Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Pharmacologic Therapies for Sickle Cell Disease
source: Journal of Sickle Cell Disease and Hemoglobinopathies (JSCDH)
year: 2016
authors: Lanetta Bronte, Kenneth I. Ataga, Kenneth Ataga, Lakiea, Bailey, Iris Buchanan, Gregory Kato, Ify Osunkwo, Betty Pace
summary/abstract:Sickle cell disease (SCD) is a multisystem disease, associated with episodes of acute illness and progressive organ damage. Despite its low prevalence in the United States, SCD is one of the most common severe monogenic disorders worldwide, with an estimated 230,000 affected children born every year in sub-Saharan Africa. As a result of its multiple complications, SCD is associated with an overall decreased life expectancy. In addition, the economic burden of SCD is enormous – a consequence of recurrent and chronic SCD-related complications, frequent absences from school and work, and physical impairment limiting the ability to contribute to society.
Although there is an increased understanding of the pathophysiology of SCD, available pharmacologic treatments remain limited. The current approach to the management of SCD includes supportive treatments with folic acid; symptomatic treatment with analgesics, intravenous fluids, oxygen and red blood cell (RBC) transfusion; disease-modifying treatment with hydroxyurea and RBC transfusion; and curative therapy with bone marrow transplantation. Despite the success of bone marrow transplantation as a curative approach in SCD, this modality is limited by its high cost, decreased availability of suitable donors and toxicity.
read moreRelated Content
-
Experimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
-
American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Management of Acute and Chronic PainObjective: These evidence-based guideli...
-
Prevention of Conversion to Abnormal Transcranial Doppler With Hydroxyurea in Sickle Cell Anemia: A Phase III Intern...Children with sickle cell anemia (SCA) a...
-
Statement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
-
Bioverativ and Bicycle Therapeutics Enter into Strategic Research Collaboration to Develop Therapies for Hemophilia ...Bioverativ Inc. (NASDAQ: BIVV), a global...
-
Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Ga...Acetylon Pharmaceuticals, Inc., the lead...
-
Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease DesignationThe U.S. Food and Drug Administration (F...
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.