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Sarcomas are a rare and highly diverse group of malignancies of mesenchymal origin. While sarcomas are generally considered resistant to immunotherapy, recent studies indicate subtype-specific differences in clinical response to checkpoint inhibitors (CPIs) that are associated with distinct immune phenotypes present in sarcoma subtypes. Oncolytic viruses (OVs) are designed to selectively infect and kill tumor cells and induce intratumoral immune infiltration, enhancing immunogenicity and thereby sensitizing tumors to immunotherapy. Herein we review the accumulated clinical data evaluating OVs in sarcoma. Small numbers of patients with sarcoma were enrolled in early-stage OV trials as part of larger solid tumor cohorts demonstrating safety but providing limited insight into the biological effects due to the low patient numbers and lack of histologic grouping. Several recent studies have investigated talimogene laherparepvec (T-VEC), an approved oncolytic herpes simplex virus (HSV-1), in combination therapy regimens in sarcoma patient cohorts. These studies have shown promising responses in heavily pre-treated and immunotherapy-resistant patients associated with increased intratumoral immune infiltration. As new and more potent OVs enter the clinical arena, prospective evaluation in subtype-specific cohorts with correlative studies to define biomarkers of response will be critical to advancing this promising approach for sarcoma therapy.
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BACKGROUND: Radiation-associated soft tissue sarcomas (RA-STS) are rare complications of patients receiving radiation therapy (RT) and are generally associated with a poor prognosis. Most of the literature surrounding RA-STS of the chest is centered on angiosarcoma. Therefore, we aim to document the management and outcome of patients with non-angiosarcoma RA-STS of the chest. METHODS: We reviewed 17 patients (all female, median age 65 years) diagnosed with RA-STS. The most common primary malignancy was breast carcinoma (n = 15), with a median RT dose of 57.9 Gy. All patients underwent surgical resection; five patients (29%) received radiotherapy; and five patients (29%) received peri-operative chemotherapy. RESULTS: The 5-year local recurrence and metastatic-free survival were 61% and 60%, while the 5-year disease-specific survival was 53%. Local recurrence was associated with death due to disease (HR 9.06, p = 0.01). Complications occurred in nine of patients, most commonly due to a wound complication (n = 7). At the most recent follow-up, the median Musculoskeletal Tumor Society Score was 63%. CONCLUSION: RA-STS involving the chest wall are aggressive tumors with a high risk of local relapse and death due to disease. Local recurrence was associated with death due to disease; as such, we recommend aggressive surgical management with evaluation for adjuvant therapies.
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Recurrencia Local de Neoplasia , Sarcoma , Humanos , Femenino , Anciano , Persona de Mediana Edad , Sarcoma/radioterapia , Sarcoma/patología , Sarcoma/mortalidad , Sarcoma/terapia , Sarcoma/cirugía , Recurrencia Local de Neoplasia/patología , Neoplasias Inducidas por Radiación/patología , Neoplasias Inducidas por Radiación/mortalidad , Neoplasias Inducidas por Radiación/etiología , Neoplasias Inducidas por Radiación/cirugía , Anciano de 80 o más Años , Estudios Retrospectivos , Adulto , Neoplasias Torácicas/radioterapia , Neoplasias Torácicas/patología , Neoplasias Torácicas/mortalidad , Pared Torácica/patología , Pared Torácica/efectos de la radiación , Estudios de Seguimiento , Neoplasias de los Tejidos Blandos/radioterapia , Neoplasias de los Tejidos Blandos/mortalidad , Neoplasias de los Tejidos Blandos/patología , Neoplasias de los Tejidos Blandos/terapia , Neoplasias de los Tejidos Blandos/cirugía , Neoplasias de la Mama/patología , Neoplasias de la Mama/radioterapia , Neoplasias de la Mama/mortalidad , Neoplasias de la Mama/terapiaRESUMEN
