Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age40-60
SponsorAssistance Publique - Hôpitaux de Paris

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

Treatment groups

52 Participants
are divided into 1 treatment group