RESUMO
Purpose: Hematopoietic stem cell transplantation (HSCT) has been an important method of treatment in the advance of pediatric acute lymphoblastic leukemia (ALL). The indications for HSCT are evolving and require updated establishment. In this study, we aimed to investigate the efficacy of HSCT on the treatment outcome of pediatric ALL, considering the indications for HSCT and subgroups. Materials and Methods: A retrospective analysis was conducted on ALL patients diagnosed and treated at a single center. Risk groups were categorized based on age at diagnosis, initial white blood cell count, disease lineage (B/T), and cytogenetic study results. Data on the patients' disease status at HSCT and indications of HSCT were collected. Indications for HSCT were categorized as upfront HSCT at 1st complete remission, relapse, and refractory disease. Results: Among the 549 screened patients, a total of 418 patients were included in the study; B-ALL (n=379) and T-ALL (n=39). HSCT was conducted on a total of 106 patients (25.4%), with a higher frequency as upfront HSCT in higher risk groups and specific cytogenetics. The overall survival (OS) was significantly better when done upfront than in relapsed or refractory state in T-ALL patients (p=0.0016). The KMT2A-rearranged ALL patients showed superior event-free survival (p=0.0023) and OS (p=0.0221) when HSCT was done as upfront treatment. Conclusion: HSCT had a substantial positive effect in a specific subset of pediatric ALL. In particular, frontline HSCT for T-ALL and KMT2A-rearranged ALL offered a better prognosis than when HSCT was conducted in a relapsed or refractory setting.
RESUMO
BACKGROUND: There has been remarkable progress in hemophilia A (HA) treatment in Korea. Viral inactivation products were developed in 1989, use of recombinant factor VIII (FVIII) concentrates started in 2002, and prophylaxis expanded thereafter. This study was conducted to identify the changes in complications in HA before and after 1989 or 2002. METHODS: The study was performed using the 2007-2019 Healthcare Big Data Hub of the Health Insurance Review and Assessment Service. RESULTS: Among 2,557 patients, 1,084 had ≥ 1 complication; 829 had joint problems, 328 had viral infections, 146 had neurologic sequelae, and 87 underwent 113 surgeries or procedures due to complications. Patients born after 1989 had a significantly lower risk of viral infections than those born before 1989; 27.1% vs. 1.4% (P < 0.001, odds ratio [OR], 0.037). Patients born after 2002 had a significantly lower risk of joint problems than those born before 2002; 36.8% vs. 24.7% (P < 0.001, OR, 0.538). Patients born after 2002 had a higher incidence of neurologic sequelae than those born before 2002; 3.7% vs. 11.1% (P < 0.001, OR, 3.210). Medical expenses for complication-associated surgeries or procedures were â©2,957,557,005. CONCLUSION: Viral infections have significantly decreased in Korean patients with HA. The degree of reduction of joint problems was lower than we expected, because it took > 10 years to expand prophylaxis widely. Neurologic sequelae have not decreased; thus, additional efforts to decrease intracranial hemorrhage are needed. We suggest personalized dosing of FVIII and more meticulous care during childbirth to further reduce the complications.
