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Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years
Sponsor: Assistance Publique - Hôpitaux de Paris
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".
Official title: Evaluation of an Optimized Allogeneic Hematopoietic Stem Cell Transplantation Protocol With Post-transplant Cyclophosphamide in Patients Aged 40 to 60 Years Old With Acquired Aplastic Anemia Refractory or in Relapse After Immunosuppression
Key Details
Gender
All
Age Range
40 Years - 60 Years
Study Type
INTERVENTIONAL
Enrollment
52
Start Date
2025-01-24
Completion Date
2030-01-24
Last Updated
2026-04-28
Healthy Volunteers
No
Conditions
Interventions
Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow
1. Conditioning regimen Thymoglobulin (0.5/mg/kg à D-9, 2 mg /kg at D-8 and 2.5 mg/kg à D-7), Fludarabine (30mg/m2/day i.v: day -6 to day -2), pre-transplant, Cyclophosphamide (14.5 mg/kg/day i.v: day -6 and day -5), and Total Body Irradiation (2 Gray on day -1). 2. Stem cell source Bone Marrow only. Target of 4 × 10\^8 nucleated cells/kg recipient body weight. Granulocyte colony stimulating factor is given subcutaneously starting on day +5 at 5 mg/ kg/day until the absolute neutrophil count is greater than 1.5 × 10\^9/L for 3 days. 3. GVHD prophylaxis Cyclophosphamide 50 mg/Kg/day at D+3 and D+4. Tacrolimus (0,2 à 0,3 mg/kg/day per os divided into 2 doses or 0.05 to 0.1 mg/kg/d IVSE) and mycophenolate (MMF) will begin from D+5. In absence of GvHD, MMF will be stopped between D35 and D45 and Tacrolimus at day 365. 4. Prevention of EBV reactivation Rituximab 150mg/m2 intravenously at Day+5 post HSCT (except patients and their donor with EBV serology and EBV PCR negative).
Locations (26)
Saint Louis hospital
Paris, France, France
CHU Amiens
Amiens, France
CHU Angers
Angers, France
CHU Besançon
Besançon, France
CHU Bordeaux
Bordeaux, France
CHU Caen
Caen, France
HNIA Percy
Clamart, France
Hôpital d'Estaing
Clermont-Ferrand, France
Hôpital Henri Mondor AP-HP
Créteil, France
CHU Grenoble Alpes
Grenoble, France
CHU Lille
Lille, France
CHU Limoges
Limoges, France
CHU Lyon Sud
Lyon, France
Institut Paoli Calmettes
Marseille, France
CHU Montpellier
Montpellier, France
CHRU Nancy
Nancy, France
CHU Nantes
Nantes, France
CHU Nice
Nice, France
Hopital Necker - APHP
Paris, France
Hôpital La Pitié Salpêtrière AP-HP
Paris, France
CHU Poitiers
Poitiers, France
CHU Rennes
Rennes, France
Henri Becquerel
Rouen, France
CHU Saint Etienne
Saint-Etienne, France
CHU Strasbourg
Strasbourg, France
CHU Toulouse
Toulouse, France