Kalwak, Krzysztof and Mielcarek, Monika and Patrick, Katharine and Styczynski, Jan and Bader, Peter and Corbacioglu, Selim and Burkhardt, Birgit and Sykora, Karl Walter and Drabko, Katarzyna and Gozdzik, Jolanta and Fagioli, Franca and Greil, Johann and Gruhn, Bernd and Beier, Rita and Locatelli, Franco and Mueller, Ingo and Schlegel, Paul Gerhardt and Sedlacek, Petr and Stachel, Klaus Daniel and Hemmelmann, Claudia and Moeller, Ann-Kristin and Baumgart, Joachim and Vora, Ajay (2020) Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. BONE MARROW TRANSPLANTATION, 55 (10). pp. 1996-2007. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dose of 10 mg/m(2)/day (7.7%), 12 g/m(2)/day (35.4%), or 14 g/m(2)/day (56.9%) according to their individual body surface area in combination with fludarabine and thiotepa. The incidence of complete donor chimerism at day +28 was 98.4% with no primary and only one secondary graft failure. At 36 months, NRM was only 3.1%, while relapse incidence was 21.7%, and overall survival was 83.0%. The cumulative incidence of acute graft-vs.-host disease was 45.3% for grades I-IV and 26.6% for grades II-IV. At 36 months, 25.8% overall and 19.4% moderate/severe chronic graft-vs.-host disease were reported. These data confirm the safe and effective use of treosulfan-based conditioning in pediatric patients with hematological malignancies. Therefore, treosulfan/fludarabine/thiotepa can be recommended for myeloablative conditioning in children with hematological malignancies.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | JUVENILE MYELOMONOCYTIC LEUKEMIA; ACUTE MYELOID-LEUKEMIA; HAPLOIDENTICAL TRANSPLANTATION; MYELODYSPLASTIC SYNDROMES; WORKING PARTY; CHILDREN; REGIMEN; TOXICITY; DISEASE; HSCT; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 29 Mar 2021 11:50 |
| Last Modified: | 29 Mar 2021 11:50 |
| URI: | https://pred.uni-regensburg.de/id/eprint/44908 |
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