A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease With no Available Sibling Donor
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
• Preexisting severe or moderate SCD related complications:
‣ Clinically significant neurological event (stroke) or deficit
⁃ Silent crisis, neurocognitive deficit
⁃ Pathological angio-MRI with TOF Sequence
⁃ TCD velocity \>200 cm/s at 2 occasions \>1 month apart
⁃ More than 5 vaso-occlusive crises (VOC) in the past 1 year or more than 20 VOC in a lifetime
⁃ Two or more episodes of acute chest syndrome (ACS) in a lifetime or one episode of ACS in the past 24 months
⁃ Chronic transfusion requirement or more than 8 transfusions or one exchange transfusion in a lifetime
⁃ Transfusion-refractory allo-immunization
⁃ More than five SCD-related hospitalizations in a lifetime
⁃ Beginning pulmonary hypertension
⁃ Osteonecrosis at more than 2 sites
⁃ Beginning SCD Nephropathy
⁃ Recurrent priapism (\>2)