Allogeneic Hematopoietic Stem Cell Transplantation After Prior Lung Transplantation for Hereditary Pulmonary Alveolar Proteinosis: A Case Report.

Pulmonary alveolar proteinosis (PAP) is a rare, diffuse lung disorder characterized by surfactant accumulation in the small airways due to defective clearance by alveolar macrophages, resulting in impaired gas exchange. Whole lung lavage is the current standard of care treatment for PAP. Lung transplantation is an accepted treatment option when whole lung lavage or other experimental treatment options are ineffective, or in case of extensive pulmonary fibrosis secondary to PAP.

Long-term use of tube feeding in children with cystic fibrosis: results from two Belgian CF centers.

Background: Enteral tube feeding (ETF) is often used in an attempt to optimize the nutritional status. The aim of this study was to observe the long term effect of ETF and to compare the start of ETF with the current European guidelines on nutrition care in CF.

Method: From all patients who received ETF (ETFp) between February 2000 and September 2016 in the Ghent University Hospital (GUH) or Brussels University Hospital (BUH), z-scores for body weight (W), height (H), growth velocity (GV) and BMI, FEV%, and FVC% were retrospectively collected from the patients' medical record, 3 years before and 5 years after the year of ETF initiation.

The nutritional status in CF: Being certain about the uncertainties.

Background: Nutritional therapy is one of the cornerstones in cystic fibrosis (CF) therapy. There is a strong association between nutritional status and pulmonary function and thus longevity. Therefore nutritional therapy should be continuously adapted to preserve or improve the nutritional status.

Point-of-care CRP matters: normal CRP levels reduce immediate antibiotic prescribing for acutely ill children in primary care: a cluster randomized controlled trial.

Objective: Antibiotics are prescribed too often in acutely ill children in primary care. We examined whether a Point-of-Care (POC) C-reactive Protein (CRP) test influences the family physicians' (FP) prescribing rate and adherence to the Evidence Based Medicine (EBM) practice guidelines.

Design: Cluster randomized controlled trial.

Risk factors and impact of allergic bronchopulmonary aspergillosis in Pseudomonas aeruginosa-negative CF patients.

Background: Allergic bronchopulmonary aspergillosis (ABPA) is a major complication in cystic fibrosis (CF) patients. Risk factors for ABPA and clinical deterioration in CF patients, negative for Pseudomonas aeruginosa (Pa), were explored.

Methods: We performed a retrospective case-control study in 73 Pa-negative patients.

Establishing the diagnosis of chronic colonization with Pseudomonas aeruginosa of cystic fibrosis patients: Comparison of the European consensus criteria with genotyping of P. aeruginosa isolates.

Unlabelled: After antibiotic eradication treatment for a first ever Pseudomonas aeruginosa isolation, the European consensus criteria (ECC) are widely used to assess colonization status with P. aeruginosa in CF-patients. We evaluated to what extent genotyping (GT) of subsequent P.

Reducing inappropriate antibiotic prescribing for children in primary care: a cluster randomised controlled trial of two interventions.

Background: Antibiotics are overprescribed for non-severe acute infections in children in primary care.

Aim: To explore two different interventions that may reduce inappropriate antibiotic prescribing for non-severe acute infections.

Design And Setting: A cluster randomised, factorial controlled trial in primary care, in Flanders, Belgium.

The effect of enteral tube feeding in cystic fibrosis: A registry based study.

Background: Long-term effect of enteral tube feeding (ETF) in cystic fibrosis (CF) remains equivocal.

Methods: A Belgian CF registry based, retrospective, longitudinal study, evaluated the pre- and post- ETF (n = 113) clinical evolution and compared each patient with 2 age, gender, pancreatic status and genotype class-matched controls.

Results: At baseline ETF had a worse BMI z-score (p < 0.

Development and validation of an LC tandem MS assay for the quantification of β-lactam antibiotics in the sputum of cystic fibrosis patients.

Objectives: Antibiotic therapy is of vital importance for the control of infectious exacerbations in cystic fibrosis (CF) patients. However, very little is known regarding the fraction of systemically administered antibiotics reaching the lower respiratory tract secretions. We developed and validated a method to measure the concentrations of piperacillin, ceftazidime, meropenem and aztreonam in CF sputum, and present the validation data.

Exercise performance and quality of life in children with cystic fibrosis and mildly impaired lung function: relation with antibiotic treatments and hospitalization.

Unlabelled: This study evaluates the impact of antibiotic treatments and hospitalization on exercise performance and health-related quality of life (QOL) in children with mild cystic fibrosis (CF) lung disease. Forty-seven children between 7 and 17 years with mild CF underwent a maximal exercise test including spiro-ergometry and filled out a QOL-questionnaire (PedsQL™). Amount of antibiotic treatments (AB) and hospitalization days in the last 3 years were reviewed.

Early-onset primary antibody deficiency resembling common variable immunodeficiency challenges the diagnosis of Wiedeman-Steiner and Roifman syndromes.

Syndromic primary immunodeficiencies are rare genetic disorders that affect both the immune system and other organ systems. More often, the immune defect is not the major clinical problem and is sometimes only recognized after a diagnosis has been made based on extra-immunological abnormalities. Here, we report two sibling pairs with syndromic primary immunodeficiencies that exceptionally presented with a phenotype resembling early-onset common variable immunodeficiency, while extra-immunological characteristics were not apparent at that time.

