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Background Pulmonary hypertension is a rare, chronic and life-threatening group of diseases. Recent advances in pulmonary hypertension management prolong survival and improve quality-of-life. However, highly complex drug therapy enhances the risk of drug-related problems. Objective To assess the impact of involving clinical pharmacists in the collaborative care of pulmonary hypertension patients. Setting Ten French University Hospital Pneumology departments, all members of the French Network for Pulmonary Hypertension. Methods This prospective multicenter randomized controlled trial included incident pulmonary hypertension patients who were followed-up for 18 months. Randomization using an adapted Zelen method allocated patients to collaborative care (n = 41) or usual care groups (n = 51). A collaborative care program involving clinical pharmacists was developed through a close partnership between with physicians, nurses and pharmacists. Besides usual care, the program includes regular one-to-one interviews between the pharmacist and the patient. These interviews had following objectives: to perform an exhaustive medication history review; to identify the patient' needs, knowledge and skills; to define educational objectives and to provide patients with relevant information when needed. Following each interview, a standardized report form containing the pharmacist's recommendations was provided to physicians and nurses and discussed collaboratively. An ancillary economic analysis was performed. Main outcome measure Number of drug-related problems and their outcomes. Results The number of drug-related problems was not significantly different between groups (1.6 ± 1.5 vs. 1.9 ± 2.4; p = 0.41). More problems were resolved in the collaborative care group than in the usual care group (86.5% vs. 66.7%, p = 0.01). Time to clinical worsening, therapeutic adherence, satisfaction or quality-of-life were not statistically different between groups. Collaborative care decreased costs of drug-related hospitalizations. Conclusion Including clinical pharmacists in the multidisciplinary care of hospitalized patients with pulmonary hypertension improved the outcome of drug-related problems and reduced the costs of related hospitalization. However, we observed no efficacy on medication errors, clinical outcomes or medication adherence. Clinical Trial Registration ClinicalTrials.gov Identifier NCT01038284. [ABSTRACT FROM AUTHOR]

Background: Recent case reports suggest that benfluorex, a fenfluramine derivative used in the management of overweight diabetic patients and dyslipidemia, is associated with cardiac valve regurgitation. Methods: We conducted a case-control study. Eligible patients were those admitted in the cardiology or the cardiac surgery units of our hospital between January, 1st 2003 and June 30th 2009, with mitral insufficiency diagnostic codes (ICD-10 I340 and I051). Patients with either a primary cause (degenerative, known rheumatic heart disease, infectious endocarditis, congenital, radiation-induced valvular disease, associated connective and/or vasculitis disease, trauma, tumor) or a secondary (functional) cause were considered as having an ‘‘explained’’ mitral regurgitation. Other patients were considered as having an ‘‘unexplained’’ mitral regurgitation and were included as cases. For each case, two controls were matched for gender and for the closest date of birth, among a list of patients with an ‘‘explained’’ mitral regurgitation. Drug exposures were assessed blindly regarding the case or control status, through contacts with patients, their family and/or their physicians. Results: Out of the 682 eligible patients, 27 cases and 54 matched controls were identified. The use of benfluorex was reported in 22 patients: 19 of the 27 cases, versus 3 of the 54 controls, odds-ratio 17.1 (3.5 to 83), adjusted for body mass index, diabetes and dexfenfluramine use. Conclusion: The use of benfluorex is associated with unexplained mitral regurgitation. [ABSTRACT FROM AUTHOR]

Cystic fibrosis (CF) is the most common autosomal lethal recessive disorder in the Caucasian population. The major cause of mortality is lung disease, owing to the failure of a functional protein from the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Today, even though the knowledge about the CFTR genomic is extensive, no efficient treatment has been developed yet. In this context, gene therapy represents a potential important advance on condition that it could develop efficient and safe transfection agents. Even though viral vectors have been used in most clinical trials owing to their high transfection efficiency, random integration and immunogenicity are still critical side effects. Consequently, all of these drawbacks brought forth the development of nonviral transfection systems. Although they engender few toxicity and immunogenicity problems, their low transfection efficiency is a hurdle that must be overcome. Over the past decade, we have developed an original family of monocationic lipids, cationic phosphonolipids, whose efficiency has been previously demonstrated both in vitro and in vivo. In this report, we observe that a new cationic phosphonolipid (KLN 30) can lead to the restoration of the CFTR protein following the ex vivo transfection of epithelial cells issuing from a ΔF508 homozygous patient. The transgene expression and the cytotoxicity correlate with the charge ratio of the lipoplex. A kinetic study was performed, and a luminescent signal was detected until 35 d after transfection. [ABSTRACT FROM AUTHOR]

During a prospective evaluation of malaria prophylaxis in pregnancy in a refugee population on the north-western border of Thailand from 1987 to 1990, an extremely high infant mortality rate (18%) was documented despite good access to health care. Infantile beri-beri was recognized as the main cause of death accounting for 40% of all infant mortality. Thereafter, severe vitamin B1 deficiency in infants was diagnosed and treated promptly. The impact of this was assessed prospectively from 1993 to 1996 in a second cohort study. The case fatality of infantile beri-beri fell from almost 100% to 7%. The overall infant mortality rates declined from 183 to 78 per 1000 live births. Post-neonatal deaths fell by 79% (95% CI 65–87%) while neonatal mortality remained unchanged. Mortality resulting from acute respiratory infections did not change (15 and 11 per 1000, respectively), whereas mortality attributable to beri-beri decreased from 73 to 5 per 1000 (P < 0.0001). Before its recognition approximately 7% of all infants in this population died from infantile beri-beri. This lethal but preventable syndrome may be more common than hitherto recognized, particularly in refugee populations, in this populous region. [Copyright &y& Elsevier]

The article presents the author's views regarding the evidences demonstrating the association between the development of valvular heart disease and the use of appetite suppressants including fenfluramine. The author is critical on the evidence for a dose-effect relationship of the drugs as well as its causal factor. She also examines the evolution of valve damage after Benfluorex cessation.