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Reduced-dose of Posttransplant Cyclophosphamide and Cotransplantation of Peripheral Blood Stem Cells and Human Umbilical Cord-derived Mesenchymal Stem Cells in Severe Aplastic Anemia
Jian Zhou
Corresponding Author
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Background Posttransplant cyclophosphamide (PTCY) as graft-versus-host disease (GVHD) prophylaxis is an effective strategie for patients receiving matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT).
Methods We evaluated the effectiveness and safety of reduced-dose cyclophosphamide, 20 mg/kg for 13 patients in the MSD-HSCT group and 25 mg/kg for 22 patients in the haplo-HSCT group, on days +3 and +4 combined with cotransplantation of peripheral blood stem cells (PBSCs) and human umbilical cord-derived mesenchymal stem cells (UC-MSCs) for patients with severe aplastic anemia (SAA) retrospectively.
Results In the MSD-PTCY group, the times to neutrophil and platelet engraftment were significantly shorter than those in the MSD-control group (P<0.05), 11 (range 10 to 14) days and 12 (range 9 to 15) days. The cumulative incidence of acute GVHD (aGVHD) at day +100 (15.4%) was lower in the MSD-PTCY group than in the MSD-control group(P=0.050). No patient developed chronic GVHD (cGVHD). The 1-year overall survival (OS) and event-free survival (EFS) rates in the MSD-PTCY group were 100% and 92.3%. In the haplo-PTCY group, the times to neutrophil and platelet engraftment were significantly shorter than those in the haplo-control group (P<0.05), 12 (range 9 to 15) days and 11.5 (range 9 to 17) days. The cumulative incidences of aGVHD at day +100 and 1-year cGVHD in the haplo-PTCY group were 31.8% and 18.2%. The 1-year OS and EFS rates in the haplo-PTCY group were 81.8% and 66.9%.
Conclusions Reduced-dose PTCY and cotransplantation of PBSCs and UC-MSCs is feasible for patients with SAA, especially for overcoming the high incidences of aGVHD and cGVHD due to PBSCs.
Keywords
Reduced, PTCY, severe aplastic anemia, peripheral blood stem cells, umbilical cord-derived mesenchymal stem cells
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Research
Reduced-dose of Posttransplant Cyclophosphamide and Cotransplantation of Peripheral Blood Stem Cells and Human Umbilical Cord-derived Mesenchymal Stem Cells in Severe Aplastic Anemia
Jian Zhou
Corresponding Author
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
History
CURRENT STATUS: Posted
Version 1
Posted 11 Jun, 2020
No community comments so far
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Hematology
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background Posttransplant cyclophosphamide (PTCY) as graft-versus-host disease (GVHD) prophylaxis is an effective strategie for patients receiving matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT).
Methods We evaluated the effectiveness and safety of reduced-dose cyclophosphamide, 20 mg/kg for 13 patients in the MSD-HSCT group and 25 mg/kg for 22 patients in the haplo-HSCT group, on days +3 and +4 combined with cotransplantation of peripheral blood stem cells (PBSCs) and human umbilical cord-derived mesenchymal stem cells (UC-MSCs) for patients with severe aplastic anemia (SAA) retrospectively.
Results In the MSD-PTCY group, the times to neutrophil and platelet engraftment were significantly shorter than those in the MSD-control group (P<0.05), 11 (range 10 to 14) days and 12 (range 9 to 15) days. The cumulative incidence of acute GVHD (aGVHD) at day +100 (15.4%) was lower in the MSD-PTCY group than in the MSD-control group(P=0.050). No patient developed chronic GVHD (cGVHD). The 1-year overall survival (OS) and event-free survival (EFS) rates in the MSD-PTCY group were 100% and 92.3%. In the haplo-PTCY group, the times to neutrophil and platelet engraftment were significantly shorter than those in the haplo-control group (P<0.05), 12 (range 9 to 15) days and 11.5 (range 9 to 17) days. The cumulative incidences of aGVHD at day +100 and 1-year cGVHD in the haplo-PTCY group were 31.8% and 18.2%. The 1-year OS and EFS rates in the haplo-PTCY group were 81.8% and 66.9%.
Conclusions Reduced-dose PTCY and cotransplantation of PBSCs and UC-MSCs is feasible for patients with SAA, especially for overcoming the high incidences of aGVHD and cGVHD due to PBSCs.
Figure 1
Figure 2
Figure 3
Figure 4