Measles virus (MV) vaccine strains have shown significant preclinical antitumor activity against glioblastoma (GBM), the most lethal glioma histology. In this first in human trial (NCT00390299), a carcinoembryonic antigen-expressing oncolytic measles virus derivative (MV-CEA), was administered in recurrent GBM patients either at the resection cavity (Group A), or, intratumorally on day 1, followed by a second dose administered in the resection cavity after tumor resection on day 5 (Group B). A total of 22 patients received study treatment, 9 in Group A and 13 in Group B. Primary endpoint was safety and toxicity: treatment was well tolerated with no dose-limiting toxicity being observed up to the maximum feasible dose (2×107 TCID50). Median OS, a secondary endpoint, was 11.6 mo and one year survival was 45.5% comparing favorably with contemporary controls. Other secondary endpoints included assessment of viremia, MV replication and shedding, humoral and cellular immune response to the injected virus. A 22 interferon stimulated gene (ISG) diagonal linear discriminate analysis (DLDA) classification algorithm in a post-hoc analysis was found to be inversely (R = -0.6, p = 0.04) correlated with viral replication and tumor microenvironment remodeling including proinflammatory changes and CD8 + T cell infiltration in post treatment samples. This data supports that oncolytic MV derivatives warrant further clinical investigation and that an ISG-based DLDA algorithm can provide the basis for treatment personalization.
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Glioblastoma , Viroterapia Oncolítica , Virus Oncolíticos , Humanos , Virus del Sarampión/genética , Antígeno Carcinoembrionario/genética , Recurrencia Local de Neoplasia/terapia , Vacuna Antisarampión , Microambiente TumoralRESUMEN
BACKGROUND: In the United States, rare disease (RD) is defined as a condition that affects fewer than 200,000 individuals. Collectively, RD affects an estimated 30 million Americans. A significant portion of RD has an underlying genetic cause; however, this may go undiagnosed. To better serve these patients, the Mayo Clinic Program for Rare and Undiagnosed Diseases (PRaUD) was created under the auspices of the Center for Individualized Medicine (CIM) aiming to integrate genomics into subspecialty practice including targeted genetic testing, research, and education. METHODS: Patients were identified by subspecialty healthcare providers from 11 clinical divisions/departments. Targeted multi-gene panels or custom exome/genome-based panels were utilized. To support the goals of PRaUD, a new clinical service model, the Genetic Testing and Counseling (GTAC) unit, was established to improve access and increase efficiency for genetic test facilitation. The GTAC unit includes genetic counselors, genetic counseling assistants, genetic nurses, and a medical geneticist. Patients receive abbreviated point-of-care genetic counseling and testing through a partnership with subspecialty providers. RESULTS: Implementation of PRaUD began in 2018 and GTAC unit launched in 2020 to support program expansion. Currently, 29 RD clinical indications are included in 11 specialty divisions/departments with over 142 referring providers. To date, 1152 patients have been evaluated with an overall solved or likely solved rate of 17.5% and as high as 66.7% depending on the phenotype. Noteworthy, 42.7% of the solved or likely solved patients underwent changes in medical management and outcome based on genetic test results. CONCLUSION: Implementation of PRaUD and GTAC have enabled subspecialty practices advance expertise in RD where genetic counselors have not historically been embedded in practice. Democratizing access to genetic testing and counseling can broaden the reach of patients with RD and increase the diagnostic yield of such indications leading to better medical management as well as expanding research opportunities.
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Enfermedades Raras , Enfermedades no Diagnosticadas , Estados Unidos , Humanos , Enfermedades Raras/diagnóstico , Enfermedades Raras/genética , Enfermedades Raras/terapia , Atención Terciaria de Salud , Medicina Genómica , Pruebas Genéticas , Asesoramiento GenéticoRESUMEN
The treatment of sarcoma necessitates a collaborative approach, given its rarity and complex management. At a single institution, multidisciplinary teams of specialists determine and execute treatment plans involving surgical, radiation, and medical management. Treatment guidelines for systemic therapies in advanced or nonresectable soft tissue sarcoma have advanced in recent years as new immunotherapies and targeted therapies become available. Collaboration between institutions is necessary to facilitate accrual to clinical trials. Here, we describe the success of the Midwest Sarcoma Trials Partnership (MWSTP) in creating a network encompassing large academic centers and local community sites. We propose a new model utilizing online platforms to expand the reach of clinical expertise for the treatment of advanced soft tissue sarcoma.
