French Patients Research Articles

The Real-World Observational Prospective Study of Health Outcomes with Dulaglutide and Liraglutide in Type2 Diabetes Patients (TROPHIES): Design and Baseline Characteristics.

IntroductionThe TROPHIES observational study enrolled patients with type 2 diabetes mellitus (T2DM) initiating their first injectable treatment with the glucagon-like peptide 1 receptor agonists (GLP-1 RAs) dulaglutide or liraglutide. This manuscript focuses on the study design, baseline characteristics of the enrolled population, and factors associated with GLP-1 RA choice.MethodsTROPHIES is a prospective, observational, 24-month study conducted in France, Germany, and Italy. Inclusion criteria include adult patients with T2DM, naïve to injectable antihyperglycemic treatments, initiating dulaglutide or liraglutide per routine clinical practice. The primary outcome is the duration of treatment on dulaglutide or liraglutide without a significant treatment change.ResultsThe analysis included 2181 patients (dulaglutide, 1130; liraglutide, 1051) (cutoff date May 15, 2019). The population was 56% male with mean [standard deviation (SD)] patient characteristics at baseline as follows: age, 59.2 (11.0) years; body mass index (BMI), 33.9 (6.6) kg/m2; T2DM duration, 8.5 (6.9) years; and glycated hemoglobin (HbA1c), 8.2 (1.3)%. Between-cohort demographic and clinical characteristics were balanced. The mean (SD) HbA1c and BMI values for French, German, and Italian patients were, respectively, 8.6 (1.4)%, 8.2 (1.4)%, 8.0 (0.8)%; 33.3 (6.1) kg/m2, 36.0 (7.2) kg/m2, and 32.6 (5.9) kg/m2.ConclusionThis study analysis at baseline provides an opportunity to evaluate between-country differences in baseline HbA1c, weight, macrovascular complications, and factors driving GLP-1 RA selection for patients with T2DM in daily practice.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13300-021-01076-0.

Adult-onset diagnosis of urea cycle disorders: Results of a French cohort of 71 patients.

Urea cycle disorders (UCD) are rare diseases that usually affect neonates or young children. During decompensations, hyperammonemia is neurotoxic, leading to severe symptoms and even coma and death if not treated rapidly. The aim was to describe a cohort of patients with adult onset of UCDs in a multicentric, retrospective and descriptive study of French adult patients with a diagnosis after 16 years of age of UCDs due to a deficiency in one of the 6 enzymes (arginase, ASL, ASS, CPS1, NAGS, OTC) or the two transporters (ORNT1 or citrin). Seventy-one patients were included (68% female, 32% male). The diagnosis was made in the context of (a) a metabolic decompensation (42%), (b) family history (55%), or (c) chronic symptoms (3%). The median age at diagnosis was 33 years (range 16-86). Eighty-nine percent of patients were diagnosed with OTC deficiency, 7% CPS1 deficiency, 3% HHH syndrome and 1% argininosuccinic aciduria. For those diagnosed during decompensations (including 23 OTC cases, mostly female), 89% required an admission in intensive care units. Seven deaths were attributed to UCD-6 decompensations and 1 epilepsy secondary to inaugural decompensation. This is the largest cohort of UCDs diagnosed in adulthood, which confirms the triad of neurological, gastrointestinal and psychiatric symptoms during hyperammonemic decompensations. We stress that females with OTC deficiency can be symptomatic. With 10% of deaths in this cohort, UCDs in adults remain a life-threatening condition. Physicians working in adult care must be aware of late-onset presentations given the implications for patients and their families.

PRO2 Burden of Aromatic L-Amino Acid Decarboxylase Deficiency (AADC-D) in France with a FOCUS on Patient Symptoms and Motor Milestones Development

AADC-d is an extremely rare autosomal recessive neuro-metabolic disease characterized by a complex syndrome of motor, behavioral, and autonomic symptoms. The aim of this study, part of a global study assessing the burden of AADC-d, was to describe symptoms and developmental motor milestones in French patients.

A validation of the 4S-AF scheme in Spanish and French patients from the EORP-AF Long-Term General Registry

Abstract Funding Acknowledgements Type of funding sources: Other. Main funding source(s): Abbott Vascular Int. (2011–2014), Amgen Cardiovascular (2009–2018), AstraZeneca (2014–2017), Bayer AG (2009–2018), Boehringer Ingelheim (2009–2019), Boston Scientific (2009–2012), The Bristol Myers Squibb and Pfizer Alliance (2011–2016), The Alliance Daiichi Sankyo Europe GmbH and Eli Lilly and Company (2011–2017), Edwards (2016–2019), Gedeon Richter Plc. (2014–2017), Menarini Int. Op. (2009–2012), MSD-Merck & Co. (2011–2014), Novartis Pharma AG (2014–2017), ResMed (2014–2016), Sanofi (2009–2011), SERVIER (2009–2018). The Atrial Fibrillation NETwork (AFNET), conducting the registry in Germany, received support from The Bristol Myers Squibb/Pfizer Alliance (2014–2018) and the German Centre for Cardiovascular Research (DZHK). Funding from Daiichi-Sankyo and Boehringer-Ingelheim have been received for conducting the registry in Spain. Funding from BMS-Pfizer Alliance was received to support the programme in France Background The 4S-AF scheme (Stroke risk, Symptom severity, Severity of atrial fibrillation [AF] burden, Substrate severity) has recently been described as a novel approach to in-depth characterization of AF, and included in the 2020 European Society of Cardiology guidelines for the management of AF. Purpose In the present study, we validated for the first time the 4S-AF scheme in the Spanish and French cohorts of the EurObservational Research Programme (EORP)-AF Long-Term General Registry. Methods The Spanish and French cohorts of the EORP-AF Long-Term General Registry, were merged and included. The baseline 4S-AF scheme was calculated as follows: Symptom severity (according to EHRA symptom score: 0-2 points), Severity of AF burden (according to AF type: 0-3 points), Substrate severity (according to comorbidities/cardiovascular risk factors: 0-7 points); and related to the primary management strategy (rhythm or rate control). According to the results for these 3 domains, four code colors have been defined. Patients with all domains in "green" should be managed by rhythm control. In patients with one domain in "yellow" or two domains in "green" categories, rhythm control can be attempted. On contrary, for patients with "red" color category, the 4S-AF scheme suggests a rate control strategy. All-cause mortality and the composite of ischemic stroke/transient ischemic attack/systemic embolism, major bleeding and all-cause death, were the primary endpoints. These outcomes were recorded during 1-year of follow-up. Results 1479 patients (36.9% females, median age of 72 [IQR 64-80] years) were included (Table 1). The median 4S-AF scheme score was 5 (IQR 4-7). The 4S-AF scheme, as continuous and as categorical, was associated with the management strategy decided for the patient (both p < 0.001). The predictive performances of the 4S-AF scheme for the actual management strategy were appropriate in its continuous (C-index: 0.77, 95% CI 0.75-0.80) and categorical (C-index: 0.75, 95% CI 0.72-0.78) forms (Figure 1A). Cox regression analyses showed that patients classified as "red" category in the 4S-AF scheme had higher risk of all-cause death (adjusted HR 1.75, 95% CI 1.02-2.99) and composite outcomes (adjusted HR 1.60, 95% CI 1.05-2.44) (Figure 1B). Thus, patients for who the 4S-AF scheme suggests that may be managed by rhythm control (recommended or considered) had a significantly lower risk of these events. Conclusion Characterization of AF by using the 4S-AF scheme may aid in identifying AF patients that would be managed by rhythm or rate control, and could also help in identifying high-risk AF patients for worse clinical outcomes in a ‘real-world’ setting. Abstract Table 1 and Figures 1A-1B

Multifactorial analysis of cancer patients’ willingness for COVID-19 vaccination.

