RESUMEN
The prognosis of relapsed/refractory (R/R) neuroblastoma (NB) is dismal, calling for new therapeutic strategies. Venetoclax (VEN) is a highly selective, potent, orally bioavailable, BCL-2 inhibitor small-molecule that showed a synergistic effect with cyclophosphamide and topotecan (Cy-Topo) in murine NB models. Our aim was to evaluate the feasibility of VEN plus Cy-Topo in children with R/R NB. Four patients, who had previously failed > 3 lines of treatment, were treated with VEN plus Cy-Topo based on a 28-day schedule in an outpatient setting. BCL-2 expression in immunochemistry on tumor samples at relapse and the BCL2 gene status was evaluated in all patients. The main toxicity was hematological, with grade 4 neutropenia and thrombocytopenia occurring in all courses and leading to transient VEN discontinuation. Grade 3 oral mucositis was observed in 1/8 courses. No other grade 2-4 toxicities were observed. BCL-2 was expressed in all tumors, while no molecular abnormalities in the BCL-2 genes were detected. A stable disease was observed in all patients, without any progression during the study period. VEN plus Cy-Topo is well tolerated, with encouraging results that may be improved by testing the schedule in less advanced patients.
Asunto(s)
Neoplasias Primarias Secundarias , Neuroblastoma , Niño , Humanos , Animales , Ratones , Topotecan , Recurrencia Local de Neoplasia/etiología , Ciclofosfamida/uso terapéutico , Neuroblastoma/patología , Compuestos Bicíclicos Heterocíclicos con Puentes/uso terapéutico , Proteínas Proto-Oncogénicas c-bcl-2/genética , Neoplasias Primarias Secundarias/etiología , Enfermedad Crónica , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversosRESUMEN
Neuroblastic tumors (NTs) represent the most common extracranial neoplasm occurring in childhood. Although ganglioneuroblastoma intermixed (GNBI) and ganglioneuroma (GN) are classified as very low-risk tumors, neuroblastoma (NB) and ganglioneuroblastoma-nodular (GNBN) may represent a serious risk to survival. Unfortunately, areas of GNBI and GNBN can coexist in the same mass, leading to incorrect risk staging when only biopsy is performed. Herein, we describe a case of multifocal NT (thoracic and abdominal localization) occurring in a 4-year-old male. Different histological subtypes, namely GNBI and GNBN, were revealed in the two lesions. We focus on the difficulties of proper diagnosis and risk stratification, underlining the usefulness of several diagnostic tools for appropriate management and therapeutic choices.
RESUMEN
BACKGROUND: Lung ultrasound (US) in the evaluation of suspected pediatric pneumonia is increasingly used and has a recognized role in evaluating pleural effusions, although there are no detailed studies specifically addressing its use in the pediatric population. OBJECTIVES: To define lung US findings of severe pediatric community-acquired pneumonia that required surgical procedures during admission. MATERIALS AND METHODS: Our prospective case-control study compared lung US findings in patients ages 1 month to 17 years admitted with community-acquired pneumonia that required surgical procedures from findings those who did not. Lung US was performed at admission and always before surgical procedures. Medical treatment, laboratory and microbiological findings, chest X-ray, computed tomography scan and surgical procedures are described. RESULTS: One hundred twenty-one children with community-acquired pneumonia were included; of these, 23 underwent surgical intervention. Compared with the control group, children requiring a surgical procedure had a significantly higher rate of large consolidations (52.2%; 95% confidence interval [CI]: 30.6% to 73.2%), larger and complicated pleural effusions (100%; 95% CI: 85.2% to 100%), and both liquid and air bronchograms (73.9%; 95% CI: 51.6% to 89.8%). CONCLUSION: Larger consolidations, larger and more complicated pleural effusions, and liquid and air bronchograms were associated with surgical treatment.
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Infecciones Comunitarias Adquiridas/diagnóstico por imagen , Derrame Pleural/diagnóstico por imagen , Neumonía/diagnóstico por imagen , Ultrasonografía/métodos , Adolescente , Estudios de Casos y Controles , Niño , Preescolar , Infecciones Comunitarias Adquiridas/cirugía , Femenino , Humanos , Lactante , Masculino , Derrame Pleural/cirugía , Neumonía/cirugía , Estudios ProspectivosRESUMEN
INTRODUCTION AND OBJECTIVES: Patients with neuromuscular disease (NMD) are often exposed to ionizing radiations which could be reduced if a noninvasive and reliable diagnostic method is identified. The major aim of this study was to compare the use of chest X-ray (CXR) with lung ultrasound (LUS) in pediatric patients with NMD, to identify pulmonary atelectasis (PA). MATERIALS AND METHODS: A prospective study was conducted on children affected by NMD. In all patients who underwent CXR, a LUS was also performed and results compared for the assessment of PA. RESULTS: Forty children affected by NMD were enrolled. Spinal muscular atrophy type 1 was the most common NMD, followed by spinal muscular atrophy type 2 and congenital myopathies. More than half of the subjects presented PA, more prevalent in the lung left lower lobes. LUS agreed with CXR results (negative or positive for atelectasis) in 31 patients (77.5%). In two patients, an agreement evaluation was not possible due to non-conclusive LUS. Conversely, LUS disagreed with CXR in seven patients, four of which were positive for atelectasis. If only complete agreement was considered, the statistical analysis between CXR and LUS showed: LUS sensitivity of 57%, LUS specificity of 82%, positive predictive value 80%, negative predictive value 61%. CONCLUSIONS: This study suggests that the use of LUS should be recommended to early identify PA and reduce frequent ionizing exposition of these fragile patients. Finally, our study also suggests that LUS can provide relevant information for clinicians and respiratory physiotherapists.