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BACKGROUND: Extraskeletal Ewing sarcoma are rare tumors within the Ewing sarcoma family. Initial staging studies for extraskeletal Ewing sarcoma historically have included imaging and bone marrow aspiration and biopsy (BMAB). However, recent studies on Ewing sarcoma of bone have questioned the utility of BMAB in the initial staging of patients, but no studies of which we are aware have evaluated the role of BMAB in extraskeletal Ewing sarcoma. We suspected that BMAB was of low diagnostic yield in patients with extraskeletal Ewing sarcoma and exposed patients to potential morbidity without an impact on their clinical course. QUESTION/PURPOSE: Is BMAB a useful test in the staging of extraskeletal Ewing sarcoma? METHODS: Between January 1996 and December 2021, our institution evaluated 109 patients with a listed diagnosis of extraskeletal Ewing sarcoma. Those patients were retrospectively reviewed for this study. Of those, we considered patients with biopsy-confirmed diagnosis of extraskeletal Ewing sarcoma. Biopsy was performed based on institutional protocols, with all diagnoses assigned by a board-certified pathologist. Based on that criteria, 96% (105 of 109) were eligible. An additional 18% (20 of 109) were excluded because records of their initial diagnostic and staging workup were not available. This left 78% (85 of 109) for analysis. Of those, 52% (44 of 85) were male. The average age was 32 ± 16 years. Primary tumor locations included extremities in 26% (22 of 85), paraspinal in 20% (17 of 85), chest in 19% (16 of 85), retroperitoneum in 13% (11 of 85), intraabdominal in 12% (10 of 85), intrapelvic in 7% (6 of 85), and head or neck in 4% (3 of 85). Initial diagnostic and staging information, including the use of PET-CT, bone scan, CT chest, and BMAB, was collected. Metastatic disease at the time of presentation or during follow-up was noted. The utility of BMAB was determined by the rate of positive tests in those undergoing BMAB during the initial staging process. Descriptive statistical analysis was sufficient to address the study question, and therefore no comparative statistics were performed. RESULTS: BMAB was obtained during the initial staging process in 64% (54 of 85) of patients. This BMAB was negative in all 54 patients, including those with known metastatic disease. CONCLUSION: Diagnosing metastatic disease in extraskeletal Ewing sarcoma is important as the presence of metastases at diagnosis adversely affects prognosis. The routine use of BMAB in the staging process of extraskeletal Ewing sarcoma is of low diagnostic yield. BMAB is unlikely to diagnose metastatic involvement even in patients with known metastases to bone. We do not have enough data to suggest whether other modalities, such as PET-CT, might be more useful. Similar studies should be pursued to determine the utility of the remainder of staging modalities in patients with extraskeletal Ewing sarcoma to elucidate the most efficient and effective staging protocol. LEVEL OF EVIDENCE: Level III, diagnostic study.
