An agent that regulates hemoglobin gene expression, used for the treatment of other haematological conditions, such as myelofibrosis and resistant to hydroxyurea polycythemia vera. A Phase 2a trial (TRUTH) in 30 transfusion-dependent β-thalassemia with splenomegaly showed that ruxolitinib therapy for 30 weeks resulted in slight improvement in pre-transfusion haemoglobin levels ...

Autologous CD34+ cells transduced with the BB305 lentiviral vector. An initial phase 1/2 study [Northstar (HGB-204)] in 22 β-thalassaemia patients showed transfusion-independency for non-β0/β0 genotypes and 60% reduction in transfuions in β0/β0 genotype. The results were published in N Engl J Med, in April 19, 2018. An ongoing phase 2 study (HGB-205) evaluates ...

The epidemiology project of TIF focuses on the collection of information on carrier rates, anticipated births and current status of haemoglobinopathy control strategies within health systems in the different country members of TIF, for β- and α-thalassaemiaσ, HbE and Sickle Cell Disease (SCD), and combinations constitute the main focus of ...

This is one of TIF’s new projects undertaken for the year 2013 and its goal is to develop a database for Haemoglobinopathy reference centres across the world, mapping the services they provide to patients. This is an extension to the information compiled by ENERCA-3 project and the deliberation of EUCERD, ...

TIF has played a leading role as a partner in the European Commission-funded project ENERCA (European Network for Rare and Congenital Anaemias). The project aims to bring experts together to improve public health services in the field of rare anaemias and, more particularly, to identify criteria and support national and ...

TIF has engaged in a project with EFGCP’s, the EU-funded European Patients’ Academy on Therapeutic Innovation’ (EUPATI). This is a patient-led academy, which will develop educational material, training courses and a public Internet library to educate patient representatives and the general public about all processes involved in medicines development. Topics ...

The ITHANET portal is a community website dedicated to thalassaemia and other haemoglobinopathies, offering a range of services including continuous updates on the upcoming conferences, interesting recent publications and talks. It also incorporates a database of organizations and networks concerned with haemoglobinopathies world-wide, and a wiki-based text interface to establish ...

Clinical care in thalassaemia major has improved dramatically over the last three decades, resulting in an impressive increase in survival rates and quality of life of patients living in countries where such clinical care is both available and affordable. However, cardiac disease still constitutes one of the most frequent and ...

DOMAINE is a European project that aims to create a safe and sufficient blood supply, by comparing and recommending good donor management practice. From 2008 to 2011, the DOMAINE project worked on improving donor management practices throughout Europe. The two main project outcomes are the Donor Management Manual and the ...