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Infant-type hemispheric glioma (IHG) is a rare pediatric brain tumor with variable response to chemotherapy and radiotherapy. Molecular insights into IHG can be useful in identifying potentially active targeted therapy. A male fetus was found to have congenital hydrocephalus at the gestational age of 37 weeks. Fetal MRI showed a 2.6 × 2.0-cm tumor located at the frontal horn of the left lateral ventricle, involving the left basal nuclei and thalamus. Tumor biopsy at the age of 2 days revealed an IHG consisting of spindle tumor cells with strong expression of GFAP and ALK. Targeted RNA sequencing detected a novel fusion gene of SOX5::ALK. After initial chemotherapy with cyclophosphamide, carboplatin, and etoposide for 2 cycles, the tumor size progressed markedly and the patient underwent a subtotal resection of brain tumor followed by treatment with lorlatinib, an ALK tyrosine kinase inhibitor with central nervous system (CNS) activity. After 3 months of treatment, reduction of tumor size was observed. After 14 months of treatment, partial response was achieved, and the infant had normal growth and development. In conclusion, we identified a case of congenital IHG with a novel SOX5::ALK fusion that had progressed after chemotherapy and showed partial response and clinical benefit after treatment with the CNS-active ALK inhibitor lorlatinib.
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Aminopiridinas , Neoplasias Encefálicas , Carcinoma de Pulmón de Células no Pequeñas , Glioma , Lactamas , Neoplasias Pulmonares , Pirazoles , Lactante , Niño , Masculino , Humanos , Recién Nacido , Neoplasias Pulmonares/tratamiento farmacológico , Carcinoma de Pulmón de Células no Pequeñas/tratamiento farmacológico , Quinasa de Linfoma Anaplásico/genética , Lactamas Macrocíclicas/uso terapéutico , Inhibidores de Proteínas Quinasas/uso terapéutico , Neoplasias Encefálicas/terapia , Neoplasias Encefálicas/tratamiento farmacológico , Glioma/terapia , Glioma/tratamiento farmacológico , Factores de Transcripción SOXDRESUMEN
BACKGROUND: Neuroblastoma varies widely in risk. Risk indicators in infants with incidental neuroblastoma refine treatment confidence for observation or intervention. The potential of functional imaging, particularly PET/CT, remains to be defined. PROCEDURE: A retrospective review of infants under 18 months diagnosed with incidental neuroblastoma from 2008 to May 2022 in our institute was conducted. Before October 2015, incidental patients were treated similarly to symptomatic cases, undergoing biopsy or surgical excision upon diagnosis (early cohort). Post October 2015 (late cohort), treatment decisions were guided by PET/CT findings, with 18F-DOPA PET/CT confirming diagnosis and staging. For tumors with low 18F-FDG uptake, an expectant observation approach was considered. Patient characteristics, diagnostic methods, image findings at diagnosis, treatment courses, and responses were compared between cohorts. RESULTS: Thirty infants less than 18 months were identified with incidental neuroblastoma and completed PET/CT at diagnosis. The early and late cohorts each comprised 15 patients. In the late cohort, nine out of 15 patients (60%) presented with localized FDG non-avid tumors were offered the option of expectant observation. Of these, seven patients opted for observation, thereby avoiding surgery. Treatment outcomes were comparable between early and late cohorts, except for one mortality of a patient who, despite showing 18F-FDG activity, declined treatment. CONCLUSIONS: This study demonstrates the potential utility of 18F-DOPA and 18F-FDG PET/CT scans in aiding clinical decision-making for infants with localized, incidental neuroblastoma. Given the concerns regarding radiation exposure, such imaging may be valuable for cases with suspected metastasis, initial large tumor size, or growth during follow-up.
