Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTR modulator, lumacaftor/ivacaftor: an assessment using linked registry data in Sweden.

Aims: We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems.

Material And Methods: This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019.

Effectiveness of manual therapy interventions in cystic fibrosis care: a pilot study.

Background: Cystic fibrosis (CF) is a severe genetic condition that affects multiple organ systems and imposes a substantial treatment burden. Regarding the lungs and airways, the progressive pathophysiological changes place a significant strain on the musculoskeletal components of the respiratory system for people with CF. This pilot study investigated the effectiveness of manual therapy interventions (MTIs) on thoracic mobility, respiratory muscle strength, lung function, and musculoskeletal pain.

Musculoskeletal aspects of respiratory function in cystic fibrosis: a cross-sectional comparative study.

Background: Respiration is an intricate interaction between visceral and musculoskeletal structures. In cystic fibrosis (CF), the airways and lungs are subject to progressive obstruction and destruction. However, knowledge about the musculoskeletal aspects of respiratory function and symptoms is still limited in this patient group.

In vitro stimulation with nontuberculous mycobacteria induced a stronger cytokine response in leukocytes isolated from individuals with latent tuberculosis compared to those isolated from active tuberculosis or cystic fibrosis patients.

Mycobacterium tuberculosis and opportunistic environmental non-tuberculous mycobacteria (NTM) can cause severe infection. Why latent tuberculosis infection advances to active disease, and why some individuals with cystic fibrosis (CF) develop pulmonary infections with NTM is still poorly understood. The aim of this study was to investigate the effector function of peripheral blood mononuclear cells (PBMC) from individuals with active or latent tuberculosis, individuals with CF with or without pulmonary NTM-infection and healthy controls, by measuring cytokine response to in vitro stimulation with different species of NTMs.

Generation of NK cells with chimeric-switch receptors to overcome PD1-mediated inhibition in cancer immunotherapy.

Multiple myeloma (MM) is an incurable hematological cancer, in which immune checkpoint inhibition (ICI) with monoclonal antibodies (mAbs) has failed due to uncontrollable immune responses in combination therapies and lack of efficacy in monotherapies. Although NK cell-specific checkpoint targets such as NKG2A and KIRs are currently being evaluated in clinical trials, the clinical impact of NK cells on the PD1 cascade is less well understood compared to T cells. Furthermore, while NK cells have effector activity within the TME, under continuous ligand exposure, NK cell dysfunctionality may occur due to interaction of PD1 and its ligand PD-L1.

Thoracic configuration in patients ageing with cystic fibrosis: A retrospective single-blinded cohort study.

Background: In patients with cystic fibrosis (CF), thoracic morphology and its role in respiratory function is conditioned by anthropometric factors, as well as by pathological changes. While the lungs are continuously monitored, examinations of potential thoracic cage adaptations to the disease are rare. Hence, the aim of this study was to investigate thoracic configuration, and its correlation to spirometry measures over time.

Autologous NK cells as consolidation therapy following stem cell transplantation in multiple myeloma.

Few approaches have been made toward exploring autologous NK cells in settings of cancer immunotherapy. Here, we demonstrate the feasibility of infusing multiple doses of activated and expanded autologous NK cells in patients with multiple myeloma (MM) post-autologous stem cell transplantation. Infused NK cells were detected in circulation up to 4 weeks after the last infusion.

EVALUATION OF TWO CHEST TOMOSYNTHESIS CYSTIC FIBROSIS SCORING SYSTEMS USING HIGH-RESOLUTION COMPUTED TOMOGRAPHY BRODY SCORING AS REFERENCE.

Purpose: To evaluate two chest tomosynthesis (CTS) scoring systems for cystic fibrosis (CF), one system developed by Vult von Steyern et al. (VvS) and one system based on the Brody scoring system for high-resolution computed tomography (HRCT) (modified Brody (mB)). Brody scoring of HRCT was used as reference.

QUANTIFICATION OF PULMONARY PATHOLOGY IN CYSTIC FIBROSIS-COMPARISON BETWEEN DIGITAL CHEST TOMOSYNTHESIS AND COMPUTED TOMOGRAPHY.

Purpose: Digital tomosynthesis (DTS) is currently undergoing validation for potential clinical implications. The aim of this study was to investigate the potential for DTS as a low-dose alternative to computed tomography (CT) in imaging of pulmonary pathology in patients with cystic fibrosis (CF).

Methods: DTS and CT were performed as part of the routine triannual follow-up in 31 CF patients.

Th17 associated cytokines in sputum samples from patients with cystic fibrosis.

Cystic fibrosis (CF) is a genetic disease leading to chronic bacterial airway infection and inflammation. T helper 17 (Th17) cells are identified by their production of interleukin (IL)-17A, which recruit neutrophils to the site of airway infection. IL-23 is an important inducer of IL-17 and IL-22 production.

Patient acceptance and outcome of mental health screening in Swedish adults with cystic fibrosis.

Purpose: Anxiety and depression are common among adults with cystic fibrosis (CF), and the International Committee on Mental Health in CF (ICMH) recommends annual screening for mental health problems. We implemented screening according to the recently published guidelines and assessed the results from the first year, as well as the patients' attitude to annual screening METHODS: Adult patients attending Gothenburg CF-center from Feb 2015 to Dec 2016 completed the GAD-7 (anxiety) and PHQ-9 (depression) forms at the time of their annual review. In addition, questions regarding the screening process and instruments used were asked.

