View clinical trials related to Bleeding Disorder.
Filter by:ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform. ENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data. Accordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when / where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies. The standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources. Moreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal. ENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670
The goal of this prospective cohort is to identified specific biological patterns in patients with a bleeding of unknown cause and to study the specific mechanisms of the bleeding disorder for each subset of patients.
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China. Method The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
This is a 3-phase mixed methods study design. A literature review (Phase 1) has been completed to determine the areas of exploration and to identify challenges faced and the impact of the blood disorder on pediatric patients. Based on Phase 1, Phases 2 and 3, as proposed in this study, will be completed and will include interviews of patients diagnosed with bleeding and thrombotic disorders (phase 2). The interviews will be individual, semi-structured, and consist of open-ended questions to elicit unbiased and in-depth responses to gain an understanding of participant's perspectives on themes predetermined in the study design phase.
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7)
Coagulation dysfunction is frequent in septic patients and it is associated with an increase risk of mortality. During sepsis platelets number usually decreases and their function is reduced and this mechanism is sustained by an inflammatory induced coagulopathy. Some recent studies evaluated the possibility to use viscoelastic whole blood tests of the haemostasis, such as thromboelastography (TEG), which analyze all blood components and their interactions during clot formation and dissolution and might be useful for assessing bleeding risk in septic patients. Maximun amplitude (MA) is one of the variables obtained from TEG analysis and it expresses the strength of the clot and the efficacy of platelet function. A low level of MA describes a lower strength of the clot determined by a lower number or a reduced function of platelet. The aim of the present study is to evaluate whether a lower level of MA and a pattern of hypocoagulability might be associated with an increased risk of bleeding and need of transfusion in patients with sepsis. We want to conduct a prospective multicenter observational study, enrolling 100 consecutive adults patients with sepsis. We will exclude patients under 18 years old of age, chronic use of oral anticoagulant and anti platelet treatment, hematologic malignancy, congenital bleeding disorders, oral contraceptives, lack of consent. Primary end point To evaluate whether a lower level of MA might be associated with an increased risk of bleeding. Secondary end points: to evaluate whether a different level of MA correlates with the biomarker of the severity of sepsis such as presepsin, with the biomarker of the severity of infection and whether a pattern of hypocoagulability might be associated with a risk of mortality. All enrolled patients will undergo a blood sample at admission (T0), after 72 hours (T1) and after 7 days (T2) and all the following parameters will be measured: Platelet count, APTT, PT, INR, fibrinogen, procalcitonin and presepsin . Additionally, all viscoelastic parameters (reaction time (R), clot formation speed (K), angle (alpha) and maximum amplitude (MA)) will be performed at bedside, at T0, T1, T2: Outcome measurements: Intensive Care Unit Length of Stay and mortality at 28 days and at 90 days.