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
Parallel
TCRα/β+ and CD19+ depleted haploidentical stem cell transplantation
Other interventionHaploidentical 5+/10 HSCT from a relative, α/β T-depleted
Matched sibling donor transplantation
Other intervention10/10 HSCT - matched family donor
Primary efficacy endpoint: Composite Endpoint: Event free survival (EFS).
Event is defined as incidence of acute GvHD (Grade III - IV), chronic GvHD (moderate/severe), graft failure (GF), or death (from any reason).
Overall survival
Overall survival rate (OS) is defined as time from transplantation to death or last follow-up and will be assessed at Day 100 and after 1 year and 2 years.
Disease free survival
• Disease-free survival (DFS) is defined as the minimum time to recurrence, to death or to the last follow-up, from the time of transplantation and will be assessed at Day 100 and after 1 year and 2 years.
Graft failure
defined as initial neutrophil engraftment followed by a decline in ANC \<500/µl that is unresponsive to growth factor therapy and/or other intervention
Quality of life: EQ-5D
Adult patients ≥18 years. The European Quality of Life 5 Dimension (EQ-5D) questionnaire has two components: health state description and evaluation. In the description part, health status is measured in terms of five dimensions (5D); mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Mobility dimension asks about the person's walking ability. Self-care dimension asks about the ability to wash or dress by oneself, and usual activities dimension measures performance in "work, study, housework, family or leisure activities". In pain/discomfort dimension, it asks how much pain or discomfort they have, and in anxiety/depression dimension, it asks how anxious or depressed they are.
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