Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study

Author:

Peffault de Latour Regis1234,Chevret Sylvie5,Jubert Charlotte36,Sirvent Anne7,Galambrun Claire38,Ruggeri Annalisa910,Gandemer Virginie11,Cornillon Jérôme12,Rialland Fanny13,Dalle Jean-Hugues314,Forcade Edouard315,Bruno Benedicte316,Paillard Catherine17,Rorlich Pierre S.18,Salmon Alexandra17,Fürst Sabine319,Sicre de Fontbrune Flore123,Rubio Marie Therese20,Bay Jacques-Olivier21,Mohty Mohamad92223,Larghero Jerome2425,Gluckman Eliane26,Socié Gerard123

Affiliation:

1. Bone Marrow Transplantation (BMT) Unit, Saint Louis Hospital, Assistance Publique—Hôpitaux de Paris (AP-HP), Paris, France;

2. University Paris Diderot, Paris, France;

3. French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital, Paris, France;

4. Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, The Netherlands;

5. Biostatistic Unit, Saint Louis Hospital, AP-HP, Paris, France;

6. Pediatric BMT Unit, Bordeaux University Hospital, Bordeaux, France;

7. Pediatric BMT Unit, Montpellier, France;

8. Pediatric BMT Unit, Marseille, France;

9. BMT Unit, Saint Antoine Hospital, AP-HP, Paris, France;

10. Department of Pediatric Hematology and Oncology, Istituto Di Ricovera e Cura a Carattere Scientifico (IRCCS) Bambino Gesù Pediatric Hospital, Rome, Italy;

11. Pediatric BMT Unit, Rennes, France;

12. BMT Unit, Lucien Neuwirth Oncology Institute, Saint-Priest-en-Jarez, France;

13. Pediatric BMT Unit, Nantes, France;

14. Pediatric BMT Unit, Robert-Debré, AP-HP, Paris, France;

15. BMT and Cell Therapy Unit, Bordeaux, France;

16. Pediatric BMT Unit, Lille, France;

17. Pediatric BMT Unit, Strasbourg, France;

18. Pediatric BMT Unit, Nice, France;

19. BMT Unit, Marseille, France;

20. BMT Unit, Nancy, France;

21. BMT Unit, Clermont-Ferrand, France;

22. Sorbonne University, Paris, France;

23. INSERM Unité Mixte de Recherche 938, Paris, France;

24. Cell Therapy Unit, Saint Louis Hospital, AP-HP, Paris, France;

25. INSERM Clinical Investigation Center–Biological Therapy (Centre d'investigation clinique en biothérapie; CBT501), Paris, France; and

26. Eurocord, Saint Louis Hospital, AP-HP, Paris, France

Abstract

Key Points CBT after FLU-CY-ATG-2-Gy TBI with at least 4 × 107 frozen NCs per kilogram leads to satisfactory OS in refractory SAA. CBT is a valuable curative option in young patients with refractory idiopathic SAA and no available matched unrelated donors.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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