Unrelated Cord Blood Transplantation in Children, Adolescents, and Young Adults with Acute Leukemia or Myelodysplastic Syndrome: A Retrospective Comparative Study from the French Society for Bone Marrow Transplantation and Cellular Therapy Between Real-World Data and Previously Reported Results of a Randomized Clinical Trial.
[en] We previously reported results of a French randomized clinical trial (RCT) comparing the risk of transplantation failure (including transplant-related mortality [TRM], engraftment failure, and autologous recovery) in single and double unrelated cord blood (UCB) transplantation in children and young adults with hematologic malignancies. We concluded that single-UCB transplantation with an adequate cell dose is the standard of care, leading to a 70% two-year overall survival (OS). It remains unclear, however, whether RCT participants have better outcomes than comparable patients not treated in the setting of a clinical trial. We compared the characteristics and outcomes of RCT participants (n = 137) to a Francophone population-based registry of patients (real-world [RW] group) fulfilling the eligibility criteria used in our RCT and transplanted with 1 or 2 UCB units after a myeloablative conditioning (MAC) regimen between March 2015 (end of inclusion in the RCT) and February 2019 (n = 141). The primary endpoint was the 2-year cumulative incidence (CI) of transplantation strategy failure as defined in our RCT. The 2 groups were comparable in terms of age, disease distribution, hematologic status at transplantation, follow-up, and HLA compatibility. Patients in the RW group were more likely to be transplanted with a single-unit UCB (87.9% versus 49.6%, P< .001) and to receive a radiation-free regimen (39.0% versus 60.6%, P< .001). The 2-year CI of transplantation strategy failure, TRM, and the 2-year probability of OS were similar between the 2 groups, although the relapse risk was higher in the RW group (31.2% ± 7.7% versus 20.4% ± 6.8%, P= .01), resulting in a significantly lower disease-free survival (DFS) (59.2% ± 8.4% versus 69.3% ± 8.0%, P= .047). This difference remained statistically significant only in the group of patients with acute lymphoid leukemia (ALL) who did not receive the conditioning regimen recommended by the RCT (fludarabine 75 mg/m2, total body irradiation 12 Gy, cyclophosphamide 120 mg/kg). The results of our RCT appear to be reproducible in real-world conditions, provided that the same cord blood selection criteria and conditioning regimen are used.
Disciplines :
Hematology
Author, co-author :
Teyssier, Anne-Charlotte ; Department of Pediatric Hematology-Oncology, Montpellier University Hospital, Montpellier, France. Electronic address: teyssier.anne-charlotte@neuf.fr
Michel, Gérard; Department of Pediatric Hematology-Oncology, APHM, Timone Hospital, Aix-Marseille University, Marseille, France
Jubert, Charlotte; Department of Pediatric Hematology-Oncology, CHU Bordeaux, F-33000, Bordeaux, France
Rialland, Fanny; Department of Pediatric Hematology-Oncology, Nantes University Hospital, Nantes, France
Visentin, Sandrine; Department of Pediatric Hematology-Oncology, APHM, Timone Hospital, Aix-Marseille University, Marseille, France
Ouachée, Marie; Institute of Pediatric Hematology-Oncology, Lyon University Hospital, Lyon, France
Bilger, Karin; Department of Hematology, Strasbourg University Hospital, Strasbourg, France
Gandemer, Virginie; Department of Pediatric Hematology-Oncology, Rennes University Hospital, Rennes, France
Beguin, Yves ; Centre Hospitalier Universitaire de Liège - CHU > > Service d'hématologie clinique
Marie-Cardine, Aude; Department of Pediatric Hematology-Oncology, Rouen University Hospital, Rouen, France
Chalandon, Yves ; Division of Hematology, Department of Oncology, Geneva University Hospitals, Geneva, Switzerland, Faculty of Medicine, University of Geneva, Geneva, Switzerland
Ansari, Marc; Department of Women, Child and Adolescent, Onco-Hematology Unit, Geneva University Hospital, Geneva, Switzerland, Department Cansearch Research platform for pediatric oncology and hematology, Faculty of Medicine, Department of Pediatrics, Gynecology and Obstetrics, University of Geneva, Geneva, Switzerland
Baumstarck, Karine; Department of Public Health, EA 3279 Research Unit, Marseille University Hospital, Aix-Marseille University, Marseille, France
Loundou, Anderson ; Department of Public Health, EA 3279 Research Unit, Marseille University Hospital, Aix-Marseille University, Marseille, France
Dalle, Jean-Hugues; Department of Pediatric Hematology, APHP, Robert Debré Hospital, Paris Diderot University, Paris, France
Sirvent, Anne; Department of Pediatric Hematology-Oncology, Montpellier University Hospital, Montpellier, France
Unrelated Cord Blood Transplantation in Children, Adolescents, and Young Adults with Acute Leukemia or Myelodysplastic Syndrome: A Retrospective Comparative Study from the French Society for Bone Marrow Transplantation and Cellular Therapy Between Real-World Data and Previously Reported Results of a Randomized Clinical Trial.
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