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Neoplasias Óseas , Sarcoma de Ewing , Humanos , Masculino , Adolescente , Adulto Joven , Adulto , Persona de Mediana Edad , Femenino , Sarcoma de Ewing/diagnóstico por imagen , Sarcoma de Ewing/patología , Neoplasias Óseas/patología , Tomografía Computarizada por Tomografía de Emisión de Positrones/métodos , Médula Ósea/patología , Estudios Retrospectivos , Relevancia Clínica , Biopsia , Tomografía Computarizada por Rayos X , Estadificación de NeoplasiasRESUMEN
PURPOSE: Extraskeletal Ewing sarcoma (EES), is a rare soft tissue sarcoma. Treatment for EES commonly involves chemotherapy and surgical resection (ST) or less commonly combined chemotherapy, surgery, and radiotherapy (ST + RT). The purpose of the current study was to evaluate our institutional experience treating EES. METHODS: We reviewed 36 (18 males:18 females) patients (mean age 30 years) with a nonretroperitoneal/visceral EES treated with either ST (n = 24, 67%) or ST + RT (n = 12, 33%). All patients were treated with chemotherapy, most commonly vincristine, doxorubicin, cyclophosphamide/ifosfamide and etoposide (VDC/IE, n = 23, 66%) Radiotherapy was mostly delivered preoperatively (n = 9). The mean follow-up was 8 years. RESULTS: The 10-year disease specific survival for patients was 78%, with no difference in the survival between patients in the ST versus the ST + RT groups (83% vs. 71%, p = 0.86). There was no difference in the 10-year local recurrence (91% vs. 100%, p = 0.29) or metastatic free survival (87% vs. 75%, p = 0.45) between the ST and ST + RT groups. CONCLUSION: The results of the current study highlight the ability to achieve excellent local control with chemotherapy and surgery for EES. We recommend for multidisciplinary management of patients with EES, including chemotherapy and surgery, with use of radiotherapy if there is concern for a potentially close margin of resection.
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Neoplasias Óseas , Sarcoma de Ewing , Sarcoma , Adulto , Femenino , Humanos , Masculino , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Ciclofosfamida , Doxorrubicina , Etopósido/uso terapéutico , Sarcoma/tratamiento farmacológico , Sarcoma de Ewing/terapia , Sarcoma de Ewing/patología , Resultado del Tratamiento , Vincristina/uso terapéuticoRESUMEN
BACKGROUND: Compared to other sarcomas, myxoid liposarcoma (ML) is known to be radiosensitive, with improved oncologic outcomes. Although these tumors "shrink" following radiotherapy, there is a paucity of data examining the degree of radiosensitivity and oncologic outcome. The purpose of the study was to evaluate pre- and postradiotherapy tumor volume to determine if size reduction impacts outcome. METHODS: We reviewed 62 patients with ML undergoing surgical resection combined with preoperative radiotherapy, with pre- and postradiotherapy MRI. This included 34 (55%) males, with a mean age of 47 ± 14 years. All tumors were deep to the fascia, and 12 (19%) patients had tumors with a >5% round-cell component. RESULTS: The mean volume reduction was 54% ± 29%. Compared to patients with >25% volume reduction, patients with reduction ≤25% had worse 10-year disease specific survival (86% vs. 37%, p < 0.01), in addition to an increased risk of metastatic disease (HR 4.63, p < 0.01) and death due to disease (HR 4.52, p < 0.01). CONCLUSION: Lack of volume reduction is a risk factor for metastatic disease and subsequent death due to disease in patients with extremity ML treated with combined preoperative radiotherapy and surgery. This data could be used to stratify patients for adjuvant therapies and follow-up intervals.
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Liposarcoma Mixoide , Liposarcoma , Sarcoma , Adulto , Femenino , Humanos , Masculino , Persona de Mediana Edad , Terapia Combinada , Extremidades/patología , Liposarcoma/patología , Liposarcoma Mixoide/radioterapia , Liposarcoma Mixoide/cirugía , Estudios Retrospectivos , Resultado del TratamientoRESUMEN
BACKGROUND: There are several modifications in the technique for the placement of mesh during laparoscopic inguinal hernia repair. One of these is a slit on the mesh to surround the cord structures which proponents of it suggest that it decreases the recurrence rate due to better anchoring of the mesh and lower risk of displacement. There is only low-level evidence in current literature examining the two methods. The present study aimed to provide stronger evidence in establishing whether the slit mesh technique is superior to the non-slit mesh technique. METHODS: The reporting of this systematic review was guided by the standards of the preferred reporting items for systematic review and meta-analysis statement and registered with PROSPERO (ID: CRD42022300629). Eligible studies had to compare the two methods of mesh placement slit Vs nonslit in laparoscopic Inguinal hernia repair and also report on at least one outcome. The outcomes were expressed in odd ratios with their 95% confidence intervals. Where significant heterogeneity existed a random effects model was used otherwise a fixed effects model was used. RESULTS: Five studies met the criteria for inclusion in quantitative analysis. Overall, there were 10 (1.5%) recurrences in the slit group compared to 12 (2.5%) in the non-slit group OR 0.62, 95% CI (0.27-1.41). There was no difference in the incidence of post-operative bleeding (OR 1.21, 95%CI 0.4-3.66), seroma formation (OR1.5, 95% CI 0.81-2.76), or post-operative neuralgia (OR 0.98, 95%CI 0.11-8.92) between the two groups. CONCLUSION: There was no difference between the two methods.
