Radiation-sparing reduced-intensity unrelated umbilical cord blood transplantation for rare hematological disorders in children
Autor: | Kohei Higuchi, Mariko Shimizu, Maho Koyama-Sato, Yosuke Okada, Masami Inoue, Shota Inoue, Kimitoshi Goto, Akihisa Sawada, Masahiro Yasui |
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Rok vydání: | 2021 |
Předmět: |
Male
medicine.medical_specialty Transplantation Conditioning Adolescent Cyclophosphamide Gastroenterology hemic and lymphatic diseases Internal medicine Humans Medicine Aplastic anemia Child Retrospective Studies Acute leukemia Leukemia Juvenile myelomonocytic leukemia business.industry Umbilical Cord Blood Transplantation Infant Hematology Bone Marrow Failure Disorders Total body irradiation Fetal Blood medicine.disease Fludarabine Regimen Child Preschool Myelodysplastic Syndromes Female Cord Blood Stem Cell Transplantation business Whole-Body Irradiation medicine.drug |
Zdroj: | International Journal of Hematology. 115:269-277 |
ISSN: | 1865-3774 0925-5710 |
Popis: | Graft failure is a major pitfall of unrelated umbilical cord blood transplantation (CBT) in children with rare hematological disorders other than acute leukemia, such as acquired and inherited bone marrow failure, myelodysplastic syndrome, juvenile myelomonocytic leukemia, and chronic myeloid leukemia. We developed a less-toxic conditioning regimen for CBT that achieves a higher rate of complete donor chimerism, and retrospectively compared it against two other conditioning regimens for CBT performed at our single institution. The engraftment rate with complete donor chimerism was 100% and 5-year event-free survival (5y-EFS) was 90.9% in patients using our latest regimen (n = 11) of reduced-intensity conditioning (RIC) containing fludarabine (Flu) 180 mg/m2, melphalan (MEL) 210 mg/m2, and low-dose rabbit anti-thymocyte globulin (LD-rATG) 2.5 mg/kg without irradiation (regimen C). Outcomes were better than in patients (n = 10) treated with previous regimens involving irradiation (5y-EFS 30.0%, p = 0.004): regimen A, consisting of myeloablative conditioning containing cyclophosphamide (CY) and total body irradiation (TBI) with 8-12 Gy, or regimen B, consisting of RIC with Flu, CY, horse ATG, and thoracoabdominal irradiation (TAI) with 6 Gy. In conclusion, Flu/MEL/LD-rATG (regimen C) without TBI/TAI may be preferable as RIC for unrelated CBT in children with rare hematological disorders. |
Databáze: | OpenAIRE |
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