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1.
Medicine (Baltimore) ; 98(43): e17572, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31651858

RESUMO

RATIONALE: Rhabdomyosarcoma (RMS) is a common soft tissue sarcoma in children with high malignancy. The prognosis of refractory recurrent RMS is extremely poor, and the 5-year survival rate is less than 20%. PATIENT CONCERNS: We reported a 2-year-old male patient with RMS who underwent 3 operations and 2 recurrences while being treated with regular multidisciplinary therapy. DIAGNOSES: A diagnosis of embryonal rhabdomyosarcoma with primary bladder (IIIa, TNM stage 2, and medium risk group) was made. INTERVENTIONS: After repeated recurrence, the patient was treated with chimeric antigen receptor T (CAR-T) cells, which had a safety mechanism and specifically bound the CD56 antigen in the fourth generation. OUTCOMES: The process of CAR-T cell transfusion was smooth, and there were no significant cytokine release syndrome manifestations after reinfusion. The patient was in complete remission at last follow-up visit after 3.5 years. CONCLUSION: CD56-CAR-T cell therapy is a safe and effective approach and may be an option for children with solid tumors who are nonresponsive to conventional radiotherapy and chemotherapy, or are unsuitable for hematopoietic stem cell transplantation.


Assuntos
Antígeno CD56/imunologia , Imunoterapia Adotiva/métodos , Recidiva Local de Neoplasia/terapia , Receptores de Antígenos Quiméricos/uso terapêutico , Rabdomiossarcoma/terapia , Neoplasias da Bexiga Urinária/terapia , Pré-Escolar , Seguimentos , Humanos , Masculino , Indução de Remissão , Rabdomiossarcoma/patologia , Neoplasias da Bexiga Urinária/patologia
2.
Medicine (Baltimore) ; 98(39): e17307, 2019 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-31574859

RESUMO

To investigate the cognitive and psychological outcomes of pediatric allogeneic HSCT survivors in China.A total of 135 3 to 18 years old children and adolescents who underwent allo-HSCT and survived at least 3 months post-HSCT were recruited and completed the assessments. Cognitive and psychological functions were assessed via age-appropriate standardized measures. Clinical information was extracted from the medical records.Forty one 3 to 6 years old children completed Psychological Questionnaires for 3 to 6 years Children. The scores of 21(51.2%) children in cognitive development dimension, 18(43.9%) in motor development dimension, 16(39.0%) in language development and social development dimension, 15(36.6%) in emotion and will dimension and 14(34.1%) in living habits dimension were less than the standard. Fifty six 8 to 16 years old children and adolescents completed the Depression Self-rating Scale for Children and 9 (16.1%) of these met the criteria of depression. Sixty nine 7 to 16 years old children and adolescents completed the screening for Child Anxiety Related Disorders and 7 (10.1%) of these met the criteria of anxiety, especially social phobia and school phobia. Eighty nine 6 to 18 years old children and adolescents completed the Symptom Checklist-90 and 43.8% to 77.5% of these experienced mild symptoms like obsession-compulsion (77.5%), hostility (64%), and interpersonal sensitivity (60.7%). Children treated with total body irradiation (TBI) showed more cognitive impairments like motor deficits than those without TBI. Also older children and adolescents had more symptoms like psychoticism.These findings demonstrated cognitive and psychological late effects of pediatric allo-HSCT survivors in a single center in China and highlighted that the survivors conditioned with TBI had more cognitive impairments and older children and adolescents had more symptoms. Early intervention in these children and adolescents might minimize the cognitive losses and psychological effects.


Assuntos
Ansiedade , Disfunção Cognitiva , Depressão , Transplante de Células-Tronco Hematopoéticas , Complicações Pós-Operatórias , Qualidade de Vida , Adolescente , Ansiedade/diagnóstico , Ansiedade/etiologia , Ansiedade/prevenção & controle , Criança , Pré-Escolar , China/epidemiologia , Cognição , Disfunção Cognitiva/diagnóstico , Disfunção Cognitiva/etiologia , Disfunção Cognitiva/prevenção & controle , Depressão/diagnóstico , Depressão/etiologia , Depressão/prevenção & controle , Intervenção Médica Precoce/métodos , Feminino , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Masculino , Destreza Motora , Complicações Pós-Operatórias/diagnóstico , Complicações Pós-Operatórias/prevenção & controle , Técnicas Psicológicas , Sobreviventes/psicologia
3.
Zhongguo Shi Yan Xue Ye Xue Za Zhi ; 27(2): 573-579, 2019 Apr.
Artigo em Chinês | MEDLINE | ID: mdl-30998173