Assuntos
Hemofilia A , Humanos , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragias Intracranianas , Seguro Saúde , República da CoreiaRESUMO
Purpose: We investigated whether nonpharmaceutical interventions (NPI) to reduce the spread of coronavirus disease 2019 (COVID-19) was associated with a change in the incidence of immune thrombocytopenia (ITP). Patients and Methods: Using the Korean Health Insurance Review and Assessment Services (HIRA) database, individuals newly diagnosed with ITP between January 2015 and December 2020 were identified. The NPI period was defined as February 2020 to December 2020. The ITP incidence in the NPI period was compared with the mean annual incidence during the same months in the pre-NPI period and the incidence predicted by the autoregressive integrated moving average model. Results: In total, 25,723 patients were identified, and the overall annual incidence of ITP was 8.28 per 100,000 persons ([95% confidence interval (CI): 8.18-8.39]. The ITP incidence in the NPI period was 6.60 per 100,000 person-years (95% CI: 6.37-6.85), 0.77 times (95% CI: 0.74-0.80) lower than that during the pre-NPI period [8.62/100,000 (95% CI: 8.50-8.74)]. With the exception for patients aged ≥70 years, the ITP incidence was significantly lower in the NPI period than in the pre-NPI period. The most significant decline in the ITP incidence during the NPI period was observed in the 0-9 years age group [25.76/100,000 vs 14.01/100,000, P <0.001; incidence rate ratio (IRR): 0.54 (95% CI: 0.51-0.58)]. The intravenous immunoglobulin-treated ITP incidence in the NPI period was 1.69/100,000 (95% CI: 1.58-1.81), 0.79 times (95% CI: 0.73-0.85) lower than that in the pre-NPI period 2.15/100,000 (95% CI: 2.09-2.21)]. The incidence of steroid-treated ITP was lower in the NPI period than in the pre-NPI period (2.73/100,000 vs 2.2/100,000, P <0.001), with an IRR of 0.80 (95% CI: 0.76-0.83). Conclusion: This nationwide study revealed a significant decrease in ITP incidence, particularly among children, after the implementation of NPI.
RESUMO
BACKGROUND: Totally implantable venous access devices (TIVADs) are frequently used in pediatric patients with cancer owing to their multiple benefits. Despite occasional infections with TIVADs, knowledge of the incidence and risk factors is limited. METHODS: This retrospective study included pediatric patients with cancer who received TIVAD at Chungbuk National University Hospital from 2001 to 2021. We collected data on demographics, diagnosis, duration of TIVAD use, pathogens, and other risk factors. RESULTS: During the study period, 55 TIVADs with 25,954 device-days were applied in 49 patients. There were 15 TIVAD infections (15/55, 27.3%), with an infection rate of 0.21 infections per TIVAD per year (0.58 cases/1,000 device-days). TIVAD infections occurred at a median of 5 months (range, 8 days-30 months) after insertion. The most common causative microorganisms were methicillin-resistant coagulase-negative staphylococci (n = 8, 53.3%) followed by Escherichia coli (n = 3, 20.0%). Infection-free TIVAD survival was higher in the group with normal platelet count at insertion (platelet counts ≥ 150,000/µL) than in the group with thrombocytopenia at insertion (platelet counts < 150,000/µL) (81.3% vs. 32.1%, P = 0.004). Device removal was the mainstay of treatment (11/15, 73.3%). CONCLUSION: TIVAD infection may be related to thrombocytopenia at the time of device insertion. Further studies are needed to identify preventive factors against TIVAD infections in children with cancer.
Assuntos
Cateterismo Venoso Central , Leucopenia , Neoplasias , Trombocitopenia , Cateterismo Venoso Central/efeitos adversos , Criança , Humanos , Incidência , Leucopenia/etiologia , Neoplasias/complicações , Neoplasias/tratamento farmacológico , Estudos Retrospectivos , Fatores de Risco , Trombocitopenia/etiologiaRESUMO
Despite the availability of therapies to treat patients with immune thrombocytopenia (ITP), there is currently little data from randomized trials to assist clinicians in managing patients. The evidence-based guidelines of the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) are intended to support patients and physicians in the management of ITP. Experts from the KSHAAWP discussed and described this guideline according to the current treatment situation for ITP in Korea and finalized the guidelines. The expert panel recommended the management of ITP in adult and pediatric patients with newly diagnosed, persistent, and chronic disease refractory to first-line therapy with minor bleeding. Management approaches include observation and administration of corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin, and thrombopoietin receptor agonists. Currently, evidence supporting strong recommendations for various management approaches is lacking. Therefore, a large focus was placed on shared decision-making, especially regarding second-line treatment.
