Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies
| Autor: | Klaus Daniel Stachel, Katarzyna Drabko, Petr Sedlacek, Bernd Gruhn, Selim Corbacioglu, Jolanta Gozdzik, Krzysztof Kałwak, Peter Bader, Katharine Patrick, Franco Locatelli, Claudia Hemmelmann, Franca Fagioli, Joachim Baumgart, Paul G. Schlegel, Rita Beier, Jan Styczyński, Ann Kristin Möller, Johann Greil, Birgit Burkhardt, Ajay Vora, Karl Walter Sykora, Ingo Müller, Monika Mielcarek |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
Male
Oncology medicine.medical_specialty Transplantation Conditioning medicine.medical_treatment Medizin Graft vs Host Disease Hematopoietic stem cell transplantation ThioTEPA Treosulfan hematopoietic stem cell transplantation children hematologic neoplasms Article 03 medical and health sciences 0302 clinical medicine Internal medicine medicine Humans Leukaemia Cumulative incidence Child Busulfan Transplantation Juvenile myelomonocytic leukemia business.industry Haematopoietic stem cells Hematopoietic Stem Cell Transplantation Myeloid leukemia Hematology medicine.disease Fludarabine Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA Hematologic Neoplasms 030220 oncology & carcinogenesis HSCT Female business Thiotepa Vidarabine 030215 immunology medicine.drug |
| Zdroj: | Bone Marrow Transplantation |
| Popis: | 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/m2/day (7.7%), 12 g/m2/day (35.4%), or 14 g/m2/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. |
| Databáze: | OpenAIRE |
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