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Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies
- Source :
- Bone Marrow Transplantation
- Publication Year :
- 2020
-
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/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.
- Subjects :
- 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
Subjects
Details
- Language :
- English
- Database :
- OpenAIRE
- Journal :
- Bone Marrow Transplantation
- Accession number :
- edsair.doi.dedup.....a007f5355a53d871e1c9b2db18af740d