The European Hematology Association Roadmap for European Hematology Research. A Consensus Document

Abstract The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at Euro 23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine sections in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients. Received December 15, 2015. Accepted January 27, 2016. Copyright © 2016, Ferrata Storti Foundatio

The European Hematology Association Roadmap for European Hematology Research: a consensus document

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at €23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients

Recommendations regarding splenectomy in hereditary hemolytic anemias.

Hereditary hemolytic anemias are a group of disorders with a variety of causes, including red cell membrane defects, red blood cell enzyme disorders, congenital dyserythropoietic anemias, thalassemia syndromes and hemoglobinopathies. As damaged red blood cells passing through the red pulp of the spleen are removed by splenic macrophages, splenectomy is one possible therapeutic approach to the management of severely affected patients. However, except for hereditary spherocytosis for which the effectiveness of splenectomy has been well documented, the efficacy of splenectomy in other anemias within this group has yet to be determined and there are concerns regarding short- and long-term infectious and thrombotic complications. In light of the priorities identified by the European Hematology Association Roadmap we generated specific recommendations for each disorder, except thalassemia syndromes for which there are other, recent guidelines. Our recommendations are intended to enable clinicians to achieve better informed decisions on disease management by splenectomy, on the type of splenectomy and the possible consequences. As no randomized clinical trials, case control or cohort studies regarding splenectomy in these disorders were found in the literature, recommendations for each disease were based on expert opinion and were subsequently critically revised and modified by the Splenectomy in Rare Anemias Study Group, which includes hematologists caring for both adults and children

Therapeutic options for patients with severe ß-thalassemia: the need for globin gene therapy

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Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy

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Robotic Resection of Giant Duodenal Hamartoma After 18Fluorodeoxyglucose Positron Emission Tomography Magnetic Resonance Imaging (18FDG PET-MRI)

Brunner\u2019s gland hamartoma is a rare duodenal lesion. Resection for benign neoplasms of the duodenum should be considered in case of malignant potential or in case of symptomatic lesions. An accurate preoperative staging is mandatory in order to allow minimally invasive surgical approach, and to avoid under- or overtreatment. Endoscopic ultrasonography (EUS), Computed tomography (CT) scan, Magnetic Resonance Imaging (MRI) and PET/CT are techniques widely used for gastrointestinal tumor staging. We report a case of a 41-year-old female presenting a giant lesion of the second portion of the duodenum. Pathological examination of multiple forceps biopsies was inconclusive for histological characterization of the lesion. After a clinical staging including Esophagusgastroduodenoscopy, EUS, and CT scan, a Hybrid 18FDG PET/MRI was performed to assess the malignant potential of the lesion and the relation between polyp base and Vater\u2019s papilla. After multidisciplinary meeting, the patient underwent robotic transduodenal excision. The post-operative course was uneventful, and the patient was discharged on post-operative day 5. Final pathologic report consists in a histologically of Brunner\u2019s Glands Hamartoma. This is the first report on the role of 18FDG PET/MRI in staging and planning treatment of bulky low malignant duodenal lesion. An accurate staging with 18FDG PET/MRI could be very useful in the planning the management of duodenal lesion with uncertain malignant potential in order to avoid under- and overtreatment

Cellular biosensors for the identification of fetal hemoglobin inducers

The development of cellular systems for the screening of molecules able to induce the production of fetal or adult hemoglobin in crythroid cells isolated from beta-thalassemia patients is very important for the identification of molecules of interest for pharmacological therapy of thalassemia. This article reports the development of a cellular system for the identification of inducers of fetal hemoglobin preferentially acting on the human gamma-globin gene promoter. To achieve this aim, green and red fluorescence protein genes were cloned under the control of gamma-globin and beta-globin promoters, respectively. The developed K562 clones were tested for the increase of fluorescence after treatment with sodium butyrate and hydroxyurea, two well-known inducers of fetal hemoglobin