Purpose: 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.
Methods: Forty-one out of 483 patients (8.5%; median age 9 years) diagnosed at the University of Leipzig with haematological and oncological diseases required HSCT from 1999 to 2011.
Results: 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 non-relapse mor-tality (NRM) of 4±4% and 13±9% at 10-years after one or more HSCT for allogeneic and autologous HSCT, respectively. One patient in complete remission (CR)1 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 (paediatric or JACIE accredited paediatric/adult) was not a determinant for survival.
Conclusion: Paediatric 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.
| Identifer | oai:union.ndltd.org:DRESDEN/oai:qucosa.de:bsz:15-qucosa-214903 |
| Date | 30 November 2016 |
| Creators | Niederwieser, Christian |
| Contributors | Universität Leipzig, Medizinische Fakultät, Prof. Dr. med Holger Christiansen, Prof. Dr. med. Wieland Kiess, Prof. Dr. med. Ulrich H. Thome |
| Publisher | Universitätsbibliothek Leipzig |
| Source Sets | Hochschulschriftenserver (HSSS) der SLUB Dresden |
| Language | English |
| Detected Language | English |
| Type | doc-type:doctoralThesis |
| Format | application/pdf |
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