Importance:Allogeneic hematopoietic stem cell transplantation(alloHSCT)is considered the only effective treatment for chronic active Epstein–Barr virus infection(CAEBV).The clinical efficacy and safety of allo-HSCT w...Importance:Allogeneic hematopoietic stem cell transplantation(alloHSCT)is considered the only effective treatment for chronic active Epstein–Barr virus infection(CAEBV).The clinical efficacy and safety of allo-HSCT with different conditioning regimens in children with CAEBV remain unclear.Objective:To evaluate the effectiveness and safety of allo-HSCT with the modified myeloablative conditioning(MAC)regimen for children with CAEBV and also the factors affecting the outcomes.Methods:We retrospectively analyzed children with CAEBV who underwent allo-HSCT with the modified MAC regimen at Beijing Children’s Hospital,Capital Medical University from October 2016 to June 2021.Data related to the clinical manifestations,engraftment,and outcome were extracted from the medical records.Results:The cohort comprised 41 patients(24 males,17 females)with a median transplantation age of 92.6(60.4,120.7)months and a median follow-up time of 28.2(15.3,40.2)months.Four patients(9.8%)died,among which three died from primary disease relapse,and one died from grade IV acute graft-versus-host diseases(aGVHD)after stopping treatment.The 3-year overall survival(OS)and 3-year event-free survival(EFS)rates were 88.8%±5.4%and 85.0%±5.7%,respectively.The 3-year OS and EFS did not significantly differ between the patients with hemophagocytic lymphohistiocytosis(HLH)and the patient without HLH(87.7%±6.8%vs.91.7%±8.0%,P=0.790;85.0%±6.9%vs.84.6%±10.0%,P=0.921),or among the patients with complete remission,partial remission,and activity disease before HSCT(all P>0.05).Multivariate analysis showed that grade III–IV aGVHD was a risk factor for mortality(Hazards ratio:11.65,95%confidence interval:1.00,136.06;P=0.050).Interpretation:Allo-HSCT with the modified MAC regimen is safe and effective for pediatric CAEBV.This treatment benefits patients with HLH or active disease.Patients with Grade III–IV aGVHD may be associated with worse outcomes.展开更多
Background:Allogeneic peripheral-blood stem-cell transplantation(SCT)from a matched related donor after myeloablative conditioning is the preferred curative treatment for patients with high-risk blood cancers.The comb...Background:Allogeneic peripheral-blood stem-cell transplantation(SCT)from a matched related donor after myeloablative conditioning is the preferred curative treatment for patients with high-risk blood cancers.The combination of a calcineurin inhibitor and an antimetabolite remains standard care for graft-versus-host disease(GVHD)prophylaxis in these patients.Data from two randomized trials have suggested that post-transplantation cyclophosphamide can reduce the risk of GVHD after SCT from a matched donor when it is added to or replaces the antimetabolite.展开更多
Since the first hematopoietic stem cell transplantation(HSCT)in 1956,HSCT has served as a cellular-level transplantation treatment that has cured tens of thousands of patients with hematologic diseases.[1]HSCT primari...Since the first hematopoietic stem cell transplantation(HSCT)in 1956,HSCT has served as a cellular-level transplantation treatment that has cured tens of thousands of patients with hematologic diseases.[1]HSCT primarily eradicates leukemia cells through myeloablative conditioning chemotherapy and the graft-versus-leukemia(GVL)effect,with subsequent developments such as donor lymphocyte infusion(DLI)and microtransplantation emerging as additional therapeutic methods.展开更多
基金Beijing Municipal Science&Technology Commission,Grant/Award Number:Z171100001017050National Science and Technology Key Projects,Grant/Award Number:2017ZX09304029。
文摘Importance:Allogeneic hematopoietic stem cell transplantation(alloHSCT)is considered the only effective treatment for chronic active Epstein–Barr virus infection(CAEBV).The clinical efficacy and safety of allo-HSCT with different conditioning regimens in children with CAEBV remain unclear.Objective:To evaluate the effectiveness and safety of allo-HSCT with the modified myeloablative conditioning(MAC)regimen for children with CAEBV and also the factors affecting the outcomes.Methods:We retrospectively analyzed children with CAEBV who underwent allo-HSCT with the modified MAC regimen at Beijing Children’s Hospital,Capital Medical University from October 2016 to June 2021.Data related to the clinical manifestations,engraftment,and outcome were extracted from the medical records.Results:The cohort comprised 41 patients(24 males,17 females)with a median transplantation age of 92.6(60.4,120.7)months and a median follow-up time of 28.2(15.3,40.2)months.Four patients(9.8%)died,among which three died from primary disease relapse,and one died from grade IV acute graft-versus-host diseases(aGVHD)after stopping treatment.The 3-year overall survival(OS)and 3-year event-free survival(EFS)rates were 88.8%±5.4%and 85.0%±5.7%,respectively.The 3-year OS and EFS did not significantly differ between the patients with hemophagocytic lymphohistiocytosis(HLH)and the patient without HLH(87.7%±6.8%vs.91.7%±8.0%,P=0.790;85.0%±6.9%vs.84.6%±10.0%,P=0.921),or among the patients with complete remission,partial remission,and activity disease before HSCT(all P>0.05).Multivariate analysis showed that grade III–IV aGVHD was a risk factor for mortality(Hazards ratio:11.65,95%confidence interval:1.00,136.06;P=0.050).Interpretation:Allo-HSCT with the modified MAC regimen is safe and effective for pediatric CAEBV.This treatment benefits patients with HLH or active disease.Patients with Grade III–IV aGVHD may be associated with worse outcomes.
文摘Background:Allogeneic peripheral-blood stem-cell transplantation(SCT)from a matched related donor after myeloablative conditioning is the preferred curative treatment for patients with high-risk blood cancers.The combination of a calcineurin inhibitor and an antimetabolite remains standard care for graft-versus-host disease(GVHD)prophylaxis in these patients.Data from two randomized trials have suggested that post-transplantation cyclophosphamide can reduce the risk of GVHD after SCT from a matched donor when it is added to or replaces the antimetabolite.
基金supported by grants from the National Natural Science Foundation of China(No.82341201)the National Key R&D Program of China(Nos.2022YFA1103300 and 2022YFA1103304)+1 种基金the Chongqing Municipal Natural Science Foundation General Project(No.2024NSCQ-MSX3299)the Special Project for Talent Construction in Xinqiao Hospital(No.2022XKRC001).
文摘Since the first hematopoietic stem cell transplantation(HSCT)in 1956,HSCT has served as a cellular-level transplantation treatment that has cured tens of thousands of patients with hematologic diseases.[1]HSCT primarily eradicates leukemia cells through myeloablative conditioning chemotherapy and the graft-versus-leukemia(GVL)effect,with subsequent developments such as donor lymphocyte infusion(DLI)and microtransplantation emerging as additional therapeutic methods.
