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Genomic sequencing has driven precision-based oncology therapy; however, the genetic drivers of many malignancies remain unknown or non-targetable, so alternative approaches to the identification of therapeutic leads are necessary. Ependymomas are chemotherapy-resistant brain tumours, which, despite genomic sequencing, lack effective molecular targets. Intracranial ependymomas are segregated on the basis of anatomical location (supratentorial region or posterior fossa) and further divided into distinct molecular subgroups that reflect differences in the age of onset, gender predominance and response to therapy. The most common and aggressive subgroup, posterior fossa ependymoma group A (PF-EPN-A), occurs in young children and appears to lack recurrent somatic mutations. Conversely, posterior fossa ependymoma group B (PF-EPN-B) tumours display frequent large-scale copy number gains and losses but have favourable clinical outcomes. More than 70% of supratentorial ependymomas are defined by highly recurrent gene fusions in the NF-κB subunit gene RELA (ST-EPN-RELA), and a smaller number involve fusion of the gene encoding the transcriptional activator YAP1 (ST-EPN-YAP1). Subependymomas, a distinct histologic variant, can also be found within the supratetorial and posterior fossa compartments, and account for the majority of tumours in the molecular subgroups ST-EPN-SE and PF-EPN-SE. Here we describe mapping of active chromatin landscapes in 42 primary ependymomas in two non-overlapping primary ependymoma cohorts, with the goal of identifying essential super-enhancer-associated genes on which tumour cells depend. Enhancer regions revealed putative oncogenes, molecular targets and pathways; inhibition of these targets with small molecule inhibitors or short hairpin RNA diminished the proliferation of patient-derived neurospheres and increased survival in mouse models of ependymomas. Through profiling of transcriptional enhancers, our study provides a framework for target and drug discovery in other cancers that lack known genetic drivers and are therefore difficult to treat.
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Elementos Facilitadores Genéticos/genética , Ependimoma/tratamento farmacológico , Ependimoma/genética , Regulação Neoplásica da Expressão Gênica , Redes Reguladoras de Genes/genética , Terapia de Alvo Molecular , Oncogenes/genética , Fatores de Transcrição/metabolismo , Animais , Sequência de Bases , Ependimoma/classificação , Ependimoma/patologia , Feminino , Humanos , Camundongos , Medicina de Precisão , Interferência de RNA , Ensaios Antitumorais Modelo de XenoenxertoRESUMO
Unlike other developed countries that hold national burn registries to monitor burn injury and care, Canada relies on single-centre secondary datasets and administrative databases as surveillance mechanisms. The objective of this study was to determine the knowledge gap faced in Canada for not having a dedicated burn registry. A comprehensive scoping review was conducted to identify the burn literature that has arisen from secondary datasets in Canada. Literature of all study designs was included with the exception of case reports and cases series. Once data extraction was concluded, a thematic framework was constructed based on the information that arose from nations that hold national burn registries. Eighty-eight studies were included. Twelve studies arose from national datasets, and 18 from provincial databases, most of which were from Ontario and British Columbia. Only seven studies were conducted using a combination of Canadian units' single-centre datasets. The majority of included studies (58%) resulted from non-collaborative use of single-centre secondary datasets. Research efforts were predominantly conducted by burn units in Ontario, British Columbia, Manitoba and Alberta. A significant number of the included studies were outdated and several provinces/territories had no published burn data whatsoever. Efforts should be made towards the development of systems to surveil burn injury and care in Canada. This study supports the development of a nation-wide burn registry to bridge this knowledge gap.
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Queimaduras , Sistema de Registros , Queimaduras/epidemiologia , Queimaduras/terapia , Humanos , Canadá/epidemiologia , Bases de Dados Factuais , Unidades de Queimados/estatística & dados numéricos , Unidades de Queimados/organização & administraçãoRESUMO
INTRODUCTION: While some countries collect burn clinical data as part of nonspecific trauma datasets, others have developed burn registries allowing for benchmarking of outcome and quality-of-care data. The objectives of this project are to characterize the current state of burn clinical data collection and analysis in Canada, and to explore the interest of Canadian burn centers in contributing to a nation-wide burn registry. METHODS: A 23-item mixed methods survey was created and delivered via REDCap® to burn directors of 22 burn centers across Canada. Quantitative items were analyzed by means of descriptive statistics, and thematic analysis was used to explore qualitative data. RESULTS: Sixteen (72 %) complete survey responses were received. All respondent units collect burn clinical data. Data are largely collected for quality improvement (69 %) and clinical research (50 %) purposes. Half of the institutions did not analyze their data, and a majority (67 %) did not benchmark their data against other datasets. The majority of respondents (93 %) demonstrated interest in contributing to a Canada-wide burn registry. CONCLUSION: Although all respondent units are currently collecting burn clinical data, there is an opportunity to improve data analysis, benchmarking, and knowledge translation. Most centers demonstrated interest in contributing to a novel Canadian burn registry.
