RecruitingPhase 2

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

France52 participantsStarted 2025-01-24

Plain-language summary

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".

Who can participate

Age range40 Years – 60 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 * With the absence of donor specific antibody detected in the patient with a MFI \< 1500 (antibodies to the distinct haplotype between donor and recipient) * Usual criteria for HSCT: * ECOG ≤ 2 * No severe and uncontrolled infection * Cardiac function compatible with high dose of cyclophosphamide * With an adequate organ function ASAT and ALAT ≤ 3N, conjugated bilirubin ≤ 2N (or total bilirubin ≤ 2N if not available), clearance creatinine ≥ 50ml / min * With health insurance coverage * Women of childbearing potential and men must use contraceptive methods during their participation to the research and for 12 months and 6 months after the last dose of cyclophosphamide, respectively. * Having signed a written informed consent NB: The authorized contraceptive methods are: For women of childbearing age and in absence of permanent sterilization: * oral, intravaginal or transdermal combined hormonal contraception, * oral, injectable or transdermal progestogen-only hormonal contraception, * intrauterine hormonal-releasing system (IUS),…

What they're measuring

GRFS (Graft Versus Host Disease (GvHD) and Relapse/rejection-Free Survival)

Timeframe: 2 years after transplantation

Trial details

NCT IDNCT06646497

SponsorAssistance Publique - Hôpitaux de Paris

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2030-01-24

Contact for this trial

Régis Peffault de Latour, MD PhD

142385073 regis.peffaultdelatour@aphp.fr