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- Title
Favorable outcome in children and adolescents with a high proportion of advanced phase disease using single/multiple autologous or matched/mismatched allogeneic stem cell transplantations.
- Authors
Niederwieser, C.; Starke, S.; Fischer, L.; Christiansen, H.; Krahl, R.; Al-Ali, H-K.; Cross, M.; Eisfeld, A-K.; Heyn, S.; Vucinic, V.; Franke, G-N.; Lange, T.; Pönisch, W.; Behre, G.; Beck, J.; Gruhn, B.; Ebell, W.; Körholz, D.; Wößmann, W.; Bader, P.
- Abstract
We determined the indication, outcome, and risk factors of single and multiple hematopoietic stem cell transplantation(s) (HSCT) in children and adolescents mostly with advanced disease. Forty-one out of 483 patients (8.5 %; median age 9 years) diagnosed at the University of Leipzig with hematological and oncological diseases required HSCT from 1999 to 2011. Patients had overall survival (OS) of 63 ± 10 and 63 ± 16 %, event-free survival (EFS) of 57 ± 10 and 42 ± 16 %, relapse incidence (RI) of 39 ± 10 and 44 ± 18 % and nonrelapse mortality (NRM) of 4 ± 4 and 13 ± 9 % at 10 years after one or more allogeneic and autologous HSCT, respectively. One patient in CR1 and five with advanced disease received two HSCT. Four of the six patients maintained/achieved CR for a median of 13 months. Three died of progression and one of NRM. Two patients had a third HSCT and one survived in CR +231 days after HSCT. Risk factors for OS and EFS were disease stage at HSCT and EBMT risk score. Center (pediatric or JACIE accredited pediatric/adult) was not a determinant for survival. Pediatric single and multiple HSCT are important curative approaches for high-risk malignant diseases with low NRM. Efforts to reduce high RI remain the major aim.
- Subjects
STEM cell transplantation research; JUVENILE diseases; GRAFT versus host disease; BLOOD diseases; CHILD mortality; PEDIATRIC surgery
- Publication
Annals of Hematology, 2016, Vol 95, Issue 3, p473
- ISSN
0939-5555
- Publication type
journal article
- DOI
10.1007/s00277-015-2569-7