Importance:Neuroblastoma is the most common extracranial malignant solid tumor in children.Multidisciplinary care is critical to improving the survival of pediatric patients with neuroblastoma.Objective:To systematica...Importance:Neuroblastoma is the most common extracranial malignant solid tumor in children.Multidisciplinary care is critical to improving the survival of pediatric patients with neuroblastoma.Objective:To systematically summarize the clinical characteristics of children with neuroblastoma and evaluate their prognosis with multidisciplinary care provided in a single center.Methods:This retrospective study analyzed the clinical data of 1041 patients with neuroblastoma who were diagnosed,treated,and followed-up in the Hematology-Oncology Center of Beijing Children’s Hospital from 2007 to 2019.Results:The median age at diagnosis was 34 months;80.8%of the patients were younger than 5 years of age.Notably,243 patients(23.3%)were classified as low-risk,249 patients(23.9%)were classified as intermediate-risk,and 549(52.7%)were classified as high-risk.Furthermore,956 patients underwent surgical resections;986(94.7%)patients received chemotherapy;and 176 patients with high-risk neuroblastoma received hematopoietic stem cell transplantation.The 5-year event-free survival(EFS)rate was 91.3%and 5-year overall survival(OS)rate was 97.5%in low-risk group;in the intermediate-risk group,these rates were 85.1%and 96.7%,respectively,while they were 37.7%and 48.9%in the high-risk group(P<0.001 for both).The 5-year EFS and OS rates were significantly higher in patients diagnosed between 2015 and 2019 than in patients diagnosed between 2007 and 2014(P<0.001).In total,278 patients(26.7%)exhibited tumor relapse or progression;the median interval until relapse or progression was 14 months.Of the 233 patients who died,83%died of relapse or progression of neuroblastoma and 4.3%died of therapy-related complications.Interpretation:The 5-year OS rate was low in high-risk patients,compared with low-and intermediate-risk patients.Multidisciplinary care is critical for improvement of survival in pediatric patients with neuroblastoma.Additional treatment strategies should be sought to improve the prognosis of patients with high-risk neuroblast展开更多
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
Importance: Allogeneic hematopoietic stem cell transplantation (HSCT) is considered to be the only curative treatment for familial hemophagocytic lymphohistiocytosis (FHLH). Treatment of pediatric FHLH with reduced-in...Importance: Allogeneic hematopoietic stem cell transplantation (HSCT) is considered to be the only curative treatment for familial hemophagocytic lymphohistiocytosis (FHLH). Treatment of pediatric FHLH with reduced-intensity conditioning (RIC)-based haploidentical donor (HID) HSCT has been rarely reported. Objective: To investigate outcomes and adverse events in patients with FHLH who received HID-HSCT. Methods: We conducted a retrospective study of five patients, including three with mutations in PRF1 and two with XIAP deficiency. Four of the five donors were heterozygous for these mutations. The conditioning regimen included fludarabine, cyclophosphamide, and antithymocyte globulin, with or without low-dose irradiation. Unmanipulated mobilized bone marrow and peripheral blood stem cells were used as the grafts. results: All five patients were successfully engrafted. Four patients survived, and one patient died. All exhibited complete response (CR) after HSCT. All of the patients who survived exhibited CR to FHLH without severe regimen-related complications at a median of 29.5 months (range: 23–34 months) after HSCT. Four of the five patients had mixed donor chimerism. Three patients had 17% to 87% mixed donor chimerism but remained free of disease. Four patients received donor lymphocyte infusion (DLI), which improved the level of mixed donor chimerism. One patient experienced a decrease in donor chimerism to 1% and relapsed;Four patients developed acute graft-versus-host disease (GvHD) (grade I or II), and one patient developed grade IV GvHD. Interpretation: HID-HSCT with RIC can be considered for treatment for patients with FHLH, but the conditions and DLI regimens need to be optimized for long-term use, and more prospective studies should be conducted.展开更多
Introduction: Neuroblastoma (NB) is the most common extracranial solid tumor among children. The 5-year event-free survival rate for high-risk (HR) NB is still poor, especially for patients with advanced NB withMYCN g...Introduction: Neuroblastoma (NB) is the most common extracranial solid tumor among children. The 5-year event-free survival rate for high-risk (HR) NB is still poor, especially for patients with advanced NB withMYCN gene amplification. Chimeric antigen receptor T (CAR-T) cell therapy is a new treatment for HR-NB.Case presentation: A 55-month-old boy with stage IV HR-NB received 4th-generation CAR-T cells that target disialoganglioside GD2, as consolidation maintenance treatment after intensive chemotherapy, surgery, and autologous stem-cell transplantation. As of February 2019, his CAR-T follow-up time was 37.5 months, indicating prolonged survival. Cranial MRI and ultrasound showed no mass;123I-metaiodobenzylguanidine (123I-MIBG) scan was negative.Conclusion: GD2-CAR-T cells may be an effective treatment option for NB patients withMYCN amplification.展开更多
