Search results: (10000)
News EHL preparations have the potential to change the quality of life for people with hemophilia
Within the therapeutic modalities for hemophilia, the current era offers a number of preparations. Newer extended half-life (EHL) replacement therapies can provide hemophiliacs with better protection against bleeding and allow for greater physical activity.
News Current Recommendations of the Expert Panel on the Management of von Willebrand Disease
Von Willebrand disease (vWD) is a common congenital bleeding disorder. The care of patients with vWD involves significant variability due to bleeding manifestations and treatment options. The new recommendations from 2021 regarding the care of people with vWD are evidence-based and supported by the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation USA (NHF), and the World Federation of Hemophilia (WFH). We bring you a concise summary of the key points.
News Smaller Volume and Faster Infusion Rate of aPCC: What Do Current Data Say About This Modification?
Administering an infusion of activated prothrombin complex concentrate (aPCC) at the standardized rate of 2 U/kg/min can be time-consuming, especially when repeated applications are needed. The authors of the recently published multicenter study cited below focused on the safety and tolerability of reducing the infusion volume by 50% and increasing the infusion rate to 4 and 10 U/kg/min.
News Estimated Worldwide Prevalence of Population with AAT Deficiency with MZ Alleles
Alpha-1-antitrypsin deficiency (AATD) with the MZ genotype was until recently associated with negligible or low risk for lung and liver tissue damage. However, growing evidence suggests that carriers of MZ alleles are at increased risk of organ damage when exposed to tobacco smoke, polluted air, or hepatotoxic substances. Moreover, according to a global epidemiological study, the prevalence of the MZ genotype is very common, especially among Caucasians in Europe and other continents.
News Repeated Episodes of Respiratory Failure Due to Undiagnosed AAT Deficiency – Case Report
The case report from authors in Naples shows that alpha-1-antitrypsin deficiency (AATD) should be considered when respiratory symptoms occur at any age. It also illustrates the diagnostic procedure in such cases and confirms the effectiveness of subsequently initiated therapy.
News Patient Evaluation of Durvalumab Treatment Results
The interim analysis results of the PACIFIC study, which monitored the efficacy of durvalumab in the treatment of locally advanced unresectable non-small cell lung cancer (NSCLC), demonstrated a significantly longer progression-free survival (PFS) with this treatment compared to placebo. The following work analyzed one of the secondary objectives of the PACIFIC study − patient-reported outcomes (PRO).
News Clinical Experiences with Reversing Dabigatran with Idarucizumab
Idarucizumab is an effective specific antidote to dabigatran, which very quickly negates its coagulative effects in cases of severe bleeding conditions or emergency surgical procedures. A Danish retrospective study examined clinical experiences with the use of idarucizumab for blocking dabigatran and related clinical outcomes.
News Comparison of Dabigatran and Vitamin K Antagonists in Patients with Atrial Fibrillation in Real-World Practice
The final 3-year results of the GLORIA-AF study conducted in real-world practice with patients newly diagnosed with atrial fibrillation show a lower incidence of major bleeding and lower overall mortality with comparably effective prevention of cerebrovascular accidents and myocardial infarction when administering dabigatran compared to vitamin K antagonists (VKAs).
News Effectiveness of the Venetoclax/Rituximab Combination in the Treatment of CLL – 5-Year Follow-Up Data
At the virtual 62nd Annual Meeting of the American Society of Hematology (ASH), updated results of a clinical evaluation of the treatment of patients with refractory/relapsed chronic lymphocytic leukemia (R/R CLL) using venetoclax in combination with rituximab were presented.
News Effectiveness of Idarucizumab Reversal Effect
It is, of course, a great advantage if doctors have a specific substance available that counteracts the effect of oral anticoagulants in urgent need. Idarucizumab is a humanized monoclonal antibody that binds dabigatran with high affinity. The drug was approved for clinical use based on an interim analysis of the results of 90 patients enrolled in the RE-VERSE AD study. What conclusions were drawn based on the results of the entire study population?
News Reverse Remodeling of LV in Patients with Diabetes/Prediabetes and Heart Failure with Reduced Ejection Fraction Treated with Empagliflozin
Authors of the British study SUGAR-DM-HF published in February 2021 examined whether empagliflozin reduces left ventricle (LV) volume in patients with type 2 diabetes mellitus (DM), prediabetes, and heart failure with reduced ejection fraction (EF). This favorable reverse remodeling of the LV could be a mechanism through which empagliflozin achieves reduced hospitalization rates for heart failure and overall mortality in these patients.
