Radiation-sparing reduced-intensity unrelated umbilical cord blood transplantation for rare hematological disorders in children.
Akihisa SawadaMariko ShimizuMaho Koyama-SatoKohei HiguchiYosuke OkadaKimitoshi GotoShota InoueMasahiro YasuiMasami InouePublished in: International journal of hematology (2021)
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.
Keyphrases
- umbilical cord
- mesenchymal stem cells
- low dose
- bone marrow
- end stage renal disease
- newly diagnosed
- chronic kidney disease
- young adults
- ejection fraction
- high dose
- traumatic brain injury
- radiation induced
- prognostic factors
- free survival
- chronic myeloid leukemia
- acute myeloid leukemia
- stem cells
- stem cell transplantation
- acute lymphoblastic leukemia
- cord blood
- radiation therapy
- peritoneal dialysis