Practical guide for disease-modifying medication management of children and adolescents with sickle cell disease

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.