A phase II trial to assess Haploidentical a/ß T-depleted stem cell transplantation in patients with sickle cell disease with no available sibling donor (T-HAPLO-SCT)

Basic data for this project

Description

Sickle cell disease (SCD) represents a worldwide health problem with over 300,000 affected children born each year and remains a disease with high morbidity and mortality. Migration makes SCD an increasingly eminent problem of western countries. The disease is overtaking haemophilia and cystic fibrosis in certain parts of Europe and is a progressive chronic multi-organ disease. Stroke is the major debilitating complication of SCD and the most frequent cause of stroke in children. 30% develop silent strokes leading to cognitive impairment in children and poor quality of life. Matched sibling donor (MSD) hematopoietic stem cell transplantation (HSCT) is the gold standard and the only curative treatment available, with a 90% survival without graft failure at 5 years. But MSD are available for only 20% of patients and HSCT with unrelated donors resulted in unacceptably high rates of graft-versus-host disease (GvHD). T-cell depleted haploidentical HSCT (T-Haplo-HSCT) from relatives, mostly parents, but also haplo-matched siblings and cousins could offer cure to the remaining 80% of affected patients. This trial aims to demonstrate that a haploidentical stem cell transplantation from a relative is equivalent to a sibling donor HSCT with regard to outcome and treatment related morbidity and mortality. Patients with an established indication for HSCT will be stratified and will receive a sibling donor transplantation if available. If not, the patient will receive a transplant from a haploidentical relative. With the exception of the stem cell source, both groups will receive an almost identical treatment. We expect to show that T-Haplo-HSCT is safe and can achieve a comparable outcome to a MSD transplantation with regard to overall, disease-free and GvHD-free survival. The intention is, to offer cure to every patient affected with sickle cell disease.