scholarly journals Avapritinib in advanced gastrointestinal stromal tumor: case series and review of the literature from a tertiary care center in India

2021 ◽  
pp. FSO676
Author(s):  
Saurav Verma ◽  
Rohit Reddy ◽  
Sheragaru Hanumanthappa Chandrashekhara ◽  
Shamim Ahmed Shamim ◽  
Sarthak Tripathy ◽  
...  

The therapeutic landscape in advanced gastrointestinal stromal tumor has evolved. Avapritinib and ripretinib have now been approved by the US FDA for platelet-derived growth factor alpha D842V-mutant and refractory gastrointestinal stromal tumor patients, respectively. Here we report five patients who have been on avapritinib under an expanded access program. Response assessment was available for four patients – a partial response in two patients and stable disease in one, while one patient had progressive disease. Though preliminary results of the VOYAGER trial have shown less activity of avapritinib and no significant difference in progression-free survival when compared with regorafenib, avapritinib may show some clinical benefit in a subset of patients refractory to approved therapies. We share our experience of five cases, with clinical benefit in three. We believe avapritinib should be further evaluated in clinical trials.

2020 ◽  
Vol 12 (1) ◽  
Author(s):  
Rafiye Ciftciler ◽  
Hakan Goker ◽  
Yahya Buyukasik ◽  
Nilgun Sayınalp ◽  
Ibrahim C. Haznedaroglu ◽  
...  

The treatment landscape and clinical outcome of multiple myeloma (MM) patients have changed in the last decades, with an improved median survival of 8-10 years. This study aimed to evaluate the bortezomib, cyclophosphamide and dexamethasone (VCD) regimen versus bortezomib and dexamethasone (VD) regimen in patients with newly diagnosed MM. This study has been performed in a retrospective manner. One hundred and three patients with newly diagnosed MM who received chemotherapy at our tertiary care center between the years of 2009 and 2018 were evaluated. A total of 103 patients were included. The 5-year overall survival (OS) for patients who received VD regimen and patients who received VCD regimen were 75% and 83%, respectively. The OS for VD patients was 113.1±12.5 versus 122.2±9.5 months for VCD patients with no statistically significant difference (P=0.47). The 5- year PFS (progression free survival) for patients who received VD regimen and patients who received VCD regimen were 66% and 75%, respectively. The PFS for VCD patients was higher than the PFS for VD patients (67.1±7.4 versus 97.7±13.4 months), but no statistically significant difference was observed (P=0.59). Relapse rate (P=0.002) and mortality rate (P=0.01) were higher in VD group than VCD group and they were statistically significant. The OS and PFS were clinically longer in patients receiving VCD regimen than in patients receiving VD regimen, although not statistically significant. Cyclophosphamide should be given to patients at physician discretion and depending on patient’s frailty function.


2006 ◽  
Vol 24 (18_suppl) ◽  
pp. 16018-16018
Author(s):  
K. K. Curtis ◽  
D. W. Northfelt

16018 Background: Second opinions (SO) are common in medical practice. Aside from case series, little evidence exists to demonstrate a clinical benefit associated with SO. Lack of relevant data limits knowledge of numbers of patients, diseases, and motivations of SO seekers. Within the field of medical hematology/oncology (H/O), no specific demographic data are available to characterize patients seeking SO in the United States. Methods: To typify such patients at Mayo Clinic in Arizona (MCA), we recorded demographic and disease-related information for patients seeking these evaluations over a six month period from 1/1 through 6/30/05. Results: A total of 683 patients contacted MCA for medical H/O SO over the 6 month study period. A complete set of demographic and disease-related information was obtained from 655 patients; the remainder were excluded from the data set. Demographically, the majority of patients were female (53%), married (66%), and lived nearby (i.e., from Arizona-58%, or bordering state-22%). Average age was 62.3 years. Among oncologic diagnoses, the majority of patients (60% of 374 reported oncologic diagnoses) stated a diagnosis of breast, lung, colorectal, pancreatic or prostate cancer. Approximately 1/3 of patients had metastatic disease at the time they sought evaluation at MCA. There were 278 patients seeking evaluation for hematologic abnormalities, with the majority (160 patients, 58%) seeking evaluation for non-malignant conditions. Of those with malignancies, lymphoma (including Hodgkin and non-Hodgkin) was the most common (45%), followed by multiple myeloma (21%) and chronic lymphocytic leukemia (15%). Conclusions: This study provides insight into demographics and disease processes of patients seeking medical H/O SO at a tertiary care center in the United States. Although no definite conclusions can be drawn about motivations for seeking H/O SO, future research should examine patient motivation to better understand factors leading to this behavior. Given a lack of evidence for clinical benefit associated with SO seeking, and its potential costliness, better knowledge of demographic and motivational factors may allow for a more constructive approach to be taken toward the needs of SO seekers. No significant financial relationships to disclose.