RESUMO

OBJECTIVE: To study the association between IL-10 gene-592(C→A) (rs1800872) single nucleotide polymorphism (SNP) and the graft versus host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children. METHODS: Ninety-seven childhood patients and seventy-one donors in the Hematology Oncology Center of Beijing Children's Hospital from Jan 2011 to Jul 2017 were enrolled in this study. The genomic DNA was extracted from peripheral blood cells and the SNP genotype was analyzed using TaqMan SNP genotyping assay. RESULTS: In malignant patients with AA genotype, the incidence of Ⅱ-Ⅳ grade acute GVHD (aGVHD) was lower than that in patients with AC and CC genotype (9.1% vs 43.5%) (P<0.01), and the gastrointestinal aGVHD rate was also lower (9.1% vs 39.1%) (P<0.05). There's no significant association between patients' genotype and Ⅱ-Ⅳ grade aGVHD in total patients and non-malignant patients. Also, the genotype in patients did not corelate with chronic GVHD (cGVHD) and 1 year transplantation-related mortality (TRM). In cases who received HSCT of donors with AA genotype, the liver aGVHD rate was higher than that in cases who received HSCT of donors with AC and CC genotype (23.1% vs 0.0%) (P<0.05), but the genotype in donors did not correlate with Ⅱ-Ⅳ grade aGVHD, cGVHD and 1 year TRM. CONCLUSION: AA genotype in the IL-10 gene-592 (C→A) (rs1800872) single nucleotide polymorphism in patients protects pediatric malignant patients against Ⅱ-Ⅳ grade aGVHD and gastrointestinal aGVHD after allo-HSCT. AA genotype in donors is a risk factor for liver aGVHD after allo-HSCT in non-malignant disease.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Interleucina-10/genética , Criança , Humanos , Polimorfismo de Nucleotídeo Único , Doadores de Tecidos
4.
J Pediatr Hematol Oncol ; 41(1): 38-41, 2019 01.
Artigo em Inglês | MEDLINE | ID: mdl-30080752

RESUMO

The delay in platelet recovery after hematopoietic stem cell transplantation (HSCT) is closely related to the overall survival rate of transplanted children. The use of platelet-producing agents such as eltrombopag and romiplostim has made great progress in treating diseases such as immune thrombocytopenia and aplastic anemia. However, the use of such drugs in patients with thrombocytopenia after transplantation, especially in children, is rare. This study aimed to report eltrombopag treatment for 3 children with primary platelet engraftment failure and secondary thrombocytopenia after allogeneic HSCT. Of these patients, 2 had platelets stabilized at ≥50×10/L after eltrombopag treatment and subsequent withdrawal of eltrombopag. All 3 patients showed no clear adverse reactions. The results indicated a wide application prospect of eltrombopag treatment in children with thrombocytopenia after allogeneic HSCT.


Assuntos
Benzoatos/administração & dosagem , Transplante de Células-Tronco Hematopoéticas , Hidrazinas/administração & dosagem , Pirazóis/administração & dosagem , Recuperação de Função Fisiológica/efeitos dos fármacos , Trombocitopenia , Adolescente , Aloenxertos , Anemia Aplástica/sangue , Anemia Aplástica/terapia , Criança , Doença de Gaucher/sangue , Doença de Gaucher/terapia , Humanos , Masculino , Contagem de Plaquetas , Receptores Fc/administração & dosagem , Proteínas Recombinantes de Fusão/administração & dosagem , Trombocitopenia/sangue , Trombocitopenia/tratamento farmacológico , Trombocitopenia/etiologia , Trombopoetina/administração & dosagem
5.
J Pediatr Ophthalmol Strabismus ; 56(1): 35-42, 2019 Jan 23.
Artigo em Inglês | MEDLINE | ID: mdl-30371911

RESUMO

PURPOSE: To investigate the difference in the optic canal diameter between children with autosomal recessive malignant infantile osteopetrosis and normal children, and to assess the influence of hematopoietic stem cell transplantation on the optic canal diameter. METHODS: Twenty pediatric patients with malignant infantile osteopetrosis and 22 normal control children were included in this study. Eleven patients with malignant infantile osteopetrosis underwent hematopoietic stem cell transplantation. The measurements included optical canal diameter and flash visual evoked potential. Comparisons of these measurements between patients with malignant infantile osteopetrosis and normal controls as well as before and after hematopoietic stem cell transplantation were performed. The correlation between age and optic canal diameter was analyzed using Pearson correlation analysis. RESULTS: The mean optic canal diameter before hematopoietic stem cell transplantation was 1.65 ± 0.54 mm in patients with malignant infantile osteopetrosis and 3.38 ± 0.60 mm in the control group (P < .001). The mean optic canal diameter after hematopoietic stem cell transplantation was 2.72 ± 0.66 mm, which was significantly different from the pre-transplantation measurement (P < .001). The P2 latency for the flash visual evoked potential after hematopoietic stem cell transplantation (152.3 ± 36.4 ms) was significantly less than that before transplantation (165.5 ± 27.7 ms; P = .051). Pearson correlation analysis revealed a significant correlation between age and optic canal diameter (r = 0.722, P < .001). CONCLUSIONS: The optic canal was narrower in pediatric patients with malignant infantile osteopetrosis than in age-matched normal controls. This condition can be relieved through hematopoietic stem cell transplantation and the impaired conductibility of the optic nerve can be improved in some cases. [J Pediatr Ophthalmol Strabismus. 2019;56(1):35-42.].


