Age/disease duration influence on activities of daily living and quality of life after levodopa-carbidopa intestinal gel in Parkinson's disease

Aim: To determine if age and Parkinson's disease duration at therapy initiation influence the efficacy of levodopa-carbidopa intestinal gel (LCIG) on quality of life and activities of daily living. Patients & methods: This post hoc analysis assessed subgroups of patients stratified by baseline age, disease duration, hours/day of 'off' time and levodopa equivalent dose. Patients' data were collected from the GLORIA study, a 24-month observational registry evaluating long-term effectiveness of LCIG. Results & conclusion: LCIG therapy led to sustained improvements in quality of life irrespective of patient age and disease duration at baseline. Improvements in activities of daily living were observed across all subgroups, particularly in younger patients, patients with shorter disease duration and in patients with the highest baseline levodopa equivalent dose

The role and structure of the multidisciplinary team in the management of advanced Parkinson's disease with a focus on the use of levodopa-carbidopa intestinal gel

A multidisciplinary team (MDT) approach is increasingly recommended in Parkinson's disease (PD) treatment guidelines, but no standard of care exists for such an approach, and the guidelines do not provide clarification on how it should be implemented. This paper reviews evidence of MDT interventions in people with PD and provides expert clinical perspectives for an MDT approach, with a focus on advanced PD and levodopa-carbidopa intestinal gel (carbidopa-levodopa enteral suspension in the USA). The key recommendations are to enable the best possible treatment of people with PD locally by facilitating a close structured collaboration of different health care professionals working in a fixed network structure; to refer people with PD to established MDT centers in a timely manner; to establish regular meetings for the MDT enabling interdisciplinary exchange and learning; to optimize individual treatment and carefully evaluate available treatment options; to ensure treatment decisions are agreed jointly between people with PD, their caregivers, family, and health care professional; and to include specialists outside of neurology from adjuvant medical departments as necessary when implementing advanced therapies

Corrigendum to "Global long-term study on motor and non-motor symptoms and safety of levodopa-carbidopa intestinal gel in routine care of advanced Parkinson's disease patients; 12-month interim outcomes" [Parkinsonism Relat. Disord. 21(3) (2015) 231-235].

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Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national sample

The psychometric properties of a health-related quality of life (HRQOL) instrument, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), were evaluated in a national sample of patients with cystic fibrosis (CF).The Epidemiologic Study of CF is a national, multicenter, longitudinal cohort study containing CFQ-R and health outcomes data. Developmentally appropriate versions of the CFQ-R were available from 7,330 patients aged 6–70 years and a proxy version from 2,728 parents of school-age children. The CFQ-R was completed during a “stable” or “sick” visit before recording health outcomes such as weight, lung function, and pulmonary exacerbations.There were few floor and ceiling effects and strong internal consistency (Cronbach alpha ≥0.70) for most scales. The CFQ-R consistently discriminated between patients seen for sick-versus-well visits, and among stages of disease severity based on lung function. As predicted, women with CF reported worse HRQOL than men on scales not related to body image and weight. Strong parent–child agreement was found on scales measuring observable behaviors (respiratory symptoms). Convergence between CFQ-R scales and health outcomes provided evidence of construct validity.The CFQ-R demonstrated robust psychometric properties and consistent associations with health outcomes in a large national sample

Current challenges and future opportunities in patient‐focused management of hereditary angioedema: A narrative review

Abstract Patients with hereditary angioedema (HAE) experience a high burden of disease due to unpredictable, painful, disfiguring, and potentially life‐threatening HAE attacks. Multiple HAE‐specific medications for the on‐demand treatment, short‐term and long‐term prophylaxis of HAE attacks have entered the market in recent years; however, the availability and access to these medications may vary between different countries. For this review, PubMed and EMBASE databases were searched for guidelines, consensus statements, and other publications on HAE management as well as publications on quality of life in patients with HAE. The current guidelines and recent literature on HAE management in specific countries are summarized with the aim to highlight the similarities and differences between guideline recommendations and the country‐specific clinical practice. Improvement in quality of life, which is a key goal in HAE management, is also discussed and the country‐specific trends are highlighted. Finally, the ways to achieve a more patient‐centric approach to HAE management within the framework set by the clinical management guidelines are examined

Erratum to: Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national, US sample

The psychometric properties of a health-related quality of life (HRQOL) instrument, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), were evaluated in a national, US sample of patients with cystic fibrosis (CF). This is the first psychometric evaluation of the revised version of this instrument. The Epidemiologic Study of CF is a national, US multicenter longitudinal cohort study containing CFQ-R and health outcomes data. Developmentally appropriate versions of the CFQ-R were available from 7,330 patients aged 6-70 years and a proxy version from 2,728 parents of school-age children. The CFQ-R was completed during a "stable" or "sick" visit before recording health outcomes such as weight, lung function, and pulmonary exacerbations. There were few floor and ceiling effects and strong internal consistency (Cronbach alpha ≥0.70) for most scales. The CFQ-R consistently discriminated between patients seen for sick-versus-well visits, and among stages of disease severity based on lung function. As predicted, women with CF reported worse HRQOL than men on scales not related to body image and weight. Strong parent-child agreement was found on scales measuring observable behaviors (respiratory symptoms). Convergence between CFQ-R scales and health outcomes provided evidence of construct validity. The CFQ-R demonstrated robust psychometric properties and consistent associations with health outcomes in a large national, US sample. Normative data are available to aid in interpretation

