Search

Objectives: This study aims to explore the impact of Charcot-Marie-Tooth disease type 1A (CMT1A) and its treatment on patients in European (France, Germany, Italy, Spain, and the United Kingdom) and US real-world practice., Methods: Adults with CMT1A (n = 937) were recruited to an ongoing observational study exploring the impact of CMT. Data were collected via CMT&Me, an app through which participants completed patient-reported outcome measures., Results: Symptoms ranked with highest importance were weakness in the extremities, difficulty in walking, and fatigue. Almost half of participants experienced a worsening of symptom severity since diagnosis. Anxiety and depression were each reported by over one-third of participants. Use of rehabilitative interventions, medications, and orthotics/walking aids was high., Conclusions: Patient-reported burden of CMT1A is high, influenced by difficulties in using limbs, fatigue, pain, and impaired quality of life. Burden severity appears to differ across the population, possibly driven by differences in rehabilitative and prescription-based interventions, and country-specific health care variability., Competing Interests: The authors report no conflicts of interest., (Copyright © 2022 The Author(s). Published by Wolters Kluwer Health, Inc.)

Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and sensory neuropathy affecting the peripheral nervous system. This study will explore the real-world impact of CMT. The trial is a digital study of approximately 2000 people in 6 countries with CMT ≥18 years. Participants will use a smartphone application to check eligibility, provide consent and contribute data. The dataset will include a personal profile, covering demographics, lifestyle, diagnosis and treatment and a selection of validated generic and disease-specific instruments. Participants will provide data for up to 2 years. Data analysis will be conducted upon registration of the 1000th participant and at 12-month intervals from launch. This study is designed to help researchers and clinicians understand the real-world impact of CMT and the unmet needs of patients. ClinicalTrials.gov identifier: NCT03782883.

Objective: To assess the use of mobile coronary care units (MCU) in hypertensive patients previously treated for cardiovascular diseases in comparison with those with no history of cardiovascular disease and to estimate the influence of the use of MCU on cardiovascular outcome in this population., Patients: We used a nationwide prospective registry of all patients admitted for AMI in French intensive care units in 2000. Patients without history of hypertension or patients admitted with pulmonary oedema or cardiogenic shock were excluded. Men (N = 514) and women (N = 291) were analysed separately., Results: The proportion of patients with history of myocardial infarction, peripheral artery disease and stroke was not significantly higher in subjects who used physician-staffed MCU as compared with patients with no history of myocardial infarction, peripheral artery disease or stroke. In each sex, revascularization (pre hospital fibrinolysis, in hospital fibrinolysis or coronary angioplasty) were more frequent in patients who used MCU. Also, one year cardiovascular mortality was lower in men who used MCU., Conclusion: Known high risk hypertensive patients did not use physician-staffed MCU more than subjects free of such condition. Education of hypertensive patients at risk during routine visits is required to increase of the use of physician-staffed MCU in case of symptoms suggestive of AMI.

Objective: Several studies underlined the worse prognosis of myocardial infarction (MI) among patients with peripheral arterial disease (PAD). We sought to describe the presentation and management modalities of a cohort of PAD patients presenting an acute MI, compared to those without PAD., Materials and Methods: The USIC 2000 registry, a nationwide database on all patients admitted to a CCU for an acute MI < 48 hours in France in November 2000 was used for this study., Results: Among the 2311 patients included, PAD was reported in 215 subjects (9.3%). In multivariate analysis, the following factors were positively related to the presence of PAD (P < or = 0.05): age >75 y (OR = 2.3), diabetes (OR = 2.0), hypertension (OR = 1.4), active smoking (OR = 4.6), renal failure (OR =3.1), and treatments with antiplatelets (OR = 3.9), anti-vitamin K (OR = 1.9), statins (OR = 1.7) and low molecular weight heparins (OR = 6.8). By introducing the data concerning the arrival in CCUs in the model, the following factors were also significantly more frequent among PAD patients: male sex (OR = 1.6), past history of coronary artery disease (OR = 2.2), left bundle branch block (OR = 1.8) and late management >6 hours (OR = 1.4). Conversely, ST-segment elevation was less frequent (OR = 0.7). When the CCU stay data were introduced in the model, a lower rate of coronary stenting (OR = 0.7) and betablockers use within 48 hours of admission (OR = 0.6) were noted., Conclusion: Beyond the presence of PAD per se, several particularities do exist, especially the coexistence of a high number of pejorative factors and an under-utilization of treatments presenting prognostic benefits.

We evaluated the association of statin initiation within 48 hours of admission for an acute myocardial infarction with a 1-year prognosis on a nationwide scale. Patients who received a statin within 48 hours of admission but not before hospitalization had an improved prognosis (hazard ratio 0.57, 95% confidence interval 0.38 to 0.86, p <0.007) after adjustment for covariates and propensity score.