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Nervo Óptico/diagnóstico por imagem , Osteopetrose/congênito , Baixa Visão/cirurgia , Acuidade Visual , Pré-Escolar , Potenciais Evocados Visuais , Feminino , Seguimentos , Humanos , Imagem Tridimensional , Lactente , Recém-Nascido , Masculino , Osteopetrose/complicações , Osteopetrose/diagnóstico , Osteopetrose/cirurgia , Estudos Retrospectivos , Tomografia Computadorizada por Raios X , Baixa Visão/diagnóstico , Baixa Visão/etiologia
6.
Zhongguo Shi Yan Xue Ye Xue Za Zhi ; 26(6): 1831-1835, 2018 Dec.
Artigo em Chinês | MEDLINE | ID: mdl-30501729

RESUMO

Transplantation-associated thrombotic microangiopathy (TA-TMA) is one of the fatal complications of hematopoietic stem cell transplantation(HSCT). The pathogenesis of TA-TMA has not been fully elucidated. The latest researches show that the abnormal activation of the complement system may lead to widespread endothelial injury which may play an important role in the pathogenesis of this disease. Incontrotable hypertension, proteinuria, increase of soluble C5b-9 concentration and early pericardial effusion are the risk factors of TA-TMA . In this review, the latest advances of pathogenesis, early diagnosis, treatment and other aspects of the progress of TA-TMA are summarized, so as to provide new ideas to early diagnosis and treatment in TA-TMA.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Microangiopatias Trombóticas , Doença Aguda , Humanos , Imunofenotipagem , Leucemia-Linfoma Linfoblástico de Células Precursoras
7.
Pediatr Transplant ; 22(5): e13204, 2018 08.
Artigo em Inglês | MEDLINE | ID: mdl-29744996

RESUMO

For young patients, HLA-MRD HSCT is the first-line treatment of SAA. However, due to China's birth control policy, few patients could find suitable sibling donors and HLA-MUD. More and more transplantation centers have used Haplo-D as the donor source for young adult and pediatric patients. However, studies with larger amount of pediatric patients are rare. We retrospectively analyzed the data of children with AA who were treated with allogeneic HSCT and compared the therapeutic efficacy of Haplo-HSCT and MRD/MUD group. A total of 62 patients were enrolled. Implantation was successfully performed in 58 patients. There was no significant difference in the time for reconstruction of hematopoietic function between patients in the two groups. Thirty-two had grade I-IV aGVHD with incidence of 51.61%. The incidence of aGVHD was 79.41% for patients in the Haplo-HSCT, significantly higher than that of 17.86% for patients in the MRD/MUD group (P < .01). However, the incidence of cGVHD was not significantly different between patients in the two groups (26.47% vs 10.71%, P = .09), the incidence of CMV infection was 28.57% and 52.94% for patients in the MRD/MUD and Haplo group, respectively, showing no significant difference (P = .053). The incidence of EBV infection was 47.06% for patients in the Haplo group and 28.57% for patients in the MRD/MUD group, showing no significant difference (P = .11). However, the 3- and 5-year cumulative OS and FFS rates showed statistically significant difference in the two groups, P = .012 and .045, respectively. Compared to Haplo-HSCT, MRD/MUD is more economic. In this study, we achieved good Haplo transplantation results. The incidences of cGVHD and CMV/EBV were not significantly different between Haplo group and MRD/MUD group. Although OS and FFS of the Haplo group were not as good as those of the MRD/MUD group, it is still acceptable as an alternative treatment under emergency.


Assuntos
Anemia Aplástica/terapia , Transplante de Células-Tronco Hematopoéticas/métodos , Adolescente , Anemia Aplástica/mortalidade , Criança , Pré-Escolar , China , Feminino , Seguimentos , Humanos , Lactente , Masculino , Estudos Retrospectivos , Irmãos , Transplante Haploidêntico , Resultado do Tratamento , Doadores não Relacionados
8.
World J Pediatr ; 14(1): 92-99, 2018 02.
Artigo em Inglês | MEDLINE | ID: mdl-29511925

