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

Hydroxyurea has his tor i cally been the sole dis ease-mod i fy ing med i ca tion (DMM) for sickle cell dis ease (SCD). However, 3 newer DMMs, L-glu ta mine, voxelotor, and crizanlizumab, were approved for chil dren and ado les cents with SCD since 2017. Despite their emer gence, treat ment bar ri ers, includ ing access, afford abil ity, and nonadherence, limit the opti mi za tion of DMMs in the clin i cal set ting. Furthermore, there is lim ited work outlining real-world use and safety of the newer DMMs, and no published guide lines advise how best to select between DMMs or to use mul ti ple in com bi na tion. Meanwhile, each DMM is asso ci ated with unique char ac ter is tics, such as tol er a bil ity, cost, and route of admin is tra tion, which must be consid ered when weighing these options with patients and fam i lies. This arti cle discusses DMMs for SCD and offers prac ti cal guid ance on using the available DMMs in real-world set tings based on published peer-reviewed stud ies and con sid er ing patient pref er ences. 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.