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Queimaduras , Gerenciamento de Dados , Humanos , Canadá/epidemiologia , Queimaduras/epidemiologia , Queimaduras/terapia , Unidades de Queimados , Melhoria de Qualidade , Sistema de RegistrosRESUMO
The COVID-19 pandemic had widespread effects on the healthcare system due to public health regulations and restrictions. The following study shares trends observed during these extraordinary circumstances to investigate the impact of the COVID-19 pandemic on the provision of pediatric burn care at an American-Burn-Association verified tertiary pediatric hospital in Ontario, Canada. Pediatric burn patient data for new burn patients between March 17th, 2019, and March 17th, 2021, was retrospectively extracted and two cohorts of patients were formed: pre-pandemic and pandemic, through which statistical analysis was performed. No significant changes in the number of admitted patients, age, and sex of patients were observed. However, a significant increase in fire/flame burns was observed during the pandemic period. Additionally, a decrease in follow-up care was observed while an increase in acute burn care (wound care and surgical interventions) was found for the pandemic cohort. Despite changes to hospital care facilities to maximize resources for COVID-19-related care, our findings demonstrate that burn care remained an essential service and significant reductions in patient volumes were not observed. Overall, this study will aid in future planning and management for the provision of pediatric burn resources during similar public health emergencies.
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Queimaduras , COVID-19 , Criança , Humanos , Queimaduras/epidemiologia , Queimaduras/terapia , Estudos Retrospectivos , Pandemias , COVID-19/epidemiologia , Ontário/epidemiologia , Unidades de QueimadosRESUMO
Recent evidence has demonstrated that silver has anti-inflammatory properties that are independent from the known antimicrobial ones. In our current model of care, non-adherent, non-silver dressings are applied for acute presentations of pediatric partial thickness burn injuries. The wounds are re-assessed after the progression phase (48-72 hours after injury) and silver dressings are applied. However, when logistical obstacles prevent re-assessment within the 48-72-hour window, nanocrystalline silver-based dressings are applied on presentation. The objective of this study was to test our model of care. We hypothesized that immediate application (< 24 hours after injury) of nanocrystalline silver-based dressings would reduce surgical interventions. This was a retrospective single-center cohort study. All patients <18 years old treated at a pediatric burn center for acute partial thickness burn injuries, between January 1, 2020, and December 31, 2021 were included. Multivariable logistic regression was used to compare surgical treatment rates between patients with different timing of nanocrystalline silver-based dressing application. Four hundred seventy-six patients were included for analysis. One hundred four (21.8%) had nanocrystalline silver-based dressings and 372 (78.2%) had non-silver non-adherent dressings applied within 24 hours of injury. Multivariable logistic regression identified three statistically significant variables as predictors for surgical treatment: age (OR = 1.14, 95% CI [1.06-1.23]), total body surface area (OR = 1.15, 95% CI [1.06-1.25]), and burns to buttocks/lower extremity (OR = 2.39, 95% CI [1.26-4.53]). Immediate (< 24 hours after injury) application of nanocrystalline silver-based dressings does not affect surgical treatment rate in pediatric patients with partial thickness burns.
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Wound infection is the most common complication among pediatric burn patients. When not treated promptly, burn wound infection may lead to delayed healing, failure of skin grafts, or death. Standard burn wound assessment includes inspection for visual signs and symptoms of infection (VSSI) and microbial sampling. To aid in the assessment of burn wound infection, the MolecuLight, a point-of-care autofluorescence imaging device, was introduced at our pediatric burn program in 2020. The MolecuLight uses violet light to illuminate the wound bed, causing clinically relevant quantities of 29 different species of bacteria (>104 CFU/g) to fluoresce in real time. The objectives of this study were to evaluate the role of the MolecuLight in the management of pediatric burn wounds and determine if the findings from the MolecuLight corresponded to VSSI and/or microbial sampling. A retrospective review of patients 0-18 years who had burn wounds assessed with the MolecuLight between November 1, 2020 and June 8, 2023 was conducted. Data were extracted from the medical records of 178 eligible patients with 218 wounds imaged with the device. Fluorescence corresponded with VSSI in 81% of wounds and microbial findings in 82% of wounds. MolecuLight fluorescence, in combination with VSSI, improved sensitivity for detecting wound infections by 39% and decreased specificity by 19% compared to visual signs and symptoms in isolation. Incorporation of the MolecuLight in standard burn wound assessments can improve the detection of infections, which may promote improved wound healing outcomes and antimicrobial stewardship.
