Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickle cell disease
source: British Journal of Haematology
year: 2015
authors: Strocchio L, Zecca M, Comoli P, Mina T, Giorgiani G, Giraldi E, Vinti L, Merli P, Regazzi M, Locatelli F
summary/abstract:Although allogeneic haematopoietic stem cell transplantation (HSCT) still represents the only consolidated possibility of cure for sickle cell disease (SCD) patients, its use has been limited by the risk of morbidity and mortality associated with conventional myeloablative therapy. The introduction of treosulfan to replace busulfan in conditioning regimens has recently been explored by virtue of its lower toxicity profile. We report our experience with a treosulfan/thiotepa/fludarabine conditioning for human leucocyte antigen (HLA)-matched sibling or unrelated donor-HSCT in 15 children with SCD, and compare patient outcomes with those of a historical cohort (15 patients) given a busulfan-based regimen. Engraftment was achieved in 28 out of 30 patients (93%), with one case of graft failure in either group. The conditioning regimen was well tolerated in both groups, with no cases of grade III-IV regimen-related toxicity. The 7-year overall survival (OS) and disease-free survival (DFS) for the whole cohort were 100% and 93%, respectively, with a 93% DFS in both busulfan and treosulfan groups. No SCD-related adverse events occurred after engraftment in patients with complete or mixed donor chimerism. This retrospective analysis suggests that a treosulfan-based conditioning regimen is able to ensure engraftment with excellent OS/DFS and low regimen-related toxicity in patients with SCD.
organization: Fondazione IRCCS Policlinico San Matteo, Pavia; Azienda Ospedaliera Papa Giovanni XXIII, Bergamo; IRCCS Ospedale Pediatrico Bambino Gesù, Roma; Università degli Studi di PaviaDOI: 10.1111/bjh.13352
read more full text
+myBinderRelated Content
-
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and TrialsWide interest in a CBS 60 Minutes story ...
-
Hydroxyurea for children with sickle cell anemia in Sub-Saharan AfricaBackground: Hydroxyurea is an effective...
-
Realizing Effectiveness Across Continents With Hydroxyurea: Enrollment and Baseline Characteristics of the Multicent...Despite its well-described safety and ef...
-
GBT announces positive top-line data from part A of the phase 3 HOPE study of Voxelotor in sickle cell diseaseGlobal Blood Therapeutics, Inc. today an...
-
Debbie’s Story on BBC2 Hospitalhttps://www.facebook.com/bbctwo/videos/1...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
-
ASH Clinical Practice Guidelines on Sickle Cell Disease (SCD)https://www.youtube.com/watch?v=tozUcTSN...
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.