INTRODUCTIONTransplantation-associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic stem cell transplantation (HSCT) characterized by small vessel endothelial damage leading to thrombosis and...INTRODUCTIONTransplantation-associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic stem cell transplantation (HSCT) characterized by small vessel endothelial damage leading to thrombosis and fibrin deposition resulting in hemolytic anemia and thrombocytopenia. The severity of TA-TMA varies from mild self-limited disease to a fulminant variant resulting in death. Here, we review two rare cases and review the literature of TA-TMA.展开更多
基金Capital's Funds for Health Improvement and Research(No.CFH2018-2-2095)。
文摘Importance:Neuroblastoma is the most common extracranial malignant solid tumor in children.Multidisciplinary care is critical to improving the survival of pediatric patients with neuroblastoma.Objective:To systematically summarize the clinical characteristics of children with neuroblastoma and evaluate their prognosis with multidisciplinary care provided in a single center.Methods:This retrospective study analyzed the clinical data of 1041 patients with neuroblastoma who were diagnosed,treated,and followed-up in the Hematology-Oncology Center of Beijing Children’s Hospital from 2007 to 2019.Results:The median age at diagnosis was 34 months;80.8%of the patients were younger than 5 years of age.Notably,243 patients(23.3%)were classified as low-risk,249 patients(23.9%)were classified as intermediate-risk,and 549(52.7%)were classified as high-risk.Furthermore,956 patients underwent surgical resections;986(94.7%)patients received chemotherapy;and 176 patients with high-risk neuroblastoma received hematopoietic stem cell transplantation.The 5-year event-free survival(EFS)rate was 91.3%and 5-year overall survival(OS)rate was 97.5%in low-risk group;in the intermediate-risk group,these rates were 85.1%and 96.7%,respectively,while they were 37.7%and 48.9%in the high-risk group(P<0.001 for both).The 5-year EFS and OS rates were significantly higher in patients diagnosed between 2015 and 2019 than in patients diagnosed between 2007 and 2014(P<0.001).In total,278 patients(26.7%)exhibited tumor relapse or progression;the median interval until relapse or progression was 14 months.Of the 233 patients who died,83%died of relapse or progression of neuroblastoma and 4.3%died of therapy-related complications.Interpretation:The 5-year OS rate was low in high-risk patients,compared with low-and intermediate-risk patients.Multidisciplinary care is critical for improvement of survival in pediatric patients with neuroblastoma.Additional treatment strategies should be sought to improve the prognosis of patients with high-risk neuroblast
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Importance: Allogeneic hematopoietic stem cell transplantation (HSCT) is considered to be the only curative treatment for familial hemophagocytic lymphohistiocytosis (FHLH). Treatment of pediatric FHLH with reduced-intensity conditioning (RIC)-based haploidentical donor (HID) HSCT has been rarely reported. Objective: To investigate outcomes and adverse events in patients with FHLH who received HID-HSCT. Methods: We conducted a retrospective study of five patients, including three with mutations in PRF1 and two with XIAP deficiency. Four of the five donors were heterozygous for these mutations. The conditioning regimen included fludarabine, cyclophosphamide, and antithymocyte globulin, with or without low-dose irradiation. Unmanipulated mobilized bone marrow and peripheral blood stem cells were used as the grafts. results: All five patients were successfully engrafted. Four patients survived, and one patient died. All exhibited complete response (CR) after HSCT. All of the patients who survived exhibited CR to FHLH without severe regimen-related complications at a median of 29.5 months (range: 23–34 months) after HSCT. Four of the five patients had mixed donor chimerism. Three patients had 17% to 87% mixed donor chimerism but remained free of disease. Four patients received donor lymphocyte infusion (DLI), which improved the level of mixed donor chimerism. One patient experienced a decrease in donor chimerism to 1% and relapsed;Four patients developed acute graft-versus-host disease (GvHD) (grade I or II), and one patient developed grade IV GvHD. Interpretation: HID-HSCT with RIC can be considered for treatment for patients with FHLH, but the conditions and DLI regimens need to be optimized for long-term use, and more prospective studies should be conducted.
文摘Introduction: Neuroblastoma (NB) is the most common extracranial solid tumor among children. The 5-year event-free survival rate for high-risk (HR) NB is still poor, especially for patients with advanced NB withMYCN gene amplification. Chimeric antigen receptor T (CAR-T) cell therapy is a new treatment for HR-NB.Case presentation: A 55-month-old boy with stage IV HR-NB received 4th-generation CAR-T cells that target disialoganglioside GD2, as consolidation maintenance treatment after intensive chemotherapy, surgery, and autologous stem-cell transplantation. As of February 2019, his CAR-T follow-up time was 37.5 months, indicating prolonged survival. Cranial MRI and ultrasound showed no mass;123I-metaiodobenzylguanidine (123I-MIBG) scan was negative.Conclusion: GD2-CAR-T cells may be an effective treatment option for NB patients withMYCN amplification.
文摘INTRODUCTIONTransplantation-associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic stem cell transplantation (HSCT) characterized by small vessel endothelial damage leading to thrombosis and fibrin deposition resulting in hemolytic anemia and thrombocytopenia. The severity of TA-TMA varies from mild self-limited disease to a fulminant variant resulting in death. Here, we review two rare cases and review the literature of TA-TMA.
