About this trial
Outcomes for adult patients with Severe Aplastic Anemia (SAA) aged more than 40 years who are refractory or in relapse after first-line IST remain poor. Hematopoietic stem cell transplantation (HSCT) is the unic valid therapeutic option but results have always been disappointing in patients aged 40 years or older. The first cause of death after HSCT in those refractory/relapse SAA patients is still graft versus host disease (GvHD). Recently, new strategies to prevent GvHD, including T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy), have revolutionized the field, notably in haplo-identical donor setting. Using marrow as source of stem cells and a PTCy strategy not only in haplo-identical donor setting but also in case of an available matched sibling or unrelated donor might prevent drastically GvHD and eventually be practice changing. Evaluating this new strategy is the main objectives of "APARR".
Eligibility criteria
Qualifiers
Aged from 40 to 60 years old
Suffering from acquired refractory severe idiopathic aplastic anemia after at least 6 months treatment with anti-thymocyte globulin, cyclosporine with Eltrombopag or in relapse
Allograft validated in the National Multidisciplinary expertise meetings of the French reference centre for aplastic anemia
With an available geno-identical donor or 10/10 matched donor or haploidentical donor
Disqualifiers
With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype).
With seropositivity for HIV or HTLV-1-2 or active hepatitis B or C and associated hepatic cytolysis
Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
Pregnant (βHCG positive) or breast-feeding
Trial design
Treatments tested in this trial
- Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow