Durable engraftment after pharmacological pre-transplant immune suppression followed by reduced-toxicity myeloablative haploidentical stem cell transplantation in highly HLA-immunized adults with sickle cell disease.

Autor: Fürst S; Department of Hematology, Institut Paoli Calmettes, Marseille, France. fursts@ipc.unicancer.fr., Bernit E; Reference Center for Sickle Cell Disease, Thalassemia and Other Red Cell Rare Diseases, CHU Guadeloupe, Pointe à Pitre, Guadelloupe, France., Legrand F; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Granata A; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Harbi S; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Devillier R; Department of Hematology, Institut Paoli Calmettes, Centre de Recherche en Cancérologie de Marseille, INSERM, CNRS, Aix-Marseille University, Marseille, France., Maisano V; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Bouchacourt B; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Pagliardini T; Department of Hematology, Institut Paoli Calmettes, Marseille, France., Mokart D; Department of Intensive Care, Institut Paoli Calmettes, Marseille, France., Lemarié C; Cell Therapy Facility, Institut Paoli Calmettes, Marseille, France., Calmels B; Cell Therapy Facility, Institut Paoli Calmettes, Marseille, France., Picard C; HLA Laboratory, Etablissement Français du Sang, Marseille, France., Basire A; HLA Laboratory, Etablissement Français du Sang, Marseille, France., Andersson BS; Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX, USA., Blaise D; Department of Hematology, Institut Paoli Calmettes, Centre de Recherche en Cancérologie de Marseille, INSERM, CNRS, Aix-Marseille University, Marseille, France.
Jazyk: angličtina
Zdroj: Bone marrow transplantation [Bone Marrow Transplant] 2024 Jul; Vol. 59 (7), pp. 918-927. Date of Electronic Publication: 2024 Mar 14.
DOI: 10.1038/s41409-024-02257-8
Abstrakt: Allogeneic stem cell transplantation (Allo-SCT) is the only rapidly available curative treatment modality in patients with severe sickle cell disease (SCD). The development of reduced-toxicity myeloablative conditioning (RT-MAC) regimen and the use of partially matched family donors with post-transplantation cyclophosphamide (PT-Cy) have widened the access to Allo-SCT. Antibodies against donor-specific HLA (DSA) increase the risk of engraftment failure in HLA mismatched Allo-SCT. We report the results of five patients with SCD, whereas three with DSA, who underwent an unmanipulated haploidentical stem cell transplantation (Haplo-SCT) after a busulfan-based RT-MAC regimen with PT-Cy. To reduce the risk of engraftment failure, a sequential two courses pharmacological pre-transplant immune suppression (PTIS) phase was added prior to the conditioning regimen. All patients engrafted successfully. The procedure was well tolerated. None of the patients developed acute GVHD, whereas one developed moderate chronic GVHD. After a median follow-up of 5 years (range, 2.2-9), all patients are free of pain with excellent quality of life. Our report shows that Haplo-SCT after a RT-MAC regimen is feasible and safe with stable long-term engraftment and excellent disease control. The risk of graft failure can be abrogated by adding a PTIS phase prior to initiating the conditioning regimen.
(© 2024. The Author(s), under exclusive licence to Springer Nature Limited.)
Databáze: MEDLINE
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