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
-
Comparative Cost Analysis of Therapy in Sickle Cell Anemia: Supportive Care Vs. Bone Marrow TransplantObjective: A comparative cost analysis o...
-
Does sickle cell disease increase risk of adverse outcomes following total hip and knee arthroplasty? A nationwide d...Sickle cell disease (SCD) is associated ...
-
Big Jump in Success for Sickle Cell TransplantsDoubling the dose of total-body irradiat...
-
MARAC Statement: Crizanlizumab (Adakveo)July 7, 2023 - SCDAA’s Medical and Res...
-
BCL11A-based gene therapy for sickle cell disease passes key preclinical testA precision-engineered gene therapy viru...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
-
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.