RESUMO

BACKGROUND: ß-Thalassemia major (ß-TM) has become a public health problem in mainland China. Hematopoietic stem cell transplantation (HSCT) has remained the only cure for ß-TM in mainland China since 1998. METHODS: This multicenter retrospective study provides a comprehensive review of the outcomes of 50 pediatric patients with ß-TM who received HSCT between 1998 and 2009 at five centers in mainland China. Both related (n = 35) and unrelated donors (n = 15) with complete human leukocyte antigen matches were included. The stem cell sources included bone marrow (BM), peripheral blood stem cells, umbilical cord blood (UCB) and a combination of BM and UCB or a combination of BM and peripheral blood stem cells from a single sibling donor. RESULTS: The probabilities of 5-year overall survival (OS) and thalassemia-free survival (TFS) after the first HSCT were 83.1 and 67.3%, respectively. Graft failure (GF) occurred in 17 patients. Univariate analyses showed that umbilical cord blood transplantation (UCBT) was one of the potential risk factors for decreased OS (P = 0.051), and that UCBT (P = 0.002) was potentially related to TFS. GF incidence was distinct between the UCBT and non-UCBT groups (P = 0.004). Four cases of UCB-BM combined transplantation led to decreased risks of mortality and recurrence. In the UCBT group, related donor transplantation produced more favorable results than unrelated donor transplantation in OS (P = 0.009) but not in TFS (P = 0.217). CONCLUSIONS: GF was the primary cause of UCBT failure. Though UCBT from related donors was not favorable, the combined transplantation of UCB and BM could improve the prognosis of UCBT.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Transplante de Células-Tronco Hematopoéticas/métodos , Talassemia beta/cirurgia , Adolescente , Análise de Variância , Criança , Pré-Escolar , China/epidemiologia , Estudos de Coortes , Intervalos de Confiança , Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/epidemiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Estimativa de Kaplan-Meier , Masculino , Estudos Retrospectivos , Medição de Risco , Índice de Gravidade de Doença , Taxa de Sobrevida , Resultado do Tratamento , Talassemia beta/diagnóstico , Talassemia beta/mortalidade
9.
Pediatr Transplant ; 22(1)2018 02.
Artigo em Inglês | MEDLINE | ID: mdl-29239497

RESUMO

This multicenter retrospective study included 184 children with malignant and non-malignant diseases who underwent UCBT between January 1998 and August 2012. The malignant disease group included 101 children with ALL, AML, CML, JMML, and MDS, and the non-malignant disease group included 83 children with PID, ß-thalassemia, IMD BMF, and HLH. The median duration to neutrophil and platelet engraftment was 16 and 35 days in the malignant disease group vs 15 and 38 days in the non-malignant disease group. The cumulative incidence of grade II-IV aGVHD and cGVHD was 25.6% and 13.5% in the malignant disease group vs 19.7% and 11.1% in the non-malignant disease group, respectively. The median duration and cumulative incidence of neutrophil and platelet engraftment, and the cumulative incidence of grade II-IV aGVHD and cGVHD were similar between the two groups. Of the 184 pediatric patients, 114 patients survived during a median follow-up period of 14 months (range 4-138). The 5-year OS and DFS were not statistically different between the two groups (56.3% and 46.1% in malignant disease group vs 68.5% and 52.8% in non-malignant disease group). The above results indicate that UCB is a viable source for HSCT for children with malignant or non-malignant diseases, especially in urgent cases.


Assuntos
Doenças da Medula Óssea/terapia , Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Síndromes de Imunodeficiência/terapia , Leucemia/terapia , Doenças Linfáticas/terapia , Doenças Metabólicas/terapia , Doadores não Relacionados , Adolescente , Doenças da Medula Óssea/mortalidade , Criança , Pré-Escolar , China , Transplante de Células-Tronco de Sangue do Cordão Umbilical/mortalidade , Feminino , Seguimentos , Humanos , Síndromes de Imunodeficiência/mortalidade , Lactente , Recém-Nascido , Leucemia/mortalidade , Doenças Linfáticas/mortalidade , Masculino , Doenças Metabólicas/mortalidade , Estudos Retrospectivos , Resultado do Tratamento
10.
Health Qual Life Outcomes ; 14(1): 142, 2016 Oct 05.
Artigo em Inglês | MEDLINE | ID: mdl-27716318

RESUMO

BACKGROUND: Long-term health-related quality of life (HRQoL) of pediatric patients after hematopoietic stem cell transplantation (HSCT) is increasingly studied worldwide. However, few studies have been performed in China, where no uniform scale is available; the PedsQL™ Cancer Module 3.0 Chinese Mandarin version has been used to evaluate HRQoL of patients after HSCT in China. This study aimed to assess the reliability and validity of the Chinese Mandarin version of PedsQL™ 3.0 Transplant Module. METHODS: Patients between 2 and 18 years old, who underwent HSCT from January 2006 to June 2014, were recruited in Beijing Children's Hospital affiliated to Capital Medical University, the First Affiliated Hospital of Southern Medical University and Beijing Daopei Hospital. 207 parent reports and 182 child self-reports of the PedsQL™ 3.0 Transplant Module Chinese Mandarin version were assigned, of which 362 were returned. RESULTS: No missing item response was observed in the returned reports. Cronbach's alpha coefficient exceeded 0.7 in total scale and every dimension. The intraclass correlation coefficient exceeded 0.8 in all dimensions of child self-reports and parent reports. Spearman's rank correlation coefficients of items and their respective dimensions were 0.6-0.94 in parent reports, and 0.62-0.93 in child self-reports, while a weak association was found between the items and other dimensions. Exploratory factor analysis indicated a good extraction effect, and construct validity of the scale was >60 %. CONCLUSIONS: The Chinese Mandarin version of PedsQL™ 3.0 Transplant Module has good feasibility, reliability and validity. Its use may help improve the HRQoL of children after HSCT in China.


