HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.
Age 2yrs to 35yrs
Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
Study specific consent given
Clinically significant neurological event (stroke) or deficit
Karnofsky or Lansky Performance Score < 70%
Patients with donor-specific antibodies (DSA) against the potential stem cell donor by either
Cell-based crossmatched assays (Complement-dependent cytotoxicity; CDC) or
Flow cytometry crossmatch test or
University of Regensburg
Lead sponsor