Functional Assessment for Clinical Use of Serum-Free Adapted NK-92 Cells.

Natural killer (NK) cells stand out as promising candidates for cellular immunotherapy due to their capacity to kill malignant cells. However, the therapeutic use of NK cells is often dependent on cell expansion and activation with considerable amounts of serum and exogenous cytokines. We aimed to develop an expansion protocol for NK-92 cells in an effort to generate a cost-efficient, xeno-free, clinical grade manufactured master cell line for therapeutic applications.

Survival after lung transplantation for cystic fibrosis in Sweden.

Objectives: In Sweden, lung transplantation has been performed in patients with end-stage lung disease since 1990. We assessed survival after lung transplantation for cystic fibrosis (CF) with focus on early mortality and outcome for patients infected with certain multiresistant bacteria, considered a relative contraindication for lung transplantation.

Methods: Review of CF and transplant databases and patient charts.

Measuring Generalized Trust: An Examination of Question Wording and the Number of Scale Points.

Survey institutes recently have changed their measurement of generalized trust from the standard dichotomous scale to an 11-point scale. Additionally, numerous survey institutes use different question wordings: where most rely on the standard, fully balanced question (asking if "most people can be trusted or that you need to be very careful in dealing with people"), some use minimally balanced questions, asking only if it is "possible to trust people." By using two survey-embedded experiments, one with 12,009 self-selected respondents and the other with a probability sample of 2,947 respondents, this study evaluates the generalized trust question in terms of question wording and number of scale points used.

VISIBILITY OF STRUCTURES OF RELEVANCE FOR PATIENTS WITH CYSTIC FIBROSIS IN CHEST TOMOSYNTHESIS: INFLUENCE OF ANATOMICAL LOCATION AND OBSERVER EXPERIENCE.

The aims of this study were to assess the visibility of pulmonary structures in patients with cystic fibrosis (CF) in digital tomosynthesis (DTS) using computed tomography (CT) as reference and to investigate the dependency on anatomical location and observer experience. Anatomical structures in predefined regions of CT images from 21 patients were identified. Three observers with different levels of experience rated the visibility of the structures in DTS by performing a head-to-head comparison with visibility in CT.

Genotyping of Pseudomonas aeruginosa reveals high diversity, stability over time and good outcome of eradication.

Background: Genotyping of Pseudomonas aeruginosa (P.a) is used for surveillance at our CF clinic.

Methods: P.

Epidemiology of nontuberculous mycobacteria among patients with cystic fibrosis in Scandinavia.

Background: Nontuberculous mycobacteria (NTM) are an emerging threat to cystic fibrosis (CF) patients but their epidemiology is not well described.

Methods: In this retrospective observational study we identified all Scandinavian CF patients with a positive NTM culture from airway secretions from 2000 to the end of 2012 and used national CF databases to describe microbiological and clinical characteristics.

Results: During the 13-year period 157 (11%) CF patients were culture positive for NTM at least once.

Slow and fast lung compartments in cystic fibrosis measured by nitrogen multiple-breath washout.

Imaging studies describe significant ventilation defects across a wide range of cystic fibrosis (CF) related lung disease severity. These are unfortunately poorly reflected by phase III slope analysis-derived Scond and Sacin from multiple-breath washout (MBW). Methodology extending previous two-lung compartment model-based analysis is presented describing size and function of fast- and slow-ventilating lung compartments from nitrogen (N2) MBW and correlation to obstructive lung disease severity.

Extracorporeal membrane oxygenation as a bridge to lung transplantation: a long-term study.

Objectives: We investigated early outcomes in patients with end-stage pulmonary disease bridged with extracorporeal membrane oxygenation (ECMO) with the intention to perform lung transplantation (LTx).

Methods: ECMO was used as a bridge to LTx in 20 patients between 2005 and 2013. Most patients suffered from rapid progress of disease and most failed to stabilize on mechanical ventilation.

Genotyping of Pseudomonas aeruginosa isolates from lung transplant recipients and aquatic environment-detected in-hospital transmission.

Lung infection with Pseudomonas aeruginosa is common in lung transplant recipients and may lead to severe complications. Bacteriological surveillance aims to detect transmission of microbes between hospital environment and patients. We sought to determine whether genotyping of P.

Development of IgG antibodies to Exophiala dermatitidis is associated with inflammatory responses in patients with cystic fibrosis.

Background: The clinical importance of airway colonisation by the fungus Exophiala dermatitidis in patients with cystic fibrosis (CF) is unclear. We have previously shown that E. dermatitidis frequently colonises the airways of patients with CF.

Towards an advanced therapy medicinal product based on mesenchymal stromal cells isolated from the umbilical cord tissue: quality and safety data.

Introduction: Standardization of mesenchymal stromal cells (MSCs) manufacturing is urgently needed to enable translational activities and ultimately facilitate comparison of clinical trial results. In this work we describe the adaptation of a proprietary method for isolation of a specific umbilical cord tissue-derived population of MSCs, herein designated by its registered trademark as UCX®, towards the production of an advanced therapy medicinal product (ATMP).

Methods: The adaptation focused on different stages of production, from cell isolation steps to cell culturing and cryopreservation.