Hemoglobinopathies (Sickle Cell Disease and Thalassemia)

Hematopoietic cell transplantation (HCT) using an HLA-matched sibling donor is a well-established curative therapy for pediatric patients with sickle cell disease (SCD) and transfusion-dependent thalassemias (TDT). In order to expand the donor pool, new approaches such as related haploidentical donor HCT have been used with encouraging results. These approaches aim for a higher overall survival, an effective reduction of acute and chronic GvHD and a reduced toxicity. Due to these alternative approaches and adult patients being increasingly transplanted, the number of HCT has dramatically increased in the last decade. Furthermore, different gene therapy and gene editing strategies are being developed in clinical trials, showing promising results.