Status and phase
Conditions
Treatments
About
multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based on cyclophosphamide administration post transplantation in patients with severe sickle cell disease.
Full description
Sickle cell disease is a severe disease with frequent occurrence of painful crises and progressive installation of a multi organ injuries. Despite the progress in its management, particularly since the introduction of hydroxycarbamide, the median age of death in sickle cell patients was about 40 years in a recent US study. Severe forms resistant to hydroxyurea or cerebral vasculopathy require transfusion programs throughout susceptible to risks of iron overload and alloimmunization. The bone marrow transplantation cures almost 95% of children and adolescents transplant from an HLA-identical siblings. In patients without HLA-identical donor, interesting results have been reported in haploidentical transplants marrow without ex vivo T cell depletion taken after non myeloablative conditioning regimen and GvHD prevention with cyclophosphamide high dose injection after bone marrow transplant . This approach performed in 14 patients was effective to cure 50% of the patients and 50% have rejected the transplant . No death or severe GvHD were related to the procedure.
DREPHAPLO protocol aims to evaluate that approach in a population of sickle cell patients with severe complications of the disease, bringing direct benefit to patients with a cure of the disease in at least half of them.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Inclusion criteria recipient:
Age: 13 years-40 years
Severe Sickle cell with at least one of the following criteria:
Not having geno-identical donor, but a haploidentical major donor (parent, sibling, adult child, or HbAA AS)
Having red and understood the information letter and signed the informed consent
Patients affiliated to a social security system (Social Security or Universal Medical Coverage)
Exclusion Criteria recipient:
Inclusion criteria donor
Primary purpose
Allocation
Interventional model
Masking
18 participants in 1 patient group
Loading...
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal