Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Nonmyeloablative Matched Sibling Donor Hematopoietic Cell Transplantation in Children and Adolescents With Sickle Cell Disease
source: Biology of Blood and Marrow Transplantation
year: 2019
authors: Guilcher GMT, Monagel DA, Nettel-Aguirre A, Truong TH, Desai SJ, Bruce A, Shah RM, Leaker MT, Lewis VA
summary/abstract:Sickle cell disease is a potentially debilitating hemoglobinopathy associated with early mortality. The only established curative therapy is hematopoietic cell transplantation (HCT) with a matched sibling donor. The National Institutes of Health nonmyeloablative regimen of alemtuzumab/300 cGy total body irradiation and prolonged sirolimus exposure for graft-versus-host disease (GVHD) prophylaxis was administered to 16 children and adolescents. Infused products were unmanipulated granulocyte colony stimulating factor mobilized peripheral blood stem cells. All patients achieved mixed donor-recipient engraftment with no cases of secondary graft failure to date. Two patients have donor myeloid chimerism in the range of 30% to 40%. No sickling crises post-HCT have been observed. Event-free and overall survival rates are 100% with median follow-up of 19.5 months. No cases of GVHD have been observed. Sirolimus weaning was possible in all but one eligible patient to date. Ongoing follow-up and a larger prospective clinical trial are required to determine the long-term safety and efficacy of this regimen in children.
organization: University of Calgary, Canada; University of Alberta, CanadaDOI: 10.1016/j.bbmt.2019.02.011
read more
+myBinderRelated Content
-
Vanderbilt University Medical CenterThe Vanderbilt-Meharry-Matthew Walker Ce...
-
Orphan Drug Designation Granted for CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy for Sickle ...Global biotherapeutics leader CSL Behrin...
-
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...
-
Altered Functional Connectivity in Sickle Cell Disease Exists at Rest and During Acute Pain ChallengeOBJECTIVES: Sickle cell disease (SCD) is...
-
Pediatric sickle cell study stopped early due to positive resultsA national sickle cell disease study inv...
-
Comparing Sickle Cell Disease Acute Pain Management in the Pediatric and Adult Emergency DepartmentBackground: Vasoocclusive pain leading ...
-
Coronavirus Preparedness for People With Chronic Diseases – WebinarEvent Date: Mar. 13, 2020 Event...
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.