Impact of posttransplant cyclophosphamide on the outcome of patients undergoing unrelated single-unit umbilical cord blood transplantation for pediatric acute leukemia

Background Umbilical cord blood transplantation (UCBT) from unrelated donors is one of the successful treatments for acute leukemia in childhood. The most frequent side effect of UCBT is peri-engraftment syndrome (PES), which is directly associated with the greater prevalence of acute and chronic gr...

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Published in:BMC cancer 2022-11, Vol.22 (1), p.1-1190, Article 1190
Main Authors: Li, Xin-Yu, Zhan, Li-Ping, Liu, Dian-Dian, Han, Xia-Wei, Chen, Han, Wu, Zheng-Zhou, Wang, Yin, Que, Li-Ping, Wu, Xiao-Jun, Liu, Su, Wang, Kai-Mei, Huang, Shao-Liang, Fang, Jian-Pei, Huang, Ke, Xu, Hong-Gui
Format: Article
Language:English
Subjects:
Acute leukemia
Bacteremia
Care and treatment
Children
Cord blood
Cyclophosphamide
Cytokines
Cytomegalovirus
Disease prevention
Dosage and administration
Epstein-Barr virus
Fetal blood
Graft rejection
Graft versus host reaction
Grafts
Hemorrhage
Hemorrhagic cystitis
Immunosuppression
Infections
Leukemia
Leukemia in children
Life sciences
Mortality
Multivariate analysis
Neutrophils
Patient outcomes
Patients
Pediatrics
Posttransplant cyclophosphamide
Prevention
Prophylaxis
Remission
Remission (Medicine)
Risk factors
Sepsis
Statistical analysis
Stem cell transplantation
Transplantation
Umbilical cord
Umbilical cord blood transplantation
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Summary:Background Umbilical cord blood transplantation (UCBT) from unrelated donors is one of the successful treatments for acute leukemia in childhood. The most frequent side effect of UCBT is peri-engraftment syndrome (PES), which is directly associated with the greater prevalence of acute and chronic graft-versus-host-disease (aGvHD and cGvHD). In haploidentical stem cell transplantation, posttransplant cyclophosphamide (PTCY) has been demonstrated to be an effective method against GvHD. However, the effects of PTCY as a GvHD prophylactic in UCBT had not been investigated. This study aimed to evaluate the effects of PTCY on the outcomes of UCBT for pediatric acute leukemia. Methods This retrospective study included 52 children with acute leukemia who underwent unrelated single-unit UCBT after myeloablative conditioning regimens. The results from the PTCY and non-PTCY groups were compared. Results The incidence of transplantation-related mortality in non-PTCY and PTCY were 5% and 10% (p = 0.525), respectively. The incidence of relapse in non-PTCY and PTCY were 5% and 23% (p = 0.095), respectively. Second complete remission status (CR2) was an independent risk factor for relapse-free survival (hazard ratio = 9.782, p = 0.001). The odds ratio for sepsis or bacteremia incidence was significantly greater in the PTCY group (9.524, p = 0.017). PTCY group had increased rates of cytomegalovirus activity and fungal infection. The incidence of PES, aGvHD, cGvHD, and hemorrhagic cystitis in the PTCY group was lower than that in the non-PTCY group, although it was not significantly different. Additionally, higher doses of PTCY (29 mg/kg and 40 mg/kg) were associated with lower incidences of aGvHD and severe GvHD (65% and 29%, respectively) than lower doses (93% and 57%, respectively). Engraftment time and graft failure incidence were similar across groups. Conclusion The results support the safety and efficiency of PTCY as part of PES controlling and GvHD prophylaxis in single-unit UCBT for children with acute leukemia. A PTCY dosage of 29 mg/kg to 40 mg/kg appears to be more effective in GvHD prophylaxis for UCBT patients. Keywords: Umbilical cord blood transplantation, Acute leukemia, Posttransplant cyclophosphamide
ISSN:1471-2407
1471-2407
DOI:10.1186/s12885-022-10309-9