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La iatrogénie médicamenteuse ne cesse de s’accroître avec l’augmentation conséquente de l’espérance de vie des patients et de la polypathologie associée. La polymédication associée à cette hausse de la polypathologie engendre une augmentation du risque d’erreurs médicamenteuses (EM). Le sujet âgé fait donc partie par corrélation des patients les plus à risque d’erreurs médicamenteuses. À ce jour, si l’on suit les recommandations données par la Haute Autorité de santé (HAS), tous les patients hospitalisés les « plus à risque » devraient recevoir une conciliation médicamenteuse.

Alors que l’utilité de la mesure de la qualité de vie dans l’évaluation des prises en charge est à ce jour consensuelle dans la communauté des médecins et des chercheurs, il n’en reste pas moins vrai que cette mesure est encore insuffisamment utilisée. Nous proposons, d’une part, d’explorer et comprendre les différents freins relatifs à la sous-utilisation de cette mesure, et, d’autre part, de promouvoir l’intérêt de cette mesure pour le clinicien, le chercheur et le décideur. Nous abordons les difficultés inhérentes au choix des questionnaires du fait de leur multiplicité, des propriétés métriques insuffisamment documentées, de leur acceptabilité. Nous mettons en évidence l’importance de la connaissance des déterminants de la qualité de vie ou de l’intérêt de cette mesure dans la prédiction de l’évolution des états de santé. Enfin, nous développons les difficultés liées à l’interprétation des résultats d’évaluation de la qualité de vie.

We evaluated the impact of central nervous system irradiation (CNSI) on long-term health status and quality of life (QoL) of childhood lymphoblastic leukemia survivors included in the French L.E.A. (Childhood and Adolescent Leukemia) multicentric cohort. QoL was self-reported in adults and assessed by parents in children and adolescents, using adapted questionnaires. From 2004 to 2009, 630 nongrafted patients were assessed after 11.8±6.3 years from diagnosis. Patients receiving CNSI (18.6%) or chemotherapy alone (81.4%) were compared. The risk of having long-term physical effects was increased with CNSI (odds ratio=3.3; 95% confidence interval, 1.8-5.9), especially regarding growth failure, second tumor, cataract, and overweight. QoL did not differ significantly according to the treatment received, despite a tendency toward lower scores with CNSI in children and adolescents (summary score 63.6±13.3 vs. 71.7±12.4, P=0.14). Compared with French norms, adult survivors had an impaired QoL, especially in mental domains (mental composite score 45.2±9.8 vs. 47.9±2.1, P<0.001). In pediatric survivors, QoL was not impaired and even tended to be higher than population norms (summary score 71.7±12.4 vs. 70.0±4.2, P=0.054), mainly in social and relational domains. In conclusion, QoL seems to be impaired by the trauma of a life-threatening illness in childhood, as well as by the treatment received.

AbstractObjective:Most of the existing studies investigating the impact of schizophrenia on utility have focused on the different stages of the disease. The objective of this study was to describe and quantify the impact of treatment-related side-effects on utility in patients with schizophrenia, using data from an observational study.Methods:This study used data from the European Schizophrenia Cohort (EuroSC), a multi-center 2-year cohort study conducted in France, England, and Germany. The EQ-5D questionnaire was completed every 6 months, as well as the Subjective Side Effect Rating Scale, assessing patient distress over extrapyramidal symptoms (EPS), weight gain, sedation, and sexual dysfunction, used to assess whether or not the patient experienced the side-effect. At first a bivariate analysis was conducted to describe utility values with and without side-effects. Then, a random effects regression analysis was performed on utility, where random effects were controlled for repeated measures on the same subjects, with potential confounding factors. Finally, findings were compared with those of previous publications.Results:This sample consisted of 1208 patients with schizophrenia. At the baseline visit, the most reported side-effect was EPS (almost 60 of patients), followed by sedation and weight gain (∼50 of patients for each), and sexual dysfunction (almost 30 of patients). Significant association with severity of symptoms, functioning abilities, and utility were found. Patients reporting none of the studied side-effects had an average EQ-5D index score of 0.81, found to be higher than scores of patients reporting EPS (0.70), sexual dysfunction (0.67), sedation (0.70), or weight gain (0.72). The random effects model reported a utility decrement of 0.042 for EPS, 0.022 for weight gain, 0.022 for sexual dysfunction, and 0.019 for sedation. Although the external validation was difficult due to the different methods or definitions of the side-effects, as well as the paucity of data for weight gain, sedation and sexual dysfunction, the results were generally consistent with previous studies.Conclusion:This study aimed at quantifying the direct impact of main side-effects associated with antipsychotics on patients’ utility. Results suggested a significant direct impact of side-effects, with EPS being the most impactful.