Assuntos
Transplante de Células-Tronco Hematopoéticas/psicologia , Neoplasias/psicologia , Qualidade de Vida , Adolescente , Grupo com Ancestrais do Continente Asiático/psicologia , Criança , Pré-Escolar , China , Análise Fatorial , Feminino , Humanos , Linguagem , Masculino , Neoplasias/terapia , Reprodutibilidade dos Testes , Inquéritos e Questionários , Traduções
11.
Biol Blood Marrow Transplant ; 22(11): 2104-2108, 2016 11.
Artigo em Inglês | MEDLINE | ID: mdl-27555533

RESUMO

We investigated the efficacy of allogeneic hematopoietic stem cell transplantation (alloHSCT) in pediatric patients with mucopolysaccharidosis (MPS). A retrospective analysis of transplantation data from 34 cases of MPS from the China Children Transplant Group, treated between December 2004 and September 2015, was conducted. Among the 34 cases, 12 cases were type I, 12 were type II, 4 were type IV, 4 were type VI, and 2 were of an unknown type. The median age at transplantation was 3.75 years (range, 1 to 7 years); the median follow-up time was 14 months (range, 2 to 119 months). Eleven patients underwent unrelated cord blood transplantation and 23 underwent peripheral blood stem cell transplantation (4 cases with an HLA-matched sibling donor, 2 cases with an HLA-mismatched related donor, and 17 cases with an unrelated donor). A busulfan-based myeloablative regimen was used as a conditioning regimen. The estimated overall survival at 3 years was 84.8% ± 6.3% and 91.2% of the patients (31 of 34) achieved full donor chimerism. Twenty-seven children were evaluable and all but 1 (carrier sibling donor; enzyme level improved but failed to reach normal) achieved normal enzyme level after transplantation. The incidence of grades II to IV acute graft-versus-host disease (aGVHD) was 41.1% (14 of 34), wherein the incidence of grades III and IV aGVHD was 11.8% (4 of 34). The incidence of moderate-to-severe chronic graft-versus-host disease was 5.9% (2 of 34). There was a significant difference in the survival rate between children who received transplantation before 2009 and those after 2009 (55.6% versus 95.7%, P = .002); the survival rate was lower in patients with pneumonia before transplantation than in those with no active infection before transplantation (66.7% versus 95.5%, P = .019), and no significant differences in survival rates were observed among children with different disease types, ages at transplantation, donor/graft source, and conditioning regimens. After transplantation, upper-airway obstruction, hepatosplenomegaly, and corneal clouding were significantly improved; hearing and motor function were improved to a certain extent; valvular heart disease was improved in some patients but progressed in others; and short stature and speech skills showed little improvement. AlloHSCT may save the lives of patients with MPS I, II, IV or VI and could improve quality of life. Pretransplantation pneumonia affects transplantation outcomes. Advances in transplantation protocols and techniques help to improve patient prognosis. Well-matched unrelated donors can also be an ideal donor source. Standardized follow-up and a multidisciplinary team contribute to accurate evaluation of long-term post-transplantation outcome and further improve the quality of life of MPS patients.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Mucopolissacaridoses/complicações , Mucopolissacaridoses/terapia , Criança , Pré-Escolar , China , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Feminino , Antígenos HLA , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Lactente , Masculino , Mucopolissacaridoses/mortalidade , Transplante de Células-Tronco de Sangue Periférico , Qualidade de Vida , Estudos Retrospectivos , Análise de Sobrevida , Condicionamento Pré-Transplante/métodos , Doadores não Relacionados
12.
Onco Targets Ther ; 9: 2557-63, 2016.
Artigo em Inglês | MEDLINE | ID: mdl-27217774

RESUMO

Hematopoietic stem cell transplantation (HSCT) is a promising method for therapy of pediatric patients with acute leukemia. However, less availability of matched donors limited its wide application. Recently, haploidentical HSCT has become a great resource. Here, we have retrospectively reported our experience of 20 pediatric patients with acute leukemia who underwent haploidentical HSCT without total body irradiation (TBI) myeloablative regimen in our center from November 2007 to June 2014. All the patients attained successful HSCT engraftment in terms of myeloid and platelet recovery. Thirteen patients developed grade I-IV acute graft-versus-host disease (a-GVHD). The incidence of grade I-II a-GVHD, grade III-IV a-GVHD, and chronic GVHD (c-GVHD) was 45%, 20%, and 25%, respectively. The mean myeloid and platelet recovery time was 13.20±2.41 and 19.10±8.37 days. The median follow-up time was 43.95±29.26 months. During the follow-up, three patients died. The overall survival (OS) rate was 85%. The present study indicated that haploidentical HSCT without TBI myeloablative regimen significantly improved the OS rate of pediatric patients with acute leukemia.