Objective: To assess actual practices and in-hospital outcome of patients with acute myocardial infarction on a nationwide scale., Methods: Of 443 intensive care units in France, 369 (83%) prospectively collected data on all cases of infarction (within < 48 hours of symptom onset) in November 2000., Results: 2320 patients (median age 68 years, 73% men) were included, of whom 83% had ST segment elevation infarction (STEMI). Patients without STEMI were older and had a more frequent history of cardiovascular disease. Median time to admission was 5.0 hours for patients with and 6.5 hours for those without STEMI. Reperfusion therapy was used for 53% of patients with STEMI (thrombolysis 28%, primary angioplasty 25%). In-hospital mortality was 8.7% (5.5% of patients without and 9.3% of those with STEMI). Multivariate analysis found that age, Killip class, lower blood pressure, higher heart rate on admission, anterior location of infarct, STEMI, diabetes mellitus, previous stroke, and no current smoking independently predicted in-hospital mortality. At hospital discharge, 95% received antiplatelet agents, 75% received beta blockers, and over 60% received statins. Angiotensin converting enzyme inhibitors were prescribed for 40% of the patients without and 52% of those with ST elevation., Conclusions: This nationwide registry, including all types of centres irrespective of their size and experience, shows continued improvement in patient care and outcomes. Time from symptom onset to admission, however, has not improved in recent years and reperfusion therapy is used for just over 50% of patients with STEMI, with an increasing use of primary angioplasty.

Background: Limited data are available on the impact of prehospital thrombolysis (PHT) in the "real-world" setting., Methods and Results: Of 443 intensive care units in France, 369 (83%) prospectively collected all cases of infarction (< or =48 hours of symptom onset) in November 2000; 1922 patients (median age, 67 years; 73% men) with ST-segment-elevation infarction were included, of whom 180 (9%) received intravenous thrombolysis before hospital admission (PHT). Patients with PHT were younger than those with in-hospital thrombolysis, primary percutaneous interventions, or no reperfusion therapy. Median time from symptom onset to hospital admission was 3.6 hours for PHT, 3.5 hours for in-hospital lysis, 3.2 hours for primary percutaneous interventions, and 12 hours for no reperfusion therapy. In-hospital death was 3.3% for PHT, 8.0% for in-hospital lysis, 6.7% for primary percutaneous interventions, and 12.2% for no reperfusion therapy. One-year survival was 94%, 89%, 89%, and 79%, respectively. In a multivariate analysis of predictors of 1-year survival, PHT was associated with a 0.49 relative risk of death (95% CI, 0.24 to 1.00; P=0.05). When the analysis was limited to patients receiving reperfusion therapy, the relative risk of death for PHT was 0.52 (95% CI, 0.25 to 1.08; P=0.08). In patients with PHT admitted in < or =3.5 hours, in-hospital mortality was 0% and 1-year survival was 99%., Conclusions: The 1-year outcome of patients treated with PHT compares favorably with that of patients treated with other modes of reperfusion therapy; this favorable trend persists after multivariate adjustment. Patients with PHT admitted very early have a very high 1-year survival rate.

We assessed the in-hospital management and short- and long-term outcomes of two series of patients admitted for acute myocardial infarction, 5 years apart, in France. The most recent cohort was younger and with a less frequent history of cardiac diseases, but was more often diabetic and with anterior infarcts. Five-day mortality significantly improved from 7.7% to 6.1% (P < 0.03) and 1-year survival was also less in the most recent period (15% versus 19%, P < 0.01). Multivariate analyses showed that the period of inclusion (2000 versus 1995) was an independent predictor of both short- and long-term mortality. In analyses restricted to the patients who were alive by day 5, initial treatment with statins was associated with a 38% decrease in the risk of death at 1 year. Likewise, in patients with left ventricular ejection fraction < or = 35%, the early prescription of ACE inhibitors was associated with a 41% reduction in the risk of 1-year mortality. Thus, in the real world setting, a continued decline in 1-year mortality is observed in patients admitted to intensive care units for recent acute myocardial infarction. This goes along with a shift in reperfusion therapy towards a broader use of coronary angioplasty and with an increased use of the early prescription of recognised secondary prevention medications.

Background: The efficacy and tolerability of oral telithromycin 800 mg once daily for 5 vs. 10 days were assessed in patients with acute maxillary sinusitis (AMS)., Methods: Adults (n = 341) with confirmed AMS diagnosed on clinical signs and symptoms and sinus X-ray showing total opacity or air-fluid level were randomized to receive oral telithromycin for 5 days (followed by placebo for 5 days; n = 170) or 10 days (n = 171). Causative pathogens were isolated by pretreatment sinus puncture (day 1). Clinical and bacteriologic outcomes, and safety and tolerability endpoints were assessed., Results: Clinical cure rates post-therapy (per-protocol; days 17-21) were comparable (91.1% in the 5-day group, n = 123; 91.0% in the 10-day group, n = 133). Bacteriologic eradication rates (per-protocol) were also similar (90.7 vs. 91.3%). Both regimens were well tolerated., Conclusions: A 5-day course of telithromycin 800 mg once daily is an effective, well-tolerated treatment for adults with AMS, comparable to a 10-day regimen., (Copyright 2002 S. Karger AG, Basel)