EBMT 2022 | Unmet needs in hemoglobinopathy care

There are several unmet medical needs in patients with hemoglobinopathies. The first one, without talking about treatment, is the need to establish adequate referral pathways for patients from one physician, for instance, from the pediatrician to eventually the adult physician, and to create a multidisciplinary team of professionals to take care of these patients. In addition to that, it’s really important to have referral centers that have adequate experience to treat these patients in the optimal conditions.
From a treatment point of view, we know that allogeneic stem cell transplantation is curative in patients with hemoglobinopathy, so in transfusion-dependent beta-thalassemia, and in sickle cell disease. But we know that in this specific subgroup of patients, GvHD should not be happening. So this question of GvL and GvHD should not have any impact when considering transplantation for patients with hemoglobinopathy. So, it’s a population of patients where the toxicity of allogeneic stem cell transplantation is more important to try to avoid. In this sense, nowadays, we are looking at really interesting data that have introduced a new therapy in this specific field.