RESUMO
PURPOSE: Children with Down syndrome (DS) show a higher risk of acute leukemia than those without DS. In this study, we investigated the nationwide incidence of acute leukemia among children with DS and compared their epidemiologic characteristics with those of children with acute leukemia but without DS. MATERIALS AND METHODS: Using the National Health Insurance Service database, we selected patients with acute leukemia aged 0-19 years at diagnosis between 2007 and 2016. RESULTS: Among the 4,697 children with acute leukemia, 54 (1.1%) had DS. The median incidence rate of leukemia with DS by year was 1.3% (range, 0.2%-2.0%). Sixteen patients with acute lymphoblastic leukemia (ALL; 29.6%) and 36 with acute myeloid leukemia (AML; 66.7%) had DS. The DS group showed younger age at diagnosis than the non-DS group, and diagnosis of AML was more frequent in the DS group than in the non-DS group (3 years vs. 9 years, p<0.001; 66.7% vs. 32.4%, P<0.001, respectively). The 5-year overall survival was comparable between the DS and non-DS groups (88.0% vs. 81.9%, p=0.375). Among all the Koreans born between 2007 and 2008, the incidences of acute leukemia, ALL, and AML were 49.25, 20.75, and 163.38 times higher, respectively, in the DS group than in the non-DS group. CONCLUSION: Our findings support the fact that the incidence of acute leukemia is higher among patients with DS than among those without DS in Korea. However, the DS and non-DS groups in this study had a comparable overall survival rate.
Assuntos
Síndrome de Down , Leucemia Mieloide Aguda , Leucemia-Linfoma Linfoblástico de Células Precursoras , Doença Aguda , Criança , Síndrome de Down/complicações , Síndrome de Down/epidemiologia , Humanos , Leucemia Mieloide Aguda/epidemiologia , Leucemia Mieloide Aguda/etiologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/epidemiologia , Taxa de SobrevidaRESUMO
PURPOSE: Acute promyelocytic leukemia (APL) is a rare disease in children and there are some different characteristics between children and adult. We aimed to evaluate incidence, clinical characteristics and treatment outcomes of pediatric APL in Korea. MATERIALS AND METHODS: Seventy-nine pediatric APL patients diagnosed from January 2009 to December 2016 in 16 tertiary medical centers in Korea were reviewed retrospectively. RESULTS: Of 801 acute myeloid leukemia children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×109/L than in those with initial WBC ≥ 10×109/L (p=0.020). CONCLUSION: This study showed that the CR rates and survival outcomes in Korean pediatric APL patients were relatively good. The initial WBC count was the most important prognostic factor and most causes of death were related to serious bleeding in the early stage of treatment.
Assuntos
Antineoplásicos/administração & dosagem , Leucemia Promielocítica Aguda/tratamento farmacológico , Leucemia Promielocítica Aguda/mortalidade , Tretinoína/administração & dosagem , Adolescente , Antineoplásicos/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Humanos , Quimioterapia de Indução/métodos , Lactente , Contagem de Leucócitos , Masculino , Intervalo Livre de Progressão , Indução de Remissão , República da Coreia/epidemiologia , Estudos Retrospectivos , Resultado do Tratamento , Tretinoína/efeitos adversosRESUMO
The diagnosis of inherited platelet function disorders (IPFDs) is challenging owing to the unavailability of essential testing methods, including light transmission aggregometry and flow cytometry, in several medical centers in Korea. This study, conducted by the Korean Pediatric Hematology Oncology Group from March 2017 to December 2020, aimed to identify the causative genetic variants of IPFDs in Korean patients using next-generation sequencing (NGS). Targeted exome sequencing, followed by whole-genome sequencing, was performed for diagnosing IPFDs. Of the 11 unrelated patients with suspected IPFDs enrolled in this study, 10 patients and 2 of their family members were diagnosed with Glanzmann thrombasthenia (GT). The variant c.1913+5G>T of ITGB3 was the most common, followed by c.2333A>C (p.Gln778Pro) of ITGB2B. Known variants of GT, including c.917A>C (p.His306Pro) of ITGB3 and c.2975del (p.Glu992Glyfs*), c.257T>C (p.Leu86Pro), and c.1750C>T (p.Arg584*) of ITGA2B, were identified. Four novel variants of GT, c.1451G>T (p.Gly484Val) and c.1595G>T (p.Cys532Phe) of ITGB3 and c.1184G>T (p.Gly395Val) and c.2390del (p.Gly797Valfs*29) of ITGA2B, were revealed. The remaining patient was diagnosed with platelet type bleeding disorder 18 and harbored two novel RASGRP2 variants, c.1479dup (p.Arg494Alafs*54) and c.813+1G>A. We demonstrated the successful application of NGS for the accurate and differential diagnosis of heterogeneous IPFDs.