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Queimaduras , Imagem Óptica , Sistemas Automatizados de Assistência Junto ao Leito , Infecção dos Ferimentos , Humanos , Queimaduras/complicações , Infecção dos Ferimentos/microbiologia , Infecção dos Ferimentos/diagnóstico , Criança , Pré-Escolar , Lactente , Masculino , Feminino , Adolescente , Estudos Retrospectivos , Recém-NascidoRESUMO
Postburn pruritus is a significant issue that can have a devastating impact on patient quality of life. Despite its known negative impact, few studies have focused on the pediatric population. Thus, the aim of this study was to determine the incidence of pruritus among pediatric burn patients as well as identify its predictive factors and commonly used treatments, including the novel use of laser therapy. A retrospective analysis of all burn patients treated at our pediatric burn center from 2009 to 2017 was conducted. The primary outcome measure was the presence or absence of pruritus at any point following the burn. One thousand seven hundred and eighty-three patients met the inclusion criteria for this study. The mean age at injury was 3.67 years (SD = 4.02) and the mean burn TBSA was 3.48% (SD = 4.81) with most burns resulting from scalds (66%). In total, 665 patients (37.3%) experienced pruritus. Following multivariable logistic regression, TBSA, age >5 years, burns secondary to fire/flame, and burn depth, were identified as significant predictors of pruritus (P < .05). Pruritus was treated with diphenhydramine (85.0%), hydroxyzine (37.3%), and gabapentin (4.2%) as well as massage (45.7%), pressure garments (20.0%), and laser therapy (8.6%). This study addresses the knowledge gap in the literature related to postburn pruritus among pediatric patients and includes one of the largest patient cohorts published to date. Moreover, the results further contribute to our understanding of postburn pruritus in children and may help us to predict which patients are most likely to be affected, so that treatment can be initiated as soon as possible.
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Queimaduras , Qualidade de Vida , Queimaduras/complicações , Queimaduras/terapia , Criança , Pré-Escolar , Humanos , Incidência , Prurido/epidemiologia , Prurido/etiologia , Prurido/terapia , Estudos RetrospectivosRESUMO
Registry science allows for the interpretation of disease-specific patient data from secondary databases. It can be utilized to understand disease and injury, answer research questions, and engender benchmarking of quality-of-care indicators. Numerous burn registries exist globally, however, their contributions to burn care have not been summarized. The objective of this study is to characterize the available literature on burn registries. The authors conducted a scoping review, having registered the protocol a priori. A thorough search of the English literature, including grey literature, was carried out. Publications of all study designs were eligible for inclusion provided they utilized, analyzed, and/or critiqued data from a burn registry. Three hundred twenty studies were included, encompassing 16 existing burn registries. The most frequently used registries for peer-reviewed publications were the American Burn Association Burn Registry, Burn Model System National Database, and the Burns Registry of Australia and New Zealand. The main limitations of existing registries are the inclusion of patients admitted to burn centers only, deficient capture of outpatient and long-term outcome data, lack of data standardization across registries, and the paucity of studies on burn prevention and quality improvement methodology. Registries are an invaluable source of information for research, delivery of care planning, and benchmarking of processes and outcomes. Efforts should be made to stimulate other jurisdictions to build burn registries and for existing registries to be improved through data linkage with administrative databases, and by standardizing one international minimum dataset, in order to maximize the potential of registry science in burn care.
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Queimaduras , Austrália/epidemiologia , Unidades de Queimados , Queimaduras/epidemiologia , Queimaduras/terapia , Humanos , Melhoria de Qualidade , Sistema de RegistrosRESUMO
There have been significant improvements in the technology available for treating extensive burns in the past decade. This case presents two unique, skin replacement technologies that were used to treat an 86% surface area flame burn in a pediatric patient. A temporary dermal replacement, known as "Novosorb™ Biodegradable Temporizing Matrix" was first used to stabilize the burn injury and remained in place for approximately 3 months. Given the large burn size and lack of available donor skin for grafting, a permanent skin replacement product known as "Self-Assembled Skin Substitute (SASS)" was then utilized to cover the burns. SASS is a novel technology that was developed to replace skin as an autologous skin graft and is currently available in Canada through a clinical trial for major burns. Ultimately, the concurrent use of these two technologies allowed for the unprecedented survival of a child following an extensive and life-threatening burn injury.