Few studies have assessed the role of cross-sex hormones on psychological outcomes during the period of hormonal therapy preceding sex reassignment surgery in transsexuals. The objective of this study was to assess the relationship between hormonal therapy, self-esteem, depression, quality of life (QoL), and global functioning. This study incorporated a cross-sectional design. The inclusion criteria were diagnosis of gender identity disorder (Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision) and inclusion in a standardized sex reassignment procedure. The outcome measures were self-esteem (Social Self-Esteem Inventory), mood (Beck Depression Inventory), QoL (Subjective Quality of Life Analysis), and global functioning (Global Assessment of Functioning). Sixty-seven consecutive individuals agreed to participate. Seventy-three percent received hormonal therapy. Hormonal therapy was an independent factor in greater self-esteem, less severe depression symptoms, and greater “psychological-like” dimensions of QoL. These findings should provide pertinent information for health care providers who consider this period as a crucial part of the global sex reassignment procedure.

L’évaluation d’une technologie de santé s’accompagne souvent d’une incertitude sur les conséquences de son introduction sur la santé de la population. Un recueil de données complémentaires, permettant une réévaluation des technologies concernées, peut être demandé par les autorités de santé. La responsabilité de recueillir ces données « post-inscription » revient aux industriels. La méthodologie proposée est évaluée par la Haute autorité de santé. Ce guide a pour objectif de donner des points de repère sur les aspects méthodologiques de ces études. Il décrit les types d’études envisageables en fonction des objectifs, incluant le recours aux bases de données et cohortes déjà existantes. Il souligne l’importance de constituer un comité scientifique, de définir clairement les objectifs de l’étude, de justifier les choix méthodologiques, de documenter la représentativité ou l’exhaustivité des centres, investigateurs et patients, de limiter les sujets perdus de vue et les données manquantes, de décrire les méthodes d’analyse statistique, les biais et leur impact possible sur les résultats. La publication des résultats de ces études est encouragée.

Health-related quality-of-life instruments are critical for assessing disease burdens. Generic tools allow comparison between diseases but do not discriminate between disease severities. Specific tools also tend to be more sensitive. No specific tool is available to assess quality of life in patients with Huntington’s disease (HD). In the context of the European study on HD burden, a specific tool was created: the Huntington Quality of Life Instrument (H-QoL-I). The aim of this study was to optimize the content and validate the H-QoL-I. After a semistructured interview with patients, caregivers and HD specialists, we conducted a patient focus group. A self-reported questionnaire was then developed in French and Italian. A total of 252 patients were recruited to answer the questionnaire. Face, internal and external validities were examined using a variety of methods. The shortened H-QoL-I that resulted from the successive analyses comprises 11 items, which are divided into three dimensions: motor functioning (four items), psychology (four items) and socializing (three items). These three domains were identified as being essential to cover the full domain of the quality of life for patients affected by HD. The H-QoL-I showed an acceptable reliability (Cronbach’s >0.84). Factor analyses demonstrated satisfactory construct validity. Moreover, the item internal consistency and item discriminant validity criteria were fulfilled. No differential item functioning was detected. External validity supported the scale’s robustness. These data support the validity of the H-QoL-I in patients with HD.

Femoral and lumbar bone mineral densities (BMDs) were measured in 159 adults enrolled in the Leucémies de l'Enfant et de l'Adolescent program, a French prospective multicentric cohort of childhood leukemia survivors. BMDs were expressed as Z-scores, and multivariate linear regression analyses were used to construct association models with potential risk factors. Mean age at evaluation and follow-up was 23 and 14.7 years, respectively. In the whole cohort, mean femoral Z-score was −0.19 ± 0.08. Two factors were associated with lower femoral BMD transplantation (−0.49 ± 0.15 vs −0.04 ± 0.10 in the chemotherapy group; P= .006) and female sex (−0.34 ± 0.10 vs −0.03 ± 0.13; P= .03). Among patients who received a transplant, the only significant risk factor was hypogonadism (−0.88 ± 0.16 vs −0.10 ± 0.23; P= .04). A slight reduction in lumbar BMD (mean Z-score, −0.37 ± 0.08) was detected in the whole cohort without difference between the transplantation and chemotherapy groups. Among patients who received a transplant, younger age at transplantation was correlated with a low lumbar BMD (P= .03). We conclude that adults who had received only chemotherapy for childhood leukemia have a slight reduction in their lumbar BMD and a normal femoral BMD. Patients who received a transplant with gonadal deficiency have a reduced femoral BMD which might increase the fracture risk later in life.

We evaluate the prevalence and risk factors of the metabolic syndrome (MS) in young adults surviving childhood leukemia. During the years 2007 to 2008, assessment of MS was proposed to all adults included in the Leucémie de l'Enfant et de l'Adolescent program, a French prospective multicentric cohort of leukemia survivors. Among 220 eligible patients, 184 (83.6%) had complete evaluation. Median age at evaluation and follow-up duration were 21.2 and 15.4 years. Overall prevalence of MS was 9.2% (95% confidence interval, 5.5-14.4). There was no association of MS with sex, age at diagnosis, leukemia subtype, steroid therapy, and central nervous system irradiation. Patients were stratified according to 4 therapeutic modalities: chemotherapy alone (n = 97), chemotherapy and central nervous system irradiation (n = 27), hematopoietic stem cell transplantation (HSCT) without (n = 17) or with (n = 43) total body irradiation (TBI). MS occurred in 5.2%, 11.1%, 5.9%, and 18.6% of them, respectively. The higher risk observed in the HSCT-TBI group was significant in univariate and in multivariate analysis (odds ratio [OR] = 3.9, P = .03). HSCT with TBI was associated with a higher rate of hypertriglyceridemia (OR = 4.5, P = .004), low level of high-density lipoprotein cholesterol (OR = 2.5, P = .02), and elevated fasting glucose (OR = 6.1, P = .04) So, TBI is a major risk factor for MS. Further studies are warranted to explain this feature.

The target population of a medicine may include different populations that may partially overlap including the population that has been evaluated in the clinical trials, the population for which the medicine provides an actual benefit (SMR), that for which the drug provides an improvement of the actual benefit (ASMR), etc. The definition of the target population in both qualitative and quantitative terms has key public health and economic implications. Recommendations are made to shed light on the definitions, to clarify the requests of the public decision makers and to improve the methods and the sources allowing the quantification of target populations.

La population cible d’un médicament en vue du remboursement peut inclure différentes populations qui peuvent se recouvrir partiellement : la population qui a été étudiée dans les essais cliniques, celle de l’autorisation de mise sur le marché (AMM), celle du service médical rendu (SMR), celle de l’amélioration du service médical rendu (ASMR), etc. Sa définition qualitative et quantitative implique des enjeux importants en termes de santé publique et économique. Des recommandations ont été faites afin de clarifier les définitions, d’éclaircir les souhaits des décideurs publics et d’améliorer les méthodes et les sources permettant une quantification des populations cibles.