13.
Pediatr Transplant ; 19(5): 563-70, 2015 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-26058853

RESUMO

The success of treating a wide variety of pediatric diseases with HSCT, hematologic malignancies in particular, has resulted in an increased number of long-term survivors. This study is the first large-scale, multicentre report that describes the evolution of pediatric HSCTs in China during the period of 1998-2012. Of all 1052 patients, 266 cases were treated with autologous HSCs and 786 used allogeneic HSCs. The disease indications for HSCTs mainly included leukemias, lymphoma, solid tumors, and non-malignant disorders. The total number of HSCTs, especially unrelated donor transplants, appeared to be increasing year by year. For patients with neuroblastoma, the therapeutic efficacy seemed to be poor, with a five-yr OS and DFS rate of 34.5 ± 14.3% and 20.7 ± 9.6%, respectively. In contrast, the survival of patients with SAA was prominently improved, and their five-yr OS and DFS rates were 82.8 ± 4% and 80.7 ± 4.1%, respectively. Patients who received cord blood transplants had a lower incidence of acute GVHD than that of PB and/or BM transplants from unrelated donors. This report offers us a valuable resource for evaluating the changes in HSCTs in China over the past 14 yr.


Assuntos
Doença Enxerto-Hospedeiro/epidemiologia , Neoplasias Hematológicas/epidemiologia , Neoplasias Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Transplante de Células-Tronco Hematopoéticas/tendências , Adolescente , Criança , Pré-Escolar , China , Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Intervalo Livre de Doença , Feminino , Geografia , Neoplasias Hematológicas/mortalidade , Humanos , Lactente , Masculino , Estudos Retrospectivos , Resultado do Tratamento , Doadores não Relacionados
14.
Chin Med J (Engl) ; 128(4): 472-6, 2015 Feb 20.
Artigo em Inglês | MEDLINE | ID: mdl-25673448

RESUMO

BACKGROUND: Infections remain a major cause of therapy-associated morbidity and mortality in children with acute lymphoblastic leukemia (ALL). METHODS: We retrospectively analyzed the medical charts of 256 children treated for ALL under the CCLG-2008 protocol in Beijing Children's Hospital. RESULTS: There were 65 infectious complications in 50 patients during vincristine, daunorubicin, L-asparaginase and dexamethasone induction therapy, including microbiologically documented infections (n = 12; 18.5%), clinically documented infections (n = 23; 35.3%) and fever of unknown origin (n = 30; 46.2%). Neutropenia was present in 83.1% of the infectious episodes. In all, most infections occurred around the 15 th day of induction treatment (n = 28), and no patients died of infection-associated complications. CONCLUSIONS: The infections in this study was independent of treatment response, minimal residual diseases at the end of induction therapy, gender, immunophenotype, infection at first visit, risk stratification at diagnosis, unfavorable karyotypes at diagnosis and morphologic type. The infection rate of CCLG-2008 induction therapy is low, and the outcome of patients is favorable.


Assuntos
Antineoplásicos/uso terapêutico , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/microbiologia , Criança , Pré-Escolar , China , Daunorrubicina/uso terapêutico , Dexametasona/uso terapêutico , Feminino , Humanos , Lactente , Masculino , Neoplasia Residual/etiologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Estudos Retrospectivos , Vincristina/uso terapêutico
15.
Pediatr Hematol Oncol ; 31(1): 39-49, 2014 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-24383400

RESUMO

The treatment of pediatric severe aplastic anemia (SAA) with allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents major challenges including the risks of graft failure, septic complications, and graft-versus-host disease (GVHD). Additive infusions of human umbilical cord derived mesenchymal stem cell (hUC-MSC) may be administered to improve patient survival. We retrospectively examined 37 pediatric patients with SAA who received allo-HSCT and subsequent infusions of hUC-MSC suspension at a dose of 1.0 × 10(6 )/kg. The times and doses of hUC-MSC infusions were increased in patients with severe GVHD. All patients received hUC-MSC infusions. The median time to post-transplantation neutrophil count of greater than 0.5 × 10(9 )/L was 14 days (range, 11-20 days) and time to post-transplantation platelet count of greater than 20 × 10(9 )/L was 19 days (14-29 days). The overall frequency of acute GVHD (aGVHD) was 45.9% (17/37). These aGVHD episodes occurred at a median time of post-transplantation 47 days (15-83 days). The frequency of chronic GVHD (cGVHD) was 18.9% (7/37); cGVHD developed from aGVHD in 10.8% (4/37) of patients. The GVHD-associated mortality rate was 18.9% (7/37) and aGVHD-specific mortality rate was 8.1% (3/37). The median overall survival time was 35 months (9-67 months) and the three-year overall survival rate was 74.2% (28/37). Seven patients died of GVHD, one patient died of a severe invasive fungal infection, and one patient died of renal failure. In conclusion, post-transplantation hUC-MSC infusions seemed to be safely infused in children with SAA who have previously received allo-HSCT.


