Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Beyond Hydroxyurea: New and Old Drugs in the Pipeline for Sickle Cell Disease
source: Blood
year: 2016
authors: Telen MJ
summary/abstract:Despite Food and Drug Administration (FDA) approval of hydroxyurea to reduce the frequency of vaso-occlusive episodes, sickle cell disease (SCD) has continued to be treated primarily with analgesics for pain relief. However, elucidation of the multiple pathophysiologic mechanisms leading to vaso-occlusion and tissue injury in SCD has now resulted in a burgeoning effort to identify new treatment modalities to prevent or ameliorate the consequences of the disease. Development of new drugs as well as investigation of drugs previously used in other settings have targeted cell adhesion, inflammatory pathways, upregulation of hemoglobin F, hemoglobin polymerization and sickling, coagulation, and platelet activation. Although these efforts have not yet yielded drugs ready for FDA approval, several early studies have been extremely encouraging. Moreover, the marked increase in clinical pharmaceutical research addressing SCD and the new and old drugs in the pipeline make it reasonable to expect that we will soon have new treatments for SCD.
organization: Division of Hematology, Department of Medicine, and Duke Comprehensive Sickle Cell Center, Duke University, Durham, NC, USADOI: 10.1182/blood-2015-09-618553
read more full text
+myBinderRelated Content
-
Sancilio Pharmaceuticals Company announces Altemia™ receives orphan drug designation from the European Medicines A...Sancilio Pharmaceuticals Company, Inc. (...
-
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approachesSickle cell disease is an inherited diso...
-
The Association Between Timely Opioid Administration and Hospitalization in Children With Sickle Cell Disease Presen...Objective: To evaluate the association ...
-
SUSTAIN Clinical Trial Results Show Crizanlizumab Reduced Sickle Cell–Related Pain CrisesResults from the Phase 2 SUSTAIN clinica...
-
New Guidelines for Managing Sickle Cell Disease Presented at ACP Internal Medicine MeetingNew guidelines for therapeutic strategie...
-
Pediatric sickle cell study stopped early due to positive resultsA national sickle cell disease study inv...
-
CRISPR could end sickle cell disease, but signing up black patients for clinical trials will be a hard sellThe first attempts to use a groundbreaki...
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
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.