Assuntos
Integrina alfa2/genética , Integrina beta3/genética , Polimorfismo de Nucleotídeo Único , Trombastenia/genética , Pré-Escolar , Feminino , Frequência do Gene , Humanos , Lactente , Recém-Nascido , Masculino , República da CoreiaRESUMO
BACKGROUND: Oral propranolol has become first-line treatment for infantile hemangiomas (IHs). This study focused on identifying cytokines related to the biology of IH and early regression indicators of IH after propranolol treatment. METHODS: For inclusion, the patients had to be aged less than 1 year and have an IH with a largest diameter ≥2 cm. Patients were scheduled to receive 1 year of propranolol treatment. Serum cytokines involved in angiogenesis, vasculogenesis, and/or chronic inflammation were analyzed at 0, 1, and/or 12 months after treatment using Multiplex Luminex assays. RESULTS: Among the 49 evaluable patients, 33 completed the 1-year treatment: 16 showed excellent response and 12 had good response to propranolol. Significant decreases in serum MMP-2, bFGF, VEGF-α, and MCP-1 levels were observed after 1 year of treatment compared to pretreatment values. The maximal diameters of the lesions significantly correlated with pretreatment serum VEGF-α, bFGF, and MMP-9. Patients with higher bFGF and VEGF levels showed better response to propranolol at 1 year. CONCLUSION: MMP-2, VEGF-α, bFGF, and MCP-1 may involve in the biology of IH and their downregulation may be associated with involution processes of IH. Pretreatment bFGF and VEGF could be novel biomarkers for predicting response to propranolol. IMPACT: We found that decreases in the concentrations of MMP-2, bFGF, VEGF, and MCP-1 were associated with regression of the hemangioma, which indicates that one of the mechanisms of propranolol in the treatment of proliferative hemangiomas may involve downregulation of those cytokines. Patients with higher bFGF and VEGF levels showed better response to propranolol at 1 year. Importantly, serum bFGF higher than 37.07 pg/mL may predict an excellent response to propranolol. Therefore, along with the patient's age and the size and visual characteristics of the lesion, bFGF levels could help determine the viability of propranolol use in the treatment of IHs. Our study represented extensive serum profiling in IH, reporting the indicators and molecules clearly related to IH regression with propranolol treatment. The authors believe that monitoring serum cytokines, including MMP-2, bFGF, VEGF, and MCP-1, in IH patients could be important, in addition to clinical follow-up, for determining when to start and end propranolol treatment.
Assuntos
Antagonistas Adrenérgicos beta/administração & dosagem , Antineoplásicos/administração & dosagem , Fator 2 de Crescimento de Fibroblastos/sangue , Hemangioma/tratamento farmacológico , Propranolol/administração & dosagem , Fator A de Crescimento do Endotélio Vascular/sangue , Administração Oral , Antagonistas Adrenérgicos beta/efeitos adversos , Antineoplásicos/efeitos adversos , Biomarcadores Tumorais/sangue , Quimiocina CCL2/sangue , Feminino , Hemangioma/sangue , Hemangioma/diagnóstico , Humanos , Lactente , Recém-Nascido , Masculino , Metaloproteinase 2 da Matriz/sangue , Valor Preditivo dos Testes , Propranolol/efeitos adversos , Estudos Prospectivos , República da Coreia , Fatores de Tempo , Resultado do TratamentoRESUMO