Gastro-intestinal manifestations in cystic fibrosis patients.

Cystic fibrosis (CF) is a life-limiting disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR). This defective chloride channel, present in different organ systems such as respiratory system, gastrointestinal tract, reproductive system and sweat glands, disturbs the ion and water transport over the membranes leading to the well known CF symptoms. CF has outgrown paediatric care, as half of CF patients are currently adults.

Recurrent spontaneous pneumomediastinum in a child with tracheomalacia.

Spontaneous pneumomediastinum in children is a very rare, benign entity. Recurrent episodes are exceptional. Identifying an underlying trigger is crucial, and very often, spontaneous pneumomediastinum occurs in association with an asthma exacerbation.

Epidemic Achromobacter xylosoxidans strain among Belgian cystic fibrosis patients and review of literature.

Background: Achromobacter xylosoxidans is increasingly being recognized as an emerging pathogen in cystic fibrosis. Recent severe infections with A. xylosoxidans in some of our cystic fibrosis (CF) patients led to a re-evaluation of the epidemiology of CF-associated A.

A case of chronic recurrent multifocal osteomyelitis associated with crohn's disease.

Chronic recurrent multifocal osteomyelitis (CRMO) is an autoinflammatory bone disease of unknown etiology, most commonly affecting the metaphysis of long bones, especially the tibia, femur and clavicle. The clinical spectrum varies from self-limited uni-or multi-focal lesions to chronic recurrent courses. Diagnosis is based on clinical, radiologic and pathological findings, is probably underdiagnosed due to poor recognition of the disease.

Colistin and neurotoxicity: recommendations for optimal use in cystic fibrosis patients.

Case description The use of i.v. colistin reappeared recently for the treatment of multidrug-resistant Gram negative organisms in the intensive care and cystic fibrosis (CF) setting.

Optimizing antibiotic prescribing for acutely ill children in primary care (ERNIE2 study protocol, part B): a cluster randomized, factorial controlled trial evaluating the effect of a point-of-care C-reactive protein test and a brief intervention combined with written safety net advice.

Background: Despite huge public campaigns, there is still overconsumption of antibiotics in children with self-limiting diseases. Possible explanations may be the physicians' and parents' uncertainty about the gravity of the disease and inadequate communication between physicians and parents leading to lack of reassurance for the parents. In this paper we describe the design and methods of a trial aiming to rationalize antibiotic prescribing by decreasing this uncertainty and parental anxiety.

Tuberculin skin test versus interferon-gamma release assays for the diagnosis of tuberculosis infection.

Objective: Accurate detection of latent tuberculosis infection (LTBI) is becoming increasingly important due to the increasing use of immunosuppressive medications and the human immunodeficiency epidemic, which have increased the risk for reactivation to active tuberculosis (TB) infection. LTBI is detected by tuberculin skin test (TST) and interferon-gamma release assays (IGRAs). The latter include T-SPOT(®).

Is the improvement of CF patients, hospitalized for pulmonary exacerbation, correlated to a decrease in bacterial load?

Background: Cystic Fibrosis (CF) patients are vulnerable to airway colonization with Pseudomonas aeruginosa. In case eradication fails after antibiotic treatment, patients become chronically colonized with P. aeruginosa, with recurrent pulmonary exacerbation, for which patients typically are hospitalized for 2 weeks and receive intravenous antibiotic treatment.

Complete factor I deficiency due to dysfunctional factor I with recurrent aseptic meningo-encephalitis.

Purpose: Complement regulators control the activated complement system. Defects in this homeostasis can result in tissue damage and autoimmune diseases with a heterogeneity in clinical presentation. Complement factor I (FI), a serine protease, is an important regulator of alternative pathway activation.

Achromobacter xylosoxidans induced bronchiolitis obliterans in cystic fibrosis.

We report a 12-year-old boy with progressive bronchiolitis obliterans caused by Achromobacter xylosoxidans (Ax) colonization after liver transplantation, resulting in a steep decline in lung function.

Genetic variations in toll-like receptor pathway and lung function decline in Cystic fibrosis patients.

The toll-like receptor (TLR) family maintains pulmonary homeostasis by pathogen recognition, clearance and regulation of inflammation. Genes affecting inflammation response play a key role in modifying Cystic fibrosis (CF) lung disease severity. We assessed the impact of single nucleotide polymorphisms (SNPs) of TLR genes (TLR1 to TLR10, CD14, lipopolyssacharide-binding protein (LBP)) on lung function in CF patients.

Spirometry-related pain and distress in adolescents and young adults with cystic fibrosis: the role of acceptance.

Objective: To investigate the occurrence of spirometry-related pain and distress in adolescents and young adults with cystic fibrosis (CF), and to investigate the role of acceptance of illness in spirometry-related pain and distress.

Methods: A total of 36 adolescents and young adults with CF (12 to 22 years of age) completed a questionnaire assessing acceptance of illness. Spirometry-related distress was assessed using self-report (ie, anxiety⁄worry about the procedure) and physiological outcomes (ie, heart rate and heart rate variability) before spirometry.