AbstractObjective:To determine the treatment pattern and impact on healthcare costs of anxiety disorders and major depressive disorder (MDD), and influence of their concomitance and subsequence.Methods:A retrospective cohort study was conducted using a US reimbursement claims database. Adult patients with an incident diagnosis of anxiety or MDD (index date) were included. Their sociodemographic data, diagnoses, healthcare resource use and associated costs were collected over the 6 months preceding and 12 months following index date.Results:A total of 599,624 patients were identified and included. Patients with phobia or post-traumatic stress disorder had the highest 12-month costs (8,442 and 8,383, respectively). Patients with social anxiety disorder had the lowest costs (3,772); generalized anxiety disorder (6,472) incurred costs similar to MDD (7,170). Costs were substantially increased with emergence of anxiety during follow-up in MDD patients (10,031) or emergence of MDD in anxiety patients (9,387). This was not observed in patients with both anxiety and MDD at index date (6,148).Conclusion:This study confirms the high burden of costs of anxiety, which were within the same range as MDD. Interestingly, the emergence of anxiety or MDD in the year following a first diagnosis of MDD or anxiety, respectively, increased costs substantially. Major limitations were short follow-up and lack of absenteeism costs.

Le circuit des données de vigilance des essais cliniques le plus largement adopté dans la recherche clinique institutionnelle nécessite une double voire une triple saisie des informations dans différentes bases de données (clinique, vigilance) et sur divers supports (électronique, papier). Celles-ci occasionnent inévitablement une étape de réconciliation complexe et chronophage. Un tel circuit augmente le risque d'erreurs et compromet la fiabilité des données, ainsi que la sécurité des patients pouvant entraîner un délai significatif entre la survenue de l’évènement indésirable grave (EIG) et sa déclaration aux autorités compétentes. Dans ce contexte, la Direction de la Recherche Santé Maladies Rares des Hôpitaux de Marseille s'est orientée vers l'implémentation d'un formulaire électronique de notification des EIG via son Electronic Data Capture REDCap®.

To examine the association between use of health care services and health status, sociodemographic, and health care system characteristics in children and adolescents from 11 European countries.

Pulmonary function tests are used to select patients with non–small-cell lung cancer (NSCLC) suitable for thoracic surgery. We studied the impact of pulmonary function tests on both quantitative (morbidity, mortality, and overall survival [OS]) and qualitative (quality of life [QOL]) outcomes of patients undergoing thoracic surgery for NSCLC.

Background: Despite dual antiplatelet therapy, the rate of major adverse cardiovascular events (MACE) after percutaneous coronary angioplasty remains high. Studies have shown interindividual variations in response to clopidogrel. Furthermore, there is an apparent link between clinical outcomes and clopidogrel resistance. Objectives: To investigate the value of platelet reactivity index (PRI), assessed by vasodilator‐stimulated phosphoprotein (VASP) phosphorylation analysis, for predicting MACE after percutaneous coronary intervention (PCI) with stent implantation.Methods: A prospective monocentric study was performed on 144 patients undergoing PCI. PR was evaluated by VASP phosphorylation analysis 24 h after they received a 300‐mg loading dose of clopidogrel. MACE were recorded during a 6‐month follow‐up. Patients were divided into quintiles according to PRI, as assessed by VASP analysis. The receiver operating characteristic (ROC) curve served to determine the optimal cut‐off value of VASP analysis to detect MACE.Results: Of the 144 patients, 34% had stable angina pectoris, 40% silent ischemia, and 26% low‐risk non‐ST‐segment elevation acute coronary syndrome. During the follow‐up, 21 MACE were observed. Patients in quintile 1 of VASP analysis had a significantly lower risk of MACE as compared with those among the four higher quintiles (0 vs. 21, P < 0.01). ROC curve analysis of VASP showed an optimal cut‐off value of 50% PR to exclude MACE. The negative predictive value of the test was 100%.Conclusions: VASP phosphorylation analysis can evaluate the individual response to clopidogrel loading dose prior to PCI and predict postprocedural MACE.