12062 Background: Public health authorities advocate vaccinations for the general population, including cancer patients and survivors. Since the onset of the COVID-19 pandemic, its vaccine has been eagerly awaited, but the extent of cancer patients’ willingness to get vaccinated is not clear. As health promotion is crucial for these individuals, it is important to measure and analyze their willingness to receive the vaccine. Methods: A few days after the regulatory approval for the first COVID-19 vaccine, the CareAcross online interactive platforms were used to evaluate the willingness of patients to get vaccinated. Through an online survey, within a few hours, 1106 cancer patients selected either “Yes, I plan to get the vaccine” or “No, I will not get the vaccine”. The patients were from the UK, Germany, France, Spain or Italy; they had been diagnosed with breast, lung, prostate or colorectal cancer. Their responses were analyzed to determine how their cancer diagnosis (including date, metastatic status, and other aspects), and their country of origin, affected their reported willingness to get vaccinated. Results: Overall, 70.6% of patients indicated willingness to get the vaccine (WTV), and 29.4% reported the opposite (NWTV). The strongest determinant of WTV was the patient’s country of origin: patients in the UK, Spain, Italy, Germany and France reported WTV of 84.1%, 64.2%, 58.7%, 47.4% and 38.3%, respectively. The next strongest determinant was the time elapsed since the patient’s diagnosis: for the largest population with available diagnosis date (451 UK patients), the average time since diagnosis for patients with WTV vs NWTV was: breast, 3.5 vs 2.5 years; lung, 1.6 vs 1.4 years; prostate, 2.4 vs 3.3 years; colorectal, 1.9 vs 1.5 years. Among patients from other countries with available diagnosis date: as the time since diagnosis increased, among 148 Italian patients WTV gradually increased; among 94 Spanish patients, WTV substantially decreased; among 85 French patients, WTV gradually decreased; among 50 German patients, WTV substantially increased. There was no significant correlation of WTV percentages with cancer type; metastatic status; triple negative vs non-triple negative among breast cancer patients; non-small cell vs small cell among lung cancer patients. Conclusions: Despite long-standing efforts of the scientific community for health promotion through the COVID-19 vaccine, a substantial percentage of cancer patients reported no willingness to get vaccinated. This appeared to depend on each patient’s country of origin, and the time elapsed since their diagnosis. This patient input was collected shortly after the first vaccine’s approval. With increasing evidence of efficacy and safety through more vaccinations of citizens and patients, willingness is expected to increase. We are in the process of conducting a follow-up survey to track these changes and update the results to be reportable during ASCO.

Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease

BackgoundElexacaftor-tezacaftor-ivacaftor partially restores cystic fibrosis transmembrane conductance regulator function, and has been shown to induce significant clinical improvement in patients with at least one Phe508del allele. Yet little data exist on patient perspectives following elexacaftor-tezacaftor-ivacaftor initiation. MethodsA mixed methods study was conducted using an online 13-item questionnaire (including 9 closed questions and 4 open questions), submitted from July 10th to August 21th 2020 to French patients aged 12 years and older with advanced CF who were treated with elexacaftor-tezacaftor-ivacaftor. Their responses were summarized as numbers (%), and free-text items were analysed using a grounded theory approach. ResultsOf 245 patients who started elexacaftor-tezacaftor-ivacaftor in France, 101 (41%) participated. Median [IQR] age was 35 [28–41] years and duration of elexacaftor-tezacaftor-ivacaftor treatment was 4.3 [3.0–5.6] months. Patients generally reported a rapid impact on respiratory symptoms, sleep quality, general well-being and physical self-esteem, and a reduction in overall treatment burden. The majority of patients contrasted treatment burden, symptom severity, depression and a closed future marked by death or transplantation before elexacaftor-tezacaftor-ivacaftor, to renewed and unexpected physical strength, leading to greater self-confidence, autonomy and long-term planning, after treatment initiation. A small number of patients expressed concerns, mainly regarding changes in body representation and/or the fear of becoming dependent on the treatment. ConclusionAfter initiation of elexacaftor-tezacaftor-ivacaftor, CF patients with advanced disease reported rapid and positive physical, psychological and social effects, which translated into improved quality of life and the formulation of new life goals.

Prognosis of French COVID-19 patients with chronic liver disease: A national retrospective cohort study for 2020