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Hernia Inguinal , Laparoscopía , Humanos , Mallas Quirúrgicas , Hernia Inguinal/cirugía , Herniorrafia/métodos , Laparoscopía/métodos , Seroma/cirugía , RecurrenciaRESUMEN
The coronavirus disease 2019 (COVID-19) pandemic had a significant impact on elective surgery for benign disease. We examined the effects of COVID-19 related delays on the outcomes of patients undergoing elective laparoscopic cholecystectomy (LC) in an upper gastrointestinal surgery unit in the UK. We have analysed data retrospectively of patients undergoing elective LC between 01/03/2019 to 01/05/2019 and 01/04/2021 to 11/06/2021. Demographics, waiting time to surgery, intra-operative details and outcome data were compared between the two cohorts. Indications for surgery were grouped as inflammatory (acute cholecystitis, gallstone pancreatitis, CBD stone with cholangitis) or non-inflammatory (biliary colic, gallbladder polyps, CBD stone without cholangitis). A p value of <0.05 was used for statistical significance. Out of the 159 patients included, 106 were operated pre-pandemic and 53 during the pandemic recovery phase. Both groups had similar age, gender, ASA-grades and BMI. In the pre-pandemic group, 68 (64.2%) were operated for a non-inflammatory pathology compared to 19 (35.8%) from the recovery phase cohort (p < 0.001). The waiting time to surgery was significantly higher amongst patients operated during the recovery phase (p = 0000.1). Less patients had complete cholecystectomy during the pandemic recovery phase (p = 0.04). There were no differences in intraoperative times and patient outcomes. These results demonstrate the impact of COVID-19 related delays to our cohort, however due to the retrospective nature of this study, the current results need to be backed up by higher evidence in order for strong recommendations to be made.
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Background The coronavirus pandemic has caused global disruption to all aspects of life. This disturbance has been most notable in the medical world. Political, societal, medical, and behavioral alterations have forced emergency surgical practices to adapt. This study investigated the impact of coronavirus 2019 (COVID-19) at a busy surgical center. Methodology This is a retrospective observational study. Three study periods were analyzed: pre-COVID, first wave, and second wave. Data were collected on referrals, diagnoses, investigations, management pathways, outcomes, patient behavior, and consultant practice. A one-way analysis of variance (ANOVA test) was used for the analysis of parametric data and the Mann-Whitney U test for non-parametric data. Results Declining numbers of patients presented across the three periods. There was a severe disruption in performing emergency general surgeries during the first wave, propagated by alterations in clinical decision-making, as well as fluctuations in societal and patient behavior. Despite the effects of the second wave being significantly more profound in terms of hospitalization and COVID-related mortality, a paradoxical, gradual return to the norm was noted, which was seen in referral pathways, imaging decisions, and management strategies. Conclusion Our data is suggestive of society, both within and outside the medical sphere, adjusting to life with COVID-19.
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Radiation-associated angiosarcoma of the breast (RAASB) is a rare and aggressive malignancy occurring after radiation therapy as part of breast cancer treatment. RAASB usually presents several years after prior radiation and typically involves the skin with or without involvement of the parenchyma. Most RAASB are detected as cutaneous changes on physical exam. Herein, we present a unique case of a clinically occult RAASB diagnosed as non-mass enhancement on annual surveillance breast MRI.
