was initiated in the study, or both. The Hydroxyurea Optimization through Precision Study (HOPS) aims to validate the feasibility and benefits of this PK-guided dosing approach in a multi-center trial. (Continued on next page)
* Correspondence: [email protected] 7 University of Cincinnati College of Medicine, Cincinnati, OH, USA 10 Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Cohen Children’s Medical Center of New York, New Hyde Park, NY, USA Full list of author information is available at the end of the article © The Author(s). 2020 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly fr
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