The role of compliance in the treatment of chronic heart failure and the ways to improve it

Despite the development and implementation of clinical guidelines, emergence of modern effective drugs for the treatment of chronic heart failure (CHF), this pathology is characterized by stable progression, directly worsening the quality of life and decreasing the life expectancy. With that, treatment efficacy directly depends on high treatment compliance in patients with CHF both at the treatment start and upon its subsequent correction. One should also not underestimate the value of patient readiness to modify their lifestyle. Besides, the complete physician compliance with clinical guidelines also plays a significant role. The absence of correspondence between principles defined in the guidelines and really administered drugs excludes the possibility of achieving control over CHF symptoms and positive impact on the prognosis. This review is aimed at evaluating treatment compliance among both patients with CHF and their physicians with a focus on Russian studies and good clinical practice (GCP) compared to studies of foreign authors. Unfortunately, the problem of compliance in CHF in the Russian Federation has not been sufficiently analyzed in large-scale studies up to the present time: CHF patient registries mainly allow to detect clinical and epidemiological disease features, while almost not covering compliance issues. It should also be noted that the problem of compliance in both physicians and patients is undoubtedly up-to-date, as it often defines further prognosis in patients with CHF, which explains the scientific practical value of large trials with subsequent thorough analysis and searches regarding increasing its efficiency.

Trusted data spaces as a viable and sustainable solution for networks of population-based patient registries (Preprint)

BACKGROUND Population-based patient registries are entities that collect summary patient data from a well-defined population. Their main function is the monitoring and surveillance of a particular disease within their population catchment area, but they are also an important data source used in epidemiology. Comparing indicators across national boundaries brings considerable extra benefit to registries’ data, especially in regions where supranational initiatives are or could be coordinated to leverage good practices; this is particularly important for the European Union. Stricter data-protection laws however can unintentionally hamper the efforts of data harmonization to ensure the removal of statistical bias in the individual data sets, thereby compromising the integrated value of registries’ data. A new paradigm is required to ensure registries can operate in an environment that is not unnecessarily restrictive and allow accurate comparison of data for better ascertaining measures and practices most conducive to the public health of societies. OBJECTIVE To propose a solution towards a viable and sustainable model for the integration of registry data at supranational level. METHODS The pan-European organisational model of cancer registries, owing to its long and successful establishment, was taken as a good starting point from which to propose a sustainable, generic model for patient registries. Drawbacks to the model, particularly with respect to scalability and resourcing, were addressed in an adapted model. RESULTS An inter-registry organisational model based along the lines of the European Network of Cancer Registries was adapted to tackle the governance and resourcing aspects essential for a generic patient-registry model. The adapted model is a proposal for how patient registries can inter operate to ensure harmonisation and quality of data for accurate comparison at supranational level. CONCLUSIONS In view of the challenges relating to accurate and unbiased inter-comparison of population-based registry data across national boundaries for disease-surveillance purposes, a sustainable, generic patient-registry model is proposed. Integrating registry data is important for understanding progression and trends of the most prevalent diseases as well as for ascertaining effective control measures. The model promises a valuable data resource for epidemiological research, whilst providing a closely regulated environment for the processing of pseudonomised patient summary data on a broader scale than has hitherto been possible.

Burden of comorbid conditions in children and young people with juvenile idiopathic arthritis: a collaborative analysis of 3 JIA registries

Objectives Burden of comorbidities are largely unknown in JIA. From 2000, national and international patient registries were established to monitor biologic treatment, disease activity and adverse events in patients with JIA. The aim of this analysis was to investigate in parallel, for the first time, three of the largest JIA registries in Europe/internationally—UK JIA Biologic Registers (BCRD/BSPAR-ETN), German biologic registers (BiKeR/JuMBO), multinational Pharmachild—to quantify the occurrence of selected comorbidities in patients with JIA. Methods Information on which data the registers collect were compared. Patient characteristics and levels of comorbidity were presented, focussing on four key conditions: uveitis, MAS, varicella, and history of tuberculosis. Incidence rates of these on MTX/biologic therapy were determined. Results 8066 patients were registered into the three JIA registers with similar history of the four comorbidities across the studies; however, varicella vaccination coverage was higher in Germany (56%) vs UK/Pharmachild (16%/13%). At final follow-up, prevalence of varicella infection was lower in Germany (15%) vs UK/Pharmachild (37%/50%). Prevalence of TB (0.1–1.8%) and uveitis (15–19%) was similar across all registers. The proportion of systemic-JIA patients who ever had MAS was lower in Germany (6%) vs UK (15%) and Pharmachild (17%). Conclusion This analysis is the first and largest to investigate the occurrence of four important comorbidities in three JIA registries in Europe and the role of anti-rheumatic drugs. Combined, these three registries represent one of the biggest collection of cases of JIA worldwide and offer a unique setting for future JIA outcome studies.