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Queimaduras/terapia , Pele Artificial , Engenharia Tecidual/métodos , Transplante Autólogo , Sobrevivência de Enxerto , Humanos , Regeneração/fisiologia , Transplante de Pele , CicatrizaçãoRESUMO
Assessment and monitoring of inflammation and tissue damage is crucial in localized scleroderma (LS), but validated diagnostic tools are lacking. We aimed to determine the feasibility of using acoustic radiation force imaging ultrasound elastography in the assessment of pediatric-onset LS lesions. Conventional ultrasound and shear-wave elastography (SWE) imaging were used to characterize changes in pre-assigned LS lesions in 13 prospectively recruited participants. Contralateral sites were used as controls. Mean SWE values were compared. LS lesions were significantly stiffer than control sites in the dermis and the hypodermis using both parametric and non-parametric tests, before and after skin-thickness normalization. We show that SWE imaging is a feasible way to discriminate between normal skin and LS lesions in the pediatric population.
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Técnicas de Imagem por Elasticidade , Esclerodermia Localizada/diagnóstico por imagem , Adolescente , Criança , Pré-Escolar , Estudos Transversais , Estudos de Viabilidade , Feminino , Humanos , Masculino , Estudos ProspectivosRESUMO
The purpose of this study was to quantify the stiffness of hypertrophic scars using acoustic radiation force impulse ultrasound elastography. Sixteen pediatric patients with hypertrophic scars resulting from burn injuries participated in this study (mean age: 5.13, standard deviation: 3.20). Values for the elastic modulus (E) of scar and control sites were obtained. Scarred areas were found to be almost four times stiffer than control sites (scar Emeanâ¯=â¯39.29 kPa compared with control Emeanâ¯=â¯10.19 kPa) (pâ¯=â¯0.0004). Correlations between scar stiffness and clinician-reported subjective scar scale scores were not observed (rsâ¯=â¯0.30, pâ¯=â¯0.27 and rsâ¯=â¯0.25, pâ¯=â¯0.35 respectively). We found that acoustic radiation force impulse imaging can discriminate between hypertrophic scars and normal skin and should be considered a potentially valuable tool in the armamentarium of objective scar measures. Future research should focus on evaluating the technology's ability to detect scar change over time in order to determine responsiveness to treatment.
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Queimaduras/complicações , Cicatriz/diagnóstico por imagem , Cicatriz/fisiopatologia , Técnicas de Imagem por Elasticidade/métodos , Acústica , Criança , Pré-Escolar , Cicatriz/etiologia , Módulo de Elasticidade , Feminino , Humanos , Lactente , Estudos Longitudinais , MasculinoRESUMO
INTRODUCTION: Despite the fact that millions of scars affect individuals annually, little is known about their psychosocial impact and overall quality of life (QOL) on individuals. Scars from multiple aetiologies may cause psychiatric and emotional disturbances, can limit physical functioning and increase costs to the healthcare system. The purpose of this protocol is to describe the methodological considerations that will guide the completion of a scoping review that will summarise the extent, range and nature of psychosocial health outcomes and QOL of scars of all aetiologies. METHODS AND ANALYSIS: A modified Arksey and O'Malley (2005) framework will be completed, namely having ongoing consultation between experts from the beginning of the process, then (1) identifying the research question/s, (2) identifying the relevant studies from electronic databases and grey literature, with (3) study selection and (4) charting of data by two independent coders, and (5) collating, summarising and reporting data. Experts will include a health information specialist (TAW), scar expert (JSF), scoping review consultant (SCK), as well as at least two independent coders (NZ, AM). ETHICS AND DISSEMINATION: Ethics approval will not be sought for this scoping review. We plan to disseminate this research through publications, presentations and meetings with relevant stakeholders.