Background & AimsThe impact of chronic liver disease on outcomes in patients with COVID-19 is uncertain. Hence, we aimed to explore this association.MethodsWe explored the outcomes of all adult inpatients with COVID-19 in France, in 2020. We computed adjusted odds ratios to measure the associations between chronic liver disease, alcohol use disorders, mechanical ventilation and day-30 in-hospital mortality.ResultsThe sample comprised 259,110 patients (median [IQR] age 70 (54–83) years; 52% men), including 15,476 (6.0%) and 10,006 (3.9%) patients with chronic liver disease and alcohol use disorders, respectively. Death occurred in 38,203 (15%) patients, including 7,475 (28%) after mechanical ventilation, and 2,941 (19%) with chronic liver disease. The adjusted odds ratios for mechanical ventilation and day-30 mortality were 1.54 (95% CI 1.44–1.64, p <0.001) and 1.79 (1.71–1.87, p <0.001) for chronic liver disease; 0.55 (0.47–0.64, p <0.001) and 0.54 (0.48–0.61, p <0.001) for mild liver disease; 0.64 (0.53–0.76; p <0.001) and 0.71 (0.63–0.80, p <0.001) for compensated cirrhosis; 0.65 (0.52–0.81, p <0.001) and 2.21 (1.94–2.51, p <0.001) for decompensated cirrhosis; 0.34 (0.24–0.50; p <0.001) and 1.38 (1.17–1.62, p <0.001) for primary liver cancer; and 0.82 (0.76–0.89; p <0.001) and 1.11 (1.05–1.17; p <0.001) for alcohol use disorders. Chronic viral hepatitis; non-viral, non-alcoholic chronic hepatitis; organ, including liver, transplantation, and acquired immunodeficiency syndrome were not associated with COVID-19-related death.ConclusionChronic liver disease increased the risk of COVID-19-related death in France in 2020. Therapeutic effort limitation may have contributed to COVID-19-related death in French residents with a liver-related complication or an alcohol use disorder.Lay summaryWe studied the outcomes, including mechanical ventilation and day-30 mortality, of all adults with COVID-19 who were discharged from acute and post-acute care in France in 2020 (N = 259,110). Patients with mild liver disease; compensated cirrhosis; organ, including liver, transplantation; or acquired immunodepression syndrome were not at increased risk of COVID-19-related mortality. Patients with alcohol use disorders, decompensated cirrhosis, or primary liver cancer were at increased risk of COVID-19-related mortality but were less likely to receive mechanical ventilation. Our results suggest that therapeutic effort limitation may have contributed to the excess mortality in French residents with a liver-related complication or an alcohol use disorder.

Outcome after hematopoietic stem cell transplantation in patients with extranodal natural killer/T-Cell lymphoma, nasal type: A French study from the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC).

We evaluated the outcome of 65 French patients with Extranodal NK/T-cell lymphoma, nasal type (ENKTL) undergoing hematopoietic stem cell transplantation (HSCT) (19 allogeneic and 46 autologous). Fifty-four patients (83%), most of which receiving L-asparaginase (L-aspa) containing regimens (81%), achieved complete or partial response at time of HCST. After a median follow-up of 79.9months, 4-years progression-free survival (PFS) and overall survival (OS) were similar in both autologous and allogeneic groups (PFS: 34% vs. 26%, p=.12 and OS: 52% vs. 53%, p=.74). Response status at HSCT was the major independent prognostic factor on survival (OS: HR: 4.013 [1.137; 14.16], p=.031 and PFS: HR: 5.231 [1.625; 16.838], p=.006). As compared to control patients receiving chemotherapy and/or radiotherapy containing regimens only, upfront HSCT did not improve the outcome of responder patients, including those treated by L-aspa. However, it tends to provide survival benefit for relapsed patients with initial high-risk clinical features who achieved second remission. Whereas the place of HSCT in upfront therapy has still to be clarified, these data confirm that HSCT should be considered for consolidation in selected patients with relapsed ENKTL. Based on a large non Asian ENKTL cohort since the L-aspa era, this study provides some insight into the survival patterns of ENKTL patients with HSCT in the Western hemisphere and may give future direction for the next clinical trial design.

Quality of Life in CKD Patients on Low-Protein Diets in a Multiple-Choice Diet System. Comparison between a French and an Italian Experience.

Prescribing a low-protein diet (LPD) is part of the standard management of patients in advanced stages of chronic kidney disease (CKD). However, studies on the quality of life (QoL) of patients on LPDs are lacking, and the impact these diets have on their QoL is often given as a reason for not prescribing one. We, therefore, decided to assess the QoL in a cohort of CKD stage 3–5 patients followed up by a multiple-choice diet approach in an outpatient nephrology clinic in France. To do so, we used the short version of the World Health Organization’s quality of life questionnaire and compared the results with a historical cohort of Italian patients. We enrolled 153 patients, managed with tailored protein restriction in Le Mans, and compared them with 128 patients on similar diets who had been followed in Turin (Italy). We found there were no significant differences in terms of age (median 73 vs. 74 years, respectively), gender, CKD stage, and comorbidities (Charlson’s Comorbidity Index 7 vs. 6). French patients displayed a greater body mass index (29.0 vs. 25.4, p < 0.001) and prevalence of obesity (41.2 vs. 15.0%, p < 0.001). Baseline protein intake was over the target in France (1.2 g/kg of real body weight/day). In both cohorts, the burden of comorbidities was associated with poorer physical health perception while kidney function was inversely correlated to satisfaction with social life, independently of the type of diet. Our study suggests that the type of LPD they follow does not influence QoL in CKD patients and that a personalized approach towards protein restriction is feasible, even in elderly patients.