2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Jonathan Shabab ◽  
Allysen Dubisky ◽  
Ambaris Singh ◽  
Megan Crippen ◽  
Khalid Abulaban ◽  
...  

Abstract Background Multisystem Inflammatory Syndrome in Children (MIS-C) is a rare hyperinflammatory condition that occurs following SARS-CoV-2 infection. There is a paucity of research describing risk factors, optimal management, and outcomes of this life-threatening condition. Methods This is a case series of 26 patients diagnosed with MIS-C in a West Michigan pediatric tertiary care center from April 2020 to February 2021. We describe the clinical, imaging, and laboratory characteristics of these patients and detail their treatments and outcomes with comparisons between Pediatric Intensive Care Unit (PICU) and non-PICU patients. Categorical testing utilized Chi-square and Fisher’s Exact tests. Comparison between groups used T-tests or Kruskal-Wallis. Results Fifteen patients (57%) required intensive care. There was no statistically significant difference in demographics between PICU and non-PICU patients, however all Black patients required intensive care. Gastrointestinal symptoms were present in 22 patients (84%). Seventeen patients (65%) had Kawasaki-like features and 12 (46%) developed coronary artery dilation. Patients requiring intensive care were less likely to have a reported history of COVID-19 disease or exposure (p = 0.0362). Statistically significant differences were also noted in peak ferritin (p = 0.0075), procalcitonin, and BNP in those who required intensive care. Conclusions Although overlap exists with other hyperinflammatory conditions, our study provides further evidence that MIS-C is a distinct, albeit heterogenous, disorder with various degrees of cardiac involvement. Anakinra, in conjunction with steroid use, appears to be effective and safe in the treatment of MIS-C. This report identifies procalcitonin, peak ferritin, and BNP as potentially useful biomarkers for severity of disease.


2021 ◽  
Vol 15 (1) ◽  
Author(s):  
Ghazal Tansir ◽  
Nihar Ranjan Dash ◽  
Saurabh Galodha ◽  
Prasenjit Das ◽  
Shamim Ahmed Shamim ◽  
...  

Abstract Background Carney’s triad is a rare syndrome comprising gastrointestinal stromal tumor, extra-adrenal paraganglioma, and pulmonary chondroma along with newer additions of adrenal adenoma and esophageal leiomyoma. The triad is completely manifest in only 25–30% cases, with most patients presenting with two out of three parts of the syndrome. Wild-type succinate-dehydrogenase-deficient gastric gastrointestinal stromal tumor forms the most common component of Carney’s triad and is usually multicentric and multifocal. It usually demonstrates indolent behavior and resistance to imatinib; hence, the management remains predominantly surgical. Pulmonary chondromas are commonly unilateral and multiple with slow-growing nature, which allows for conservative management. Adrenocortical adenomas are found in 20% of patients and are usually detected as incidentalomas. Case presentation A 49-year-old Asian male presented with upper gastrointestinal bleed and was diagnosed with multiple gastric succinate-dehydrogenase-deficient gastrointestinal stromal tumors. On evaluation, he was found to have left pulmonary chondroma and non-secretory adrenal adenoma, thus completing the Carney’s triad. He underwent surgery with sleeve gastrectomy and excision of the antral tumor nodule, while the adrenal and pulmonary tumors have been under close follow-up. Conclusion Literature regarding Carney’s triad is scarce, especially from the Indian setting. Our report aims to highlight the various manifestations of this syndrome with emphasis on management of wild-type succinate-dehydrogenase-deficient gastrointestinal stromal tumor. Radical gastric surgeries do not offer a survival advantage in this condition; hence, more conservative modalities of resection can be adopted.


2021 ◽  
pp. 019459982110089
Author(s):  
Quinn Dunlap ◽  
James Reed Gardner ◽  
Amanda Ederle ◽  
Deanne King ◽  
Maya Merriweather ◽  
...  

