Background Several studies have assessed safety and efficacy outcomes for lumacaftor/ivacaftor therapy. We report on lumacaftor/ivacaftor's impact on lung function, physical performance, and health‐related quality of life (HRQOL) in a subpopulation of Danish people with Cystic Fibrosis (CF; PWCF) with advanced pulmonary disease who would not fulfill inclusion criteria for these studies. Methods This follow‐up study examined lumacaftor/ivacaftor's effect in a highly selected CF population. Inclusion criteria included low percent predicted forced expiratory volume in one second (ppFEV1), fast deteriorating ppFEV1, low body mass index (BMI), and difficult‐to‐treat infections. Primary endpoints included change in ppFEV1 slope, cardiopulmonary exercise testing (CPET), and all domains of the Cystic Fibrosis Questionnaire‐Revised (CFQ‐R). Secondary outcomes included change in ppFEV1, BMI Z‐score, and sweat chloride concentration. Results A total of 21 patients homozygous for the F508del mutation and a median ppFEV1 of 38.7 were included. We found significant improvements in ppFEV1 (+4.2 p < .01, +5.8 p < .01, +4.8 p < .01 and +3.8 p = .03 ppFEV1 after 3, 6, 9, and 12 months of treatment compared to baseline), ppFEV1 slope (+6.84 ppFEV1/year between the year before and the year after treatment initiation; p = .02), and saturation at CPET initiation (+1.4%, p < .02) and termination (+2.6%, p < .01) after 6 months of treatment. Finally, HRQOL improved significantly in all CFQ‐R domains except Emotion and Treat. Conclusions Our findings suggest that lumacaftor/ivacaftor reduces lung function decline, improves lung function, physical performance, and HRQOL to a greater extent in PWCF with severe lung disease than previously recognized.
Background: Intracranial abscess formation secondary to chronic rhinosinusitis (CRS) is a rare, but life-threatening infection with a poor outcome. Case presentation: A 63-year old Caucasian male with a history of CRS presented with one day of fever (40,3 C), repeated vomiting, two episodes of generalized seizures, and later became unresponsive. The patient was diagnosed with pansinusitis and a cerebral abscess, and he was treated successfully with surgery and antibiotic therapy. At follow-up nearly 9 years later, the patient had only minor complaints despite severe sinus pathology on the follow-up computed tomography scan. Conclusion: Early diagnosis and treatment of intracranial complications are essential to reduce subsequent morbidity and mortality. After an acute exacerbation, imaging findings and subjective complaints may differ. Treatment should, therefore, be based on a combination of objective and subjective findings.
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