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Stem Cell Investig. 2019 Dec 5;6:39. doi: 10.21037/sci.2019.10.04. eCollection 2019.

Cord blood transplantation for bone marrow failure syndromes: state of art.

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Hematology and Transplantation Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
Eurocord-Monacord, Hôpital Saint Louis, Paris, France.
Cellular Therapy and Immunobiology Working Party of EBMT, Leiden, The Netherlands.


Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing IST still have a poor prognosis. Cord blood transplantation (CBT) has extended the possibility of HSCT for many patients in case of the absence of an eligible donor, and although in the last years, this procedure is less used in several hematological diseases, it remains an option for the treatment of patients with BMF syndromes. Nevertheless, optimization of conditioning regimen and cord blood unit selection is warranted to reduce the risk of graft failure and transplant-related mortality. This review summarizes the state of art of CBT in the field of BMF diseases, focusing on historical and recent issues in idiopathic aplastic anemia and inherited disorders.


Cord blood transplantation (CBT); Fanconi anemia (FA); bone marrow failure (BMF); severe aplastic anemia (SAA)

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Conflicts of Interest: The authors have no conflicts of interest to declare.

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