1,721,359 research outputs found
Fourth international workshop on haploidentical transplants, Naples, Italy, July 8-10, 2004
No full textAbstract: Many patients with high-risk hematological malignancies or with incurable inborn errors do not have an HLA-matched sibling and cannot find an HLA-matched donor for an allogeneic hematopoietic stem cell transplantation (SCT). Transplantation strategies using mismatched haploidentical family donors have been an important development. Although the procedure has saved patients from certain death, it is still beset by major problems like life-threatening infections-due to profound immunodeficiency following T-cell depletion and to disease relapse. At every International Workshop on Haploidentical Transplants, new data are presented, showing how scientists are attempting to improve the outcomes of mismatched transplants while reducing the severity of complications. The fourth Workshop continues in this tradition of presenting ground-breaking research. It opened with presentations of the current results with unrelated volunteer and umbilical cord blood transplants and proceeded to a session with the results of haploidentical transplants in the world with series of patients with high-risk acute leukemia, ranging in number from well over 100 in Perugia, Italy, and 80 in Haifa, Israel, to smaller groups in Europe and the United States. The session on graft engineering presented the latest results in the search for the optimal graft. The graft-vs.-leukemia effect in the haploidentical transplant was discussed in depth. Subsequently, attention focussed on one of the major problems in haploidentical transplant, that is, the delay in immunological recovery. The Workshop closed with presentations on tolerance induction that was followed by the results of ongoing registration studies being performed by the Italian GIMEMA group and the European Bone Marrow Transplant group
Improving laboratory diagnosis of von Willebrand Disease
No full textAbstract: In von Willebrand disease (VWD), the most common inherited bleeding disorder, patients have a quantitative or qualitative defect of von Willebrand factor (VWF). VWD is widely misdiagnosed because both its diagnosis and classification involve several pitfalls. The complexity and heterogeneity of VWF, (pre)analytical variables, limited repertoire of laboratory tests, intra-individual variation, complex interpretation of results and lack of expertise contribute to the diagnostic challenge. To provide the reader of the general knowledge on VWD, background information regarding biosynthesis, structure and function of VWF and epidemiology, classification, diagnosis and treatment of the disease is provided. Subsequently, the various published articles are presented. Recent developments in VWD diagnosis and classification are highlighted. All available laboratory tests are described, including their mechanisms and pitfalls, and are reviewed whether they are able to improve the VWD characterization. Two new automated VWF:GPIb binding activity assays, HemosIL VWF:RCo (ISTH nomenclature VWF:GPIbR) and INNOVANCE VWF Ac (ISTH nomenclature VWF:GPIbM), are compared in an extensively typed VWD population to evaluate whether they are more able to clearly distinguish type 1 and 2 VWD by using the VWF:GPIb binding activity / VWF antigen ratio, and also whether they are able to improve the quality of diagnosis and subtyping of VWD within this VWD cohort. The semi-automatic Hydragel von Willebrand Factor multimer technique, after correlation with the "gold standard" method for VWD diagnosis, is standardized and quantified by the establishment of reference intervals. Finally, we discuss the cross-sectional studies in VWD which were set up in collaboration with the Czech Republic and Slovakia (University Hospital Brno and Bratislava, F.D. Roosevelt Hospital Bansk\ue1 Bystrica). These studies aim to improve the understanding of the VWD epidemiology and laboratory phenotype/genotype correlation. Pitfalls in VWD diagnosis and classification are also highlighted in these studies. Based on all our findings from this thesis, it is emphasized that for the most accurate and complete VWD diagnosis/classification, an extensive test panel, analyzing different aspects of VWF, is required. Methodologies with the best balance between accessibility and accuracy are recommended, with automated testing being preferred above more manual methods. However, the currently available diagnostic tests are not infallible, which means that often unreliable results can lead to a misdiagnosis. The VWD cohort studies provide unique information on the laboratory phenotype-genotype relationship in VWD, and on the presence of certain causal mutations in a specific geographic region, which invite comparisons with other regions
Interleukin-13 as a master regulator of alternative microglia/macrophage activation : modulation of allograft- and pathology-associated immune responses
No full textBreaching the barrier-targeting dendritic cell migration in the treatment of multiple sclerosis
No full textOptimizing dendritic cells for cancer vaccination through introduction of ribonucleic acid for neo-expression of the tumor-associated antigen "receptor for hyaluronic acid-mediated motility" and the cytokine interferon-\u3b1
No full textAbstract: abstract not availabl
Therapeutic objectives and treatment strategy in myelodysplastic syndromes
No full textAbstract: Myelodysplastic syndromes or neoplasms (MDS) are a group of complex hematological malignancies that result in malfunctioning of the bone marrow. They all carry a risk of progression towards acute myeloid leukemia. Allogeneic stem cell transplantation is the only curative treatment for this group of malignancies. With a median age of 70 at diagnosis, most patients are considered not to be eligible for transplantation. Since most treatments have no curative potential and minimal impact on overall survival, quality of life is uniformly recognized as a treatment goal. Although recognized as a therapeutic objective, data on quality of life in patients with MDS outside clinical trials where lacking at the start of this project. We set up a national study to evaluate the impact of current available therapies on quality of life in MDS patients. This observational trial demonstrated a significant correlation between the patients\u2019 perception of MDS and health related quality of life in MDS patients. However, the drop in inclusion due to the COVID pandemic did not allow a firm conclusion on the therapeutic impact on quality of life. Low inclusion rates into clinical trials are the most frequent reason for trial failures. We explored the hematology patients\u2019 drivers to participate in clinical trials during a collaborative effort in the province of Antwerp and hematologists\u2019 drivers for trial inclusion in a national survey. We concluded that patients express a high willingness to trial participation but a knowledge-gap concerning general understanding of clinical trial concepts was present. We therefore created an information brochure in collaboration with the Patient Committee of the Belgian Hematology Society. The brochure is available online and as a hard-copy. Physicians have an important role in trial inclusions. The physicians\u2019 interest in the study objective is an important motivator for trial inclusion. In an opinion letter we discussed the current attitude towards quality of life as a study endpoint. To our opinion quality of life is undervalued as a study endpoint during different phases of clinical trials. The importance of health related quality of life (HRQoL) was demonstrated during another national collaboration during which we collected real-life data on the use of luspatercept, a relatively new molecule in the field of MDS. We demonstrated the importance of quality of life in MDS patients, an assumption we made incorrectly at the start of this project. We hereby missed our primary objective that was to explore treatment impact on quality of life in MDS. We hope this work can be an incentive for future projects
Meeting report: HTLV molecular biology and pathogenesis, Airlie Center, Warrenton, Va, USA, 17-19 March 2000
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