Demographic characteristics, clinical and laboratory features, and the distribution of pathogenic variants in the CFTR gene in the Cypriot cystic fibrosis (CF) population demonstrate the utility of a national CF patient registry

Background Specialized clinical care for cystic fibrosis (CF) in Cyprus, a small island country, has been implemented since the 1990s. However, only recently, a national CF patient registry has been established for the systematic recording of patients’ data. In this study, we aim to present data on the epidemiological, genotypic and phenotypic features of CF patients in the country from the most recent data collection in 2019, with particular emphasis on notable rare or unique cases. Results Overall, data from 52 patients are presented, 5 of whom have deceased and 13 have been lost to follow-up in previous years. The mean age at diagnosis was 7.2 ± 12.3 years, and the mean age of 34 alive patients by the end of 2019 was 22.6 ± 13.2 years. Patients most commonly presented at diagnosis with acute or persistent respiratory symptoms (46.2%), failure to thrive or malnutrition (40.4%), and dehydration or electrolyte imbalance (32.7%). Sweat chloride levels were diagnostic (above 60 mmol/L) in 81.8% of examined patients. The most common identified mutation was p.Phe508del (F508del) (45.2%), followed by p.Leu346Pro (L346P) (6.7%), a mutation detected solely in individuals of Cypriot descent. The mean BMI and FEV1 z-scores were 0.2 ± 1.3 and − 2.1 ± 1.7 across all age groups, respectively, whereas chronic Pseudomonas aeruginosa colonization was noted in 26.9% of patients. The majority of patients (74.5%) were eligible to receive at least one of the available CFTR modulator therapies. In 25% of patients we recovered rare or unique genotypic profiles, including the endemic p.Leu346Pro (L346P), the rare CFTR-dup2, the co-segregated c.4200_4201delTG/c.489 + 3A > G, and the polymorphism p.Ser877Ala. Conclusions CF patient registries are particularly important in small or isolated populations, such as in Cyprus, with rare or unique disease cases. Their operation is necessary for the optimization of clinical care provided to CF patients, enabling their majority to benefit from evolving advances in precision medicine.

A prospective investigation of developmental trajectories of psychosocial adjustment in adolescents facing a chronic condition - study protocol of an observational, multi-center study

Background Relatively little is known about protective factors and the emergence and maintenance of positive outcomes in the field of adolescents with chronic conditions. Therefore, the primary aim of the study is to acquire a deeper understanding of the dynamic process of resilience factors, coping strategies and psychosocial adjustment of adolescents living with chronic conditions. Methods/design We plan to consecutively recruit N = 450 adolescents (12–21 years) from three German patient registries for chronic conditions (type 1 diabetes, cystic fibrosis, or juvenile idiopathic arthritis). Based on screening for anxiety and depression, adolescents are assigned to two parallel groups – “inconspicuous” (PHQ-9 and GAD-7 < 7) vs. “conspicuous” (PHQ-9 or GAD-7 ≥ 7) – participating in a prospective online survey at baseline and 12-month follow-up. At two time points (T1, T2), we assess (1) intra- and interpersonal resiliency factors, (2) coping strategies, and (3) health-related quality of life, well-being, satisfaction with life, anxiety and depression. Using a cross-lagged panel design, we will examine the bidirectional longitudinal relations between resiliency factors and coping strategies, psychological adaptation, and psychosocial adjustment. To monitor Covid-19 pandemic effects, participants are also invited to take part in an intermediate online survey. Discussion The study will provide a deeper understanding of adaptive, potentially modifiable processes and will therefore help to develop novel, tailored interventions supporting a positive adaptation in youths with a chronic condition. These strategies should not only support those at risk but also promote the maintenance of a successful adaptation. Trial registration German Clinical Trials Register (DRKS), no. DRKS00025125. Registered on May 17, 2021.

Physical Activity Is Associated With Lower Long-Term Incidence of Anxiety in a Population-Based, Large-Scale Study

Physical activity may prevent anxiety, but the importance of exercise intensity, sex-specific mechanisms, and duration of the effects remains largely unknown. We used an observational study design to follow 395,369 individuals for up to 21 years to investigate if participation in an ultralong-distance cross-country ski race (Vasaloppet, up to 90 km) was associated with a lower risk of developing anxiety. Skiers in the race and matched non-skiers from the general population were studied after participation in the race using the Swedish population and patient registries. Skiers (n = 197,685, median age 36 years, 38% women) had a significantly lower risk of developing anxiety during the follow-up compared to non-skiers (adjusted hazard ratio, HR 0.42). However, among women, higher physical performance (measured as the finishing time to complete the race, a proxy for higher exercise dose) was associated with an increased risk of anxiety compared to slower skiing women (HR 2.00). For men, the finishing time of the race did not significantly impact the risk of anxiety. Our results support the recommendations of engaging in physical activity to decrease the risk of anxiety in both men and women. The impact of physical performance level on the risk of anxiety requires further investigations among women.

