Sickle cell disease (SCD)‐related organ complications are a major cause of morbidity and
mortality in patients with SCD. We sought to assess whether hematopoietic stem cell …

Haematopoietic stem cell transplantation (HSCT) is curative in sickle cell disease (SCD);
however, the lack of available matched donors makes this therapy out of reach for the …

Hematopoietic stem cell transplantation (HSCT) is curative for many non-malignant
disorders. As HSCT and supportive care technologies improve, this life-saving treatment …

Gonadal impairment is an important late effect with a significant impact on quality of life of
transplanted patients. The aim of this study was to compare gonadal function after busulfan …

Introduction Hematopoietic stem cell transplant (HSCT) is the only readily available curative
option for sickle cell disease (SCD). Cure rates following human leukocyte antigen (HLA) …

Thalassemia and sickle cell disease (SCD) are the most common monogenic diseases in
the world and represent a growing global health burden. Management is limited by a paucity …

Hemoglobinopathies are autosomal recessive disorders that occur when genetic mutations
negatively impact the function of hemoglobin. Common hemoglobinopathies that are …

Background Patients with sickle cell disease (SCD) have just one recognized curative
therapy option: hematopoietic stem cell transplantation (HSCT), which results in a long …

The β-hemoglobinopathies, transfusion-dependent β-thalassemia and sickle cell disease,
are the most prevalent inherited disorders worldwide and affect millions of people. Many of …

Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have
myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non …