INTRODUCTION: Under the hypothesis that early natural killer cell infusion (NKI) following haploidentical stem cell transplantation (haplo-SCT) will reduce relapse in the early post-transplant period, we conducted a pilot study to evaluate the safety and feasibility of NKI following haplo-SCT in children with recurrent neuroblastoma who failed previous tandem high-dose chemotherapy and autologous SCT. METHODS: We used the high-dose 131I-metaiodobenzylguanidine and cyclophosphamide/fludarabine/anti-thymocyte globulin regimen for conditioning and infused 3 × 107/kg of ex-vivo expanded NK cells derived from a haploidentical parent donor on days 2, 9, and 16 post-transplant. Interleukin-2 was administered (1 × 106 IU/m2/day) subcutaneously to activate infused donor NK cells on days 2, 4, 6, 9, 11, 13, 16, 18, and 20 post-transplant. RESULTS: Seven children received a total of 19 NKIs, and NKI-related acute toxicities were fever (n = 4) followed by chills (n = 3) and hypertension (n = 3); all toxicities were tolerable. Grade ≥II acute GVHD and chronic GVHD developed in two and five patients, respectively. Higher amount of NK cell population was detected in peripheral blood until 60 days post-transplant than that in the reference cohort. Cytomegalovirus and BK virus reactivation occurred in all patients and Epstein-Barr virus in six patients. Six patients died of relapse/progression (n = 5) or treatment-related mortality (n = 1), and one patient remained alive. CONCLUSION: NKI following haplo-SCT was relatively safe and feasible in patients with recurrent neuroblastoma. Further studies to enhance the graft-versus-tumor effect without increasing GVHD are needed.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Reconstituição Imune , Imunoterapia Adotiva , Células Matadoras Naturais/imunologia , Células Matadoras Naturais/transplante , Neuroblastoma/imunologia , Neuroblastoma/terapia , Transplante Haploidêntico , Biomarcadores , Contagem de Células , Criança , Pré-Escolar , Feminino , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Imunofenotipagem , Imunoterapia Adotiva/efeitos adversos , Imunoterapia Adotiva/métodos , Lactente , Masculino , Estadiamento de Neoplasias , Neuroblastoma/diagnóstico , Neuroblastoma/mortalidade , Quimeras de Transplante , Resultado do TratamentoRESUMO
BACKGROUND: The global burden of seasonal influenza on medical care has been one of the greatest in the pediatric population. The attention drawn to influenza B was relatively low compared to influenza A, probably because the influenza B virus was thought to be less virulent and have a lower pandemic potential. This study aimed to compare the clinical features of influenza A and B in children. METHODS: This retrospective study included children diagnosed and treated for influenza as inpatients or outpatients during the 2017/18 influenza season at a tertiary referral hospital. Data regarding clinical characteristics, diagnoses, laboratory results, and vaccination histories were collected and reviewed. RESULTS: Over the study period, 128 patients with influenza A and 109 patients with influenza B were identified. The mean age of patients with influenza B was significantly higher than that of patients with influenza A (5.6 ± 4.4 vs 4.1 ± 4.4 years, p = 0.010). Fever was the most common manifestation of influenza followed by respiratory symptoms. No single symptom was specifically associated with either type of influenza. The total duration of fever (4.3 ± 2.3 vs 3.7 ± 2.6 days), 'time from fever onset to initiation of antivirals', and 'time from initiation of antivirals to defervescence' were similar between the two influenza types, even though all three time periods tended to be longer for influenza B. The platelet counts and proportions of neutrophils were higher for influenza A than for influenza B infections, although the values were within normal limits for both influenza types. CONCLUSIONS: We found overall clinical similarities between influenza A and B with no less clinical significance or severity of influenza B compared to those of influenza A. Equal levels of awareness and attention should be paid to both influenza types.
