Outcome of first or second transplantation using unrelated umbilical cord blood without ATG conditioning regimen for pediatric bone marrow failure disorders

Xia Chen; Fang Liu; Yuanyuan Ren; Luyang Zhang; Yang Wan; Wenyu Yang; Xiaojuan Chen; Li Zhang; Yao Zou; Yumei Chen; Xiaofan Zhu; Ye Guo

doi:10.1016/j.bcmd.2023.102793

Outcome of first or second transplantation using unrelated umbilical cord blood without ATG conditioning regimen for pediatric bone marrow failure disorders

Blood Cells Mol Dis. 2024 Jan:104:102793. doi: 10.1016/j.bcmd.2023.102793. Epub 2023 Aug 25.

Authors

Xia Chen¹, Fang Liu¹, Yuanyuan Ren¹, Luyang Zhang¹, Yang Wan¹, Wenyu Yang¹, Xiaojuan Chen¹, Li Zhang¹, Yao Zou¹, Yumei Chen¹, Xiaofan Zhu¹, Ye Guo²

Affiliations

¹ Department of Pediatrics, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
² Department of Pediatrics, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. Electronic address: guoye@ihcams.ac.cn.

PMID: 37659255
DOI: 10.1016/j.bcmd.2023.102793

Abstract

Background: Unrelated umbilical cord blood transplantation (UCBT) for bone marrow failure (BMF) disorders using conditioning regimens without Anti-Thymocyte Globulin (ATG) has been used as an alternative transplantation for emerging patients without matched-sibling donors. Experience with this transplant modality in children is limited, especially as a secondary treatment for transplant failure patients.

Procedure: We retrospectively reviewed 17 consecutive bone marrow failure patients who underwent unrelated umbilical cord blood transplantation in our center and received conditioning regimens of Total Body Irradiation (TBI) or Busulfan (BU) + Fludarabine (FLU) + Cyclophosphamide (CY).

Results: Among the 17 BMF patients, 15 patients were treated with first cord blood transplantation and another 2 with secondary cord blood transplantation because of graft failure after first haploidentical stem cell transplantation at days +38 and +82. All patients engrafted with a median donor cell chimerism of 50 % at days +7 (range, 16 %-99.95 %) and finally rose to 100 % at days +30. Median time to neutrophil engraftment was 19 days (range, 12-30) and time to platelet engraftment was 32 days (range, 18-61). Pre-engraftment syndrome (PES) was found in 16 patients (94.11 %, 16/17). Cumulative incidence of grades II to IV acute GVHD was 58.8 % (95 % CI: 32.7-84.9 %), and 17.6 % (95 % CI: 2.6-37.9 %) of patients developed chronic GVHD. The 3-year overall survival (OS) and failure-free survival (FFS) rates were 92.86 ± 6.88 %.

Conclusion: UCBT is an effective alternative treatment for bone marrow failure pediatric patients. TBI/BU + FLU + CY regimen ensure a high engraftment rate for unrelated umbilical cord blood transplantation, which overcomes the difficulty of graft failure. Secondary salvage use of cord blood transplantation may still be useful for patients who have failed after other transplantation.

Keywords: Bone marrow failure disorders; Graft failure; Unrelated umbilical cord blood transplantation.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Antilymphocyte Serum / therapeutic use
Bone Marrow Failure Disorders / therapy
Busulfan / therapeutic use
Child
Cyclophosphamide
Fetal Blood
Graft vs Host Disease* / etiology
Hematopoietic Stem Cell Transplantation*
Humans
Retrospective Studies
Transplantation Conditioning

Substances

Antilymphocyte Serum
Cyclophosphamide
Busulfan