Objective Neck dissection (ND) is one of the most commonly performed procedures in head and neck surgery. We sought to compare the morbidity of elective ND (END) versus therapeutic ND (TND). Study Design Retrospective chart review. Setting Academic tertiary care center. Methods Retrospective chart review of 373 NDs performed from January 2015 to December 2018. Patients with radical ND or inadequate chart documentation were excluded. Demographics, clinicopathologic data, complications, and sacrificed structures during ND were retrieved. Statistical analysis was performed with χ2 and analysis of variance for comparison of categorical and continuous variables, respectively, with statistical alpha set a 0.05. Results Patients examined consisted of 224 males (60%) with a mean age of 60 years. TND accounted for 79% (n = 296) as compared with 21% (n = 77) for END. Other than a significantly higher history of radiation (37% vs 7%, P < .001) and endocrine pathology (34% vs 2.6%, P < .001) in the TND group, no significant differences in demographics were found between the therapeutic and elective groups. A significantly higher rate of structure sacrifice and extranodal extension within the TND group was noted to hold in overall and subgroup comparisons. No significant difference in rate of surgical complications was appreciated between groups in overall or subgroup analysis. Conclusion While the significantly higher rate of structure sacrifice among the TND population represents an increased morbidity profile in these patients, no significant difference was found in the rate of surgical complications between groups. The significant difference seen between groups regarding history of radiation and endocrine pathology likely represents selection bias.


Author(s):  
Ruo S. Chen ◽  
Laurel O’Connor ◽  
Matthew R. Rebesco ◽  
Kara L. LaBarge ◽  
Edgar J. Remotti ◽  
...  

Abstract Introduction: Emergency Medical Services (EMS) providers are trained to place endotracheal tubes (ETTs) in the prehospital setting when indicated. Endotracheal tube cuffs are traditionally inflated with 10cc of air to provide adequate seal against the tracheal lumen. There is literature suggesting that many ETTs are inflated well beyond the accepted safe pressures of 20-30cmH2O, leading to potential complications including ischemia, necrosis, scarring, and stenosis of the tracheal wall. Currently, EMS providers do not routinely check ETT cuff pressures. It was hypothesized that the average ETT cuff pressure of patients arriving at the study site who were intubated by EMS exceeds the safe pressure range of 20-30cmH2O. Objectives: While ETT cuff inflation is necessary to close the respiratory system, thus preventing air leaks and aspiration, there is evidence to suggest that over-inflated ETT cuffs can cause long-term complications. The purpose of this study is to characterize the cuff pressures of ETTs placed by EMS providers. Methods: This project was a single center, prospective observational study. Endotracheal tube cuff pressures were measured and recorded for adult patients intubated by EMS providers prior to arrival at a large, urban, tertiary care center over a nine-month period. All data were collected by respiratory therapists utilizing a cuff pressure measurement device which had a detectable range of 0-100cmH2O and was designed as a syringe. Results including basic patient demographics, cuff pressure, tube size, and EMS service were recorded. Results: In total, 45 measurements from six EMS services were included with ETT sizes ranging from 6.5-8.0mm. Mean patient age was 52.2 years (67.7% male). Mean cuff pressure was 81.8cmH2O with a range of 15 to 100 and a median of 100. The mode was 100cmH2O; 40 out of 45 (88.9%) cuff pressures were above 30cmH2O. Linear regression showed no correlation between age and ETT cuff pressure or between ETT size and cuff pressure. Two-tailed T tests did not show a significant difference in the mean cuff pressure between female versus male patients. Conclusion: An overwhelming majority of prehospital intubations are associated with elevated cuff pressures, and cuff pressure monitoring education is indicated to address this phenomenon.


2021 ◽  
pp. 1-8
Author(s):  
Halil Celik ◽  
Sadettin Burak Acikel ◽  
Fatih Mehmet Akif Ozdemir ◽  
Erhan Aksoy ◽  
Ulkuhan Oztoprak ◽  
...  

<b><i>Background and Aim:</i></b> Although anyone can be affected by the COVID-19 pandemic, it may cause additional concern for people with chronic conditions. Epilepsy is the most common neurological disease in childhood and adolescence. The aim of this study was to determine anxiety levels among the mothers of children under follow-up for epilepsy in our clinic during the COVID-19 pandemic. <b><i>Methods:</i></b> The study group consisted of the mothers of epilepsy patients who were under follow-up in the pediatric neurology outpatient clinic of the tertiary care center and were scheduled for a routine examination during the COVID-19 pandemic. The mothers’ anxiety levels according to the Beck Anxiety Inventory and their opinions about COVID-19 in relation to their child were assessed and compared based on whether the mother/patient attended their appointments in person and whether the child had frequent or infrequent seizures. <b><i>Results:</i></b> There was no statistically significant difference in anxiety level between the mothers of 64 children with epilepsy who attended their appointment during the pandemic and those of the mothers of 52 who did not attend their appointment. However, the mothers of children with frequent seizures had significantly higher anxiety levels. <b><i>Conclusion:</i></b> Anxiety level of mothers whose children have frequent seizures was significantly higher compared to mothers whose children have infrequent seizures. It is important to be aware about this point and using telemedicine approach in suitable population and postpone routine outpatient follow-up appointments as much as possible.


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