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, on behalf of the members of the Belgian CF Registry ABSTRACT: A recent American registry analysis in cystic fibrosis (CF) children showed less lung function decline after starting inhaled corticosteroid (ICS) use. We therefore examined the influence of ICS treatment on lung function in Belgian CF patients.Data from patients o6 yrs of age were eligible, provided entries on lung function, height and ICS use were available in two consecutive years. Data after oral steroid use or transplant were excluded.852 subjects contributed data with 2,976 data pairs analysed, 44.9% concerning years of ICS use. Yearly % predicted decline in forced expiratory volume in 1 s (FEV1) was 1.07% lower during ICS use (p50.001). Subgroup analysis for age revealed that the lower FEV1 decline rate during ICS use was only statistically significant in children 6-12 yrs of age (2.56%; p50.0003). Baseline FEV1 was lower by 5.89% (p,0.0001) in ICS users for all age groups combined, but there was no difference in baseline lung function in the children 6-12 yrs of age.In 6-12-yr-old children with CF, baseline lung function was similar in ICS users and nonusers, but annualised FEV1 decline was 2.56% pred lower in ICS users. Our data therefore support recent American findings.

CFTR mutations are grouped according to disease-causing mechanism. Several studies demonstrated that patients having at least one mutation of class IV/V, present with a milder phenotype, but little is known about their relative treatment burden. We compared treatment burden between patients with two class I, II, or III mutations and patients with at least one mutation of class IV/V in the 2010 database of the Belgian CF Registry. We calculated a "Treatment Burden Index" (TBI) by assigning long term therapies to categories low, medium and high intensity, for differential weighing in the total score. There were 779 patients with two known class I/II/III mutations and 94 patients with at least one class IV/V mutation. Compared to class I/II/III, class IV/V patients had a lower median number of clinic visits (4 vs. 5; P < 0.001), a lower risk of hospitalization (24.7% vs. 50.8%; P < 0.001) and intravenous antibiotic treatment (23.5% vs. 46.0%; P < 0.001) and a lower median TBI (6 vs. 9; P < 0.001). These differences remained significant when only class IV/V patients with pancreatic insufficiency (n = 31) were considered. This study clearly demonstrates the significantly lower treatment burden in patients with CF and at least one class IV/V mutation compared to patients with two class I/II/III mutations and contributes to providing better individual counseling at time of diagnosis.

Bronchial damage cannot be considered as a facilitating factor for ABPA. ABPA causes a significant increase in bronchial damage. In patients with ABPA, further bronchial damage can be controlled by an increase in hospitalization days and use of IV antibiotics. rhDNase and inhaled corticosteroids were associated with the development of ABPA.

BackgroundGeneral Practitioners (GPs) are well placed to care for patients with (chronic) substance use problems. This pilot was carried out to study the feasibility and usefulness of a continuous surveillance of substance use problems among general practice patients. The objectives were (i) to describe variables with missing values exceeding 1% and whether patients were reported without substance-related problems; (ii) the profile and the magnitude of the patient population that is treated for substance use problems.MethodsObservational study by the Belgian Network of Sentinel General Practices (SGP) in 2013. Baseline (at the first encounter) and 7-month follow-up data were reported of all patients treated for substance use problems. Two main measurements were type of substance use and patient status at follow-up. Multiple logistic regression analysis was used to examine patient status at follow-up.ResultsOf 479 patients, 47.2% had problems with alcohol alone, 20.3% with prescription drugs, 16.7% with illicit drugs other than heroin or methadone and 15.9% with heroin or methadone. Problems with alcohol alone were more prevalent in Flanders (53.0%; 95% confidence interval (CI) 46.8–59.1%) than in Wallonia-Brussels (39.8%; 95% CI 33.1–46.8%), while problems with heroin or methadone were more prevalent in Wallonia-Brussels (27.0%; 95% CI 21.1–33.5%) than in Flanders (7.1%; 95% CI 4.3–10.9%). At follow-up, 32.8% of the patients had dropped out, 29.0% had discontinued GP treatment and 38.2% had continued GP treatment. Overall, 32.4% of 479 patients had continued GP treatment for substance use problems during the study period. In Wallonia-Brussels, this proportion was higher (42.7%; 95% CI 35.9–49.6%) than in Flanders (24.3%; 95% CI 19.2–29.8%).ConclusionsA continuous surveillance of the general practice population treated for substance use problems seems to be feasible and useful. The latter is suggested by the specific profile and the relative magnitude of the population. Inter-regional health system differences should be taken into account to estimate the epidemiology of substance use problems among general practice patients.Electronic supplementary materialThe online version of this article (doi:10.1186/s12889-016-3885-0) contains supplementary material, which is available to authorized users.

Cystic fibrosis newborn screening (NBS), has not yet become a reality in all countries. We therefore compared the demographic and clinical characteristics of young children included in national cystic fibrosis registries from countries with and without NBS. We hypothesised that registries from countries with NBS would contain a higher proportion of milder and equivocal cases.

BackgroundCFTR2 provides clinical and functional information of the most common CFTR-mutations. Rare mutations (RMs) occur in only a few patients with limited reported clinical data. Their role in CF-disease liability is hardly documented.MethodsBelgian CF-Registry 2013 data were analyzed to identify CF with at least 1 RM (CF+RM). Clinical data and sweat chloride of CF+RM were compared to CF-controls, carrying 2 class 1 to 3 mutations (CFclassic). Disease severity was compared between both groups. To avoid bias in the comparison, transplanted patients were excluded from each group.ResultsSeventy-seven CF+RM were identified (77/1183 = 6.5%). Sixty-four different RM were detected, of which 21 had not been previously reported. All RMs, corresponding to HGVS (Human Genome Variation Society) nomenclature, were listed in supplementary data.Seven transplanted CF+RM were excluded for further analysis. CF+RM had higher age at diagnosis [median (IQR)] [3.7 y (0.3–18.3) vs. 0.3y (0.1–2,0) (p < 0.0001)], lower sweat chloride [96 mmol/L (64–107) vs. 104 mmol/L (97–115) (p < 0.0001)], higher FEV1%pred [77%pred (58–96) vs. 68%pred (48–86) (p = 0.017)], were less frequently pancreatic insufficient [56% vs. 98% (p < 0.0001)], Pseudomonas aeruginosa colonized [24% vs. 44% (p = 0.0093)] and needed fewer IV antibiotics [36% vs. 51% (p = 0.041)] than CFclassic. However, a wide spectrum of disease severity was seen amongst CF+RM.ConclusionsCF-patients with a RM cover 6.5% of the Belgian CF-population. Rare mutations can be found in severely ill patients, but more often in late diagnosed, pancreatic sufficient patients.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-017-0694-1) contains supplementary material, which is available to authorized users.

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