L’analyse des données de vie réelle à partir des systèmes d’information hospitaliers pourrait permettre de trancher sur l’efficacité et la sécurité des traitements prescrits dans le Covid-19 en évitant les écueils des études préliminaires et des essais cliniques randomisés. Les différentes thérapeutiques testées dans les essais cliniques en cours sont déjà largement prescrites aux patients par les médecins dans les hôpitaux, et peuvent donc faire l’objet d’analyses immédiates selon des standards méthodologiques validés.

Contrast nephropathy (CN) is a common cause of renal dysfunction that may be prevented by saline hydration and by drugs such as theophylline or furosemide. Whether oral saline hydration is as efficient as intravenous saline hydration is unknown. The preventive efficacy of theophylline and furosemide for CN remains controversial. The purpose of the current study was to evaluate the efficacy of oral saline hydration and of intravenous saline hydration plus theophylline or furosemide for the prevention of CN.

Study objectives:To compare the quality of non-small cell lung cancer (NSCLC) surgical care with patient satisfaction.

BACKGROUND: Approximately 30% of lung cancer cases are diagnosed in patients > 70 years of age. Standard chemotherapy regimens are generally considered too toxic for elderly patients. We conducted a multicenter phase II trial to determine the efficacy and safety of carboplatin combined with vinorelbine every 4 weeks as first-line treatment for advanced non–small-cell lung cancer (NSCLC) in elderly patients. PATIENTS AND METHODS: Patients were eligible if they were aged ≤ 70 years, had stage IIIB (with pleural effusion) or stage IV NSCLC, had a performance status of 0/1, had not previously received chemotherapy, and had normal organ function. Forty patients (31 men and 9 women) were enrolled and received 3-5 courses of treatment. Median age was 72 years (range, 70-82 years). Eighty percent of patients had stage IV NSCLC, with squamous cell (n = 21), adenocarcinoma (n = 12), and undifferentiated (n = 7) histologies. RESULTS: Forty patients were assessable for toxicity and 32 for treatment response. Among these 32 patients, 8 had a partial response (intent-to-treat response rate, 20%), and 10 (25%) had stable disease. The median survival was 7.8 months (range, 4-11.6 months). The 1- and 2-year survival rates were 25% and 7%, respectively; median time to progression was 4.3 months (range, 0.2-13.8 months). Grade 3/4 neutropenia was seen in 27 patients (68%), and grade 3/4 anemia was seen in 5 patients (13%). One patient died of febrile neutropenia during treatment. The main nonhematologic adverse effect was fatigue (grade 3/4 in 18% of patients). CONCLUSION: Carboplatin/vinorelbine is well tolerated by elderly patients with extensive-stage NSCLC. Efficacy is low but similar to that of other treatments used in this setting.

This study describes the development and reports the first psychometric results of the European KIDSCREEN-52 generic health-related quality-of-life questionnaire for children and adolescents. The KIDSCREEN-52, including ten dimensions, was applied in a European survey involving 12 countries (i.e., Austria, Switzerland, Czech Republic, Germany, Greece, Spain, France, Hungary, The Netherlands, Poland, Sweden and the UK) and 22,110 children and adolescents aged between 8 and 18 years of age. Questionnaire development included a literature search, expert consultation, and focus group discussions with children and adolescents. After definition of dimensions and collection of items, a translation process following international translation guidelines, cognitive interviews and a pilot test were performed. Analysis regarding psychometric properties showed Cronbach-α ranged from 0.77 to 0.89. Correlation coefficients between KINDLRand KIDSCREEN-52 dimensions were high for those assessing similar constructs (r = 0.51–0.68). All KIDSCREEN-52 dimensions showed a gradient according to socioeconomic status and most dimensions showed a gradient according to psychosomatic health complaints. The first results demonstrate that the KIDSCREEN-52 questionnaire is a promising cross-cultural measure of health-related quality-of-life assessment for children and adolescents in Europe.

To develop a brief French-language, generic, self-administered questionnaire to measure inpatient satisfaction.