Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches
source: British Journal of Haematology
year: 2016
authors: Arnold SD, Bhatia M, Horan J, Krishnamurti L
summary/abstract:Sickle cell disease is an inherited disorder that affects over 5 million people worldwide. Current maintenance therapy has been successful in reducing complications and enhancing life expectancy; yet subclinical complications persist. To date, allogeneic haematopoietic stem cell transplant (HSCT) remains the only available curative therapy for sickle cell disease. With declining incidences of rejection and transplant- related mortality, disease-free survival after human leucocyte antigen-identical sibling transplant exceeds 90%. However, the majority of individuals with sickle cell disease do not have an human leucocyte antigen (HLA)-identical sibling; therefore, research is expanding to focus on new approaches to alternative donor transplant. Advances in supportive care and conditioning regimens have led to expansion of the pool of donors to unrelated donors and haploidentical donors. Challenges remain in improving the safety and efficacy of HSCT from alternate donors. Early results from gene therapy may provide another curative option in patients with sickle cell disease. These approaches show early promise, but larger, longitudinal studies are needed to better determine the optimal clinical circumstances for transplant in sickle cell disease.
organization: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta; Emory University School of Medicine, Atlanta; Columbia University Medical CenterDOI: 10.1111/bjh.14167
read more full text
+myBinderRelated Content
-
Treatments for priapism in boys and men with sickle cell diseaseBACKGROUND: Sickle cell disease compris...
-
Hydroxyurea improves lung function in children with sickle cell diseaseFor the first time, researchers were abl...
-
Association of Antibiotic Choice With Hospital Length of Stay and Risk Factors for Readmission in Patients With Sick...Study objective: We determine the assoc...
-
Sickle cell patient with severe anemia rapidly improves with voxelotor, case study showsGlobal Blood Therapeutics (GBT) reported...
-
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...
-
APS Scientific Meeting 2019The American Pain Society will hold its ...
-
A new Sickle Cell Disease Drug Holds Much Promise but Most Sufferers Won’t be Able to Afford itThe regulatory approval of a groundbreak...
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.