Analyses of Clinical and Biological Data for French and Belgian Immunocompetent Patients Infected With Hepatitis E Virus Genotypes 4 and 3.

Hepatitis E virus (HEV) genotypes 3 and 4 are the major causes of acute hepatitis in industrialized countries. Genotype 3 is mainly found in Europe and America, while genotype 4 is predominant in Asia. Several Japanese studies have suggested that genotype 4 is more virulent than genotype 3. We investigated this aspect by analyzing the clinical and biological data for 27 French and Belgian immunocompetent patients infected with HEV genotype 4. Their infections were probably acquired locally, since none of these patients reported traveling outside France or Belgium during the 2–8 weeks before symptoms onset. Each patient was matched for age (±5 years) and gender with two patients infected with HEV genotype 3. Bivariate analysis indicated that the HEV genotype 4-infected patients had significantly higher alanine aminotransferase (ALT) (2067 IU/L) and aspartate aminotransferase (AST) (1581 IU/L) activities and total bilirubin concentrations (92.4 μmol/L) than did those infected with HEV genotype 3 (1566 IU/L, p = 0.016; 657 IU/L, p = 0.003 and 47 μmol/L, p = 0.046) at diagnosis. In contrast, more patients infected with HEV genotype 3 reported dark urine (71% vs. 39%, p = 0.02) and experienced asthenia (89% vs. 58%, p < 0.01) than did those infected with HEV genotype 4. Two HEV genotype 4-infected patients died of multi-organ failure, while none of the genotype 3-infected patients died (p = 0.035). Finally, stepwise regression analysis retained only a greater increase in ALT (odds-ratio: 1.0005, 95% confidence interval: 1.00012–1.00084) and less frequent fever (odds-ratio = 0.1244; 95% confidence interval: 0.01887–0.82020) for patients infected with HEV genotype 4. We conclude that HEV-4 infections are likely to be associated with higher ALT activity than HEV-3 infections. Additional immunological and virological studies are required to confirm these findings and better understand the influence, if any, of genotype on HEV pathophysiology.

ST-segment elevation myocardial infarction: Management and association with prognosis during the COVID-19 pandemic in France

BackgroundSystems of care have been challenged to control progression of the COVID-19 pandemic. Whether this has been associated with delayed reperfusion and worse outcomes in French patients with ST-segment elevation myocardial infarction (STEMI) is unknown.AimTo compare the rate of STEMI admissions, treatment delays, and outcomes between the first peak of the COVID-19 pandemic in France and the equivalent period in 2019.MethodsIn this nationwide French survey, data from consecutive STEMI patients from 65 centres referred for urgent revascularization between 1 March and 31 May 2020, and between 1 March and 31 May 2019, were analysed. The primary outcome was a composite of in-hospital death or non-fatal mechanical complications of acute myocardial infarction.ResultsA total of 6306 patients were included. During the pandemic peak, a 13.9 ± 6.6% (P = 0.003) decrease in STEMI admissions per week was observed. Delays between symptom onset and percutaneous coronary intervention were longer in 2020 versus 2019 (270 [interquartile range 150−705] vs 245 [140−646] min; P = 0.013), driven by the increase in time from symptom onset to first medical contact (121 [60−360] vs 150 [62−420] min; P = 0.002). During 2020, a greater number of mechanical complications was observed (0.9% vs 1.7%; P = 0.029) leading to a significant difference in the primary outcome (112 patients [5.6%] in 2019 vs 129 [7.6%] in 2020; P = 0.018). No significant difference was observed in rates of orotracheal intubation, in-hospital cardiac arrest, ventricular arrhythmias and cardiogenic shock.ConclusionsDuring the first peak of the COVID-19 pandemic in France, there was a decrease in STEMI admissions, associated with longer ischaemic time, exclusively driven by an increase in patient-related delays and an increase in mechanical complications. These findings suggest the need to encourage the population to seek medical help in case of symptoms.