Assuntos
Vírus da Influenza A , Vírus da Influenza B , Influenza Humana/virologia , Gravidade do Paciente , Distribuição por Idade , Antivirais/uso terapêutico , Criança , Pré-Escolar , Feminino , Hospitalização , Humanos , Lactente , Vírus da Influenza A/isolamento & purificação , Vírus da Influenza B/isolamento & purificação , Influenza Humana/sangue , Influenza Humana/tratamento farmacológico , Influenza Humana/epidemiologia , Masculino , Reação em Cadeia da Polimerase , República da Coreia , Estudos RetrospectivosRESUMO
RATIONALE: Diamond-Blackfan anemia (DBA) is a rare inherited marrow disorder, characterized by erythrocyte aplasia and is associated with congenital anomalies and a susceptibility to cancer. Although congenital abnormalities have been observed in â¼50% of DBA patients, the occurrence of an associated congenital diaphragmatic hernia (CDH) has rarely been reported. PATIENT CONCERNS: A 19-month-old male child was referred to our pediatric hematology-oncology outpatient clinic with anemic appearance. He presented to us with recurrent anemia, short stature, and developmental delay. DIAGNOSIS: On bone marrow examination, only erythropoietic cells were markedly decreased in number, whereas other cell lines were unaffected. An abdominal computed tomography scan revealed a Bochdalek type of CDH. A genetic analysis revealed heterozygous mutation of RPS19; therefore, he was diagnosed as having DBA with CDH. INTERVENTIONS: The patient received an initial packed red blood cell transfusion, followed by an administration of oral prednisone. OUTCOMES: The patient is maintained on oral prednisone administered at a dose of 0.3âmg/kg every alternate day and has since a hemoglobin level of >9.0âg/dL without further RBC transfusions. LESSONS: We learned that a Bochdalek type of CDH can manifest in a DBA patient with RPS19 gene mutation. Therefore, patients diagnosed with the latter disorder should also be screened for an early detection of potential CDHs.
Assuntos
Anemia de Diamond-Blackfan , Células da Medula Óssea/patologia , Transfusão de Eritrócitos/métodos , Hérnias Diafragmáticas Congênitas/diagnóstico , Prednisona/administração & dosagem , Proteínas Ribossômicas/genética , Anemia de Diamond-Blackfan/genética , Anemia de Diamond-Blackfan/fisiopatologia , Exame de Medula Óssea/métodos , Glucocorticoides/administração & dosagem , Humanos , Masculino , Mutação , Radioterapia Assistida por Computador/métodos , Resultado do Tratamento , Adulto JovemRESUMO
BACKGROUND: Children with cancer may be at an increased risk of infection with hepatitis B virus (HBV) when levels of hepatitis B antibodies are reduced owing to chemotherapy-induced immunosuppression. This study evaluated the changes in HBV antibody status and HBV infections after chemotherapy in children with acute lymphoblastic leukemia (ALL). PROCEDURE: The data of patients with ALL diagnosed between April 2007 and March 2013 were retrospectively collected. Hepatitis B surface antibody (HBsAb) titers were defined as negative at levels <10 IU/L. The HBsAb titers were individually compared before and after chemotherapy. RESULTS: A total of 88 patients were included in this study. At the time of diagnosis, 32 (36.4%) and 56 (63.6%) patients were HBsAb negative and HBsAb positive, respectively. The 56 HBsAb-positive patients were categorized into two groups, namely, group A with 44 patients (78.6%, 44/56) who became HBsAb negative after chemotherapy, and group B with 12 patients (21.4%) who remained HBsAb positive. On multivariate analysis, lower initial levels of HBsAb titers were associated with HBsAb negativity after chemotherapy (relative risk: 1.003, 95% confidence interval: 1.001-1.006; P = .009). CONCLUSION: This study demonstrated that patients with a low level of prechemotherapy HBsAb titers were likely to become HBsAb negative after chemotherapy. Therefore, evaluation of HBsAb status may be necessary after the completion of chemotherapy in children with ALL.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Anticorpos Anti-Hepatite B/sangue , Vírus da Hepatite B/efeitos dos fármacos , Hepatite B/sangue , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , Hepatite B/etiologia , Hepatite B/patologia , Anticorpos Anti-Hepatite B/imunologia , Antígenos de Superfície da Hepatite B/metabolismo , Humanos , Lactente , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/imunologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/virologia , Prognóstico , Estudos Retrospectivos , Adulto JovemRESUMO
A 12-year-old boy who underwent gastric wedge resection was transferred to our hospital because of vomiting, growth failure, and weight loss in January, 2016. We tried to restore his general condition by maintaining additional nutritional supply through peripheral parenteral nutrition (PN). However, continuous vomiting, weight loss, and superior mesenteric artery syndrome persisted because of low treatment compliance. The findings of hyponatraemia and bicytopenia did not improve. Bone marrow biopsy was performed, and it revealed copper deficiency. PN with additional micronutrient agents, including copper, were administered. In particular, invasive diagnosis and treatment, and adequate education improved the treatment compliance of the child. His copper deficiency and bicytopenia improved, and his weight and dietary intake also increased. We confirmed that treatment compliance is important in paediatric patients with malnutrition. In chronic malnutrition, attention should also be paid to deficiency of micronutrients such as copper, which can lead to haematologic problems.
