RÉSUMÉ
Pharmacotherapy options for chronic lymphocytic leukaemia (CLL) have expanded significantly in recent years. These options include chemotherapy, chemoimmunotherapy and signalling pathway inhibitors. A notable shift in the treatment landscape began with the widespread adoption of ibrutinib in 2016. This analysis of claims data focuses on understanding how the use of novel therapies has evolved in clinical practice over the past decade in Germany. Anonymized claims data (2010-2022) from German statutory health insurance was used, covering patient demographics, treatments, and prescriptions. The study population included patients with two confirmed CLL diagnoses. Treatment patterns were analysed, and survival outcomes were compared using time-to-event analyses. In the analysed cohort of 2983 incident CLL patients, 1041 started first-line therapy between 2011 and 2022, with a median duration of 18 months from diagnosis to the first prescription. Chemoimmunotherapy, the predominant 1L therapy until 2019, decreased significantly, while targeted therapy usage increased from 3% in 2015 to 77% in 2022. Targeted therapies became dominant in patients receiving treatment for relapsed or refractory disease after 2016. Median treatment durations were: 122 days for chemo, 176 days for chemo-immuno, and 373 days for targeted therapy. The overall survival for patients diagnosed in or after 2016 was significantly better (hazard ratio 0.56, 95% confidence interval, 0.44-0.69)). The adoption of targeted therapies like ibrutinib and venetoclax has transformed CLL treatment in Germany, leading to improved patient outcomes. Additionally, we demonstrate successful adherence to evolving clinical guidelines.
RÉSUMÉ
BACKGROUND: Sudden unexpected death in epilepsy (SUDEP) is in most cases probably due to a fatal complication of tonic-clonic seizures and plays a significant role in the premature mortality of individuals with epilepsy. The reported risks of SUDEP vary considerably depending on the study population, so that an up-dated systematic review of SUDEP incidence including most recent studies is required to improve the estimated SUDEP risk and the counseling of individuals with epilepsy. OBJECTIVE: To provide an overview of the current research landscape concerning SUDEP incidence across different patient populations and discuss potential conclusions and existing limitations. MATERIAL AND METHODS: A systematic literature review on SUDEP incidence was conducted in MEDLINE and EMBASE, supplemented by a manual search in June 2023. Out of a total of 3324 publications, 50 were reviewed for this study. RESULTS: The analyzed studies showed significant heterogeneity concerning cohorts, study design and data sources. Studies conducted without specific criteria and relying on comprehensive registers indicated an incidence of 0.78-1.2 per 1000 patient-years. Research providing incidences across various age groups predominantly show an increase with age, peaking in middle age. DISCUSSION: Due to varying methods of data collection and incidence calculation, comparing between studies is challenging. The association with age might be due to an underrepresentation of children, adolescents and patients over 60 years. CONCLUSION: Considering all age groups and types of epilepsy it is estimated that about 1 in 1000 individuals with epilepsy dies of SUDEP annually. With an assumed epilepsy prevalence of 0.6% in Germany, this could lead to more than one SUDEP case daily. Standardization of research methods is essential to gain more profound insights.
Sujet(s)
Mort subite et inexpliquée en épilepsie , Humains , Mort subite/épidémiologie , Épilepsie/épidémiologie , Épilepsie/mortalité , Épilepsie/complications , Allemagne/épidémiologie , Incidence , Facteurs de risque , Mort subite et inexpliquée en épilepsie/épidémiologieRÉSUMÉ
BACKGROUND: Myasthenia gravis (MG) is a rare chronic autoimmune disease caused by autoantibodies directed against postsynaptic antigens of the neuromuscular junction. Over the last decades, increasing incidence and prevalence rates have been reported. Epidemiological data on prevalence and incidence in Germany are lacking. Furthermore, the MG treatment landscape is rapidly changing due to the continued approval of novel monoclonal antibodies. METHOD: This is a retrospective study assessing incidence, prevalence, and hospitalization rates of MG as well as treatment patterns in Germany over 10 years based on medical claims data covering 6.1 million insured persons. RESULTS: Between 2011 and 2020, the prevalence rate of MG increased from 15.7 to 28.2 per 100,000 person-years. The age-adjusted incidence rate was 2.8 per 100,000 person-years within the study period (95% confidence interval, 2.43-3.22) and decreased dramatically in 2020, the year of the COVID-19 pandemic. Similarly, the hospitalization rate fluctuated within the study period but reached an overall low of 8.3% in 2020 (mean hospitalization rate 11.5%). Treatment patterns showed that most MG patients are treated with base therapy. However, crisis intervention is necessary for 2-5% of MG patients, and therapeutic monoclonal antibodies, including rituximab and eculizumab, are increasingly used. CONCLUSION: This is the first study on MG prevalence and incidence rates in Germany. Data show an increase in prevalence by 1.8-fold over 10 years. Decreasing incidence and hospitalization rates in 2020 hint at the impact of the COVID-19 pandemic. Treatment patterns in MG are changing with the advent of therapeutic monoclonal antibodies in this indication.