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Pulmón/diagnóstico por imagen , Enfermedades Neuromusculares/diagnóstico por imagen , Atelectasia Pulmonar/diagnóstico por imagen , Niño , Preescolar , Femenino , Humanos , Masculino , Estudios Prospectivos , Radiografía , UltrasonografíaRESUMEN
Neuroblastoma is the most common extracranial solid tumor in children and accounts for 8% to 10% of all childhood cancers. The spread of metastases is both lymphatic and hematogenous. Hematogenous route occurs most commonly in bone marrow and cortical bone; pancreatic metastases are very rare, and there are few reports about this topic in medical literature. We report the case of a 14-year-old boy who suffers a third relapse of neuroblastoma: in this patient I-MIBG confirms that lesions detected by CT were due to a relapse in pancreatic tissue and influenced subsequent therapeutic management.
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3-Yodobencilguanidina , Neuroblastoma/patología , Neoplasias Pancreáticas/diagnóstico por imagen , Neoplasias Pancreáticas/secundario , Tomografía Computarizada por Tomografía Computarizada de Emisión de Fotón Único , Adolescente , Médula Ósea/diagnóstico por imagen , Médula Ósea/patología , Humanos , Masculino , Neuroblastoma/terapia , RecurrenciaRESUMEN
Fungal infections of the lungs are relatively common and potentially life-threatening conditions in immunocompromised children. The role of imaging in children with lung mycosis is to delineate the extension of pulmonary involvement, to assess response to therapy, and to monitor for adverse sequelae such as bronchiectasis and cavitation. The aim of this paper is to show imaging findings in a series of patients with fungal pneumonia from two tertiary children's hospitals, to discuss differential diagnoses and to show how imaging findings can vary depending on the host immune response.
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Enfermedades Pulmonares Fúngicas/diagnóstico por imagen , Enfermedades Pulmonares Fúngicas/microbiología , Niño , Diagnóstico Diferencial , Humanos , Huésped InmunocomprometidoRESUMEN
INTRODUCTION: Hepatic regenerating nodules (HRN) and focal nodular hyperplasia (FNH) are benign regenerating lesions of the liver that rarely occur in children. An increased incidence of these lesions is reported in children treated for cancer. MATERIAL AND METHODS: Eight children who developed FNH and HRN after treatment for malignancies in the Oncology unit at the "Bambino Gesù" Pediatric Hospital in Rome, were retrospectively analyzed. RESULTS: The lesions, considered in the differential diagnosis with metastatic relapse of the primitive disease, have been monitored with US or other available imaging techniques. Evolution of the lesions was observed in only 1 patient three years after the initial diagnosis of FNH. CONCLUSION: In conclusion serial monitoring with imaging techniques is sufficient to rule out liver metastasis and to monitor the evolution of the lesions. Surgery is suggested only in the case of complications.
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Antineoplásicos/efectos adversos , Diagnóstico por Imagen , Hiperplasia Nodular Focal/diagnóstico , Neoplasias Hepáticas/diagnóstico , Regeneración Hepática , Sobrevivientes , Adolescente , Factores de Edad , Niño , Preescolar , Diagnóstico por Imagen/métodos , Femenino , Hiperplasia Nodular Focal/inducido químicamente , Hiperplasia Nodular Focal/cirugía , Humanos , Neoplasias Hepáticas/inducido químicamente , Neoplasias Hepáticas/cirugía , Imagen por Resonancia Magnética , Masculino , Valor Predictivo de las Pruebas , Pronóstico , Estudios Retrospectivos , Factores de Riesgo , Ciudad de Roma , Factores de Tiempo , Tomografía Computarizada por Rayos X , Ultrasonografía Doppler en ColorRESUMEN
BACKGROUND: Acoustic radiation force impulse (ARFI) is a novel technique for the measurement of hepatic stiffness, which could be valuable in clinical follow-up of patients affected by cystic fibrosis liver disease (CFLD). METHODS: Seventy-five patients with suspected CFLD (35 males) underwent clinical and ultrasonographic evaluations, liver and pulmonary function tests, ARFI investigation, and upper gastrointestinal endoscopy. Ten ARFI measurements were taken at the deep right hepatic lobe to compute median values of Shear Wave Velocity (SWV) for each individual. RESULTS: SWV increased progressively from 1.02m/s (95%, Confidence Interval, CI, 0.92-1.126) in patients with no evidence of CFLD at ultrasonography (N=16), to 1.12 (95%CI 1.049-1.19) in patients with CFLD and no signs of portal hypertension (PHT, N=23), and to 1.25 (95%CI 1.14-1.358) in those with CFLD and signs of PHT (N=28). SWV was 1.63 (95%CI 1.26-1.99) in patients with oesophageal varices (N=8) (p<0.0001). CONCLUSIONS: ARFI may represent an easy, fast and non-invasive tool for the clinical follow-up of patients with cystic fibrosis associated liver disease.