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 /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.
Can an / depleted T-Haplo-HSCT with regard to disease free survival, adverse events and
safety be considered equivalent to a matched sibling donor transplantation (MSD), in order to
offer cure for the majority of patients with sickle cell disease.
The main questions of this trial are:
Safety of a / T-depleted haploidentical HSCT
Incidence of acute and chronic GvHD
Rate of rejection
Fertility It is expected that the use of TCR+ and CD19+ depleted haploidentical cell
grafts in combination with the less aggressive and well tolerated conditioning regimen
needed for patient preparation will be associated with a low risk of grade II-IV aGVHD
and no extensive cGvHD, no graft failure and increase speed, spectrum and functionality
of immune system reconstitution. This is supposed to reduce the incidence of severe
infections leading to lower rates of transplantation related mortality (TRM).
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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