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Dihidroxifenilalanina , Fluorodesoxiglucosa F18 , Neuroblastoma , Tomografía Computarizada por Tomografía de Emisión de Positrones , Radiofármacos , Humanos , Neuroblastoma/diagnóstico por imagen , Neuroblastoma/terapia , Neuroblastoma/patología , Lactante , Masculino , Femenino , Tomografía Computarizada por Tomografía de Emisión de Positrones/métodos , Estudios Retrospectivos , Dihidroxifenilalanina/análogos & derivados , Recién Nacido , Hallazgos Incidentales , Estudios de Factibilidad , Estudios de Seguimiento , Toma de Decisiones Clínicas , PronósticoRESUMEN
BACKGROUND: This study analyzed data from two consecutive protocols for children newly diagnosed with acute lymphoblastic leukemia (ALL) to determine the clinical impact of minimal/measurable residual disease (MRD) and recently identified tumor genetic subtypes. METHODS: Genetic subtypes were determined by sequential approaches including DNA indexing, reverse transcriptase-polymerase chain reaction, multiplex ligation-dependent probe amplification, and RNA-sequencing. MRD was assessed by flow cytometry. The Taiwan Pediatric Oncology Group TPOG-ALL-2013 study enrolled patients who received MRD-directed therapy. RESULTS: The 5-year event-free survival (EFS) and overall survival rates in the 2013 cohort were 77.8% and 86.9% compared to those of the 2002 cohort, which were 62.4% and 76.5%. Among patients treated with MRD-guided therapy, those with ETV6-RUNX1 fusion and high hyperdiploidy had the highest 5-year EFS (91.4% and 89.6%, respectively). The addition of dasatinib improved outcomes in patients with BCR-ABL1 ALL. Recently identified subtypes like DUX4-rearranged, ZNF384-rearranged, MEF2D-rearranged, and PAX5alt subtypes were frequently positive for MRD after remission induction, and these patients consequently received intensified chemotherapy. Treatment intensification according to the MRD improved the outcomes of patients presenting DUX4 rearrangements. In high-risk or very-high-risk subtypes, the TPOG-ALL-2013 regimen did not confer significant improvements compared to TPOG-ALL-2002, and the outcomes of BCR-ABL1-like, MEF2D-rearranged, and KMT2A-rearranged ALL subtypes (in addition to those of T-cell ALL) were not sufficiently good. Novel agents or approaches are needed to improve the outcomes for these patients. CONCLUSIONS: The TPOG-ALL-2013 study yielded outcomes superior to those of patients treated in the preceding TPOG-ALL-2002 study. This study provides important data to inform the design of future clinical trials in Taiwan. PLAIN LANGUAGE SUMMARY: MRD-directed therapy improved the outcomes for pediatric ALL, especially standard-risk patients. Genomic analyses and MRD might be used together for risk-directed therapy of childhood ALL. Our work provides important data to inform the design of future clinical trials in Taiwan.
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Leucemia-Linfoma Linfoblástico de Células Precursoras , Niño , Humanos , Neoplasia Residual/genética , Neoplasia Residual/diagnóstico , Pronóstico , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Leucemia-Linfoma Linfoblástico de Células Precursoras/patología , Dasatinib/uso terapéutico , Inducción de RemisiónRESUMEN
BACKGROUND: Medical clowning for children has been found to be effective at enhancing parents' psychological well-being during preoperative preparation, but has not been found during cancer treatment. This study aimed to examine whether and how medical clowning influenced the emotions of parents of children undergoing cancer treatment. METHODS: In this quasi-experimental study, 96 parents of children receiving inpatient cancer treatment were recruited, from June 2018 through April 2020. A demographic questionnaire measuring characteristics of parent and dyadic child, Brief Symptom Rating Scale measuring psychological distress of the parent, and Mood Assessment Scale measuring emotional status of parent and child were administered 1 day before a clowning service. The day after the clowning service, the Mood Assessment Scale again collected emotional status for parent and child. Descriptive analysis, bivariate analysis, and structural equation modeling to fit the actor-partner, cross-lagged model were used. FINDINGS: Parents experienced a low degree of psychological distress that called for emotional management. The indirect effect of medical clowning on parents' emotions through children's emotions was significant, as were the direct effect and total effect of medical clowning on parents' emotions. DISCUSSION: Parents experienced psychological distress during their child's inpatient cancer treatment. Medical clowning can directly improve children's emotions and through this pathway indirectly improve their parents' emotions. APPLICATION TO PRACTICE: There is need to monitor psychological distress and provide interventions for parents of children undergoing cancer treatment. Medical clowns should continue to serve parent-child dyads in pediatric oncology practice and become members of multidisciplinary health care teams.