Sujet(s)
COVID-19 , Myasthénie , Humains , Incidence , Études rétrospectives , Prévalence , Pandémies , COVID-19/épidémiologie , Myasthénie/traitement médicamenteux , Myasthénie/épidémiologie , Anticorps monoclonaux/usage thérapeutique , Analyse de donnéesRÉSUMÉ
Reimbursement decisions on new oncology drugs are now often made while uncertainty remains about a drug's risk-benefit profile. One consequence of this is a delay in patient access to valuable new medicines. We share our perspectives on strategies to mitigate sources of uncertainty in the health technology assessment process. These include flexible approaches for evaluating the additional benefit, such as better use of surrogate endpoints and health-related quality of life data, and renewed research efforts to define the optimal target population and generate real-world evidence post-authorisation.
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This review provides an overview of the role of long-term treatment of severe asthma with oral corticosteroids (OCS) and its associated side-effects in adults. It is based on a systematic literature search conducted in MEDLINE, Embase and the Cochrane Library to identify relevant studies. After a short overview of severe asthma and its treatment we present studies showing a dose-response relationship in asthmatic patients treated with OCS and then consider by organ systems the undesired effects demonstrated in clinical and epidemiological studies in patients with OCS-dependent asthma. It was found that the risk of developing various OCS-related complications, including infections, diabetes and osteoporosis as well as psychiatric disorders, was higher for patients with long-term exposure to OCS compared with control groups. In addition, studies showed a significant increase in healthcare resource utilisation due to OCS treatment. Therefore, it is incumbent on every clinician to carefully weigh the potential beneï¬t of preventing loss of asthma control against this risk before opting to prescribe long-term OCS therapy. Effective corticosteroid-sparing strategies must be used and should aim at short-term use with the lowest effective dose and start tapering as soon as possible until OCS therapy is terminated.
Sujet(s)
Hormones corticosurrénaliennes/administration et posologie , Hormones corticosurrénaliennes/effets indésirables , Asthme/traitement médicamenteux , Administration par voie orale , Adulte , Relation dose-effet des médicaments , Humains , Essais contrôlés randomisés comme sujetRÉSUMÉ
BACKGROUND: Physical training has been shown to improve exercise capabilities in patients with asthma. Most studies focused on children and younger adults. Previously, the maximum program duration was six months. It is not known whether the same results may be obtained with lower intensity programs and sustained for time periods longer than 6 months. This controlled study was undertaken to investigate the effects of a moderate intensity outpatient training program of one year duration on physical fitness and quality of life in adults with asthma. METHODS: 21 adult asthmatics (mean age 56 ± 10 years) were allocated to outpatient training (n = 13) or standard care (n = 8). Exercise consisted of once weekly, 60-minute sessions of moderate intensity. Assessments at baseline and after one year included cardiopulmonary exercise testing and Short Form-36 and Asthma Quality of Life Questionnaires. RESULTS: Following one year of exercise, relevant improvements were observed in the training group for maximum work capacity (p = 0.005), peak oxygen uptake (p < 0.005), O2pulse (p < 0.05), maximum ventilation (p < 0.005), and most of the quality of life domains. No changes were observed in the control group. CONCLUSIONS: A physiotherapist-led, long-term, moderate-intensity exercise program of one year duration can induce clinically relevant improvements in exercise capabilities and health-related quality of life in well-motivated adults with asthma. TRIAL REGISTRATION: clinicaltrials.gov NCT01097473 . Date trial registered: 31.03.2010.
Sujet(s)
Asthme/rééducation et réadaptation , Traitement par les exercices physiques/méthodes , Aptitude physique , Qualité de vie , Adulte , Sujet âgé , Tolérance à l'effort , Femelle , Humains , Mâle , Adulte d'âge moyen , Consommation d'oxygène , Ventilation pulmonaire , Résultat thérapeutiqueRÉSUMÉ
OBJECTIVES: To assess the quality of health care for RA patients in the general population of Germany. METHODS: A three-stage population survey was conducted to identify individuals with RA using a health care access panel (18-79 years; n = 70,112). A 20-item postal screening questionnaire of musculoskeletal symptoms and diagnoses was followed by a detailed questionnaire for those who indicated the possibility of having RA. Respondents who fulfilled the modified ACR decision tree, who reported an RA diagnosis, care by a rheumatologist or the use of DMARDs were asked to participate in a clinical examination by rheumatologists who diagnosed the participants and rated the adequacy of treatment. RESULTS: RA could not be ruled out in 1177 cases, of which 643 agreed to participate in the clinical examination, which was finally attended by 317 participants. Attendees did not differ with regard to any health or treatment measure from those who did not attend. Forty-one RA patients were detected. Of them, 93% had seen a rheumatologist at least once and 63% within the last 12 months. A total of 73% had received DMARD therapy at some time and 59% were currently receiving it. An unmet need for DMARDs was discovered in 29% of the RA attendees. It pertained almost exclusively to the seronegative cases of which 29% had a need to start and 17% to increase a DMARD therapy according to the opinion of the examining rheumatologist. CONCLUSION: Health care for RA patients has improved significantly since the last German RA survey in 1989. However, DMARD prescription still does not meet clinical recommendations, specifically in RF-negative patients. Since seronegative RA is a treatable disease, this group should not be overlooked.