Specifics of chronic fatigue syndrome coping strategies identified in a French flash survey during the COVID-19 containment.

The COVID‐19 pandemic has focused health systems on supporting patients affected by this virus. Meanwhile in the community, many other contained patients could only use self‐care strategies, especially in countries that have set up a long and strict containment such as France. The study aimed to compare coping strategies deployed by patients with Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS; a poorly recognised syndrome) to those with better known and referenced chronic conditions. An online flash survey was conducted during the containment period in partnership with French Patients Organizations including ME/CFS national association. Therefore, ‘Brief COPE’ version of Lazarus and Folkman's Ways of Coping Check List has been adapted to the specificity of the containment. The survey was e‐distributed in France from 15 April to 11 May 2020. Differences of coping strategies were analyzed using Wilcoxon–Mann–Withney test. Amongst 637 responses, 192 were complete, presenting a wide variety of diseases, including 93 ME/CFS. The latter have significantly different coping strategies than recognised diagnosed diseases patients: similar uses of emotion focused coping but less uses of seek social support and problem‐focused copings. In conclusion, coping strategies are different for those who deal with the daily experience of ME/CFS, highly disabling chronic condition with diagnostic ambiguity, low degree of medical and social recognition and without treatment. Better understanding of those strategies is needed to provide the means for health promotion researchers, managers and clinicians, to accompany those patients.

Successive Osteosarcoma Relapses after the First Line O2006/Sarcome-09 Trial: What Can We Learn for Further Phase-II Trials?

Simple SummaryOsteosarcoma is the most common primary malignant bone tumour in adolescents and young adults. The survival of osteosarcoma patients has not improved for four decades. The purpose was to describe first and subsequent relapses in patients from the OS2006/Sarcome-09 trial, to help future trial design. Among the 434 patients with a confirmed osteosarcoma who achieved CR1 during first line treatment, 157 patients experienced at least one relapse. The 3-year progression-free and overall survival rates were 21% and 37%, respectively. Only a quarter of the patients were included in clinical trials at first recurrence. We want to promote randomised phase-II trials in osteosarcoma relapses, with broad inclusion criteria at study entry in terms of age and disease status, and PFS as primary endpoint. Surgery/local treatment of all residual lesions should be allowed when feasible. Single-arm trial design could be used for subsequent relapses.The purpose was to describe first and subsequent relapses in patients from the OS2006/Sarcome-09 trial, to help future trial design. We prospectively collected and analysed relapse data of all French patients included in the OS2006/Sarcome-09 trial, who had achieved a first complete remission. 157 patients experienced a first relapse. The median interval from diagnosis to relapse was 1.7 year (range 0.5–7.6). The first relapse was metastatic in 83% of patients, and disease was not measurable according to RECIST 1.1 criteria in 23%. Treatment consisted in systemic therapy (74%) and surgical resection (68%). A quarter of the patients were accrued in a phase-II clinical trial. A second complete remission was obtained for 79 patients. Most of them had undergone surgery (76/79). The 3-year progression-free and overall survival rates were 21% and 37%, respectively. In patients who achieved CR2, the 3y-PFS and OS rates were 39% and 62% respectively. Individual correlation between subsequent PFS durations was poor. For osteosarcoma relapses, we recommend randomised phase-II trials, open to patients from all age categories (children, adolescents, adults), not limited to patients with measurable disease (but stratified according to disease status), with PFS as primary endpoint, response rate and surgical CR as secondary endpoints.

Can we learn from others? The Swedish intensive care unit database for COVID-19 cases.

Can we learn from others? The Swedish intensive care unit database for COVID-19 cases.

