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This retrospective matched-cohort analysis compared health-economic burdens of adults (≥18 years; n = 377) with phenylketonuria (PKU) and age/gender-matched non-PKU controls (n = 3770) in Germany. Healthcare costs and resource-utilization were analyzed for the year 2015. Differences between groups were tested using 95% CI of mean differences (MD). PKU patients had significantly higher mean costs in total (MD €3307, 95% CI €1736–€4879), for pharmaceuticals (MD €1912, 95% CI €1195–€2629) [including dietary amino-acid supplements (MD €1268, 95% CI €864–€1672)], and outpatient costs (MD €395, 95% CI €115–€675). Inpatient costs (MD €904, 95% CI -€293 to €2100) and costs for aids and remedies (MD €97, 95% CI -€10 to €203) were also higher in PKU patients. PKU patients had more outpatient visits and stayed longer in hospital. Adult PKU patients incur higher total healthcare costs than non-PKU controls, especially regarding pharmaceuticals and outpatient costs, and more frequent resource-utilization, resulting in higher health-economic burden for the statutory healthcare system.

Background Phenylketonuria (PKU) is an inherited deficiency in the enzyme phenylalanine hydroxylase (PAH), which, when poorly-managed, is associated with clinical features including deficient growth, microcephaly, seizures, and intellectual impairment. The management of PKU should start as soon as possible after diagnosis to prevent irreversible damage and be maintained throughout life. The aim of this study was to assess the burden of illness in PKU patients in general and in PKU patients born before and after the introduction of newborn screening in Germany. Methods This retrospective matched cohort analysis used the Institut für angewandte Gesundheitsforschung Berlin (InGef) research database containing anonymized healthcare claims of approximately 4 million covered lives. PKU patients were compared with matched controls from the general population within the same database (1:10 ratio via direct, exact matching on age and gender without replacement). PKU patients were included if they were aged ≥18 years on 01/01/15 and were continuously enrolled from 01/01/10 to 31/12/15. The 50 most commonly reported comorbidities and 50 most commonly prescribed medications in the PKU population were analyzed. Differences between groups were tested using 95% confidence interval (CI) of prevalence ratio (PR) values. Results The analysis included 377 adult PKU patients ( 1.5 vs controls included major depressive disorders (PR = 2.3), chronic ischemic heart disease (PR = 1.7), asthma (PR = 1.7), dizziness and giddiness (PR = 1.8), unspecified diabetes mellitus (PR = 1.7), infectious gastroenteritis and colitis (PR = 1.7), and reaction to severe stress and adjustment disorders (PR = 1.6). The most commonly prescribed Anatomical Therapeutic Chemical (ATC) subcodes among PKU patients (vs the control population) are for systemic antibacterials (34.7% vs 32.8%), anti-inflammatory and antirheumatic (29.4% vs 27.5%), renin-angiotensin agents (30.0% vs 27.0%), acid-related disorders (29.4% vs 20.2%), and beta-blockers (24.9% vs 19.9%). Conclusion The overall clinical burden on patients with PKU is exacerbated by a significantly higher risk of numerous comorbidities and hence, prescribing of the requisite medication, both for recognized (e.g. major depressive disorders) and more unexpected comorbidities (e.g. ischemic heart disease). Electronic supplementary material The online version of this article (10.1186/s13023-019-1153-y) contains supplementary material, which is available to authorized users.