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Hodgkin lymphoma (HL) patients who relapse after autologous-stem-cell- transplantation (auto-SCT) have traditionally had a poor prognosis. We analyzed 1781 adult HL patients who relapsed between 2006 and 2017 after a first auto-SCT. The 4-year overall survival (OS) after relapse continuously increased from 32% for patients relapsing in 2006-2008, to 63% for patients relapsing in 2015-2017 (p = 0.001). The improvement over time was predominantly noted in patients who had an early relapse (within 12 months) after auto-SCT (p = 0.01). On multivariate analysis, patients who relapsed in more recent years and those with a longer interval from transplant to relapse had a better OS, whereas increasing age, poor performance status, bulky disease, extranodal disease and presence of B symptoms at relapse were associated with a worse OS. Brentuximab vedotin (BV), checkpoint inhibitors (CPI) and second transplant (SCT2; 86% allogeneic) were used in 233, 91 and 330 patients respectively. The 4-year OS from BV, CPI, and SCT2 use was 55%, 48% and 55% respectively. In conclusion, the outcome after post-transplant relapse has improved significantly in recent years, particularly in the case of early relapse. These large-scale real-world data can serve as benchmark for future studies in this setting.
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Trasplante de Células Madre Hematopoyéticas , Enfermedad de Hodgkin , Inmunoconjugados , Adulto , Brentuximab Vedotina , Enfermedad de Hodgkin/terapia , Humanos , Inmunoconjugados/efectos adversos , Recurrencia Local de Neoplasia/inducido químicamente , Recurrencia Local de Neoplasia/terapia , Estudios Retrospectivos , Trasplante AutólogoRESUMEN
BACKGROUND: Sarcomas are rare diagnoses but are seen with relative frequency in adolescents and young adults and thus can present in pregnancy. We sought to study the administration of anthracyclines and/or ifosfamide in pregnancy-associated sarcomas. PATIENTS AND METHODS: We conducted a multi-institutional retrospective study, identifying sarcoma patients who received anthracyclines and/or ifosfamide during pregnancy. Chart review identified variables related to demographics, cancer diagnosis, therapies, and outcome of the patient and fetus. Wilcoxon rank-sum test compared two independent samples. RESULTS: We identified 13 patients at seven institutions with sarcoma who received anthracyclines and/or ifosfamide during pregnancy, including four bone sarcomas and nine soft tissue sarcomas diagnosed at a mean gestational age of 16.7 ± 5.9 weeks. Only nine patients had live births (9/13, 69.2%), with mean gestational age of 30.8 ± 3.8 weeks at delivery. The four patients with pregnancy loss all received both doxorubicin and ifosfamide, with chemotherapy initiated at 15.5 weeks as compared with 21.3 weeks for those patients with live births (p = 0.016). CONCLUSION: In this multi-institutional study of sarcoma chemotherapy regimens administered during pregnancy, we found a high rate of fetal demise that was seen only in patients receiving both doxorubicin and ifosfamide and statistically more likely with chemotherapy initiation earlier in the second trimester. While limited by a small sample size, our study represents the largest study of sarcoma patients that received anthracyclines and/or ifosfamide in pregnancy thus far reported and supports development of an international registry to study concerns raised by our study.
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Sarcoma , Neoplasias de los Tejidos Blandos , Adolescente , Antraciclinas/efectos adversos , Antibióticos Antineoplásicos/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Doxorrubicina/uso terapéutico , Femenino , Humanos , Ifosfamida/efectos adversos , Lactante , Embarazo , Resultado del Embarazo , Estudios Retrospectivos , Sarcoma/tratamiento farmacológico , Neoplasias de los Tejidos Blandos/tratamiento farmacológico , Resultado del Tratamiento , Adulto JovenRESUMEN
Primary pulmonary artery sarcomas are rare tumors and are commonly misdiagnosed as pulmonary embolism. Primary pulmonary sarcomas demonstrate intraluminal growth into the vessel, rather than through the wall; require complete resection to enhance survival; and require complex surgical planning. The purpose of this case report is to describe an optimal team approach with multidisciplinary planning facilitated by a customized 3-dimensional model to guide intervention and enhance communication.