Assuntos
Anemia/etiologia , Transtornos da Nutrição Infantil/complicações , Cobre/deficiência , Deficiências Nutricionais/complicações , Leucopenia/etiologia , Anorexia , Criança , Transtornos da Nutrição Infantil/terapia , Doença Crônica , Deficiências Nutricionais/terapia , Suplementos Nutricionais , Nutrição Enteral , Gastrectomia , Humanos , Ileostomia , Masculino , Nutrição Parenteral , Cooperação do Paciente , Síndrome da Artéria Mesentérica Superior , Vômito , Redução de PesoRESUMO
The role of unrelated donor HSCT for children with de novo AML in CR1 is controversial. We performed this study to investigate the feasibility of unrelated donor HSCT who initially had intermediate- or high-risk cytogenetics. We retrospectively reviewed medical records of patients with AML who received unrelated HSCT in CR1 at Samsung Medical Center between November 2001 and January 2012. Patients were allocated based on karyotype at diagnosis as follows: (a) low-risk: inv(16), t(16;16), t(8;21), and t(15;17); (b) high-risk: -5, 5q-, -7, 3q abnormalities, t(8;16), t(6;9), t(6;11), t(6;21), t(10;11), complex karyotype (≥3 abnormalities), and acute megakaryocytic leukemia without t(1;22); and (c) IR: all the other karyotypes including normal. Patients in intermediate- or high-risk group who were transplanted with either unrelated CB or matched unrelated BM/mobilized PB in their CR1 were included in this study. The projected OS and EFS rates were 74.9% and 71.1%, respectively, with a median follow-up of 87.3 months after transplantation. The EFS was 70.1%, 80.7%, and 73.9% for CB, BM, and mobilized PB groups, respectively (P = 0.89), and 73.9% and 70.6% for IR and high-risk groups (P = 0.76). The leading cause of death was relapse (n = 8), and only one patient died from non-relapse cause. Unrelated donor HSCT seems a feasible approach for children with intermediate- or high-risk AML in CR1. Relapse remains the leading cause of treatment failure among these patients.
Assuntos
Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda/genética , Leucemia Mieloide Aguda/terapia , Doadores não Relacionados , Adolescente , Criança , Pré-Escolar , Aberrações Cromossômicas , Inversão Cromossômica , Intervalo Livre de Doença , Feminino , Humanos , Lactente , Cariotipagem , Masculino , Neutrófilos/metabolismo , Recidiva , Indução de Remissão , Estudos Retrospectivos , Risco , Fatores de Tempo , Translocação Genética , Resultado do TratamentoRESUMO
[This corrects the article e46 in vol. 34, PMID: 30787679.].