IS THE CONTROL OF CARDIOVASCULAR RISK FACTORS IS DIFFERENT IN PRIMARY AND SECONDARY PREVENTION? COMPARISON OF 2 COHORTS OF HYPERTENSIVE PATIENTS WITHIN 10 YEARS

Objective: Euroaspire surveys highlighted the insufficiency of control of cardiovascular risk factor (CVRF) in coronary patient in secondary prevention. Patient profiles have changed, making it difficult to analyse these data over time. Our aim was to establish the state of CVRF control in two cohorts of French hypertensive patients in different clinical situations of primary and secondary prevention by following the variables assessed in Euroaspire. Design and method: Of each of the 22 participating hypertension specialist consultations, the first 20 patients (followed for at least one year) were included. Theoretical objectives were notified for each patient according to clinical profile. Two cohorts were formed, Cohort 1 (C1) in 2009 (n = 185) and Cohort 2 (C2) in 2019 (n = 260). Patients were classified according to their prevention (P) profile: primary (P1) and secondary (P2). Results: Baseline data are summarized in Table 1. The main statistically significant results between C2 vs C1 respectively are: younger people (58.8 vs 63.9 y) and more women in P1 (50% vs 32%); higher systolic blood pressure (BP) in P2 (143.7 vs 134.7 mm Hg), whereas higher diastolic BP in P1 (81.7 vs 76.6 mm Hg); fewer diabetic patients in P1 (19% vs 41%); poorly BP targets met in P1 (43% vs 63%); lower number of antihypertensive drugs in both groups (P1: 2.17 vs 2.66; P2: 2.62 vs 3.16); prescription of statins felt by half in P1 (30% vs 43%) within 10y. Conclusions: The high prevalence of unhealthy lifestyles, modifiable CVRF and inadequate use of drug therapies to achieve BP and lipid goals, are documented findings of the Euroaspire surveys in coronary patient, which we confirm in our hypertensive patients. Indeed, the P1 patient is less well managed than 10 years. In P2, there is no difference between the two cohorts, no improvement in BP control, possibly related with a reduction in the number of antihypertensive drugs. Ambition targets allocated for global prevention by combating the clinical inertia in titrating treatments must be a medical priority in clinical practice. Thanks to patients and Club des Jeunes Hypertensiologues.

A formalized transition program for cystic fibrosis: A 10-year retrospective analysis of 97 patients in Lyon.

The prognosis of people diagnosed with cystic fibrosis (CF) has dramatically improved over the past decade in France, largely due to advances in CF care management, including an emphasis on chronic maintenance medications. Currently, the majority of French CF patients are adults, which means that they went through a transition process from receiving care at a pediatric CF center to receiving care at an adult CF center. To determine the impact of the transfer on clinical evolution, we report the transition procedure of our CF center in Lyon. From January 2006 to December 2016, 97 CF patients underwent a standardized process of transitioning from the pediatric to the adult CF center in Lyon. We compared the clinical evolution of these patients duringthreeperiods, starting the year before transition and ending the year after transition. Clinical data taken into account were forced expiratory volume in 1 s (FEV1 in liters), body mass index (BMI in kg/m2 ), pulmonary colonization, number of antibiotic courses, number of days of hospitalization per year, and outpatient visits per year. No significant differences were observed between respiratory and nutritional status, respiratory microbiome, number of antibiotic courses, or number of hospitalizations or visits when comparing the threeperiods of observation around transition (the year before, the first year after, and the second year after transfer). The standardized transition procedure used in Lyon is associated with the clinical stability of our CF patients.

Untreated patients with multiple sclerosis: A study of French expert centers.

Disease-modifying therapies (DMTs) have an impact on relapses and disease progression. Nonetheless, many patients with multiple sclerosis (MS) remain untreated. The objectives of the present study were to determine the proportion of untreated patients with MS followed in expert centers in France and to determine the predictive factors of nontreatment. We conducted a retrospective cohort study. Data were extracted from the 38 centers participating in the European Database for Multiple Sclerosis (EDMUS) on December 15, 2018, and patients with MS seen at least once during the study period (from June 15, 2016 to June 14, 2017) were included. Of the 21,189 patients with MS (age 47.1±13.1 years; Expanded Disability Status Scale (EDSS) score 3.4±2.4), 6,631 (31.3%; 95% confidence interval [CI] 30.7-31.9) were not receiving any DMT. Although patients with a relapsing-remitting course (n=11,693) were the most likely to receive DMT, 14.8% (95% CI14.2-15.4) were still untreated (6.8% never treated). After multivariate analysis among patients with relapsing-remitting MS, the main factors explaining never having been treated were: not having ≥9 lesions on brain magnetic resonance imaging (odds ratio [OR]0.52 [95% CI0.44-0.61]) and lower EDSS score (OR 0.78 [95% CI0.74-0.82]). Most patients with progressive MS (50.4% for secondary and 64.2% for primary progressive MS) did not receive any DMT during the study period, while 11.6% of patients with secondary and 34.0% of patients with primary progressive MS had never received any DMT. A significant proportion of patients with MS did not receive any DMT, even though such treatments are reimbursed by the healthcare system for French patients. This result highlights the unmet need for current DMTs for a large subgroup of patients with MS.