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Neoplasias , Padres , Humanos , Padres/psicología , Emociones , Neoplasias/terapia , Neoplasias/psicología , Encuestas y Cuestionarios , Hospitalización , Relaciones Padres-HijoRESUMEN
PURPOSE: To examine the effectiveness of one-time medical clowning on improving short-term positive emotions among hospitalized children undergoing cancer treatment, and to analyze whether age moderates this effect. DESIGN: In this quasi-experimental research study, we recruited a pooled sample of 96 children who were undergoing cancer treatment in pediatric oncology/hematology wards at three university-affiliated medical centers in Taiwan from June 2018 through April 2020. METHODS: Children's demographic characteristics, symptom distress, quality of life, and pretest emotional status were collected at T1. At T2, we collected only posttest emotional status. We adapted generalized estimating equation models to evaluate the effectiveness of medical clowning on enhancing positive emotions. FINDINGS: Changes in the probabilities of positive emotion were significantly different across groups (51.84% for the experimental group, 15.76% for the control group; Δ = 36.08, p = 0.001), and the change was more than two times larger for the experimental group (effect ratio = 3.28, p < 0.05) than for the control group. When evaluating the moderating effect of age on the intervention, none of the coefficients reached the significant (p < 0.05) levels, suggesting that age may not moderate the intervention effect. CONCLUSION: This study demonstrates the core value of medical clowning in child-friendly health care. Our findings clearly support the benefit of the one-time medical clowning program on enhancing short-term emotional well-being across age groups of children. Medical clowning programs should be strongly encouraged and supported in pediatric oncology wards. CLINICAL RELEVANCE: Medical clowning programs should be widely and continuously implemented in pediatric oncology wards as a routine clinical practice for enhancing emotional well-being among children receiving cancer treatment. Nurses need to be aware of medical clowning's equal effectiveness across age groups, not only or better for younger children.
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Niño Hospitalizado , Neoplasias , Niño , Niño Hospitalizado/psicología , Emociones , Humanos , Neoplasias/terapia , Calidad de Vida , TaiwánRESUMEN
BACKGROUND: Patients with childhood cancer are at increased risk for the development of second cancers. METHODS: A national multicenter survey of second cancers conducted by the Taiwan Pediatric Oncology Group retrieved retrospective data from the database at the Children Cancer Foundation in Taiwan beginning in 1995. The characteristics of second cancers and associations of patient demographic and clinical characteristics with time to death due to a second cancer were analyzed. RESULTS: We examined the records of 8782 patients with a primary cancer diagnosed between January 1, 1995 and December 31, 2013, and a total of 99 patients with a second cancer were identified. The most common type of second cancer was acute myeloid leukemia (n = 35), followed by acute lymphoblastic leukemia (n = 15), central nervous system (CNS) tumors (n = 15), and sarcomas (n = 10). Secondary hematological malignancies occurred earlier than other secondary cancers. The frequencies of second CNS tumors and second bone cancers and sarcomas were notably increased when prior radiation doses increased from zero, low dose to high dose. The overall 5-year survival of patients with a second cancer was poor (33.7%). Multivariate survival analysis revealed that the year of primary diagnosis ≤2002, secondary hematological malignancies, and age at second cancer diagnosis ≤9.3 years or >26.8 years increased the risk of death following second cancer. CONCLUSION: Children who develop a second cancer have an unfavorable outcome. Early detection and improved treatment for second cancers are needed.