RESUMO
BACKGROUND: The impact of early peripheral blood chimerism on the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) is unclear. We aimed to determine whether day 14 peripheral blood chimerism after allo-HSCT predicts outcomes in patients with non-malignant diseases. METHODS: Data from 56 patients who received allo-HSCT between April 2007 and March 2016 were retrospectively analyzed. Chimerism was evaluated using short-tandem repeat polymerase chain reaction, with mixed chimerism (MC) defined as greater than 1% recipient cells which was further categorized into low-level MC (> 1% and < 15% of recipient-derived cells) and high-level MC (≥ 15% of the recipient-derived cells). RESULTS: Thirty-six patients showed complete donor chimerism (CC), 14 low-level MC, and 6 high-level MC at day 14 post-transplant. The estimated 5-year event-free survival (EFS) was higher in the CC or low-level MC groups than in the high-level MC group (86.1% vs. 71.4% vs. 33.3%; P = 0.001). In BM or peripheral blood stem cell (BM/PBSC) transplants, the 5-year EFS was higher in the CC or low-level MC group than in the high-level MC group (93.1% vs. 66.7% vs. 0%; P < 0.001). However, in cord blood transplants, the 5-year OS and EFS according to the day 14 peripheral blood chimerism did not reach statistical significance. CONCLUSION: Although CC is not always necessary after allo-HSCT for non-malignant diseases, our data suggest that day 14 peripheral blood chimerism may predict outcomes in patients with non-malignant diseases who underwent BM/PBSC transplants.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Quimeras de Transplante/genética , Adolescente , Adulto , Anemia Aplástica/mortalidade , Anemia Aplástica/patologia , Anemia Aplástica/terapia , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Doença Enxerto-Hospedeiro , Humanos , Lactente , Recém-Nascido , Masculino , Aplasia Pura de Série Vermelha/mortalidade , Aplasia Pura de Série Vermelha/patologia , Aplasia Pura de Série Vermelha/terapia , Estudos Retrospectivos , Taxa de Sobrevida , Quimeras de Transplante/sangue , Transplante Homólogo , Doenças por Imunodeficiência Combinada Ligada ao Cromossomo X/mortalidade , Doenças por Imunodeficiência Combinada Ligada ao Cromossomo X/patologia , Doenças por Imunodeficiência Combinada Ligada ao Cromossomo X/terapia , Adulto JovemRESUMO
BACKGROUND: Splenic hemangioma is the most common benign tumor of the spleen. However, it remains a rare medical condition in children. Although the natural course of splenic hemangioma is slow growth, treatment for large splenic hemangiomas has been recommended due to the risk of spontaneous rupture causing life-threating hemorrhage. However, the optimal treatment for splenic hemangioma in children is unclear. CASE PRESENTATION: An 11-year-old girl had an enhancing mass, 61 × 54 × 65 mm in size and numerous daughter nodules throughout the entire spleen on a contrast-enhanced computed tomography scan of the abdomen and angiography. The patient was treated by complete embolization at the distal level of splenic artery, which resulted in total splenic infarction. Treatment-related complications were thrombocytosis and postembolization syndrome, including abdominal pain and, intermittent fever below 39 °C. There were no other serious complications, including bleeding. CONCLUSION: Splenic embolization may be a safe and less invasive intervention for children with a large splenic hemangioma. Further studies are needed to confirm the effectiveness of our approach.
Assuntos
Embolização Terapêutica/métodos , Hemangioma/terapia , Neoplasias Esplênicas/terapia , Angiografia , Criança , Embolização Terapêutica/efeitos adversos , Feminino , Hemangioma/diagnóstico por imagem , Humanos , Artéria Esplênica/diagnóstico por imagem , Neoplasias Esplênicas/diagnóstico por imagem , Tomografia Computadorizada por Raios XRESUMO
RATIONALE: Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disease characterized by an excessive systemic inflammatory response. HLH is classified as primary or secondary, where the latter may occur in association with many infections. However, no case of HLH has been previously associated with group b streptococcus (GBS) sepsis. PATIENT CONCERNS: We present a fatal case of HLH in a 5-year-old girl with GBS sepsis. DIAGNOSIS: The present patient met 5 of the HLH criteria: fever, splenomegaly, bicytopenia, hypertriglyceridemia and/or hypofibrinogenemia, and hyperferritinemia. GBS was identified in 2 sets of peripheral blood bacterial cultures. INTERVENTIONS: Empirical antibiotics, inotropes, and immunoglobulins were administered. OUTCOMES: The clinical course of the patient was fulminant and the patient died of septic shock 10âhours after admission to the hospital. LESSONS: We suggest GBS infection can cause HLH and early awareness of HLH associated with GBS infection and proper effective treatment are necessary to reduce mortality.
Assuntos
Linfo-Histiocitose Hemofagocítica/microbiologia , Sepse/microbiologia , Infecções Estreptocócicas/microbiologia , Streptococcus agalactiae , Pré-Escolar , Evolução Fatal , Feminino , HumanosRESUMO
The original version of this article unfortunately contained a mistake in the 7th author's given name. The correct version is presented above.