AIMS: The aim of this study was to assess the burden of heart failure (HF) patients with/without iron deficiency/iron deficiency anaemia (ID/A) from the health insurance perspective.; METHODS AND RESULTS: We conducted a retrospective claims database analysis using the Institut fur angewandte Gesundheitsforschung Berlin research database. The study period spanned from 1 January 2012 to 31 December 2014. HF patients were identified by International Statistical Classification of Diseases and Related Health Problems, 10th revision, German Modification codes (I50.-, I50.0-, I50.00, I50.01, I50.1-, I50.11, I50.12, I50.13, I50.14, I50.19, and I50.9). HF patients were stratified into HF patients without ID/A and HF patients with ID/A (D50.-, D50.0, D50.8, D50.9, and E61.1). HF patients with ID/A were stratified into three subgroups: no iron treatment, oral iron treatment, and intravenous iron treatment. A matching approach was applied to compare outcomes for HF patients without ID/A vs. HF patient with untreated incident ID/A without iron treatment and for HF patients receiving no iron treatment vs. oral iron treatment vs. intravenous iron treatment. Matching parameters included exact age, sex, and New York Heart Association functional class. An optimization algorithm was used to balance total health care costs in the baseline period for the potential matched pairs without sample size reduction. In total, 172394 (4537.4 per 100000) HF patients were identified in the Institut fur angewandte Gesundheitsforschung Berlin research database in 2013. Of these, 11.1% (19070; 501.9 per 100000) were diagnosed with ID/A and/or had a prescription for iron medication in 2013. The mean age of HF patients was 77.0years (±12.0years). Women were more frequently diagnosed with HF (54.6%). HF patients with untreated incident ID/A (1.77%) had a significantly higher all-cause mortality than HF patients without ID/A (33.1% vs. 24.1%, P

AIMS: Several psychosocial factors have been shown to increase the risk of Type2 diabetes mellitus. This study investigated the association between structural social support and incidence of Type2 diabetes mellitus in men and women. METHODS: Data were derived from three population-based MONICA/KORA surveys conducted in 1984-1995 in the Augsburg region (southern Germany) and followed up by 2009. The study population comprised 8952 participants (4669 men/4283 women) aged 30-74years without diabetes at baseline. Structural social support was assessed using the Social Network Index. Sex-specific hazard ratios were estimated from Cox proportional hazard models. RESULTS: Within follow-up, 904 incident Type2 diabetes mellitus cases (558 men, 346 women) were observed. Crude incidence rates for Type2 diabetes mellitus per 10000person-years were substantially higher in poor compared with good structural social support (men: 94 vs. 69, women: 58 vs. 43). After adjustment for age, survey, parental history of diabetes, smoking status, alcohol intake, physical activity, hypertension, dyslipidaemia, BMI, education, sleep complaints and depressed mood, risk of Type2 diabetes mellitus for participants with poor compared with good structural social support was 1.31 [95% confidence interval (CI)=1.11-1.55] in men and 1.10 (95% CI=0.88-1.37) in women. Stratified analyses revealed a hazard ratio of 1.50 (95% CI=1.23-1.83) in men with a low level of education and 0.87 (95% CI=0.62-1.22) in men with a high level of education (P for interaction: 0.0082). CONCLUSIONS: Poor structural social support is associated with Type2 diabetes mellitus in men. This association is independent of risk factors at baseline and is particularly pronounced in men with a low level of education.

biologics. This Register will also be basis of a sustainable and comprehensive UK-wide IBD Register. The intention is to incorporate this into the clinical environment so that data can be captured at the point of care. Methods: The first objective was to define the scope and purpose of the Register. To do so, an information group was formed, including representatives from the British Society of Gastroenterology, British Society of Paediatric Gastroenterology Hepatology and Nutrition, IBD Audit group, Crohn’s and Colitis UK, the Primary Care Society for Gastroenterology, the Association of Coloproctology of Great Britain and Ireland, and the Royal College of Physicians in London. It was agreed that the data should answer four main questions about biologics treatment: Appropriateness of use, efficacy, safety and variability of access to biologics. The requirements were refined, and data identified, before the data collection structure and content was described, ensuring that this complied with the Royal College of Physicians’ recordkeeping standards. A generic clinical information system (GeneCIS), currently in use to support clinical care and research, is providing the IT infrastructure. GeneCIS allows data to be collected and used locally, but is remotely hosted and accessed via the secure National NHS Network (N3). Participating hospitals can collect and view their data, but data for national aggregate and analysis is pseudo-ananoymised and held separately. Local outputs such as individual history summaries and outpatient letters will provide support for day-to-day patient care. The Register will start in selected NHS trusts as part of the 3rd UK IBD audit, and incremently rolled out UK-wide. The information requirements, data to be collected, and overall structure have been agreed. Results: Implementation has started in pilot sites within the UK. Conclusions: The Biologics Register will provide a secure and practical data collection tool to assess safety and efficacy.