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Neoplasias Primarias Secundarias , Neoplasias , Niño , Humanos , Neoplasias/epidemiología , Neoplasias Primarias Secundarias/epidemiología , Estudios Retrospectivos , Taiwán/epidemiologíaRESUMEN
BACKGROUND: The use of social media in communications regarding cancer prevention is rapidly growing. However, less is known about the general population's social media use related to cancer screening awareness and behavior for different cancers. OBJECTIVE: We aimed to examine the relationship between social media use and cancer screening awareness and behavior among people without a cancer diagnosis. METHODS: Data were collected from the Health Information National Trends Survey 5 Cycle 1 to 3 in the United States (n=12,227). Our study included 10,124 participants without a cancer diagnosis and 3 measures of screening awareness (those who had heard of hepatitis C virus [HCV], human papillomavirus [HPV], and the HPV vaccine) and 4 measures of behavior (those who had prostate-specific antigen tests, Papanicolaou tests for cervical cancer, as well as breast cancer and colon cancer tests). Propensity-score matching was conducted to adjust for the sociodemographic variables between the social media user and nonuser participants. Multivariable logistic regression was used to assess the association of social media use by gender. Jackknife replicate weights were incorporated into the analyses. RESULTS: Of the 3794 matched participants, 1861 (57.6% weighted) were male, and the mean age was 55.5 (SD 0.42) years. Compared to social media nonusers, users were more likely to have heard of HCV (adjusted odds ratio [aOR]=2.27, 95% CI, 1.29-3.98 and aOR=2.86, 95% CI, 1.51-5.40, for male and female users, respectively) and HPV (aOR=1.82, 95% CI, 1.29-2.58 and aOR=2.35, 95% CI, 1.65-3.33, for male and female users, respectively). In addition, female users were more likely to have heard of the HPV vaccine (aOR=2.06, 95% CI, 1.41-3.00). No significant associations were found between social media use and prostate-specific antigen tests in males, Papanicolaou tests and breast cancer tests in females, or colon cancer tests in both male and female users. CONCLUSIONS: While social media services can potentially promote cancer screening awareness in the general population, but they did not improve screening behavior after adjusting for socioeconomic status. These findings strengthened our understanding of social media use in targeting health communications for different cancers.
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Infecciones por Papillomavirus , Vacunas contra Papillomavirus , Medios de Comunicación Sociales , Neoplasias del Cuello Uterino , Estudios de Cohortes , Detección Precoz del Cáncer , Femenino , Conocimientos, Actitudes y Práctica en Salud , Humanos , Masculino , Persona de Mediana Edad , Estados Unidos , Neoplasias del Cuello Uterino/diagnóstico , Neoplasias del Cuello Uterino/prevención & controlRESUMEN
Neuroblastoma is a neural crest-derived embryonal tumor and accounts for about 15% of all cancer deaths in children. MYCN amplification is associated with aggressive and advanced stage of high-risk neuroblastoma, which remains difficult to treat and exhibits poor survival under current multimodality treatment. Here, we analyzed the transcriptomic profiles of neuroblastoma patients and showed that aurora kinases lead to poor survival and had positive correlation with MYCN amplification and high-risk disease. Further, pan-aurora kinase inhibitor (tozasertib) treatment not only induces cell-cycle arrest and suppresses cell proliferation, migration, and invasion ability in MYCN-amplified (MNA) neuroblastoma cell lines, but also inhibits tumor growth and prolongs animal survival in Th-MYCN transgenic mice. Moreover, we performed quantitative proteomics and identified 150 differentially expressed proteins after tozasertib treatment in the Th-MYCN mouse model. The functional and network-based enrichment revealed that tozasertib alters metabolic processes and identified a mitochondrial flavoenzyme in fatty acid ß-oxidation, ACADM, which is correlated with aurora kinases and neuroblastoma patient survival. Our findings indicate that the aurora kinase inhibitor could cause metabolic imbalance, possibly by disturbing carbohydrate and fatty acid metabolic pathways, and ACADM may be a potential target in MNA neuroblastoma.