Assuntos
Anemia Aplástica/cirurgia , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Transplante de Células-Tronco Hematopoéticas , Transplante de Células-Tronco Mesenquimais , Aloenxertos , Contagem de Células Sanguíneas , Transplante de Medula Óssea/estatística & dados numéricos , Criança , Pré-Escolar , Transplante de Células-Tronco de Sangue do Cordão Umbilical/estatística & dados numéricos , Feminino , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro/diagnóstico , Doença Enxerto-Hospedeiro/epidemiologia , Mobilização de Células-Tronco Hematopoéticas , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Humanos , Lactente , Masculino , Transplante de Células-Tronco Mesenquimais/estatística & dados numéricos , Micoses/etiologia , Micoses/mortalidade , Transplante de Células-Tronco de Sangue Periférico/estatística & dados numéricos , Complicações Pós-Operatórias/epidemiologia , Insuficiência Renal/etiologia , Insuficiência Renal/mortalidade , Estudos Retrospectivos , Sepse/etiologia , Sepse/mortalidade , Taxa de Sobrevida , Condicionamento Pré-Transplante , Resultado do Tratamento
16.
Zhonghua Yan Ke Za Zhi ; 49(6): 541-6, 2013 Jun.
Artigo em Chinês | MEDLINE | ID: mdl-24119969

RESUMO

OBJECTIVE: To detect long-term ocular alteration of children with malignant osteopetrosis after hematopoietic stem cell transplantation. METHODS: Children diagnosed as osteopetrosis from 5 months to 31 months underwent hematopoietic stem cell transplantation. Computed tomography of optic canal, FVEP, ERG and fundus examination were applied to assess the visual function. RESULTS: Bone marrow transplantation was successful. Peripheral blood test, splenohepatomegalia and osteosclerosis improved gradually. The mean optic canal diameters of right eyes before transplantation was (1.7 ± 0.4)mm. The mean optic canal diameters of right eyes was (3.2 ± 0.7)mm after transplantation. The mean optic canal diameters of left eyes before transplantation was (1.9 ± 0.5)mm . The mean optic canal diameters of left eyes was (3.1 ± 0.8)mm after transplantation. The difference between above two groups was statistically significant(t = -5.204, -4.211;P < 0.05). P2 latency period of FVEP prolonged in 7 cases before transplantation. Mean P2 latency period of FVEP decreased 21.13 ms in 5 cases after transplantation. Mean P2 latency period of FVEP prolonged 22.25 ms in 2 cases after transplantation. Under light adaptation and dark adaptation, ERG amplitude depressed obviously in 2 cases. Two cases with optic nerve atrophy did not change after transplantation. CONCLUSIONS: Hematopoietic stem cell transplantation is an effective way to deal with malignant osteopetrosis. Successful transplantation has been shown to arrest visual deterioration in some cases.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Osteopetrose/fisiopatologia , Osteopetrose/cirurgia , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Visão Ocular , Acuidade Visual
17.
Zhonghua Er Ke Za Zhi ; 50(11): 807-12, 2012 Nov.
Artigo em Chinês | MEDLINE | ID: mdl-23302608

RESUMO

OBJECTIVE: Osteopetrosis is a rare genetic disorder and the malignant infantile osteopetrosis (MIOP) is the worst subtype of this disease. Seventy percent of patients die in six years of life without proper treatment. Hematopoietic stem cell transplantation (HSCT) offers the only chance of cure for MIOP. METHOD: Retrospective analysis was performed on 8 patients with MIOP who underwent HSCT in Beijing Children's Hospital during the period from 2006 to 2011. RESULT: Eight cases (4 male and 4 female, mean age at HSCT 13.5 months) were diagnosed as malignant infantile osteopetrosis. Conditioning regimen included fludarabine, busulfan and cyclophosphamide. All patients received cyclosporin for prophylaxis of graft vs. host disease (GvHD). A UMD recipient underwent CD34(+) cell selection. ATG/ALG, mycophenolate mofetil (MMF) and methotrexate (MTX) used for recipients with unrelated cord donor (2) and recipients with haplo-identical donors (5). Average time for neutrophil engraftment was 15.7 day (9 - 36), platelet engraftment was 43.3 day (10 - 68). The patients were followed up from 47 days to 5 years, 1 patient died of post-transplant complications. Seven cases presented better in clinical manifestation. Acute GvHD I°-II° was observed in 6 patients, III°-IV° in 2 patients. It was controlled by anti-GvHD therapy. CONCLUSION: Non-allogenic stem cell transplantation treatment of infantile MIOP showed high survival rate and restoration of hematopoiesis in haploid transplant patients, therefore, non-allogenic HSCT may be an option to treat MIOP in children.


