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Emerging therapies in sickle cell disease

Research output: Contribution to journalReview article

Amina Nardo-Marino, Valentine Brousse, David Rees

Original languageEnglish
Pages (from-to)149-172
Number of pages24
JournalBritish Journal of Haematology
Volume190
Issue number2
DOIs
Publication statusPublished - 1 Jul 2020

King's Authors

Abstract

Despite sickle cell disease (SCD) being the most common and severe inherited condition worldwide, therapeutic options are limited. To date, hydroxyurea remains the main treatment option in SCD. However, in the last decade the numbers of interventional clinical trials focussing on therapies for SCD have increased significantly. Many new drugs with various pharmacological targets have emerged and, although the majority have failed to show benefit in clinical trials, some have produced encouraging results. It seems probable that more drugs will soon become available for the treatment of SCD. Furthermore, promising clinical trials with improved outcomes have recently changed the perspective of curative therapies in SCD. Nevertheless, the application of novel therapeutic agents and potential curative treatments will most likely be limited to high-income countries and, thus, will remain unavailable for the majority of people with SCD in the foreseeable future.

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