Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC)

Marica Rossi; Sarah Szepetowski; Karima Yakouben; Catherine Paillard; Anne Sirvent; Martin Castelle; Charline Pegon; Christophe Piguet; Audrey Grain; Marie Angoso; Marie Robin; Nathalie Dhedin; Corinne Pondarré; Cécile Dumesnil de Maricourt; Ana Berceanu; Pauline Simon; Ambroise Marcais; Maryline Poirée; Virginie Gandemer; Dominique Plantaz; Stéphanie Nguyen; Gérard Michel; Anderson Loundou; Jean-Hugues Dalle; Isabelle Thuret

doi:10.1038/s41409-023-02079-0

Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC)

Bone Marrow Transplant. 2023 Nov;58(11):1254-1256. doi: 10.1038/s41409-023-02079-0. Epub 2023 Aug 4.

Authors

Marica Rossi¹, Sarah Szepetowski², Karima Yakouben³, Catherine Paillard⁴, Anne Sirvent⁵, Martin Castelle⁶, Charline Pegon⁷, Christophe Piguet⁸, Audrey Grain⁹, Marie Angoso¹⁰, Marie Robin¹¹, Nathalie Dhedin¹², Corinne Pondarré¹³, Cécile Dumesnil de Maricourt¹⁴, Ana Berceanu¹⁵, Pauline Simon¹⁶, Ambroise Marcais¹⁷, Maryline Poirée¹⁸, Virginie Gandemer¹⁹, Dominique Plantaz²⁰, Stéphanie Nguyen^{21

22}, Gérard Michel², Anderson Loundou²³, Jean-Hugues Dalle³, Isabelle Thuret²

Affiliations

¹ Department of Pediatric Hematology, Robert Debré Hospital, GHU APHP Nord, Université Paris Cité, Paris, France. marica.rossi@aphp.fr.
² Department of Pediatric Hematology and Oncology, Rare Disease Center for Thalassemia, La Timone Hospital, Marseille, France.
³ Department of Pediatric Hematology, Robert Debré Hospital, GHU APHP Nord, Université Paris Cité, Paris, France.
⁴ Department of Pediatric Hematology and Oncology, Hautepierre Hospital, Strasbourg, France.
⁵ Department of Pediatric Hematology and Oncology, Arnaud de Villeneuve Hospital, Montpellier, France.
⁶ Department of Pediatric Immunology and Hematology, Necker-Enfants Malades Hospital, Paris, France.
⁷ Department of Pediatric Hematology and Oncology, Estaing Hospital, Clermont-Ferrand, France.
⁸ Department of Pediatric Hematology and Oncology, Mother and Child University Hospital, Limoges, France.
⁹ Department of Pediatric Hematology and Oncology, University Hospital of Nantes, Nantes, France.
¹⁰ Department of Pediatric Hematology and Oncology, Pellegrin Hospital, Bordeaux, France.
¹¹ Department of Stem Cell Transplantation, Saint-Louis Hospital, GHU APHP Nord, Université Paris Cité, Paris, France.
¹² Unit of Hematology for Adolescents, Saint-Louis Hospital, Paris, France.
¹³ Rare Disease Center for Sickle Cell Disease, Centre Hospitalier Intercommunal de Créteil, Créteil, INSERM U955, Paris Est Créteil University, Créteil, France.
¹⁴ Department of Pediatric Hematology and Oncology, Charles Nicolle Hospital, Rouen, France.
¹⁵ Department of Adult Hematology, Jean Minjoz Hospital, Besançon, France.
¹⁶ Department of Pediatric Hematology and Oncology, Jean Minjoz Hospital, Besançon, France.
¹⁷ Department of Adult Hematology, Necker-Enfants Malades Hospital, Paris, France.
¹⁸ Department of Pediatric Hematology and Oncology, University Hospital of Nice, Nice, France.
¹⁹ Department of Pediatric Hematology and Oncology, University Hospital of Rennes, Rennes, France.
²⁰ Department of Pediatric Hematology and Oncology, University Hospital of Grenoble, Grenoble, France.
²¹ Department of Hematology, La Pitié-Salpêtrière Hospital, Paris, France.
²² Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC), Paris, France.
²³ Unit for Clinical and Epidemiological Research, DRRC/AP-HM Faculté de Médecine de Marseille, Marseille, France.

PMID: 37542188
DOI: 10.1038/s41409-023-02079-0

No abstract available

Publication types

Letter

MeSH terms

Busulfan / therapeutic use
Chimerism
France
Hematopoietic Stem Cell Transplantation* / methods
Humans
Retrospective Studies
Societies, Medical
Stem Cell Transplantation
Thalassemia* / therapy
Transplantation Conditioning / methods

Substances

Busulfan