Foell, Juergen and Schulte, Johannes H. and Pfirstinger, Beatrix and Troeger, Anja and Wolff, Daniel and Edinger, Matthias and Hofmann, Petra and Aslanidis, Charalampos and Lang, Peter and Holler, Ernst and Eggert, Angelika and Corbacioglu, Selim (2019) Haploidentical CD3 or alpha/beta T-cell depleted HSCT in advanced stage sickle cell disease. BONE MARROW TRANSPLANTATION, 54 (11). pp. 1859-1867. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Despite significant improvements in the supportive care, sickle cell disease (SCD) leads to significant morbidity and mortality. Allogeneic hematopoietic stem cell transplantation (HSCT), the only curative option, is limited due to matched donor availability. This could be met with T-cell-depleted haploidentical HSCT. Twenty advanced-stage SCD patients, median age 15 years, and 9 patients, median age 14 years, were transplanted with CD3/CD19- or TCR alpha beta/CD19-depleted grafts and from matched sibling donors (MSDs). The conditioning consisted of ATG, thiotepa, fludarabine, and treosulfan. The median follow-up in the T-haplo-HSCT and the MSD patients was 21 (9-62) and 25 (7-60) months, respectively. The OS in the T-haplo-HSCT and MSD was 90% and 100%, respectively. In the T-haplo-HSCT group, two patient succumbed to a CMV pneumonitis and a macrophage activation syndrome (MAS). One patient in the T-haplo-HSCT group requires renal replacement therapy because of BK virus nephritis. None developed grade III-IV acute GvHD. In the T-haplo-HSCT and in the MSD, 20% and 22%, respectively, developed a mild or moderate chronic GvHD. These results demonstrate the feasibility, safety, and efficacy of T-haplo-HSCT also for adult advanced stage SCD patients.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | MARROW-TRANSPLANTATION; CHILDREN; DONORS; THALASSEMIA; STRATEGIES; ALLOGRAFTS; MORTALITY; TRIAL; US; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation Medicine > Lehrstuhl für Klinische Chemie und Laboratoriumsmedizin |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 25 Mar 2020 10:21 |
| Last Modified: | 06 Apr 2020 06:07 |
| URI: | https://pred.uni-regensburg.de/id/eprint/25922 |
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