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Acil-CoA Deshidrogenasa/metabolismo , Redes y Vías Metabólicas/efectos de los fármacos , Proteína Proto-Oncogénica N-Myc/metabolismo , Neuroblastoma/metabolismo , Inhibidores de Proteínas Quinasas/farmacología , Proteómica/métodos , Acil-CoA Deshidrogenasa/genética , Animales , Aurora Quinasas/antagonistas & inhibidores , Aurora Quinasas/genética , Aurora Quinasas/metabolismo , Línea Celular Tumoral , Proliferación Celular/efectos de los fármacos , Proliferación Celular/genética , Progresión de la Enfermedad , Perfilación de la Expresión Génica/métodos , Regulación Neoplásica de la Expresión Génica/efectos de los fármacos , Humanos , Redes y Vías Metabólicas/genética , Ratones de la Cepa 129 , Ratones Transgénicos , Proteína Proto-Oncogénica N-Myc/genética , Neuroblastoma/tratamiento farmacológico , Neuroblastoma/genética , Piperazinas/farmacología , Análisis de SupervivenciaRESUMEN
Glioblastoma multiforme represents one of the deadliest brain tumor types, manifested by a high rate of recurrence and poor prognosis. The presence of glioma stem cells (GSCs) can repopulate the tumor posttreatment and resist therapeutics. A better understanding of GSC biology is essential for developing more effective interventions. We established a CD133 promoter-driven dual reporter, expressing green fluorescent protein (GFP) and firefly luciferase (CD133-LG), capable for in vitro and in vivo imaging of CD133+ GSCs. We first demonstrated the reporter enabled in vitro analyses of GSCs. DBTRG-05MG (Denver Brain Tumor Research Group 05) carrying CD133-LG (DBTRG-05MG-CD133-LG) system reported increased GFP/luciferase activities in neurospheres. Additionally, we identified and isolated CD133+/GFP+ cells with increased tumorigenic properties, stemness markers, Notch1, ß-catenin, and Bruton's tyrosine kinase (Btk). Furthermore, prolonged temozolomide (TMZ) treatment enriched GSCs (reflected by increased percentage of CD133+ cells). Subsequently, Btk inhibitor, ibrutinib, suppressed GSC generation and stemness markers. Finally, we demonstrated real-time evaluation of anti-GSC function of ibrutinib in vivo with TMZ-enriched GSCs. Tumorigenesis was noninvasively monitored by bioluminescence imaging and mice that received ibrutinib showed a significantly lower tumor burden, indicating ibrutinib as a potential GSC inhibitor. In conclusion, we established a dual optical imaging system which enables the identification of CD133+ GSCs and screening for anti-GSC drugs.
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Glioma/diagnóstico por imagen , Células Madre Neoplásicas/citología , Imagen Óptica/métodos , Antígeno AC133/metabolismo , Adenina/análogos & derivados , Agammaglobulinemia Tirosina Quinasa/antagonistas & inhibidores , Animales , Neoplasias Encefálicas/diagnóstico por imagen , Neoplasias Encefálicas/tratamiento farmacológico , Neoplasias Encefálicas/patología , Línea Celular Tumoral , Supervivencia Celular/efectos de los fármacos , Electroforesis en Gel de Poliacrilamida , Citometría de Flujo , Glioma/tratamiento farmacológico , Glioma/patología , Humanos , Luciferasas de Luciérnaga , Ratones , Piperidinas , Pirazoles/uso terapéutico , Pirimidinas/uso terapéutico , Temozolomida/uso terapéuticoRESUMEN
BACKGROUND: Infection is a major complication in pediatric patients with acute lymphoblastic leukemia during chemotherapy. In this study, the infection characteristics were determined and risk factors analyzed based on the Taiwan Pediatric Oncology Group (TPOG) acute lymphoblastic leukemia (ALL) protocol. PROCEDURE: We retrospectively reviewed fever events during chemotherapy in 252 patients treated during two consecutive clinical trials at a single institution between 1997 and 2012. Patients were classified as standard, high, and very high risk by treatment regimen according to the TPOG definitions. We analyzed the characteristics and risk factors for infection. RESULTS: Fever occurred in 219 patients (86.9%) with a mean of 2.74 episodes per person. The fever events comprised 64% febrile neutropenia, 39% clinically documented infections, and 44% microbiologically documented infections. The microbiologically documented infections were mostly noted during the induction phase and increased in very high risk patients (89 vs. 24% and 46% in standard-risk and high-risk patients, respectively). Younger age and higher risk (high-risk and very high risk groups) were risk factors for fever and microbiologic and bloodstream infections. Female gender and obesity were additive risk factors for urinary tract infection (odds ratios = 3.52 and 3.24, P < 0.001 and P = 0.004, respectively). CONCLUSIONS: Infections developed primarily during the induction phase, for which younger age and higher risk by treatment regimen were risk factors. Female gender and obesity were additive risk factors for urinary tract infection.
