Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Emerging Pharmacotherapeutic Approaches for the Management of Sickle Cell Disease
source: Expert Opinion on Pharmacotherapy
year: 2019
authors: Torres L, Conran N
summary/abstract:Introduction:
Sickle cell disease (SCD) is an inherited disease with lifelong morbidity, whose complications include frequent acute painful vaso-occlusive episodes (VOEs) that often require hospitalization. The only pharmacotherapy currently in regular use for SCD management is hydroxyurea (hydroxycarbamide).
Areas Covered:
We review recent advances in pharmacotherapy for SCD and summarize promising synthetic agents that are in late-stage development (phase 3) for SCD.
Expert Opinion:
Emerging SCD therapies have been developed to target specific pathophysiological mechanisms of the disease, as either preventative or abortive approaches to VOEs. Continuous-use pharmacotherapeutics in late-phase development for VOE prevention include voxelotor (GBT440), which elevates hemoglobin oxygenation, and prasugrel, a platelet activation inhibitor. However, at least in the near future, it is probable that biological molecules will play a primary role in SCD preventative therapy; in combination with hydroxyurea, crizanlizumab, an anti-P-selectin monoclonal antibody, appears to reduce VOE frequency, while L-glutamine was the first substance licensed by the FDA for use in SCD in 20 years. Synthetic drugs, however, may represent key approaches for the management of individuals upon hospitalization for VOE, a major challenge for SCD. For example, rivipansel (GMI-1070), a pan-selectin inhibitor, has shown encouraging effects on hospitalization time and opioid use.
DOI: 10.1080/14656566.2018.1548610
read more
+myBinderRelated Content
-
Elliott Vichinsky, MDElliott Vichinsky, MD diagnoses and trea...
-
Perceptions of US Adolescents and Adults With Sickle Cell Disease on Their Quality of CareImportance: Sickle cell disease (SCD) i...
-
APHON/ASPHO Policy Statement for the Transition of Patients With Sickle Cell Disease From Pediatric to Adult Health ...With advances in medical care, the major...
-
Marsha Treadwell, PhDMarsha J. Treadwell, PhD, is a clinical ...
-
Listening to Sickle Cell Patients is Crucial During a CrisisI recently was admitted to the hospital ...
-
CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
-
Diggs-Kraus Sickle Cell CenterThe Diggs-Kraus Sickle Cell Center at Re...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.