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Cicatriz/psicologia , Qualidade de Vida , Queimaduras/complicações , Humanos , Projetos de Pesquisa , Procedimentos Cirúrgicos Operatórios , Ferimentos e Lesões/complicaçõesRESUMO
PURPOSE: To conduct a systematic review of studies on non-surgical and surgical interventions for elbow flexion contractures secondary to brachial plexus birth injury (BPBI). METHODS: MEDLINE, EMBASE, PsycINFO, and CINAHL databases were searched for randomized controlled trials, observational studies, and case series studies on treatment of elbow flexion contractures secondary to BPBI. Study quality was evaluated using the Effective Public Health Practice Project tool. RESULTS: Of the 950 records found, 132 full text articles were reviewed, and 3 cohort studies and 8 case series were included. The overall methodological quality of included studies was weak. The weak quality evidence demonstrated that significant gains in elbow extension passive range of motion (ROM) can be achieved with serial casting (range: 15 to 34.5 degrees) or elbow release surgery (range: 28.4 to 30.0 degrees). At best, a reduction to an elbow contracture between -15.0 and -18.8 degrees (casting) and -8.0 and -43.6 (elbow release surgery) can be achieved. Insufficient outcomes on elbow flexion ROM and strength were found in both non-surgical and surgical studies. CONCLUSION: The quality of evidence on the effectiveness of interventions for an elbow flexion contracture secondary to BPBI is weak. In the context of insufficient evidence on the risks of pursuing such interventions, it is prudent to attempt non-surgical interventions prior to surgery. LEVEL OF EVIDENCE: III - systematic review of level IV studies.
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Tratamento Conservador/métodos , Contratura , Cotovelo , Paralisia do Plexo Braquial Neonatal/complicações , Procedimentos Ortopédicos/métodos , Contratura/etiologia , Contratura/reabilitação , Contratura/terapia , Humanos , Resultado do TratamentoRESUMO
Treatment with laser therapy has the potential to greatly improve hypertrophic scarring in individuals who have sustained burn injuries. More specifically, recent research has demonstrated the success of using pulsed dye laser therapy to help reduce redness and postburn pruritus and using ablative fractional CO2 laser therapy to improve scar texture and thickness. This study describes our early experience using laser therapy in our pediatric burn program and details our specific treatment approach when using each laser individually and in combination during the same procedure. A retrospective before-after study of patients with hypertrophic burn scars who were treated with laser therapy at our pediatric institution was performed. One hundred and twenty-five patients were treated over a total of 289 laser sessions with more than 50% of patients under the age of 5 years at the first treatment. The majority of procedures were performed using both the pulsed dye and CO2 lasers in combination. Before-after Vancouver Scar Scale scores decreased from 7.37 (SD, 2.46) to 5.76 (SD, 2.29) after a single treatment. The results obtained from this study support the use of laser therapy to improve hypertrophic burn scars in the pediatric population. Rigorous randomized controlled trials are needed to confirm the effectiveness of this therapy.
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Queimaduras/complicações , Cicatriz Hipertrófica/cirurgia , Terapia a Laser/métodos , Canadá , Criança , Pré-Escolar , Cicatriz Hipertrófica/etiologia , Terapia Combinada , Feminino , Humanos , Lactente , Lasers de Corante , Lasers de Gás , Masculino , Estudos RetrospectivosRESUMO
The objective of this systematic review was to assess the effectiveness of laser therapy for the treatment of hypertrophic burn scars. Improvements in scar symptoms following laser treatment were reported in 11 of the 12 included studies. However, issues related to overall quality and risk of bias were present in all studies. As a result, there is insufficient scientific evidence to determine the effectiveness of laser therapy from this systematic review. Future studies that use more rigorous study designs such as randomized controlled trials are needed to support the use of laser therapy for hypertrophic burn scars.
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Queimaduras/complicações , Cicatriz Hipertrófica/cirurgia , Terapia a Laser , Cicatriz Hipertrófica/etiologia , Humanos , Resultado do TratamentoRESUMO
The use of ablative fractional carbon dioxide laser therapy and pulsed dye laser therapy has led to significant improvements in the rehabilitation of hypertrophic burn scars. However, laser procedures are associated with appreciable pain among pediatric patients. Clinical consensus suggests using general anesthesia for pediatric laser procedures; however, guidelines for perioperative care are lacking. The objective of this quality improvement study is to determine whether a difference exists in postoperative pain outcomes in pediatric patients who receive intraoperative opioid regimens compared with patients who receive opioid-sparing regimens for laser therapy of hypertrophic burn scars. A retrospective review of patients who received laser therapy at a pediatric burn center from April 2014 to May 2015 was performed. Overall, 88 of the 92 procedures reviewed were included. A statistically significant difference was not found between the likelihood of postoperative pain when intraoperative opioid regimens (n = 63) were given compared with opioid-sparing regimens (n = 25) X (1, n = 88) = 2.870, P = .0902. There was also no difference between short-acting (n = 48), long-acting (n = 9), or combination (n = 6) intraoperative opioids compared with opioid-sparing regimens (n = 25) in the likelihood of postoperative pain. Despite the small sample size, the low number of postoperative pain cases is encouraging. Ultimately, these data provide a foundation for developing anesthetic guidelines for pediatric laser procedures. Specifically, clinicians should consider the potential to deliver adequate perioperative care via an opioid-sparing regimen ± adjuvant.