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Protocolos de Quimioterapia Combinada Antineoplásica , Neutropenia Febril , Leucemia-Linfoma Linfoblástico de Células Precursoras , Infecciones Urinarias , Adolescente , Factores de Edad , Protocolos de Quimioterapia Combinada Antineoplásica/administración & dosificación , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Niño , Preescolar , Neutropenia Febril/inducido químicamente , Neutropenia Febril/epidemiología , Femenino , Humanos , Lactante , Recién Nacido , Masculino , Obesidad , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/epidemiología , Factores de Riesgo , Factores Sexuales , Infecciones Urinarias/inducido químicamente , Infecciones Urinarias/epidemiologíaRESUMEN
INTRODUCTION: Clinical presentations of childhood leukaemia have been reported in case-only studies. The timing when these presentations start to occur prior to diagnosis is less clear. METHODS: In this nested case-control study, 1,025 and 334 children with lymphoid and myeloid leukaemia, respectively, were matched (1:30) to population-based controls by sex, region and year of birth. An index date was assigned for each control when the matched case was diagnosed. Healthcare access records of cases and controls in the year before the index date were extracted. RESULTS: Children with lymphoid leukaemia started to visit doctors more often at least 2 months before leukaemia diagnosis (P < 0.05). Various presentations were recorded in these visits: rates of haematological presentations, musculoskeletal presentations, and injuries started to increase significantly at least 3 months before diagnosis; rates of respiratory, gastrointestinal and urinary tract presentations did not increase significantly until the last month. The findings for myeloid lymphoma were less clear, but children appeared to visit doctors more often at least 4 months before diagnosis, and the rate of haematological presentations also started to increase at least 4 months before leukaemia diagnosis. Although haematological presentations were most strongly associated with undiagnosed leukaemia (odds ratio > 290 in the last month), the majority (>96%) of children with haematological presentations did not have leukaemia if they had not been diagnosed in their first visit. CONCLUSIONS: We described a clinical picture in the year before leukaemia diagnosis. These findings revealed ongoing difficulties in early diagnosis of childhood leukaemia in healthcare settings.
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Enfermedades Gastrointestinales/diagnóstico , Leucemia Linfoide/diagnóstico , Leucemia Mieloide/diagnóstico , Enfermedades Pulmonares/diagnóstico , Enfermedades Urológicas/diagnóstico , Adolescente , Estudios de Casos y Controles , Niño , Preescolar , Humanos , Leucemia Linfoide/patología , Leucemia Mieloide/patología , Sistema de Registros , TaiwánRESUMEN
Treatment abandonment, the failure to complete therapy that is required for definitive disease control, frequently causes treatment failure for pediatric patients in low- and middle-income countries with chronic conditions, particularly cancer. Other forms of incomplete treatment affecting children in all settings, such as nonadherence and loss to follow-up, are often confused with treatment abandonment. Unclear definitions of incomplete treatment dramatically affect reported outcomes. To facilitate disease-specific and cross-sector analyses, we outline a practical approach to categorize forms of incomplete treatment, present distinct semantic categories with case examples and provide an algorithm that could be tailored to disease- and context-specific needs.
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Algoritmos , Atención a la Salud/economía , Atención a la Salud/organización & administración , Renta , Neoplasias/economía , Neoplasias/terapia , Niño , Preescolar , Enfermedad Crónica , Humanos , Masculino , Neoplasias/epidemiologíaRESUMEN
Medulloblastoma (MB) is a type of malignant tumor arising only in the cerebellum that was first defined by Cushing and Bailey in 1920s. In this review paper, we trace the evolution of risk stratification and the correlated changing concept of management in the past years. Outcome analysis of the hospital series of the Taipei Veterans General Hospital, Cheng Hsin General Hospital, and Taipei Medical University Hospital was performed to correlate prognostic indicators with reported studies. The purpose is to provide clues for age-specific and risk-adjusted optimal, effective, but beneficial and protective treatment strategies of these tumors in children.