Should treatment of ALK-positive anaplastic large cell lymphoma be stratified according to minimal residual disease?

In anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALK+ ALCL), positive minimal residual disease (MRD+) after the first chemotherapy course was proven of strong prognostic significance. We aimed to validate these results in 138 French patients. Eighty-seven patients had a detectable minimal disseminated disease at diagnosis (MDD+). Early MRD assessment was performed in 33 of 87 patients and was positive in 18 and negative in 15 (MRD-). Three-year progression-free survival was significantly correlated with the MDD/MRD status: 81.1% in MDD-, 69.6% in MDD+/MRD-, and 15.2% in MDD+/MRD+ patients. In conclusion, we confirmed on an independent cohort that the MDD/MRD status has strong prognosis significance in ALK+ ALCL.

Incidence and risk factors for bilateral proximal femoral fractures

BackgroundProximal femoral fractures (PFFs) are a public health issue due to their high frequency. The frequency of a second PFF on the other side is estimated at 10%. This estimation is controversial, however, and the risk factors have not been evaluated in a large population of French patients. The objective of this retrospective case-control study was to determine: (1) the incidence of second PFFs and (2) their risk factors. HypothesisThe incidence of second PFFs is >2% after 1 year and >5% after 3 years. Material and methodsWe conducted a case-control study in a population of consecutive patients managed surgically for PPF at the Lyon Sud Hospital between 2013 and 2014. We analysed the following clinical factors: age, sex, body mass index (BMI), institutionalisation, the Parker score, the American Society of Anesthesiologists score (ASA), comorbidities, and the use of psychoactive drugs. ResultsWe included 474 PFFs (trochanter, n=240 and neck, n=234) of which 36 were bilateral. The contralateral fracture occurred within 1 year of the first fracture in 6/474 (1.3%) cases and within 3 years in all 36 cases (7.6%). The case-control study comprised 49 cases with bilateral PFF and 161 controls with no second hip fracture within 3 years. Risk factors for a second hip fracture were age older than 90 years (odds ratio [OR]=5.44; 95% confidence interval [95%CI], 112–2642 (p=0.002)) and a history of heart disease (OR, 2.18; 95%CI, 1.06–4.47 [p=0.03]). A Parker score≥6 was protective (OR, 0.84; 95%CI, 0.71–0.99 [p=0.03]). Mortality after 3 years was 42% (201/474), and 13% (63/474) of patients were lost to follow-up. DiscussionAge older than 90 years, a Parker score below 6, and a history of heart disease are risk factors for a second PFF within 3 years after the first PFF. Level of evidenceIII; case-control study.

Health state utility and quality of life measures in patients with chronic myeloid leukemia in France.

Tyrosine kinase inhibitors (TKIs) have dramatically improved the prognosis of chronic myeloid leukemia (CML). We aimed to assess health state utility and quality of life (QoL) in French patients with CML in real-life setting, to study the determinants of utility score and to compare health-related QoL values to general population norms. We conducted a cross-sectional study in 412 patients with CML. Data were collected by electronic survey. Three patient-reported outcomes questionnaires were used: EORTC QLQ-C30, EORTC QLQ-CML24 and EuroQol EQ-5D-3L. Health state utility values were computed using the French value set. We computed deviations from reference norms from the general population. We studied the determinants of health utility score using multiple regression models. The mean utility score (SD) was 0.72 (0.25) in the chronic phase and 0.84 (0.21) in treatment-free remission, with marked variations by gender. Patients with CML had a deviation from the reference norm of -0.15 on average (SD: 0.25). In terms of QoL, social functioning, role functioning and cognitive functioning were notably impacted with a mean difference of -16.0, -13.1 and -11.7 respectively. Fatigue, dyspnea and pain were the symptoms with the highest deviation from general population norms (mean difference of 20.6, 14.0 and 8.3 respectively). In the multiple regression analysis, fatigue was the most important independent predictor of the utility score. Although TKIs prevent the disease from progressing and even allow remission without treatment, QoL in patients with CML is notably altered. The utility scores deteriorate with CML symptoms.