What Are Patient Registries and Why Are They Needed (Through a Number of Examples of Psoriasis Registries)

The article substantiates the relevance of registries of patients as illustrated by registries of patients with psoriasis, gives a comparative characteristic and description of the advantages and disadvantages of randomized controlled and observational registry studies, considers the main goals of patient registers. The importance of patient registers as sources of additional knowledge about diseases is described. Review of the existing dermatological patient registers is given. The paper provides the detailed description of the most significant registries of patients with psoriasis: PSOLAR, Badbir, Corrona, BIOBADADERM and others. An overview of publications and main results of the research based on registries of psoriasis patients is presented. A description of the register of patients with psoriasis as part of the Database of patients with chronic diseases of the skin and subcutaneous tissue, maintained by the Russian Society of Dermatovenereologists and Cosmetologists since 2018 is given. The article substantiates the relevance of patient registries as illustrated by registries of patients with psoriasis, gives a comparative characteristic and description of the advantages and disadvantages of randomized controlled studies and observational registry studies, argues in favor of complementarity of data from randomized controlled trials and registry-based studies. Consideration of the main goals of patient registers is given. The importance of patient registers as sources of additional knowledge about diseases and quality of medical care is described. Review of the existing dermatological patient registers is given. The paper provides the detailed description of the most significant registries of patients with psoriasis: PSOLAR, Badbir, Corrona, BIOBADADERM and others. An overview of publications and main results of the research based on registries of patients with psoriasis is presented. A description of the register of patients with psoriasis as part of the Database of patients with chronic diseases of the skin and subcutaneous tissue, maintained by the Russian Society of Dermatovenereologists and Cosmetologists since 2018 is given, as are inclusion criteria. In addition, the main federal registries of patients that are maintained in the Russian Federation are listed, the goals of their maintenance are indicated.

Growth in children on kidney replacement therapy: a review of data from patient registries

Growth retardation is a major complication in children with chronic kidney disease (CKD) and on kidney replacement therapy (KRT). Conversely, better growth in childhood CKD is associated with an improvement in several hard morbidity–mortality endpoints. Data from pediatric international registries has demonstrated that improvements in the overall conservative management of CKD, the search for optimal dialysis, and advances in immunosuppression and kidney transplant techniques have led to a significant improvement of final height over time. Infancy still remains a critical period for adequate linear growth, and the loss of stature during the first years of life influences final height. Preliminary new original data from the European Society for Paediatric Nephrology/European Renal Association-European Dialysis and Transplant Association (ESPN/ERA-EDTA) Registry confirm an association between the final height and the height attained at 2 years in children on KRT.

Characterisation of Pediatric Pulmonary Hypertensive Vascular Disease from the PPHNet Registry

BackgroundThere are limited data about the range of diseases, natural history, age-appropriate endpoints and optimal care for children with pulmonary hypertension (PH), including the need for developing high quality patient registries of children with diverse forms of PH to enhance care and research.ObjectiveTo characterise the distribution and clinical features of diseases associated with pediatric PH, including natural history, evaluation, therapeutic interventions and outcomes, as defined by the WSPH Classification.Methods1475 patients were enrolled into a multisite registry across the Pediatric Pulmonary Hypertension Network (PPHNet), comprised of 8 interdisciplinary PH programs.ResultsWSPH Groups 1 (PAH) and 3 (lung disease) were the most common primary classifications (45% and 49% of subjects, respectively). The most common Group 3 conditions were BPD and CDH. Group 1 disease was predominantly associated with congenital heart disease (60%) and idiopathic (23% of Group 1 cases). In comparison with Group 1, Group 3 subjects had better disease resolution (HR=3.1, p<0.001), tended to be younger at diagnosis (0.3 (0.0,0.6) versus 1.6 (0.1,6.9) years (median (IQR); p<0.001), and were more often male (57% versus. 45%, p<0.001). Down syndrome (DS), the most common genetic syndrome in the registry, constituted 11% of the entire PH cohort.ConclusionsWe find a striking proportion of pediatric PH patients with Group 3 disorders, reflecting the growing recognition of PH in diverse developmental lung diseases. Greater precision of clinical phenotyping based on disease-specific characterization may further enhance care and research of pediatric PH.

Recurrent deep venous thromboses in a patient with adult-onset Still’s disease

Adult-onset Still’s disease (AOSD) is a rare inflammatory disorder affecting just over one in a million people. Due to its rarity, understanding of its pathophysiology and the spectrum of its clinical associations are limited. Improved case identification and creation of patient registries have begun to reveal sporadic reports of deep venous thromboses associated with AOSD. Herein, we report the first case of recurrent deep venous thrombosis in a patient with AOSD despite treatment with therapeutic dose anticoagulant medication. This case points for a judicious approach to the selection of an anticoagulation strategy for deep venous thromboses in the setting of active AOSD. This case is of contemporary interest in its clinical similarity with COVID-19 symptoms and pathophysiology for which a careful diagnostic approach with a broad differential should be considered given the limitations of SARS-CoV-2 testing and the risk associated with treatment in the event of misdiagnosis.