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Neoplasias Pulmonares , Neoplasias de Tejido Vascular , Sarcoma , Neoplasias Vasculares , Humanos , Arteria Pulmonar/diagnóstico por imagen , Arteria Pulmonar/cirugía , Arteria Pulmonar/patología , Sarcoma/diagnóstico por imagen , Sarcoma/cirugía , Neoplasias Pulmonares/diagnóstico por imagen , Neoplasias Pulmonares/cirugía , Neoplasias Pulmonares/patología , Impresión Tridimensional , Neoplasias Vasculares/diagnóstico por imagen , Neoplasias Vasculares/cirugíaRESUMEN
PURPOSE: Idiopathic intracranial hypertension is a significant cause of preventable blindness. Patients suffer from debilitating headaches, pulsatile tinnitus, nausea, vomiting, photophobia and radicular pain. At this rate, treatment cost will increase to 462.7 million pounds sterling annually by 2030. Weight loss is the only proven disease-modifying therapy for reversal of idiopathic intracranial hypertension. Bariatric surgery leads to superlative weight loss and reversal of related comorbidities. The case series and literature review aim to raise awareness of bariatric surgery as a safe and effective treatment modality for idiopathic intracranial hypertension. MATERIAL AND METHODS: The literature review comprises three systematic analysis and one randomised control trial which were identified after a PubMed search. In the case series, we have included four patients with a preoperative diagnosis of long-standing idiopathic intracranial hypertension. They were referred to our department for bariatric surgery by the neuro-ophthalmologist between January and December 2018. They were followed up for 2 years after bariatric surgery. RESULTS: All four patients were women with a mean age of 34 years. Mean body mass index reduced from 47.3 kg/m2 before surgery to 30 kg/m2 at the end of 2 years after surgery. They showed significant improvement or resolution in their symptoms related to idiopathic intracranial hypertension, and none of them required further cerebrospinal fluid pressure reducing procedures. CONCLUSION: Bariatric surgery is a safe and effective method of treating idiopathic intracranial hypertension. It is superior compared to medical management and cerebrospinal fluid pressure reducing procedures which have high rates of recurrence.
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Cirugía Bariátrica , Obesidad Mórbida , Seudotumor Cerebral , Adulto , Índice de Masa Corporal , Femenino , Humanos , Obesidad Mórbida/cirugía , Seudotumor Cerebral/cirugía , Ensayos Clínicos Controlados Aleatorios como Asunto , Pérdida de PesoRESUMEN
PURPOSE: Angiosarcomas represents a diverse group of aggressive high-grade vascular tumours with limited therapeutic options. We sought to determine the safety and efficacy of regorafenib, a small-molecule multikinase inhibitor, in the treatment of metastatic or locally advanced unresectable angiosarcoma. PATIENTS AND METHODS: In this single-arm multicentre, open-label phase II clinical trial, 31 patients were enrolled and received regorafenib 160 mg PO daily for 21 days of a 28-day cycle. The primary endpoint for the study was progression-free survival at 4 months. Secondary endpoints included overall survival, response rate, and safety. Patients (≥18 years) with an Eastern Cooperative Oncology Group (ECOG) score of 0-1, a life expectancy of at least 4 months who had progressed on at least one but no more than 4 prior lines of therapy were eligible. RESULTS: Of the 23 patients evaluable for efficacy, 2 had a complete response (8.7%), and 2 had a partial response (8.7%), for a total overall response rate of 17.4%. Median PFS was 5.5 months, and 12/23 patients (52.2%) had a PFS of greater than 4 months. 10/31 (32.3%) patients evaluable for toxicity had a grade 3 or higher adverse events. CONCLUSIONS: Regorafenib is a safe and active treatment for refractory metastatic and unresectable angiosarcoma. Rates of adverse events were comparable to prior studies of regorafenib for other tumour types. Regorafenib, the single agent, could be considered as therapy for patients with metastatic or unresectable AS.