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

Abstract

T he 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.

Research at the heart of hematology: thrombocytopenias and platelet function disorders

“…These uncertainties are primarily derived from the fact that the pathogenic mechanisms of ITP, probably different in different patients, are not yet fully known. So, as recognized by the "EHA Roadmap for European Hematology Research" recently published in this Journal, 11 both basic research and clinical studies are required to further improve care for ITP patients.…”

A Novel High-Throughput Screening Platform Reveals an Optimized Cytokine Formulation for Human Hematopoietic Progenitor Cell Expansion

“…Delays in early neutrophil recovery post-CB HSCT can be overcome by using in vitro pretreatment strategies to either increase specific progenitor cell numbers [11,[13][14][15][16][17] or to improve their adhesive, homing, and BM retention properties [2][3][4][44][45][46]. CB can be used as a source of stem cells to generate blood cells ex vivo for use in difficult-to-transfuse patients and also for gene editing to modify their phenotype (eg, to make the HSPCs resistant to HIV infection) or correct defective genes in monogenic diseases [8,9,10,12,47], therefore it is important to enhance HSPC numbers in CB while maintaining potentiality. Different approaches to increase HSPC numbers in single CB units include use of cytokine cocktails, stromal coculture, chemicals, and small molecules [13,14,16,17,[34][35][36][37][38]40,42,45,[48][49][50][51][52][53].…”

“…A single CB unit is generally sufficient for pediatric, but not adult, transplant recipients [7]. CB HSPCs can also be used for generating mature blood cells ex vivo for transfusion into difficult-to-transfuse patients or modulating resistance to specific acquired infections and correcting monogenic gene disorders, in dividing HSCs, using new genome editing technologies [8][9][10].…”

Noninvasive detection of F8 int22h-related inversions and sequence variants in maternal plasma of hemophilia carriers

“…7 The European Hematology Association Roadmap for European Hematology Research has identified development in NIPT for hemophilia as a research priority, in particular for severe and common sequence variants such as F8 int22h-related inversions. 8 The well-established hemophilia A and hemophilia B databases gather detailed descriptions of more than 2000 sequence variants occurring in the F8 gene and approximately 1000 sequence variants in the F9 gene, respectively. [9][10][11] Notably, approximately half of the patients with severe hemophilia A harbor the inversion mutations associated with intron 22 in the F8 gene, the int22h-related inversions, located on chromosome X.…”