Multiple antibiotic-resistant Pseudomonas aeruginosa and lung function decline in patients with cystic fibrosis

AbstractBackgroundThe goal of this study was to determine the association of multiple antibiotic-resistant Pseudomonas aeruginosa (MARPA) acquisition with lung function decline in patients with cystic fibrosis (CF).MethodsUsing data from Epidemiologic Study of Cystic Fibrosis (ESCF), we identified patients with spirometry data and MARPA, defined as PA (1) resistant to gentamicin and either tobramycin or amikacin, and (2) resistant to ≥1 antipseudomonal beta lactam. MARPA had to be detected in a respiratory culture after ≥2years of PA-positive but MARPA-negative respiratory cultures. Multivariable piecewise linear regression was performed to model the annual rate of decline in forced expiratory volume in 1second (FEV1) % predicted 2 calendar years before and after the index year of MARPA detection, adjusting for patient characteristics and CF therapies.ResultsIn total, 4349 patients with chronic PA and adequate PFT data were identified; 1111 subsequently developed MARPA, while 3238 patients were PA positive but MARPA negative. Compared with patients who did not acquire MARPA, MARPA-positive patients had lower FEV1 and received more oral (p<0.013) and inhaled (p<0.001) antibiotic therapy. Mean FEV1 decline did not change significantly after MARPA detection (−2.22% predicted/year before detection and −2.43 after, p=0.45). There was no relationship between persistent infection or FEV1 quartile and FEV1 decline.ConclusionsNewly detected MARPA was not associated with a significant change in the rate of FEV1 decline. These results suggest that MARPA is more likely to be a marker of more severe disease and more intensive therapy, and less likely to be contributing independently to more rapid lung function decline

Levodopa-carbidopa intestinal gel in advanced Parkinson's: Final results of the GLORIA registry

INTRODUCTION: This registry evaluated the 24-month safety and efficacy of levodopa-carbidopa intestinal gel (LCIG) treatment in advanced Parkinson's disease (PD) patients under routine clinical care. METHODS: Motor fluctuations, dyskinesia, non-motor symptoms, quality of life, and safety were evaluated. Observations were fully prospective for treatment-naïve patients (60% of patients) and partially retrospective for patients with ≤12 months of pre-treatment with LCIG (40% of patients). Hours of "On" and "Off" time were assessed with a modified version of the Unified Parkinson's Disease Rating Scale part IV items 32 and 39. RESULTS: Overall, 375 patients were enrolled by 75 movement disorder centers in 18 countries and 258 patients completed the registry. At 24 months LCIG treatment led to significant reductions from baseline in "Off" time (hours/day) (mean ± SD = -4.1 ± 3.5, P < 0.001), "On" time with dyskinesia (hours/day) (-1.1 ± 4.8, P = 0.006), Non-Motor Symptom Scale total (-16.7 ± 43.2, P < 0.001) and individual domains scores, and Parkinson's Disease Questionnaire-8 item total score (-7.1 ± 21.0, P < 0.001). Adverse events deemed to have a possible/probable causal relationship to treatment drug/device were reported in 194 (54%) patients; the most frequently reported were decreased weight (6.7%), device related infections (5.9%), device dislocations (4.8%), device issues (4.8%), and polyneuropathy (4.5%). CONCLUSIONS: LCIG treatment led to sustained improvements in motor fluctuations, non-motor symptoms particularly sleep/fatigue, mood/cognition and gastrointestinal domains, as well as quality of life in advanced PD patients over 24 months. Safety events were consistent with the established safety profile of LCIG

Key findings and clinical implications from The Epidemiology and Natural History of Asthma: Outcomes and Treatment Regimens (TENOR) study

Patients with severe or difficult-to-treat asthma are an understudied population but account for considerable asthma morbidity, mortality, and costs. The Epidemiology and Natural History of Asthma: Outcomes and Treatment Regimens (TENOR) study was a large, 3-year, multicenter, observational cohort study of 4756 patients (n = 3489 adults ≥18 years of age, n = 497 adolescents 13-17 years of age, and n = 770 children 6-12 years of age) with severe or difficult-to-treat asthma. TENOR's primary objective was to characterize the natural history of disease in this cohort. Data assessed semiannually and annually included demographics, medical history, comorbidities, asthma control, asthma-related health care use, medication use, lung function, IgE levels, self-reported asthma triggers, and asthma-related quality of life. We highlight the key findings and clinical implications from more than 25 peer-reviewed TENOR publications. Regardless of age, patients with severe or difficult-to-treat asthma demonstrated high rates of health care use and substantial asthma burden despite receiving multiple long-term controller medications. Recent exacerbation history was the strongest predictor of future asthma exacerbations. Uncontrolled asthma, as defined by the 2007 National Heart, Lung, and Blood Institute guidelines’ impairment domain, was highly prevalent and predictive of future asthma exacerbations; this assessment can be used to identify high-risk patients. IgE and allergen sensitization played a role in the majority of severe or difficult-to-treat asthmatic patients