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Anestesia/métodos , Anestésicos/uso terapêutico , Queimaduras/complicações , Cicatriz Hipertrófica/cirurgia , Terapia a Laser/métodos , Adolescente , Biópsia por Agulha , Unidades de Queimados , Queimaduras/diagnóstico , Queimaduras/terapia , Canadá , Criança , Pré-Escolar , Cicatriz Hipertrófica/patologia , Cicatriz Hipertrófica/reabilitação , Estética , Feminino , Humanos , Imuno-Histoquímica , Lasers de Gás/uso terapêutico , Masculino , Medição da Dor , Segurança do Paciente , Assistência Perioperatória/métodos , Medição de Risco , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
Diffuse intrinsic pontine glioma (DIPG) is a fatal brain cancer that arises in the brainstem of children, with no effective treatment and near 100% fatality. The failure of most therapies can be attributed to the delicate location of these tumors and to the selection of therapies on the basis of assumptions that DIPGs are molecularly similar to adult disease. Recent studies have unraveled the unique genetic makeup of this brain cancer, with nearly 80% found to harbor a p.Lys27Met histone H3.3 or p.Lys27Met histone H3.1 alteration. However, DIPGs are still thought of as one disease, with limited understanding of the genetic drivers of these tumors. To understand what drives DIPGs, we integrated whole-genome sequencing with methylation, expression and copy number profiling, discovering that DIPGs comprise three molecularly distinct subgroups (H3-K27M, silent and MYCN) and uncovering a new recurrent activating mutation affecting the activin receptor gene ACVR1 in 20% of DIPGs. Mutations in ACVR1 were constitutively activating, leading to SMAD phosphorylation and increased expression of the downstream activin signaling targets ID1 and ID2. Our results highlight distinct molecular subgroups and novel therapeutic targets for this incurable pediatric cancer.
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Receptores de Ativinas Tipo I/genética , Neoplasias do Tronco Encefálico/genética , Regulação Neoplásica da Expressão Gênica/genética , Genoma Humano/genética , Glioma/genética , Animais , Neoplasias do Tronco Encefálico/classificação , Criança , Variações do Número de Cópias de DNA , Metilação de DNA , Perfilação da Expressão Gênica , Glioma/classificação , Humanos , Proteína 1 Inibidora de Diferenciação/metabolismo , Proteína 2 Inibidora de Diferenciação/metabolismo , Fosforilação , Análise de Sequência de DNA , Proteínas Smad/metabolismo , Peixe-ZebraRESUMO
BACKGROUND: This study aimed to obtain hemodynamic measurements of nesiritide in children with dilated cardiomyopathy. METHODS: A prospective, randomized, double-blinded, placebo-controlled pilot study was conducted in the pediatric intensive care unit at the University of California, Los Angeles. All subjects younger than 21 years admitted to the pediatric intensive care unit with a diagnosis of dilated cardiomyopathy and submitted to cardiac catheterization were randomized to receive either nesiritide or placebo. Right heart catheterization with Swan-Ganz catheter placement was performed. Nesiritide was infused over 24 h. Hemodynamic data were obtained before, during, and after the 24-h nesiritide infusion. The measures obtained included pulmonary capillary wedge pressure (PCWP), central venous pressure, mean pulmonary arterial pressure (MPAP), systolic arterial blood pressure (SBP), cardiac index, and systemic vascular resistance. RESULTS: The study included 20 children: 9 randomized to nesiritide and 11 to placebo. At 24 h, the mean decreases in PCWP, MPAP, and SBP were significantly greater for nesiritide than for placebo: PCWP (-5.3 vs. 1.2 mmHg; p = 0.02), MPAP (-8.0 vs. 0.4 mmHg; p = 0.006), SBP (-7.9 vs. 2.6 mmHg; p = 0.04). CONCLUSIONS: Nesiritide significantly decreases PCWP, MPAP, and SBP in children with dilated cardiomyopathy.