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Updated: Jun 5, 2025
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Practical guide for disease-modifying medication management of children and adolescents with sickle cell disease
1Department of Pediatrics, The Research Institute at Nationwide Children's Hospital, Columbus, OH.
Insights
Newer medications for sickle cell disease (SCD) offer alternatives to hydroxyurea, but access and adherence challenges persist. This review provides guidance on optimizing treatment with these disease-modifying medications (DMMs) in real-world settings.
Area of Science:
- Hematology
- Pharmacology
- Pediatric Medicine
Background:
- Hydroxyurea has been the primary disease-modifying medication (DMM) for sickle cell disease (SCD).
- Since 2017, L-glutamine, voxelotor, and crizanlizumab have been approved as newer DMMs for pediatric SCD patients.
- Treatment barriers like access, cost, and adherence limit the effective use of these DMMs.
Purpose of the Study:
- To discuss available DMMs for SCD.
- To offer practical guidance on real-world DMM utilization.
- To consider patient preferences and unique DMM characteristics.
Main Methods:
- Review of published peer-reviewed studies.
- Analysis of real-world use and safety data for newer DMMs.
- Consideration of DMM tolerability, cost, and administration routes.
Main Results:
- Limited data exist on the real-world application and safety of newer SCD DMMs.
- No established guidelines exist for selecting between DMMs or for combination therapy.
- Patient preferences and individual DMM profiles are critical for treatment decisions.
Conclusions:
- Optimizing DMMs in SCD requires addressing access and adherence barriers.
- Evidence-based guidelines are needed for DMM selection, combination therapy, and transition to curative treatments.
- The withdrawal of voxelotor underscores the need for ongoing DMM development and research.
Abstract:
Hydroxyurea has historically been the sole disease-modifying medication (DMM) for sickle cell disease (SCD). However, 3 newer DMMs, L-glutamine, voxelotor, and crizanlizumab, were approved for children and adolescents with SCD since 2017. Despite their emergence, treatment barriers, including access, affordability, and nonadherence, limit the optimization of DMMs in the clinical setting. Furthermore, there is limited work outlining real-world use and safety of the newer DMMs, and no published guidelines advise how best to select between DMMs or to use multiple in combination. Meanwhile, each DMM is associated with unique characteristics, such as tolerability, cost, and route of administration, which must be considered when weighing these options with patients and families. This article discusses DMMs for SCD and offers practical guidance on using the available DMMs in real-world settings based on published peer-reviewed studies and considering patient preferences. The recent withdrawal of one of these DMMs (voxelotor) from the market highlights the need for additional DMMs and evidence-based practices for adding DMMs and when to progress towards curative therapies.
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