A new era of hemoglobinopathies treatment began 40 years ago when allogeneic haematopoietic stem cell transplantation provided the first cure for these genetic conditions. Over 10,000 patients have received transplants since then and currently, gene therapy is at the beginning of a new era. As a result, patients suffering from hemoglobinopathies now have several therapeutic options to choose from.
In the new Hemoglobinopathies Working Party (HWP), we have the potential to redefine transplant journey modalities and compare different options in order to choose the best option for our patients.
The main objectives of the HWP will be to:
- Promote scientific activity by involving investigators around the world, especially from areas where these diseases are prevalent
- Collaborate closely with other WPs and Thalassemia and Sickle Cell Disease specialists and associations
- Improve data collection quality with a view to long-term clinical benefits