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Neoplasias Cerebelosas , Manejo de la Enfermedad , Meduloblastoma , Adolescente , Factores de Edad , Neoplasias Cerebelosas/epidemiología , Neoplasias Cerebelosas/mortalidad , Neoplasias Cerebelosas/terapia , Niño , Preescolar , Femenino , Humanos , Imagen por Resonancia Magnética , Masculino , Meduloblastoma/epidemiología , Meduloblastoma/mortalidad , Meduloblastoma/terapia , Estudios Retrospectivos , Análisis de Supervivencia , Taiwán/epidemiología , Resultado del TratamientoRESUMEN
BACKGROUND: Myeloablative, high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation (PBSCT) improves outcome in some high-risk malignant solid tumors and lymphomas in children and young adults. METHODS: We performed 16 peripheral blood stem cell (PBSC) harvests in 12 children and 2 young adult patients with a high-risk malignant solid tumor or refractory/relapsed Hodgkin's lymphoma from August 2015 to December 2020. In our chemotherapy mobilization protocol, we used an absolute neutrophil count (ANC) of >1 × 109/L following the nadir after chemotherapy as the criterion for undertaking the apheresis. RESULTS: The median CD34+ cell count per kg body weight of the 33 apheresis products was 4.92 × 106 cells/kg (range, 0.34-22.53 × 106 cells/kg). Thirteen of the 14 patients (93%) had successful PBSC collections that met their goals for PBSCT. Three patients did not receive PBSCT due to disease progression prior to transplantation. Prompt engraftment occurred in all the remaining 11 patients with 17 PBSCTs. CONCLUSION: Our data suggest that ANC can be helpful as a surrogate parameter in clinical decision-making when the peripheral blood CD34+ count is unavailable.
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In this study, we investigated the prevalence of mental health problems among patients with cancer and whether oncology nurse navigation improved their mental health outcomes and medical experience. In this randomized controlled clinical trial, we recruited 128 outpatients with cancer via purposive sampling from a teaching hospital in northern Taiwan. Participants were randomly assigned to the navigation group (N = 61) or the usual care group (N = 67). Data were collected from January 2019 to July 2020 using questionnaires, including the self-reported Distress Thermometer, Hospital Anxiety and Depression Scale, Demoralization Scale, and Patient Assessment of Chronic Illness Care. Data were collected at baseline and after three and six months of the intervention. Descriptive and analytical statistical analyses were performed. The prevalence rates of anxiety, depression, distress, and demoralization were 17.9%, 15.7%, 29.7%, and 29.7%, respectively. After three months, the participants in the navigation group exhibited significantly reduced levels of anxiety, demoralization, and emotional distress (reduced by 92%, 75%, and 58%, respectively) and reported a better medical experience (odds ratio = 1.40) than those in the usual care group.
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Neoplasias , Enfermería Oncológica , Humanos , Femenino , Masculino , Taiwán , Neoplasias/psicología , Persona de Mediana Edad , Enfermería Oncológica/métodos , Salud Mental , Ansiedad , Navegación de Pacientes , Adulto , Anciano , DepresiónRESUMEN
BACKGROUND: Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a progressive fatal disorder caused by deposition of mutant transthyretin (TTR) amyloids mainly in the nerves and heart. Autonomic dysfunction is a major disabling manifestation, affecting 90% of patients with late-onset ATTRv-PN. The current study aimed to investigate brain functional alterations associated with dysautonomia due to peripheral autonomic nerve degeneration in ATTRv-PN. METHODS: Resting-state functional MRI data were acquired from 43 ATTRv-PN patients predominantly of A97S (p.A117S) genotype, and the functional connectivity of central autonomic regions was assessed. RESULTS: Compared with age-matched healthy controls, the ATTRv-PN patients exhibited (1) reduced functional connectivity of the central autonomic regions such as hypothalamus, amygdala, anterior insula, and middle cingulate cortex with brain areas of the limbic, frontal, and somatosensory systems, and (2) correlations of reduced functional autonomic connectivity with the severity of autonomic dysfunction especially orthostatic intolerance, decreased heart rate variability, and greater clinical disability. CONCLUSIONS: Our findings provide evidence linking peripheral autonomic dysfunction with altered connectivity in the central autonomic network in ATTRv-PN.
