Cystic fibrosis (CF) is a potentially fatal genetic disease that causes serious lung damage. With time, researchers have a more complete understanding of the molecular-biological defects that underlie CF. This knowledge is leading to alternative approaches regarding the treatment of this condition. Trikafta is the third FDA-approved drug that targets the F508del mutation of the CFTR gene. The drug is a combination of three individual drugs which are elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA). This trio increases the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and reduces the mortality and morbidity rates in CF patients. The effectiveness of Trikafta, seen in clinical trials, outperforms currently available therapies in terms of lung function, quality of life, sweat chloride reduction, and pulmonary exacerbation reduction. The safety and efficacy of CFTR modulators in children with CF have also been studied. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. In this study, we will focus on reviewing data from clinical trials regarding the benefits of CFTR modulator therapy. We address the impact of Trikafta on lung function, pulmonary exacerbations, and quality of life. Adverse events of the different CFTR modulators are discussed.

Background: Community-acquired pneumonia (CAP) is associated with significant morbidity and mortality. Non-invasive ventilation (NIV) and invasive mechanical ventilation (IMV) are most important interventions for patients with severe CAP associated with respiratory failure. We analysed utilization trends and predictors of non-invasive and invasive ventilation in patients hospitalized with CAP.Methods: Nationwide Inpatient Sample and Healthcare Cost and Utilization Project data for years 2008-2017 were analysed. Adult hospitalizations due to CAP were identified by previously validated International Classification of Diseases, 9th Revision, Clinical Modification (ICD-9-CM) and International Classification of Diseases, 10th Revision, Clinical Modification (ICD-10-CM) codes. We then utilized the Cochran-Armitage trend test and multivariate survey logistic regression models to analyse temporal incidence trends, predictors, and outcomes. We used SAS 9.4 software (SAS Institute Inc., Cary, NC, USA) for analysing data.Results: Out of a total of 8,385,861 hospitalizations due to CAP, ventilation assistance was required in 552,395 (6.6%). The overall ventilation use increased slightly; however, IMV utilization decreased, while NIV utilization increased. In multivariable regression analysis, males, Asian/others and weekend admissions were associated with higher odds of any ventilation utilization. Concurrent diagnoses of septicemia, congestive heart failure, alcoholism, chronic lung diseases, pulmonary circulatory diseases, diabetes mellitus, obesity and cancer were associated with increased odds of requiring ventilation assistance. Ventilation requirement was associated with high odds of in-hospital mortality and discharge to facility. Conclusion:The use of NIV among CAP patients has increased while IMV use has decreased over the years. We observed numerous factors linked with a higher use of ventilation support. The requirement of ventilation support is also associated with very high chances of mortality and morbidity.

Tumefactive multiple sclerosis comprises a rare subset of multiple sclerosis that often poses a diagnostic challenge to physicians. It is unique in its presentation as a solitary lesion, usually larger than 2 cm, with surrounding vasogenic edema, commonly mimicking a primary intracranial malignancy. We present a case of a 25-year-old female with no significant past medical history who presented to our institution with homonymous superior quadrantanopia. During her admission, she underwent a magnetic resonance imaging (MRI) of the brain, which revealed a large lesion in the left temporal area surrounded by marked edema. A thorough workup revealed a diagnosis of tumefactive multiple sclerosis. Subsequently, she was initiated on intravenous immunoglobulin rather than stress dose steroids, given the concern for a superimposed infection. Interestingly, the patient had a paradoxical progression of her symptoms as well as expansion of the vasogenic edema on a repeat MRI. In our case, we highlight the key differences in tumefactive multiple sclerosis diagnosis and management.

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The most common reason for seeing a gastroenterologist is irritable bowel syndrome (IBS). IBS was thought to be a functional disease; however, there are now numerous alternative pathophysiologic pathways that can explain the symptoms. The pathophysiology of IBS is diverse and not well understood. Most current first-line treatments for IBS target the primary symptom and mainly impact one symptom in the symptom complex. The purpose of this study was to summarize the data on new medicines used to treat IBS. We conducted a bibliographic search in Google Scholar and PubMed focused on medication clinical trials in IBS with diarrhea. New medications for IBS with diarrhea target important pathways in the pathophysiology of these disorders, improving both the abnormal bowel habit and other significant symptoms such as abdominal pain and bloating.