News Factors Predicting Longer Survival in Patients with Metastatic Colorectal Cancer Treated with Trifluridine/Tipiracil
A recently published non-interventional analysis evaluated overall survival (OS) and progression-free survival (PFS) in patients with metastatic colorectal cancer (mCRC) with favorable prognostic factors before initiating treatment with trifluridine/tipiracil compared to patients with unfavorable factors. It also identified which specific prognostic factors are favorable in terms of therapy effectiveness.
News What can we base the decision on for the choice of initial treatment modality for CLL?
We now have a range of highly effective treatment options for patients with chronic lymphocytic leukemia (CLL). New drugs have significantly contributed to improved overall survival (OS). However, there are still questions about the most appropriate treatment regimen or criteria for choosing the initial therapeutic modality. Some of these questions are posed—and answers sought—by Professor John F. Seymour from the University of Melbourne in his recent communication.
News Young man with nephrotic syndrome and portal vein thrombus successfully treated with edoxaban − case report
Venous thrombosis is one of the main complications of nephrotic syndrome (NS), though it is most often located in the renal vein. The following case report describes an adult patient with relapsing corticosteroid-dependent minimal change disease complicated by portal vein thrombosis and subsequent successful treatment with edoxaban.
News When Size Matters or What Is Small Is Effective
The systemic effect of an orally administered drug can be expected, among other things, if it has suitable physicochemical properties. How can insufficient absorption or poor solubility be solved? We provide the answer below.
News Insufficient dosing of enoxaparin is common in patients post-orthopedic surgery and increases the risk of thromboembolic disease
Orthopedic and traumatological procedures involving the pelvis and lower limbs are associated with a significant increase in the risk of thromboembolic disease. Therefore, prophylaxis with anticoagulants is recommended in the perioperative and postoperative period for these procedures. The most commonly administered anticoagulants are low molecular weight heparins, among which enoxaparin is included. A recently published study by authors from the University of Utah focused on insufficient dosing of enoxaparin and its impact on the development of thromboembolic disease within 90 days following the procedure.
News Presence of Risk Factors Associated with Colectomy in Patients with Ulcerative Colitis in Studies with Tofacitinib
One of the important goals of active ulcerative colitis therapy is the prevention of colectomy. A clinical study, the results of which were recently presented at the Vienna UEG (United European Gastroenterology) congress, evaluated the association of risk factors and the use of tofacitinib with colectomy in patients with active ulcerative colitis in the OCTAVE clinical trial program.
News Need for Early Intervention in COPD − A Suitable Time for Mucolytic Treatment
Chronic Obstructive Pulmonary Disease (COPD) ranks third among the leading causes of morbidity and mortality worldwide, with more than 210 million people expected to be affected by 2030. While common, it is preventable and treatable. The mucolytic erdosteine has proven to be an effective helper in COPD therapy.
News Efficacy and Safety of Fenfluramine in the Treatment of Lennox-Gastaut Syndrome – An Overview of Current Knowledge
In a recently published article, British authors summarize the current knowledge on the efficacy and safety of fenfluramine (FFA) in the treatment of Lennox-Gastaut syndrome (LGS, a developmental and epileptic encephalopathy). In addition to the reduction in seizure frequency demonstrated in clinical studies, they discuss the impact of fenfluramine on reducing the risk of sudden unexpected death in epilepsy (SUDEP), improving cognitive function in patients, and its safety concerning weight loss and heart disorders.
News Critical Overview of Clinical Studies on EHL FVIII Preparations
An international team of experts recently published a comprehensive work evaluating the methodology used in clinical trials of various extended half-life (EHL) factor VIII (FVIII) products. Their work does not directly compare the individual products but aims to assess the results of key registration clinical trials from the perspective of the hematologist and everyday clinical practice.
News Role and Possibilities of Telemedicine in the Management of Hemophilia Care
A team of Irish experts recently evaluated the current possibilities of telemedicine in the care of individuals with hemophilia – not only from the perspective of care provided by specialized comprehensive centers but also considering physiotherapy and dental care.
News Even in Severe Cases of HAE, Individualized Treatment is Crucial – Case Study
As part of the case seminar from the HAExpert series, held on December 1, 2021, which specifically focused on the treatment of hereditary angioedema (HAE) in specific patients, Dr. Roman Hakl from the Institute of Clinical Immunology and Allergology at MU Faculty of Medicine and St. Anne's University Hospital in Brno presented the case of a now 34-year-old woman with HAE and obesity (BMI 37 kg/m2), who additionally has a positive family history – the disease was also diagnosed in her mother, sister, and son.