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BACKGROUND: The decision to treat children with benign epilepsy with centrotemporal spikes (BECTS) using antiseizure medications (ASM) is controversial. Our goal is to compare the effect of ASM treatment on the alteration of electroencephalographic (EEG) functional connectivity and power across four frequency bands in children with BECTS. METHODS: Children with BECTS with two-year follow-up were retrospectively divided into ASM versus non-ASM groups. The network properties of the EEGs as based on network-based statistic and graph theory were evaluated by the following indices: global efficiency, clustering coefficient, betweenness centrality, and nodal strength in four frequency bands (delta, theta, alpha, and beta). EEG power including absolute power (AP) and relative power (RP) was analyzed in four frequency bands. RESULTS: In children with BECTS with ASM treatment, there was no significant change in EEG connectivity across all bands before and after two years of ASM. In children with BECTS without ASM treatment, there was a significant increase of global efficiency, clustering coefficient, and nodal strength but not the betweenness centrality in the delta band after two years of follow-up. A decrease in AP in the delta and theta bands and a decrease in RP in the theta band were found in the ASM group after two years of treatment. CONCLUSIONS: Our results suggest that ASM may play a role in modulating the development of increasing overall brain connectivity and in downregulating overt synaptic activity, but not intrinsic focal connectivity, in the early years of BECTS. The changes in the EEG power indicate that ASM significantly normalized slow-wave band power.
Asunto(s)
Anticonvulsivantes , Electroencefalografía , Epilepsia Rolándica , Humanos , Epilepsia Rolándica/tratamiento farmacológico , Epilepsia Rolándica/fisiopatología , Femenino , Niño , Masculino , Anticonvulsivantes/farmacología , Estudios Retrospectivos , Preescolar , Estudios de Seguimiento , Ondas Encefálicas/efectos de los fármacos , Ondas Encefálicas/fisiología , Red Nerviosa/efectos de los fármacos , Red Nerviosa/fisiopatología , Encéfalo/fisiopatología , Encéfalo/efectos de los fármacosRESUMEN
Encapsulated cell therapy (ECT) shows significant potential for treating neurodegenerative disorders including Alzheimer's and Parkinson's, which currently lack curative medicines and must be managed symptomatically. This novel technique encapsulates functional cells with a semi-permeable membrane, providing protection while enabling critical nutrients and therapeutic substances to pass through. Traditional ECT procedures, on the other hand, pose difficulties in terms of cell survival and retrieval. We introduce the Microtube Array Membrane (MTAM), a revolutionary technology that solves these constraints, in this comprehensive overview. Microtube Array Membrane has distinct microstructures that improve encapsulated cells' long-term viability by combining the advantages of macro and micron scales. Importantly, the MTAM platform improves biosafety by allowing the entire encapsulated unit to be retrieved in the event of an adverse reaction. Our findings show that MTAM-based ECT has a great potential in a variety of illness situations. For cancer treatment, hybridoma cells secreting anti-CEACAM 6 antibodies inhibit triple-negative breast cancer cell lines for an extended period of time. In animal brain models of Alzheimer's disease, hybridoma cells secreting anti-pTau antibodies successfully reduce pTau buildup, accompanied by improvements in memory performance. In mouse models, MTAM-encapsulated primary cardiac mesenchymal stem cells dramatically improve overall survival and heart function. These findings illustrate the efficacy and adaptability of MTAM-based ECT in addressing major issues such as immunological isolation, cell viability, and patient safety. We provide new possibilities for the treatment of neurodegenerative illnesses and other conditions by combining the potential of ECT with MTAM. Continued research and development in this subject has a lot of promise for developing cell therapy and giving hope to people suffering from chronic diseases.
Asunto(s)
Encéfalo , Tratamiento Basado en Trasplante de Células y Tejidos , Animales , Ratones , Humanos , Transporte Biológico , Línea Celular , Modelos Animales de EnfermedadRESUMEN
The purpose of this study was to identify the correlates of depressive symptoms and the prevalence of depression, distress, and demoralization among patients with cancer in Taiwan in relation to their sociodemographics. A cross-sectional study design with convenience sampling was used to recruit 191 consecutive patients with cancer from the Cancer Center of a teaching hospital in northern Taiwan. Multiple linear regression was applied to analyze the determinants of depressive symptoms. The prevalence rates of depression (including suspected cases), distress, and demoralization were 17.8%, 36.1%, and 32.5%, respectively. The regression model explained 42.2% of the total variance, with significant predictors including marital status, life dependence, comorbidity, demoralization, and distress. The results demonstrated that higher levels of distress and demoralization were associated with more depressive symptoms. Demoralization and distress played vital roles in moderating depressive symptoms among patients with cancer. Nursing interventions should integrate appropriate mental health services, such as alleviating distress and demoralization, to prevent the occurrence of depression in patients with cancer.