Assuntos
Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/métodos , Osteopetrose/terapia , Condicionamento Pré-Transplante/métodos , Transplante de Medula Óssea/efeitos adversos , Transplante de Medula Óssea/métodos , Pré-Escolar , Feminino , Sangue Fetal/citologia , Seguimentos , Predisposição Genética para Doença , Doença Enxerto-Hospedeiro/tratamento farmacológico , Doença Enxerto-Hospedeiro/epidemiologia , Haploidia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Lactente , Masculino , Osteopetrose/mortalidade , Estudos Retrospectivos , Análise de Sobrevida , Transplante Homólogo , Resultado do Tratamento
18.
Artigo em Chinês | MEDLINE | ID: mdl-21604575

RESUMO

OBJECTIVE: To study the incidence of human cytomegalovirus (CMV) and human herpesvirus 6 (HHV-6) infection in pediatric patients with hemopoietic stem cell transplantation (HSCT), and to explore the relationship between CMV and HHV-6 infection in pediatric patients with HSCT. METHODS: Pediatric patients with HSCT in hemotology center of Beijing Children's Hospital were enrolled into this study from June 2007 to October 2009. Peripheral blood were collected every week after HSCT, and Fluorescent quantitation PCR and conventional PCR were used to detect CMV DNA load in serum and HHV-6 DNA in peripheral blood respectively. Genetic typing was conducted on HHV-6. RESULTS: Fifty two pediatric patients with HSCT were enrolled into this study, and six hundreds and thirty six specimens were collected totally. CMV DNA was detected in fifty two specimens from twenty cases. The median time was 56 days after HSCT. The incidence of CMV infection was 38.5% (20/52) in all HSCT patients and 47.6% (20/42) in allogene HSCT patients. The incidence of late CMV infection was 22.2% (6/27) in allogene HSCT. Three patients died of CMV infection,and two died of CMV interstitial pneumonia. HHV-6 DNA was detected in thirty three specimens from fourteen cases. The median time was 23 days after HSCT. The incidence of HHV-6 infection was 26.9% (14/52)in all HSCT patients and 31% (13/42) in allogene HSCT patients. The genotype of HHV6 was all type B. HHV-6 DNA was positive in six of twenty cases with CMV infection. The incidence of co-infection was 30% (6/20). CONCLUSIONS: There was a substantial incidence of CMV and HHV6 infection after HSCT. The relationship between earlier HHV6 infection and later CMV infection in pediatric patients with HSCT need further study.


Assuntos
Infecções por Citomegalovirus/etiologia , Citomegalovirus/genética , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Infecções por Herpesviridae/etiologia , Herpesvirus Humano 6/genética , Adolescente , Criança , Pré-Escolar , Infecções por Citomegalovirus/imunologia , Feminino , Infecções por Herpesviridae/imunologia , Humanos , Lactente , Masculino , Tipagem Molecular/métodos
20.
Zhongguo Shi Yan Xue Ye Xue Za Zhi ; 16(6): 1361-4, 2008 Dec.
Artigo em Chinês | MEDLINE | ID: mdl-19099644

RESUMO

The aim of this study was to explore a safe method collecting peripheral blood stem/progenitor cell (PBSPC) from the infants of body weight less than 20 kg by using the COBE Spectra Blood Cell Separator through Auto-PBSC procedure. After washing tube by normal saline, one unit of irradiated RBC was infused into the apheresis set. When the collection terminated, only the concentrated RBC in the apheresis set was returned to the infant. The peripheral mononuclear cells (PBMNCs) and CD34+ cells were counted, the cell viability was determined. The results showed that 13 PBSPC collections were carried out successfully from 7 infants of body weight<20 kg. The average count of MNCs was 4.44x10(8)/kg [(3.46-6.45)x10(8)/kg], the CD34+ count was 2.20x10(6)/kg [(1.34-3.79)x10(6)/kg] and the cell viability was 98.45% (97%-100%) respectively. The vital signs of all the infants went smoothly during collection of PBSPCs. In conclusion, with the aid of COBE Spectra blood cell separator and other measures, the collection of PBSPCs from infants of body weight<20 kg is safe and effective, the PBMNCs containing enough PBSPC can be harvested for transplantation.


Assuntos
Separação Celular/métodos , Mobilização de Células-Tronco Hematopoéticas/métodos , Células-Tronco Hematopoéticas , Pré-Escolar , Feminino , Humanos , Lactente , Masculino
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