Erythropoiesis Control and Ineffective Erythropoiesis: from Bench to Bedside
Chairs: Maria Domenica Cappellini (Milan), Stuart Orkin (Boston), Valeria Santini (Florence)
March 15-17, 2019 – Budapest, Hungary
A. Programme description
The conference on erythropoiesis control and ineffective erythropoiesis which will be held in Budapest in March 2019, as the title underlines “from bench to bedside” has the main objective of translating the most recent basic science acquisitions related to the origin and the developmental control of mega/erythroid progenitors in haematopoiesis to clinical practice. Erythropoiesis is a complex multistep process going from committed erythroid progenitors to mature red cells, involving a talk to talk process with other haematopoietic progenitors. Although some recent advances allow the characterization of some components of erythropoiesis, much still remains to be investigated particularly on pathological erythropoiesis and in any haematological condition characterized by ineffective erythropoiesis. During erythropoiesis efficient cytoprotective systems, are required to limit possible ROS-related toxic effects: these systems play an even more important role in conditions of ineffective erythropoiesis (IE), such as thalassaemias, sickle cell disease and congenital dyserythropoietic anaemias (CDAs) but also in myelodysplastic syndromes (MDS). Blood transfusion therapy or treatments with erythropoiesis stimulating agents (ESAs), such as recombinant EPO, are so far the frontline therapies for aneamia associated with ineffective erythropoiesis (IE). However, both treatments are not without risks, and in some cases they are not effective, therefore, there is a clinical need for novel agents supporting IE with different mechanism(s) of action from existing ESAs.
The two and half days program includes 9 sessions alternating basic science to clinical practice including specific diseases such as CDAs, thalassaemia, SCD, haemolytic anaemias and MDS. More specifically session one on erythropoiesis control will elucidate the role of transcription factors, the single cell gene expression in erythroid commitment/development and the proteolytic remodeling of red cell. Session 2 will focus on the weight of ineffective erythropoiesis in different diseases in determining the phenotype expression of the disease (i.e. thalassaemia, SCD, MDS). Session III will complete the insights of molecular causes of ineffective erythropoiesis. Session IV will be very interactive presenting cases characterized by the clinical consequences of IE. Session V is focused on hemoglobin switching that represents an ideal model for understanding different mechanisms of gene regulation especially the mechanisms of switch genes off and on. Session VI will be very clinical with practical advises to care emergency events in patients with different red cell disorders. The last 3 sessions are dedicated to the new therapeutic approaches either pharmacological or molecular to erythroid disorders mainly targeting IE. The speakers are eminent experts in their field, making the faculty very attractive.
B. Learning objectives
1. To understand the mechanisms that control erythropoiesis in the context of hematopoiesis
2. To identify the different causes of ineffective erythropoiesis
3. To translate the basic information in clinical practice in order to understand the different phenotype expressions of diseases such as thalassaemias, SCD, haemolytic anaemias and MDS
4. To be updated on the development of new drugs targeting ineffective erythropoiesis and the state of the art in clinical setting
5. To acquire skills in diagnosis and treatment of erythropoietic abnormalities in different haematological disorders
C. Why the meeting will be valuable for participants?
The meeting will offer a unique opportunity to discuss basic science in a clinical contest which will be created by presenting challenging clinical cases. The meeting is structured with the specific aim to be translational “from bench to bedside” involving participants in alive discussion
This meeting will be of interest to:
– Doctoral students and resident still in-training
– Junior and senior biologists and clinicians (MD, PhD, MD-PhD)
– Fully trained, early career as well as senior biologists and clinicians including experts in the field